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Ovarian Rhabdomyosarcoma

An ultra-rare ovarian sarcoma with skeletal-muscle differentiation. Management borrows from soft-tissue/rhabdomyosarcoma playbooks plus ovary-specific surgical principles.

Educational only: This page is not medical advice. Coordinate decisions with your oncology team.

Reviewed Jun 2026 · site editor · How we review →

AI extractedhuman reviewedsources checkedretractions suppressed· last updated Jun 2026

Evidence at a glanceHuman · observationalMixed results⚠ Studies disagree
6 published studies that name Ovarian Rhabdomyosarcoma2 human studies approved & graded (trial, observational, or meta-analysis)7 human clinical studies in the Ovarian Rhabdomyosarcoma corpus222 source documents in the Ovarian Rhabdomyosarcoma corpus

last checked June 19, 2026

Why this grade?

Human · observational — Human observational evidence only — no trials.

  • 2 human · 1 animal · 0 lab · 3 review/other
  • Most authoritative study: Ovarian Rhabdomyosarcoma in Children
  • Studies disagree on the reported direction (conflict flagged).
  • Findings conflict across studies
  • Effect sizes reported in only 2 of 6 studies
  • All studies are small (n < 30)

Computed deterministically from the studies’ types and reported outcomes — not written by AI, and not a claim that anything works.

What the guidelines say

NCI PDQESMONCCNASCO

We link the authoritative guidelines rather than reproduce them. Below, the treatments on this page are split into standard care, guideline or regulatory options, supportive care, and studied but not standard so established care is not mixed with experimental or supportive items.

Standard care - guideline-backed
  • Aim for complete macroscopic resection when feasible
  • Unilateral salpingo-oophorectomy often required
  • Nodal evaluation considered, especially for alveolar RMS given higher nodal risk
  • Fertility-sparing approaches only in highly selected early cases with multidisciplinary input
  • Plan en-bloc resection to avoid capsular rupture or tumor spill
  • Mark close/deep margins with clips to guide adjuvant radiation planning
  • If an unplanned (‘whoops’) resection occurred, restage and consider re-excision to achieve R0 before RT
  • In bulky disease, consider neoadjuvant chemotherapy to downstage before definitive surgery
  • For oligometastatic disease, discuss metastasectomy or ablation (lung/liver) in tumor board after systemic control
  • Coordinate ureteral stents or bowel resection with peri-chemo timing to minimize infectious complications
  • Consider for positive margins, nodal involvement, or unresectable/local recurrence
  • Pelvic RT planning must balance organ tolerance and prior surgeries
  • SBRT
  • Use IMRT/VMAT to spare bowel, bladder, rectum, and ovaries/uterus when organ preservation matters
  • Post-op RT
  • Spine/bone mets: consider SBRT for pain control and local control
  • Time RT around systemic therapy to minimize overlapping toxicities (e
  • Lung mets: SBRT or wedge resection discussed case-by-case after systemic response
  • VAC-based regimens (vincristine, actinomycin, cyclophosphamide) standard in pediatric/AYA RMS
  • Adult options
  • Platinum/taxane regimens
  • Clinical trial enrollment strongly encouraged due to rarity
  • Risk-adapted intensity with early response assessment (typically after 2–3 cycles)
  • Consider VAC/IVA variants or VDC/IE-style intensity in AYA/fit adults when tolerated—individualize to comorbidity and goals
  • Anthracycline cardioprotection (dexrazoxane) and dose-capping strategies
  • Ifosfamide protocols: ensure mesna, aggressive hydration, and CNS/renal monitoring (encephalopathy, proximal tubulopathy)
  • Maintenance concepts (e.g., low-dose alkylator/vinca)
  • Anti-angiogenic TKIs (e
  • VAC (vincristine/actinomycin/cyclophosphamide) (pediatric/AYA)
  • Doxorubicin/Ifosfamide variants (adult STS)
  • Gemcitabine/Docetaxel (recurrent/palliative)
  • VDC/IE (vincristine/doxorubicin/cyclophosphamide ↔ ifosfamide/etoposide) (high-risk/AYA-fit adult)
  • IVA (ifosfamide/vincristine/actinomycin) (neoadjuvant/anthracycline-avoidant)
  • VIT (vincristine/irinotecan/temozolomide) (relapsed/chemo-pretreated)
  • High-dose Ifosfamide (salvage)
  • Doxorubicin/Dacarbazine (± Ifosfamide) (adult STS legacy)
  • Oral maintenance (vinorelbine + low-dose cyclophosphamide) (post-response/investigational)
  • MSI-H/dMMR (rare)
  • IGF1R/PI3K/AKT/mTOR: pathway alterations support trial eligibility
  • ctDNA/NGS
  • NTRK fusion (rare): TRK inhibitors (tumor-agnostic)
  • TFCP2-fusion subset: ALK overexpression—consider ALK-focused trials and keratin-positive RMS recognition
  • PAX–FOXO1 epigenetic dependency: BET/BRD4 inhibition trials and transcriptional-complex disruption strategies
  • YAP/TEAD (Hippo dysregulation): early-phase TEAD inhibitor programs for resistance/stemness biology
  • p53–MDM2 axis: MDM2 antagonists in development—consider when TP53 wild-type and MDM2-high
  • DDR targeting (PARP/ATR) as radiosensitizers/combos in refractory disease—trial contexts only
Guideline / FDA options - context-specific
  • Hedgehog/GLI, MEK/ERK: investigational targets
  • Checkpoint inhibitors: modest activity overall
  • FGFR4 activation/overexpression: trial-focused FGFR4 inhibitors and emerging CAR-T approaches
  • Rational combos to blunt feedback (e.g., PI3K/mTOR + MEK)
Supportive care - symptom / survivorship support
  • Pain management with multimodal analgesia
  • Nutrition optimization to counter cachexia
  • Management of bowel obstruction risk and postoperative adhesions
  • Psychosocial support and AYA-focused services when applicable
  • Growth-factor support per regimen risk and prior cycle neutropenia to preserve dose intensity
  • VTE prophylaxis/education individualized by surgery, immobilization, and TKI use
  • Fertility preservation consults (oocyte/embryo banking) before gonadotoxic therapy when feasible
  • Cardio-oncology, nephrology, and pain/spine referrals early when organ-specific risks emerge
  • Neuropathy prevention/rehab: dose adjustments, PT/OT for balance and fine-motor function
  • Sleep, mood, and anxiety management—optimize antiemetics, pain plan, and behavioral supports to maintain resilience
  • Vaccination review (avoid live vaccines on active chemo)
  • Clear home plan for fever, dehydration, and port issues
Studied, not standard - investigational
  • vincristine/doxorubicin/cyclophosphamide
  • total abdominal radiotherapy
  • surgery
  • RMS regimen
  • complete resection
  • chemotherapy
  • radiation
  • adriamycin
  • hysterectomy with bilateral salpingo-oophorectomy
  • Stem cell transplantation
  • EET therapy (IFN-gamma or TNF-SAM2 followed by OK-432)
  • camptothecin group drugs
  • bleomycin + etoposide + cisplatin
  • carbotaxol (carboplatin + paclitaxel)
  • vincristine + dactinomycin + cyclophosphamide/ifosfamide (VAC)
  • ?
  • pazopanib
  • melphalan (intravenous)
  • High-dose-rate intravaginal brachytherapy (IVRT)
  • Jasplakinolide + pulsed electric fields (PEFs)
  • CDX0239-PBD (ALK-directed ADC)
  • Irinotecan
  • Pelvic proton beam therapy
  • Ovarian transposition (OT)
  • percutaneous nephrostomy
  • ureteral stenting
  • double-J catheter
  • vincristine + Adriamycin (doxorubicin) + actinomycin D + cyclophosphamide (VAC)
  • polypectomy
  • limited resection
  • Verteporfin
  • Actinomycin D

Read the guidelines

Cancer-specific deep links aren’t curated yet — these search the authoritative sources for Ovarian Rhabdomyosarcoma.

Treatment map: Ovarian Rhabdomyosarcoma

Open as a full page →

Standard care plus every compound studied in the literature (each cited) and graded by evidence, organized by clinical readiness. A category, not a verdict that anything works — confirm anything here with your oncology team.

82
Interventions
46
Standard of care
2
Tested in people
1
Lab / animal
29
Named in lit.
7
Classes
Standard of care (46) Guideline option (4) Tested in people (2) Lab / animal only (1) Named in the literature (29)

Tested in people, by trial phase: phase not reported ×2

Clinical evidence
Preclinical evidence
Standard of care
Guideline option
Tested in people
Lab / animal only
Named in the literature
Surgery & procedures
10
—
—
—
10
Radiotherapy
8
—
—
—
4
Chemotherapy
18
—
1
—
10
Targeted therapy
9
3
—
—
2
Immunotherapy
1
1
1
—
—
Repurposed drugs
—
—
—
—
1
Other
—
—
—
1
2

Columns group into clinical evidence (used in, or tested on, people) and preclinical evidence (lab/animal, or only named in the literature). Cell = number of interventions; a dashed cell means none recorded there.

Established care — detail (50)
Surgery & procedures
Aim for complete macroscopic resection when feasible
Aim for complete macroscopic resection when feasible.
CurativeStandardCurated
Unilateral salpingo-oophorectomy often required
Unilateral salpingo-oophorectomy often required; hysterectomy/contralateral oophorectomy based on age, stage, and fertility goals.
CurativeStandardCurated
Nodal evaluation considered, especially for alveolar RMS given higher nodal risk
Nodal evaluation considered, especially for alveolar RMS given higher nodal risk.
CurativeStandardCurated
Fertility-sparing approaches only in highly selected early cases with multidisciplinary input
Fertility-sparing approaches only in highly selected early cases with multidisciplinary input.
CurativeStandardCurated
Plan en-bloc resection to avoid capsular rupture or tumor spill
Plan en-bloc resection to avoid capsular rupture or tumor spill; use a specimen bag for extraction to limit peritoneal seeding.
CurativeStandardCurated
Mark close/deep margins with clips to guide adjuvant radiation planning
Mark close/deep margins with clips to guide adjuvant radiation planning.
CurativeStandardCurated
If an unplanned (‘whoops’) resection occurred, restage and consider re-excision to achieve R0 before RT
If an unplanned (‘whoops’) resection occurred, restage and consider re-excision to achieve R0 before RT.
CurativeStandardCurated
In bulky disease, consider neoadjuvant chemotherapy to downstage before definitive surgery
In bulky disease, consider neoadjuvant chemotherapy to downstage before definitive surgery.
CurativeStandardCurated
For oligometastatic disease, discuss metastasectomy or ablation (lung/liver) in tumor board after systemic control
For oligometastatic disease, discuss metastasectomy or ablation (lung/liver) in tumor board after systemic control.
CurativeStandardCurated
Coordinate ureteral stents or bowel resection with peri-chemo timing to minimize infectious complications
Coordinate ureteral stents or bowel resection with peri-chemo timing to minimize infectious complications.
CurativeStandardCurated
Radiotherapy
Consider for positive margins, nodal involvement, or unresectable/local recurrence
Consider for positive margins, nodal involvement, or unresectable/local recurrence.
—StandardCurated
Pelvic RT planning must balance organ tolerance and prior surgeries
Pelvic RT planning must balance organ tolerance and prior surgeries.
—StandardCurated
SBRT
SBRT can be considered for oligometastatic lung/bone disease.
Advanced / metastaticStandardCurated
Use IMRT/VMAT to spare bowel, bladder, rectum, and ovaries/uterus when organ preservation matters
Use IMRT/VMAT to spare bowel, bladder, rectum, and ovaries/uterus when organ preservation matters.
—StandardCurated
Post-op RT
Post-op RT is guided by margin status (R1/R2) and nodal disease; pre-op RT is an option for downstaging in select cases.
—StandardCurated
Spine/bone mets: consider SBRT for pain control and local control
Spine/bone mets: consider SBRT for pain control and local control; screen for cord compression symptoms.
—StandardCurated
Time RT around systemic therapy to minimize overlapping toxicities (e
Time RT around systemic therapy to minimize overlapping toxicities (e.g., ifosfamide renal, anthracycline cardiac).
—StandardCurated
Lung mets: SBRT or wedge resection discussed case-by-case after systemic response
Lung mets: SBRT or wedge resection discussed case-by-case after systemic response.
—StandardCurated
Chemotherapy
VAC-based regimens (vincristine, actinomycin, cyclophosphamide) standard in pediatric/AYA RMS
VAC-based regimens (vincristine, actinomycin, cyclophosphamide) standard in pediatric/AYA RMS.
—StandardCurated
Adult options
Adult options include doxorubicin/ifosfamide or gemcitabine/docetaxel (extrapolated from soft-tissue sarcoma).
—StandardCurated
Platinum/taxane regimens
Platinum/taxane regimens are not standard for pure RMS unless mixed histology is present.
—StandardCurated
Clinical trial enrollment strongly encouraged due to rarity
Clinical trial enrollment strongly encouraged due to rarity.
—StandardCurated
Risk-adapted intensity with early response assessment (typically after 2–3 cycles)
Risk-adapted intensity with early response assessment (typically after 2–3 cycles); switch or escalate if inadequate response.
—StandardCurated
Consider VAC/IVA variants or VDC/IE-style intensity in AYA/fit adults when tolerated—individualize to comorbidity and goals
Consider VAC/IVA variants or VDC/IE-style intensity in AYA/fit adults when tolerated—individualize to comorbidity and goals.
—StandardCurated
Anthracycline cardioprotection (dexrazoxane) and dose-capping strategies
Anthracycline cardioprotection (dexrazoxane) and dose-capping strategies may preserve intensity over longer courses.
—StandardCurated
Ifosfamide protocols: ensure mesna, aggressive hydration, and CNS/renal monitoring (encephalopathy, proximal tubulopathy)
Ifosfamide protocols: ensure mesna, aggressive hydration, and CNS/renal monitoring (encephalopathy, proximal tubulopathy).
—StandardCurated
Maintenance concepts (e.g., low-dose alkylator/vinca)
Maintenance concepts (e.g., low-dose alkylator/vinca) are investigational in adults—prefer within trials.
MaintenanceStandardCurated
VAC (vincristine/actinomycin/cyclophosphamide) (pediatric/AYA)
Foundation for pediatric RMS; adult tolerance varies. Use growth-factor prophylaxis; early response check at 2–3 cycles to decide on escalation or switch.
—StandardCurated
Doxorubicin/Ifosfamide variants (adult STS)
Common adult sarcoma backbone; monitor cardiac/renal toxicity. Consider dexrazoxane for cardioprotection; strict mesna/hydration and CNS/renal monitoring for ifosfamide.
—StandardCurated
Gemcitabine/Docetaxel (recurrent/palliative)
Soft-tissue sarcoma option; activity varies in RMS. Useful for symptom control and disease stabilization when curative options are limited.
Advanced / metastaticStandardCurated
VDC/IE (vincristine/doxorubicin/cyclophosphamide ↔ ifosfamide/etoposide) (high-risk/AYA-fit adult)
Intensified alternating regimen; consider for bulky/biologically adverse disease to enable resection. Requires G-CSF, cardioprotection strategy, and tight toxicity monitoring.
—StandardCurated
IVA (ifosfamide/vincristine/actinomycin) (neoadjuvant/anthracycline-avoidant)
Option when avoiding anthracyclines (cardiac risk) or as bridge to surgery; ensure mesna/hydration and neuro/renal surveillance.
NeoadjuvantStandardCurated
VIT (vincristine/irinotecan/temozolomide) (relapsed/chemo-pretreated)
Pediatric-relapse–derived; can debulk or stabilize to open local control windows. Watch GI toxicity and myelosuppression.
—StandardCurated
High-dose Ifosfamide (salvage)
STS-standard salvage with occasional RMS responses; neuro/nephrotoxicity vigilance is essential. Consider when aiming to downstage for consolidative RT/surgery.
—StandardCurated
Doxorubicin/Dacarbazine (± Ifosfamide) (adult STS legacy)
Historic backbone with modest RMS activity; consider case-by-case when other regimens contraindicated. Cardio and marrow safety planning required.
—StandardCurated
Oral maintenance (vinorelbine + low-dose cyclophosphamide) (post-response/investigational)
Exploratory disease-control concept after good response; adult RMS data limited—prefer on protocol.
MaintenanceStandardCurated
Targeted therapy
Anti-angiogenic TKIs (e
Anti-angiogenic TKIs (e.g., pazopanib) have limited RMS-specific data; reserve mainly for refractory settings or trials.
—StandardCurated
IGF1R/PI3K/AKT/mTOR: pathway alterations support trial eligibility
IGF1R/PI3K/AKT/mTOR: pathway alterations support trial eligibility; single-agent activity historically limited.
—StandardCurated
Hedgehog/GLI, MEK/ERK: investigational targets
Hedgehog/GLI, MEK/ERK: investigational targets; consider baskets.
—Guideline optionCurated
ctDNA/NGS
ctDNA/NGS can inform trial matching and detect resistance patterns.
—StandardCurated
NTRK fusion (rare): TRK inhibitors (tumor-agnostic)
NTRK fusion (rare): TRK inhibitors (tumor-agnostic) can be high-yield when present.
—StandardCurated
TFCP2-fusion subset: ALK overexpression—consider ALK-focused trials and keratin-positive RMS recognition
TFCP2-fusion subset: ALK overexpression—consider ALK-focused trials and keratin-positive RMS recognition.
—StandardCurated
FGFR4 activation/overexpression: trial-focused FGFR4 inhibitors and emerging CAR-T approaches
FGFR4 activation/overexpression: trial-focused FGFR4 inhibitors and emerging CAR-T approaches.
—Guideline optionCurated
PAX–FOXO1 epigenetic dependency: BET/BRD4 inhibition trials and transcriptional-complex disruption strategies
PAX–FOXO1 epigenetic dependency: BET/BRD4 inhibition trials and transcriptional-complex disruption strategies.
—StandardCurated
YAP/TEAD (Hippo dysregulation): early-phase TEAD inhibitor programs for resistance/stemness biology
YAP/TEAD (Hippo dysregulation): early-phase TEAD inhibitor programs for resistance/stemness biology.
—StandardCurated
p53–MDM2 axis: MDM2 antagonists in development—consider when TP53 wild-type and MDM2-high
p53–MDM2 axis: MDM2 antagonists in development—consider when TP53 wild-type and MDM2-high.
—StandardCurated
DDR targeting (PARP/ATR) as radiosensitizers/combos in refractory disease—trial contexts only
DDR targeting (PARP/ATR) as radiosensitizers/combos in refractory disease—trial contexts only.
—StandardCurated
Rational combos to blunt feedback (e.g., PI3K/mTOR + MEK)
Rational combos to blunt feedback (e.g., PI3K/mTOR + MEK) should be pursued in trials, not empirically off-label.
—Guideline optionCurated
Immunotherapy
MSI-H/dMMR (rare)
MSI-H/dMMR (rare): may enable PD-1 blockade under tumor-agnostic approvals.
—StandardCurated
Checkpoint inhibitors: modest activity overall
Checkpoint inhibitors: modest activity overall; combinations under study.
—Guideline optionCurated

Established care shown from OncoForge editorial curation · reviewed September 15, 2025 — authoritative citations (NCI PDQ / FDA) are being added.

Supportive care (12)
  • Pain management with multimodal analgesia; early palliative care involvement improves QoL.
  • Nutrition optimization to counter cachexia; involve oncology dietitian early.
  • Management of bowel obstruction risk and postoperative adhesions.
  • Psychosocial support and AYA-focused services when applicable.
  • Growth-factor support per regimen risk and prior cycle neutropenia to preserve dose intensity.
  • VTE prophylaxis/education individualized by surgery, immobilization, and TKI use; fast evaluation of leg swelling/SOB.
  • Fertility preservation consults (oocyte/embryo banking) before gonadotoxic therapy when feasible; discuss ovarian suppression limitations.
  • Cardio-oncology, nephrology, and pain/spine referrals early when organ-specific risks emerge.
  • Neuropathy prevention/rehab: dose adjustments, PT/OT for balance and fine-motor function.
  • Sleep, mood, and anxiety management—optimize antiemetics, pain plan, and behavioral supports to maintain resilience.
  • Vaccination review (avoid live vaccines on active chemo); prompt antiviral/antibacterial prophylaxis per risk.
  • Clear home plan for fever, dehydration, and port issues; provide direct lines and thresholds to avoid delays in care.
Investigational & adjunct compounds — detail (32)
Tested in people (2)
EET therapy (IFN-gamma or TNF-SAM2 followed by OK-432)melphalan (intravenous)· First-line (advanced disease)
Named in the literature
vincristine/doxorubicin/cyclophosphamidetotal abdominal radiotherapysurgeryRMS regimencomplete resectionchemotherapyradiationadriamycinhysterectomy with bilateral salpingo-oophorectomyStem cell transplantationcamptothecin group drugsbleomycin + etoposide + cisplatin· Adjuvant (after surgery)carbotaxol (carboplatin + paclitaxel)· Adjuvant (after surgery)vincristine + dactinomycin + cyclophosphamide/ifosfamide (VAC)?pazopanibHigh-dose-rate intravaginal brachytherapy (IVRT)Jasplakinolide + pulsed electric fields (PEFs)CDX0239-PBD (ALK-directed ADC)· biomarker-selectedIrinotecanPelvic proton beam therapyOvarian transposition (OT)percutaneous nephrostomyureteral stentingdouble-J cathetervincristine + Adriamycin (doxorubicin) + actinomycin D + cyclophosphamide (VAC)polypectomylimited resectionVerteporfin
Lab / animal only
Actinomycin D

"Tested in people" rows show the highest trial phase found in that compound's cited human studies (Phase I–IV; "phase not reported" = a human study with no phase tag). "Studied" = named in the cited literature for this cancer. "FDA ✓" = FDA-approved for this cancer; "off-label" = an FDA-approved drug used outside its approved indications (per openFDA). Not a claim that anything works.

Reported figures

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Snapshot

The essentials in ~60 seconds — every line is drawn from the cited sources below.

What it is
A very rare, often aggressive primary ovarian rhabdomyosarcoma that is usually reported at an advanced stage and for which no standardized treatment exists. [1][2][3][4][5]
Survival
Overall prognosis is generally poor; reported survival ranges from 18 days to 15 months after diagnosis, and in one review 7 of 11 patients with follow-up died 10 days to 26 months after surgery. [4][6]
Standard treatment
Management is multimodal, typically combining surgery with multiagent chemotherapy and often radiation; aggressive combination therapy is commonly used. [2][4][7]
Key test
Testing for DICER1 mutations (including germline testing when indicated) and molecular confirmation of DICER1‑mutated sarcoma. [8][9][10]
Biggest challenge
Late presentation with advanced disease and the absence of standardized therapeutic approaches, contributing to poor outcomes. [2][1]

Ask about Ovarian Rhabdomyosarcoma

Answers come only from the cited sources on this page — with the supporting evidence shown. If the sources here don't cover your question, it will say so. Educational information, not medical advice.

Key numbers & factors

Risk factors

  • ▲ increases riskDICER1 syndrome / pathogenic DICER1 variants — Associated with embryonal rhabdomyosarcoma of the female genital tract; can be germline or somatic. [10][11][12]
  • ▲ increases riskLynch syndrome (germline MSH2 variants) — In systematic reviews of Lynch patients, a higher proportion of sarcomas were rhabdomyosarcoma. [13]
  • ▲ increases riskPathogenic BRCA2 variants (heterozygous, reported) — Reported in children with rhabdomyosarcoma in germline testing series. [14]

Biomarkers

  • DICER1 mutationsActionableMolecular diagnosis and germline testing to identify syndromic risk and guide surveillance/testing [8][9][11]
  • Myogenin / MYOD1 / desmin (rhabdomyoblastic markers) · Tissue immunohistochemistry to confirm rhabdomyoblastic differentiation (diagnosis) [8][11]
  • Serum CK‑MB and LD‑2 (with CK and LDH) · Serum markers that may be elevated in rhabdomyosarcoma (reported in metastatic ovarian case) [15]
  • ALK overexpression · Reported as uniformly overexpressed in fusion‑positive rhabdomyosarcoma (diagnostic/biologic marker) [16]
  • miR‑20a · Circulating/tissue prognostic marker associated with unfavorable overall survival in pooled analyses [17]

10 sections — tap any heading to expand its cited detail. Key points are above.

Overview17 points
  • Primary ovarian rhabdomyosarcoma is described as a rare ovarian neoplasm and as a very rare primary neoplasm of the ovary. [3][4]
  • Rhabdomyosarcoma can appear as a heterologous element in ovarian sex cord-stromal tumors; ovarian Sertoli-Leydig cell tumor (SLCT) with heterologous rhabdomyosarcoma (RMS) is exceptionally rare. [18][9]
  • Reports describe ovarian rhabdomyosarcoma as having a poor prognosis. [19][7]
  • Epithelial carcinoma of the ovary is one of the most common gynecologic malignancies, with almost 50% of all cases occurring in women older than 65 years; stromal and germ cell tumors of the ovary are relatively uncommon and comprise fewer than 10% of ovarian cancer cases. [20]
  • Sources describe ovarian rhabdomyosarcoma as rare, and ovarian involvement by rhabdomyosarcoma as rare. [2]
  • Sources describe ovarian rhabdomyosarcoma as often presenting at an advanced stage. [2]
  • The source states that rhabdomyosarcoma predominantly arises in parameningeal and periorbital regions and in other areas that do not typically contain striated muscle. [2]
  • A DICER1-mutated rhabdomyosarcoma of the ovary has been described as a rare, emerging entity with a predilection for the gynecologic and genitourinary tracts. [8]
  • Primary ovarian rhabdomyosarcomas are described as extremely rare in children. [5]
  • Breast metastases from ovarian carcinoma have been described as typically having microlobulated margins and posterior enhancement on imaging. [21]
  • Metastatic rhabdomyosarcoma is included among the nonmammary malignancies of the breast described in an imaging series. [22]
  • Embryonal rhabdomyosarcoma is discussed as one of the (rare) uterine mesenchymal neoplasms in recent reviews of uterine mesenchymal tumors. [23]
  • Rhabdomyosarcoma (RMS) is the most common sarcoma arising from soft tissues in the pediatric population, and approximately 15–20% of RMS are located in the urogenital system, with the urinary bladder and prostate being the most common origin points followed by the vagina and uterus; RMS was also one of the histopathologies found among pediatric pelvic malignant tumors in a retrospective series. [24][25]
Show 4 lab & early-research findings
  • Metastasized rhabdomyosarcoma cells can be present in human ovarian cortex tissue fragments and were the target of an ex vivo purging strategy in a laboratory study. [26]
  • Reported cases include a 16-year-old female, a 41-year-old premenopausal woman, and a 10-year-old female English pointer. [27][28][29]3 sources
  • Primary ovarian rhabdomyosarcoma has been reported as an extremely rare and aggressive malignancy with limited reported cases and no standardized treatment protocol. [1]
  • A case report described pure ovarian rhabdomyosarcoma in a 16-year-old female. [27]
Epidemiology12 points

Key figures

Survival & outcomes
OutcomeValue95% CI
proportion of sarcomas that were rhabdomyosarcoma in the pooled Lynch-syndrome sarcoma literature10%—
Source quotes
  • “a higher proportion of rhabdomyosarcoma (10 %, especially pleomorphic rhabdomyosarcoma) is reported.”
  • One review of 13 primary ovarian rhabdomyosarcoma cases reported ages from 7 to 79 years, and another review noted cases in a 60-year-old, a 13-year-old, and a 14-year-old. [6][11]
  • In a 13-case review, 11 tumors were embryonal and 2 were alveolar rhabdomyosarcomas. [30][6]
  • In systematic reviews of Lynch syndrome patients who developed sarcomas, a higher proportion of those sarcomas were rhabdomyosarcoma (reported as 10%, especially pleomorphic rhabdomyosarcoma). [13]
  • In the 13-case review, abdominal pain and swelling were reported presentations, tumor laterality included right ovary, left ovary, both ovaries, and unknown laterality, tumor stage ranged from stage I to stage IV, and tumor size ranged from 10 to 19.5 cm. [6]
  • One review reported that about 50% of cases were diagnosed with disease beyond the ovary. [4]
  • In one series of 11 ovarian Sertoli–Leydig cell tumors (SLCTs) with heterologous rhabdomyosarcoma the patients showed a bimodal age distribution with seven patients (64%) aged 33 years or younger (mean 20) and four patients (36%) aged 52 years or older (mean 60); in that series all tumors were unilateral. [9]
  • DICER1 syndrome is an inherited disorder, described as inherited in an autosomal dominant pattern, that is associated with several rare tumours including embryonal rhabdomyosarcoma, botryoid type. [10]
  • Rhabdomyosarcoma accounts for approximately 3% of new cancer cases in children aged 0–14 years per year. [24]
  • In a 10-year pathology series from northern Ghana, rhabdomyosarcoma accounted for 46.8% of the soft tissue cancers identified in children and adolescents. [31]
  • Pediatric gynecological cancers are uncommon and data are limited in many settings. [32]
  • In a large series of pediatric rhabdomyosarcoma, a small fraction arose from the female genital tract. [12]
  • In a separate cohort of ovarian SLCTs the reported median age at diagnosis was 21 years (range 14-57) and the prevalence of DICER1 germline variants was reported as 33.3% (4/12). [33]
Key biomarkers17 points
  • GLI1 expression is considered a reliable biomarker of Hedgehog pathway activation, and GLI gene amplification has been reported as a mechanism of ligand-independent HH-GLI pathway activation in cancers including rhabdomyosarcoma. [34][35]
  • High serum CK-MB and LD-2 may serve as a marker of rhabdomyosarcoma, and one metastatic ovarian rhabdomyosarcoma case had increased serum creatine kinase, CK-MB, lactate dehydrogenase, and LD-2. [15]
  • The 2023 report described an ovarian DICER1-mutated rhabdomyosarcoma with two DICER1 mutations identified by sequencing. [8]
  • The sarcomatous component in the 2023 report was Myogenin and MYOD1 positive. [8]
  • DICER1 mutations have been reported in ovarian and fallopian tube embryonal rhabdomyosarcoma, and in one tubal case the mutation was germline while in the other two cases it was somatic. [11]
  • Rhabdomyoblastic differentiation in these tumors was positive for desmin, myogenin, and myoD1. [11]
  • Reported tumor cells expressed desmin, muscle-specific actin, and myoglobin in one case. [28]
  • Reported tumor cells expressed myosin, desmin, vimentin, and CD10 in one dog case, and were negative for cytokeratin, placental alkaline phosphatase, inhibin-alpha, and smooth muscle actin. [29]
  • Ultrastructural findings in one case included Z-band-like material and alternating thin and thick filaments. [27]
  • In tissue-based studies, alveolar rhabdomyosarcoma showed upregulated miR-20a expression in tumorous tissue compared with nontumorous tissue; across multiple tumor types, high circulating miR-20a expression was reported to be significantly associated with unfavorable overall survival (HR = 1.71, 95% CIs: 1.43-2.04, p < 0.01). [17]
  • In one molecularly tested series, all tumors tested (7/7, 100%) harbored hotspot DICER1 mutations; of those tumors, six cases (86%) also carried a second nonsense or frameshift loss-of-function DICER1 mutation, and component-specific analysis in two cases revealed shared common DICER1 hotspot mutations in both the SLCT and RMS components, supporting a clonal origin. [9]
  • In a larger institutional series, 60% (47/78) of moderate-to-poorly differentiated SLCTs harbored DICER1 mutations (including 80% [16/20] with heterologous elements) while all well-differentiated tumors (20/20) were DICER1 wild-type. [33]
  • In that series, TERT c.-124C>T promoter mutations (4 cases) or TP53 mutations (3 cases) were present and were reported as mutually exclusive. [9]
  • Somatic RNase IIIb domain missense mutations in DICER1 have been identified in tumors including one ovarian embryonal rhabdomyosarcoma, and germline pathogenic variants in DICER1 have been found in some patients with female genital tract rhabdomyosarcoma. [12]
  • Heterozygous pathogenic variants in BRCA2 have been reported in children with rhabdomyosarcoma in germline testing series. [14]
Show 2 lab & early-research findings
  • Expression of the human Piwil2 gene was detected in tumors examined, including ovarian cancer and rhabdomyosarcoma (in mouse), and Piwil2 expression was causally linked to inhibition of apoptosis and promotion of proliferation via Stat3/Bcl-X(L) signaling in experimental systems. [36]
  • Sources report that ALK is uniformly overexpressed with tumor cell surface expression in fusion-positive rhabdomyosarcoma. [16]
Biology & pathways18 points

Key figures

Survival & outcomes
OutcomeValue95% CI
proportion of sarcomas in Lynch-syndrome patients with MSH2 mutations57%—
Source quotes
  • “It seems that most sarcomas developed in patients with a germline mutation of MSH2 (57 %) exhibit a dMMR (81 %) or MSI (77 %) phenotype, as in other LS-tumors.”
  • Sources describe the Hedgehog-GLI pathway as highly conserved in development; its aberrant activation is implicated across many cancers and is a target for therapeutic inhibition. Non-canonical, SMO-independent GLI activation is also described. [35][34]
  • In sarcomas that developed in Lynch syndrome patients, most tumors arising in patients with a germline MSH2 mutation exhibited mismatch-repair deficiency or microsatellite instability (reported as dMMR 81% and MSI 77%). [13]
  • The source states that rhabdomyosarcoma predominantly arises in parameningeal and periorbital regions and in other areas that do not typically contain striated muscle. [2]
  • The occurrence of ovarian rhabdomyosarcoma may be attributed to the presence of ovarian stromal fibroblasts or endometriotic stroma. [2]
  • DICER1-mutated ovarian rhabdomyosarcomas may show cartilage foci, teratoid mature glands, immature blastematous-like tubes, and/or neuroectodermal components. [8]
  • Molecular testing remains necessary to confirm the diagnosis of DICER1-mutated sarcomas. [8]
  • One review states that DICER1 mutations are associated with a variety of uncommon neoplasms including cervical and genitourinary embryonal rhabdomyosarcoma. [11]
  • One review states that ERMS arising at unusual sites, especially when they contain cartilage or bone/osteoid, are especially likely to be associated with DICER1 mutations. [11]
  • One source states that rhabdomyogenesis recapitulates skeletal muscle differentiation during early fetal life. [4]
  • One source suggests rhabdomyoblasts in the ovary may arise from uncommitted stromal fibroblasts or fibroblasts of endometriotic stroma. [4]
  • One report states that the tumor recapitulates primitive embryonal testis and rhabdomyogenesis. [19]
  • One case had extensive peripheral blood and bone marrow involvement and presented with a leukemia-like picture. [27]
  • Topoisomerase I catalytic activity was much more variable in human malignancies and ranged from a low in a rhabdomyosarcoma to a high in a poorly differentiated ovarian carcinoma; the authors suggested that high topo I levels might indicate that those tumors would be sensitive to drugs that target topo I. [37]
  • Rhabdomyosarcoma heterologous elements were described as primitive mesenchyme with rhabdomyoblastic differentiation and perivascular condensation of tumor cells observed in all cases. [33]
  • Some analyses have noted an association of VPS13A with rhabdomyosarcoma and with ovarian cancer. [38]
  • The combination of jasplakinolide and electric pulses synergistically affected RMS cells, causing significant cytoskeletal changes and reduced viability in vitro. [39]
  • One case report described ovarian rhabdomyosarcoma arising from a prior endometrioma, with focal areas suggesting a possible underlying ovarian adenosarcoma with stromal overgrowth. [30]
  • One case report described vascular invasion and widespread abdominal metastases. [29]
Standard management13 points
  • Management of pediatric gynecologic rhabdomyosarcoma is complex and generally requires a multidisciplinary approach; for patients with metastatic disease this typically consists of systemic multiagent chemotherapy and local therapy (surgery with or without radiotherapy), and for tumors developing in the uterus or vagina a tailored approach combining surgery, radiation therapy, and chemotherapy is considered to preserve fertility. [40][32][41]3 sources
  • The source states that management requires a multimodal treatment approach; in this series, some patients also received abdominopelvic radiotherapy. [2]
  • Sarcomas should be treated in tertiary care centers with good pathology support and expertise. [42]
  • One review states that aggressive combination therapy including surgery, radiation, and chemotherapy appears to be the only hope for improved survival. [4]
  • One reported patient underwent hysterectomy with bilateral salpingo-oophorectomy and staging biopsies. [28]
  • One report states that despite two cycles of chemotherapy, disease persisted in the pelvis 4 months after diagnosis. [28]
  • Most patients with botryoid vaginal rhabdomyosarcoma are classified as low- or intermediate-risk and are treated with limited-intensity chemotherapy, but local treatment is required to minimize the risk of local relapse. [43]
  • Tumor removal in pediatric oncology is increasingly performed by minimally invasive approaches, which allow for quicker postoperative recovery and less postoperative pain. [44]
  • Bilateral upper urinary tract obstructions and unilateral severe obstructions from pelvic tumors are managed as oncological emergencies and may require emergent urinary diversion such as nephrostomy or ureteral stenting; in the reported series percutaneous nephrostomy was favored as the primary option for urinary diversion in patients with obstruction from pelvic malignant tumors. [25]
  • For embryonal rhabdomyosarcoma of the cervix, the reported series indicates a good prognosis at that site and that conservative surgical approaches can be reasonable in selected cases. [45]
  • Surgical resection (oophorectomy) was performed in 100% (14/14) of cases in one reported cohort. [33]
Show 2 lab & early-research findings
  • Autotransplantation of ovarian cortex fragments that contain metastasized tumour cells may reintroduce malignancy to the recipient, posing a safety concern for fertility-preservation procedures; an ex vivo 24 h treatment with the YAP/TAZ inhibitor Verteporfin was used experimentally to attempt eradication of induced tumor foci in human ovarian cortex tissue fragments. [26]
  • Surgery, chemotherapy, and radiation have been used in reported cases. [7][4]
Treatments & compounds studied32 treatments

Chemotherapy

  • vincristine/doxorubicin/cyclophosphamide · 3 findings
    • One pediatric report described chemotherapy including vincristine, doxorubicin, and cyclophosphamide with good response to therapy; the 2025 series says chemotherapy regimens were selected by risk stratification and aligned with international protocols, but does not name specific drugs or outcomes for those regimens. [2][5]
    • Vincristine, Adriamycin (doxorubicin) and actinomycin D (alternating) with cyclophosphamide (VAC) chemotherapy was used in patients with rhabdomyosarcoma in the reported pediatric series. [32]
      patients treated 2patients alive with no evidence of disease 1
      Source quotes
      • “Vincristine, Adriamycin (alternating with actinomycin D) and cyclophosphamide (VAC) and carboplatin and paclitaxel (TC) were used in 2 patients each with rhabdomyosarcoma and epithelial ovarian tumors respectively.”
      • “One of the patients with embryonal rhabdomyosarcoma (1/2) and the one with cervical cancer (IIIc1r) were diagnosed in the last 2 years and are still on follow up, also alive with no evidence of disease at the time of study completion.”
    • Standard systemic chemotherapy for metastatic RMS in the described pediatric case included a three-drug combination of vincristine, dactinomycin, and cyclophosphamide/ifosfamide (VAC). [40]
  • RMS regimen: One review described an excellent response to an RMS regimen in a young girl with ovarian rhabdomyosarcoma. [42]
  • chemotherapy · 2 findings
    • Chemotherapy was reported in case literature without a specific regimen being identified in the provided sources. [7][46][4]3 sources
    • Chemotherapy was administered in one reported case, and the disease persisted in the pelvis 4 months after diagnosis. [28]
  • camptothecin group drugs: The authors noted that high topoisomerase I levels might indicate sensitivity to the camptothecin group of anticancer drugs. [37]
  • bleomycin + etoposide + cisplatin: Adjuvant (after surgery)Adjuvant chemotherapy with bleomycin, etoposide, and cisplatin was administered in 42.9% (6/14) of patients in one cohort. [33]
    proportion receiving surgery 100%proportion receiving BEP adjuvant chemotherapy 42.9%proportion receiving carbotaxol adjuvant chemotherapy 7.1%
    Source quotes
    • “Surgical resection (oophorectomy)100 (14/14)”
    • “Adjuvant chemotherapy (bleomycin, etoposide, cisplatin)42.9 (6/14)”
    • “Adjuvant chemotherapy (carbotaxol)7.1 (1/14)”
  • carbotaxol (carboplatin + paclitaxel): Adjuvant (after surgery)Adjuvant chemotherapy with carbotaxol was administered in 7.1% (1/14) of patients in the cohort. [33]
    proportion receiving surgery 100%proportion receiving BEP adjuvant chemotherapy 42.9%proportion receiving carbotaxol adjuvant chemotherapy 7.1%
    Source quotes
    • “Surgical resection (oophorectomy)100 (14/14)”
    • “Adjuvant chemotherapy (bleomycin, etoposide, cisplatin)42.9 (6/14)”
    • “Adjuvant chemotherapy (carbotaxol)7.1 (1/14)”
  • melphalan (intravenous): First-line (advanced disease)Intravenous melphalan has been reported to show "striking activity" in previously untreated patients with rhabdomyosarcoma. [47]
  • Irinotecan: Irinotecan was included in the chemotherapy regimen used in a reported pediatric case of cervical/vaginal rhabdomyosarcoma. [41]
Show 1 lab & early-research entry
  • adriamycin: Adriamycin was associated with a transient tumor response in one reported case. [46]

Targeted therapy

  • pazopanib: The same pediatric patient additionally received targeted treatment with pazopanib, which is described as currently undergoing clinical trials for treatment of large unresected intermediate- to high-grade soft tissue sarcomas. [40]
Show 1 lab & early-research entry
  • CDX0239-PBD (ALK-directed ADC): biomarker-selectedA humanized ALK-directed antibody-drug conjugate (CDX0239-PBD) showed potent antitumor efficacy in ALK-expressing fusion-positive rhabdomyosarcoma xenograft models. [16]

Immunotherapy

  • EET therapy (IFN-gamma or TNF-SAM2 followed by OK-432): EET therapy (intravenous administration of either IFN-gamma or TNF-SAM2 followed 3 hours later by OK-432) produced objective responses of lung metastases in patients with multiple organ metastases of cervical cancer, ovarian cancer and uterine rhabdomyosarcoma. [48]
    One of the side effects, transitory hypotension, was observed in 46% of the cases. 46%
    Source quote
    • “One of the side effects, transitory hypotension, was observed in 46% of the cases.”

Radiotherapy

  • total abdominal radiotherapy: In the 2025 series, three patients received intensity-modulated abdominopelvic radiotherapy at a total dose of 2,400 cGy in 16 sessions. [2]
  • radiation: Radiation was reported in case literature. [7][4]
  • High-dose-rate intravaginal brachytherapy (IVRT): High-dose-rate intravaginal brachytherapy (IVRT) is reported as an option for local control of vaginal RMS with few long-term risks. [43]
    Five-year local control 75% (95% CI 34–91)Five-year overall survival 86% (95% CI 33–98)
    Source quotes
    • “Five-year local control was 75% (95% CI, 34%-91%).”
    • “Two of 3 patients who experienced treatment failure were cured with salvage therapy resulting in a 5-year overall survival of 86% (95% CI, 33%-98%).”
  • Pelvic proton beam therapy: Pelvic proton beam therapy was used after ovarian transposition in a pediatric patient, with a reported total dose of 50 Gy. [41]

Repurposed drugs

Show 1 lab & early-research entry
  • Verteporfin: Verteporfin, used as a YAP/TAZ inhibitor ex vivo, enabled eradication of experimentally induced rhabdomyosarcoma tumour foci from human ovarian cortex fragments in the study. [26]
    treatment duration 24
    Source quote
    • “followed by a 24 h ex vivo treatment with the YAP/TAZ inhibitor Verteporfin to eradicate the cancer cells.”

Procedures & devices

  • surgery: Reported surgical approaches included resection, unilateral oophorectomy or adnexectomy, ovarian-sparing staging, and primary cytoreduction. [2]
  • Stem cell transplantation: Stem cell transplantation was performed at a Tehran center for various diseases including ovarian cancer and rhabdomyosarcoma. [49]
    About 74.9% of the patients (1530 of 2042) remained alive between one to 168 months after stem cell transplantation. 74.9%Nearly 25.1% (512 of 2042) of our patients died after stem cell transplantation. 25.1%
    Source quotes
    • “About 74.9% of the patients (1530 of 2042) remained alive between one to 168 months after stem cell transplantation.”
    • “Nearly 25.1% (512 of 2042) of our patients died after stem cell transplantation.”
  • Ovarian transposition (OT): Sources describe ovarian transposition as a procedure performed to preserve ovarian function in patients undergoing pelvic or abdominal radiotherapy. [41]
  • percutaneous nephrostomy: In this small retrospective series of pelvic tumors causing upper urinary tract obstruction, nephrostomies were placed in four patients; eGFR was significantly improved at the end of treatment in patients with nephrostomy, whereas it was not improved in those without. [25]
    rate of eGFR improvement (nephrostomy group) 74.9 vs -1.39%, p = 0.0416 vs non-nephrostomy group
    Source quote
    • “The rate of eGFR improvement was significantly greater in patients undergoing nephrostomy than those who did not (− 1.39 ± 4.09 versus 74.9 ± 44.8%, P = 0.0416).”
  • ureteral stenting: Ureteral stenting is listed among emergent urinary-diversion options for severe upper urinary tract obstruction from pelvic malignancy. [25]
    rate of eGFR improvement (nephrostomy group) 74.9 vs -1.39%, p = 0.0416 vs non-nephrostomy group
    Source quote
    • “The rate of eGFR improvement was significantly greater in patients undergoing nephrostomy than those who did not (− 1.39 ± 4.09 versus 74.9 ± 44.8%, P = 0.0416).”
  • polypectomy: Polypectomy was one of the surgical treatments performed for embryonal rhabdomyosarcoma of the cervix in a reported series. [45]
    patients 6patients 26patients 29
    Source quotes
    • “Treatment information was available in 62 and included polypectomy in 6 patients (2 also received chemotherapy), limited resection in 26 (14 also received chemotherapy), hysterectomy in 29 (15 with adjuvant chemotherapy), and biopsies only in 1 (with chemotherapy).”
    • “Treatment information was available in 62 and included polypectomy in 6 patients (2 also received chemotherapy), limited resection in 26 (14 also received chemotherapy), hysterectomy in 29 (15 with adjuvant chemotherapy), and biopsies only in 1 (with chemotherapy).”
    • “Treatment information was available in 62 and included polypectomy in 6 patients (2 also received chemotherapy), limited resection in 26 (14 also received chemotherapy), hysterectomy in 29 (15 with adjuvant chemotherapy), and biopsies only in 1 (with chemotherapy).”
  • limited resection: Limited resection was performed in a series of patients with embryonal rhabdomyosarcoma of the cervix. [45]
    patients 6patients 26patients 29
    Source quotes
    • “Treatment information was available in 62 and included polypectomy in 6 patients (2 also received chemotherapy), limited resection in 26 (14 also received chemotherapy), hysterectomy in 29 (15 with adjuvant chemotherapy), and biopsies only in 1 (with chemotherapy).”
    • “Treatment information was available in 62 and included polypectomy in 6 patients (2 also received chemotherapy), limited resection in 26 (14 also received chemotherapy), hysterectomy in 29 (15 with adjuvant chemotherapy), and biopsies only in 1 (with chemotherapy).”
    • “Treatment information was available in 62 and included polypectomy in 6 patients (2 also received chemotherapy), limited resection in 26 (14 also received chemotherapy), hysterectomy in 29 (15 with adjuvant chemotherapy), and biopsies only in 1 (with chemotherapy).”
Show 3 lab & early-research entries
  • complete resection: Complete resection of the primary tumors before chemotherapy was described in one case report. [5]
  • hysterectomy with bilateral salpingo-oophorectomy: Hysterectomy with bilateral salpingo-oophorectomy and staging biopsies were performed in one reported case. [28]
  • double-J catheter: Double-J catheters were placed during tumor resection in the reported cases to prevent ureteral injury or as a stent through an anastomosis. [25]
    rate of eGFR improvement (nephrostomy group) 74.9 vs -1.39%, p = 0.0416 vs non-nephrostomy group
    Source quote
    • “The rate of eGFR improvement was significantly greater in patients undergoing nephrostomy than those who did not (− 1.39 ± 4.09 versus 74.9 ± 44.8%, P = 0.0416).”

Other

  • ?: Reported pediatric patients received radiotherapy targeting the primary lesion in addition to systemic chemotherapy, and in a Kenyan pediatric gynecologic oncology series one of two patients with embryonal rhabdomyosarcoma was alive with no evidence of disease at study completion. [40][32]
    patients treated 2patients alive with no evidence of disease 1
    Source quotes
    • “Vincristine, Adriamycin (alternating with actinomycin D) and cyclophosphamide (VAC) and carboplatin and paclitaxel (TC) were used in 2 patients each with rhabdomyosarcoma and epithelial ovarian tumors respectively.”
    • “One of the patients with embryonal rhabdomyosarcoma (1/2) and the one with cervical cancer (IIIc1r) were diagnosed in the last 2 years and are still on follow up, also alive with no evidence of disease at the time of study completion.”
Show 1 lab & early-research entry
  • Jasplakinolide + pulsed electric fields (PEFs): Jasplakinolide combined with pulsed electric fields (PEFs) disrupted the actin cytoskeleton and reduced viability in rhabdomyosarcoma cells in vitro with minimal effects on normal cells. [39]
Staging & risk8 points
  • The Toronto Childhood Cancer Stage Guidelines include rhabdomyosarcoma among the childhood cancers they were developed to cover; in the registry study assessing the Toronto Guidelines, stage could be assigned according to Tier 2 criteria for 1318 (93%) of 1412 childhood cancer cases. [50]
  • The 2025 report says ovarian rhabdomyosarcoma often presents at an advanced stage; in its series, four of six patients were clinical stage and group IV due to distant metastases. [2]
  • The 2025 series also reported clinical group IV disease with metastatic involvement. [2]
  • In the 13-case review, tumors were reported across stages I to IV. [6]
  • One biopsy was reported as rhabdomyosarcoma with FIGO stage IIB. [28]
  • In registry-based analyses of poor-prognosis metastatic cancers, metastatic disease is denoted by the SEER coding rule as 'distant' stage disease. [51]
Show 2 lab & early-research findings
  • In the reported cohort FIGO stage at presentation included IA in 68.8% (11/16) of cases; several of the reported rhabdomyosarcoma cases were recorded as Stage 3 (Group III) in the case series. [33][25]
  • One case report described metastatic spread to the splenic flexure of the colon and both lungs. [5]
Prognosis15 points
  • In one institutional cohort the relapse rate was 30.8% (4/13) and the median progression-free survival was 60 months (range 8-188). [33]
  • After adjustment for cancer site, race/ethnicity, sex, and SES, the risk of death for all adolescents and young adults combined was 25% lower than for older adults (aHR 0.75; 95%CI 0.73–0.77). [51]
  • For rhabdomyosarcoma specifically, the adjusted hazard ratio for death comparing AYAs with older adults was reported as 0.33 (95%CI 0.26–0.42). [51]
  • In the series of embryonal rhabdomyosarcoma of the cervix, 11 of 79 patients had extrauterine recurrences (14%) and 9 died of disease (11%); older age was associated with extrauterine recurrence and decreased disease-specific survival. [45]
  • For alveolar rhabdomyosarcomas, overall survival was reported as remaining around or under 50% in the reviewed period. [52]
  • In a single-institution series of 12 patients treated with high-dose-rate IVRT for vaginal RMS the 5-year overall survival was reported as 86% (95% CI, 33%–98%). [43]
  • Two pediatric cases were alive 8 and 9 months post-operatively after complete resection and chemotherapy. [5]
  • In the 13-case review, 7 of 11 patients with follow-up died of disease 10 days to 26 months after surgery, and 4 of 11 patients with follow-up were alive 2 to 9 months after surgery. [6]
  • One review reported survival ranging from 18 days to 15 months after diagnosis. [4]
  • One report states that prognosis is poor when rhabdomyosarcoma is a significant or predominant component of Sertoli-Leydig cell tumor. [19]
  • One case review concluded that prognosis is poor and similar to that of primary ovarian rhabdomyosarcoma when rhabdomyosarcomatous recurrence develops in ovarian immature teratoma. [7]
  • In one case, disease persisted in the pelvis 4 months after diagnosis despite two cycles of chemotherapy. [28]
  • One case was diagnosed at autopsy after a leukemia-like presentation with extensive peripheral blood and bone marrow involvement. [27]
  • In UK population-based data for teenagers and young adults, survival from rhabdomyosarcoma did not significantly change across the studied periods. [52]
  • In the retrospective nephrostomy series, the median follow-up period for the cohort was 40 months (range 9 to 239 months). [25]
What we don't know yet12 points
  • Awareness of the association between female genital tract rhabdomyosarcoma and pathogenic germline DICER1 variants is important because detecting such mutations can enable surveillance for related conditions, but preventive screening of carriers with causative DICER1 mutations is complicated. [12][10]
  • There is an urgent need for standardized therapeutic approaches to improve patient outcomes in ovarian RMS. [1]
  • Exact categorization of sarcoma is essential for an individualized approach. [42]
  • Pseudo-Meigs’ syndrome associated with primary rhabdomyosarcoma of the ovary had never been described to date. [53]
  • The literature discusses the pathogenesis of ovarian rhabdomyosarcoma. [19]
  • Although aberrant miR-20a levels have been observed across multiple human cancers, the pooled analysis authors concluded that the efficacy of miR-20a as a prognostic biomarker remains inconsistent. [17]
  • The expression of Piwil2 in a wide variety of tumors could be a useful prognostic factor and might have diagnostic and therapeutic implications. [36]
  • The findings in one series suggest that molecular alterations other than DICER1, specifically TERT promoter and TP53 mutations, may contribute to component-specific oncogenic transformation. [9]
  • Longer follow-up is needed to assess preservation of ovarian, reproductive, and sexual function after high-dose-rate intravaginal radiation therapy (IVRT) for vaginal rhabdomyosarcoma. [43]
  • The actual effect of upper urinary tract obstruction itself, as well as urinary diversion, on the function of the affected ipsilateral kidney in childhood cancer remains unknown. [25]
  • The causal relationship between pathogenic variants in adult-onset cancer predisposition genes and childhood cancer is still under investigation. [14]
  • The ovarian cortex purging model used human cancer cell lines, and it is unclear whether those cells reflect the behaviour of malignant cells that have metastasized to the ovary during natural disease progression; additionally, the functionality of ovarian tissue after ex vivo purging with Verteporfin requires further investigation in vivo. [26]
Safety & interactions6 points
  • EET therapy was associated with transitory hypotension observed in 46% of treated cases. [48]
  • High-dose melphalan-containing regimens have been reported to yield high response rates and improved survival in some nonhematologic tumors but were noted to be associated with considerable toxicity. [47]
  • Reported complications of ovarian transposition include ovarian torsion and cyst formation. [41]
  • In the reported series, nephrostomies were placed in four patients and were successfully managed without severe infections. [25]
  • In pooled case data, neoadjuvant platinum plus paclitaxel during the second and third trimesters was reported as a feasible and safe choice for managing cervical and ovarian cancer in pregnancy. [54]
Show 1 lab & early-research finding
  • In the ex vivo Verteporfin purging experiments, ovarian tissue integrity assays did not show statistically significant differences compared with control-treated tissue (P > 0.05). [26]

Sources

Every statement above is drawn from these reviewed sources. This page reports what they describe. Sources last checked June 19, 2026.

  1. Case reportPrimary ovarian rhabdomyosarcoma: A diagnostic dilemma in an uncommon tumor · 2025
  2. StudyOvarian Rhabdomyosarcoma in Children · 2025
  3. Case reportPrimary ovarian rhabdomyosarcoma with heterologous elements: a case report · 2008
  4. Case reportRhabdomyosarcoma of the ovary: ultrastructural study of a case and review of literature · 1983
  5. Case reportPrimary ovarian rhabdomyosarcoma in children · 2008
  6. Case reportPrimary ovarian rhabdomyosarcoma: a report of 13 cases · 1998
  7. Case reportImmature teratoma of the ovary with a minor rhabdomyosarcomatous component and fatal rhabdomyosarcomatous metastases: the first case in a child · 2002
  8. Case reportDICER1-mutated rhabdomyosarcoma of the ovary with teratoid features · 2023
  9. Review articleOvarian Sertoli-Leydig cell tumors with heterologous rhabdomyosarcoma: Clinicopathologic features and molecular analysis highlighting recurrent genetic alterations · 2026
  10. Review articleDICER1 Syndrome · 2019
  11. Case reportEmbryonal Rhabdomyosarcoma of the Ovary and Fallopian Tube: Rare Neoplasms Associated With Germline and Somatic DICER1 Mutations · 2020
  12. Review articleChildhood Rhabdomyosarcoma of the Female Genital Tract: Association with Pathogenic DICER1 Variation, Clinicopathological Features, and Outcomes · 2021
  13. Systematic reviewSpecial features of sarcomas developed in patients with Lynch syndrome: A systematic review · 2023
  14. Review articleAdult-Onset Cancer Predisposition Syndromes in Children and Adolescents-To Test or not to Test? · 2024
  15. Clinical trialIncreased activities of creatine kinase and lactate dehydrogenase isoenzymes in a patient with metastatic ovarian tumor · 1987
  16. Review articleA humanized anaplastic lymphoma kinase (ALK)-directed antibody-drug conjugate with pyrrolobenzodiazepine payload demonstrates efficacy in ALK-expressing cancers · 2025
  17. Meta-analysisChange of Circulating and Tissue-Based miR-20a in Human Cancers and Associated Prognostic Implication: A Systematic Review and Meta-Analysis · 2018
  18. Clinical trialSex cord-stromal tumors of the ovary and testis: their similarities and differences with consideration of selected problems · 2005
  19. Case reportOvarian Sertoli-Leydig cell tumor with rhabdomyosarcoma: an ultrastructural study · 1982
  20. GuidelinePDQ(R) Ovarian Epithelial, Fallopian, & Peritoneal Cancer Treatment — National Cancer Institute · n.d.
    Source: PDQ(R) Adult Treatment Editorial Board. PDQ Cancer Information Summaries. Bethesda, MD: National Cancer Institute. The NCI does not endorse this site or its content.
  21. Meta-analysisIntramammary metastases: comparison of mammographic and ultrasound features · 2013
  22. Clinical trialNonmammary malignancies of the breast: ultrasound, CT, and MRI · 2000
  23. Review articleThe "Other" Uterine Mesenchymal Neoplasms: Recent Developments and Emerging Entities · 2024
  24. Review articleAtypical Pelvic Tumors in Children · 2025
  25. Review articleEffect of emergent nephrostomy on long-term total and split renal function in patients with upper urinary tract obstruction due to pelvic malignant tumors · 2024
  26. Review articleEnhancing the safety of ovarian cortex autotransplantation: cancer cells are purged completely from human ovarian tissue fragments by pharmacological inhibition of YAP/TAZ oncoproteins · 2019
  27. Case reportOvarian rhabdomyosarcoma presenting as leukemia. Case report · 1983
  28. Review articlePrimary ovarian rhabdomyosarcoma associated with clear cell carcinoma of the ovary: a case report and review of the literature · 2000
  29. StudyPrimary ovarian rhabdomyosarcoma in a dog · 2012
  30. Case reportEndometriosis Malignant Transformation Review: Rhabdomyosarcoma Arising From an Endometrioma · 2019
  31. Review articleHistopathological Review of Childhood and Adolescent Cancers in Northern Ghana · 2022
  32. Review articleEpidemiological profile and clinico-pathological features of pediatric gynecological cancers at Moi Teaching & Referral Hospital, Kenya · 2022
  33. Review articleMolecular Relationship Between Ovarian Sertoli-Leydig Cell Tumors and Their Heterologous Elements: Emphasis on the Possible Prognostic Significance of TERT Pathogenic Variants · 2026
  34. Review articleHedgehog Signaling and Truncated GLI1 in Cancer · 2020
  35. Review articleNon-canonical Hedgehog Signaling Pathway in Cancer: Activation of GLI Transcription Factors Beyond Smoothened · 2019
  36. Clinical trialStem-cell protein Piwil2 is widely expressed in tumors and inhibits apoptosis through activation of Stat3/Bcl-XL pathway · 2006
  37. Clinical trialElevations of DNA topoisomerase I catalytic activity and immunoprotein in human malignancies · 1996
  38. Review articleBiological Function and Clinical Value of VPS13A in Pan-Cancer Based on Bioinformatics Analysis · 2021
  39. Review articleOptimizing Jasplakinolide delivery in rhabdomyosarcoma cells using pulsed electric fields (PEFs) for enhanced therapeutic impact · 2025
  40. Review articleThe reproductive system and breast metastases - a narrative review and case series of metastases from soft tissue and bone sarcomas in girls · 2025
  41. Review articleRhabdomyosarcoma Requiring Ovarian Transposition Release for Recurrent Severe Ovulation Pain Following Laparoscopic Ovarian Transposition: A Case Report · 2025
  42. Case reportPrimary rhabdomyosarcoma in ovary - Pathologist clinches it all · 2018
  43. Review articleHigh-Dose-Rate Brachytherapy for Vaginal Rhabdomyosarcoma (RMS): Lessons Learned at a Single Institution · 2025
  44. Review articleMinimally invasive and robotic-assisted approaches applied to pediatric surgical oncology · 2025
  45. Review articleEmbryonal Rhabdomyosarcoma of the Uterine Cervix: A Clinicopathologic Study of 94 Cases Emphasizing Issues in Differential Diagnosis Staging, and Prognostic Factors · 2022
  46. Case reportImmature ovarian teratoma in a postmenopausal woman · 1987
  47. Clinical trialThe systemic administration of intravenous melphalan · 1988
  48. Clinical trialClinical experience of EET therapy for 75 advanced cancer patients · 1998
  49. Clinical trialStem cell transplantation; Iranian experience · 2009
  50. Review articleAssessing the feasibility and validity of the Toronto Childhood Cancer Stage Guidelines: a population-based registry study · 2018
  51. Review articleSurvival of Adolescents and Young Adults with Prevalent Poor-Prognosis Metastatic Cancers: A Population-Based Study of Contemporary Patterns and Their Implications · 2022
  52. Clinical trialTrends in survival for teenagers and young adults with cancer in the UK 1992-2006 · 2015
  53. Case reportPrimary ovarian rhabdomyosarcoma coexisting with Pseudo-Meigs' syndrome in a young patient: a case report and brief literature review · 2020
  54. Review articleThe fetal outcomes after neoadjuvant platinum and paclitaxel chemotherapy during pregnancy: analysis of three cases and review of the literature · 2022

What supports this page

The kinds of sources behind this page, strongest at the top. Faint rungs show what is not here yet.

Guideline
1
Meta-analysis
2
Systematic review
1
Randomized trial
0
Clinical trial
10
Observational
1
Case report
69
Review
136
Preclinical
0
Other
2

Living document — last change June 19, 2026: Cancer page updated. 3 recent updates logged.

Compounds compared by evidence

PubMed

How to read this: Ranked by the strength and volume of the evidence — NOT by how well a treatment works. A higher rank means a compound has been studied more, or in stronger study designs (e.g. randomized trials over lab studies), not that it produces better outcomes. The effect column shows the largest pooled figure reported, not a head-to-head comparison.

#CompoundEvidence strengthStudiesLargest pooled effect
1Cyclophosphamide OtherInsufficient evidence2—
2Vincristine OtherInsufficient evidence2—
3Actinomycin D OtherInsufficient evidence1—
4Doxorubicin OtherInsufficient evidence1—

Ranked across 4 compounds with published studies for this cancer. Updated 6/16/2026.

Medicines & supplements studied for Ovarian Rhabdomyosarcoma

PubMedFDAClinicalTrials.gov

Every drug, supplement, and other agent the published studies cover for Ovarian Rhabdomyosarcoma, ranked by how strong the evidence is — what studies report, not a recommendation. Tap any to see its full profile.

Medicines · 4

CyclophosphamideInsufficient evidenceReported positive

No primary experimental studies yet.

Most authoritative study: Primary ovarian rhabdomyosarcoma: A diagnostic dilemma in an uncommon tumor

No human studies yet · No numeric effect sizes reported · All studies are small (n < 30).
Other2 studiesFull profile →
VincristineInsufficient evidenceReported positive

No primary experimental studies yet.

Most authoritative study: Primary ovarian rhabdomyosarcoma: A diagnostic dilemma in an uncommon tumor

No human studies yet · No numeric effect sizes reported · All studies are small (n < 30).
Other2 studiesFull profile →
Actinomycin DInsufficient evidenceInconclusive

No primary experimental studies yet.

Most authoritative study: Primary ovarian rhabdomyosarcoma: A diagnostic dilemma in an uncommon tumor

No human studies yet · No numeric effect sizes reported · Based on a single study.
Other1 studyFull profile →
DoxorubicinInsufficient evidenceReported positive

No primary experimental studies yet.

Most authoritative study: Primary ovarian rhabdomyosarcoma in children

No human studies yet · No numeric effect sizes reported · Based on a single study.
Other1 studyFull profile →

What recent studies report in Ovarian Rhabdomyosarcoma

These are reviewed studies whose abstracts concern Ovarian Rhabdomyosarcoma. Each describes only what that study reported. This is not a claim by OncoForge that any compound helps or harms Ovarian Rhabdomyosarcoma. Most are early lab, animal, or small human studies, and findings often conflict.

6 studies2 human1 animal⚠ Conflicting evidenceMechanism (1)Trial (1)

Tracking 6 published studies of Ovarian Rhabdomyosarcoma: 2 in humans, 1 in animals, 3 reviews/other.

Reported direction across studies: 1 positive, 2 mixed, 3 inconclusive.

Findings conflict — both supportive and negative/mixed results exist (see below). Human evidence is limited.

These counts summarize what the studies reported; they are not a measure of whether anything works for Ovarian Rhabdomyosarcoma.

Compounds with studies mentioning Ovarian Rhabdomyosarcoma

Cyclophosphamide (2)Vincristine (2)Actinomycin d (1)Doxorubicin (1)
Case reportInconclusiveLimited evidenceTier 3 · early humann = 1

Primary ovarian rhabdomyosarcoma: A diagnostic dilemma in an uncommon tumor

Journal of cancer research and therapeutics · Oct 2025 · case report

This case report describes a 17-year-old girl with a very rare ovarian rhabdomyosarcoma. The tumor was diagnosed by imaging, pathology, and immunohistochemistry, and it came back within 3 months after surgery. The authors report that she then received VAC chemotherapy (vincristine, actinomycin D, and cyclophosphamide), and they emphasize the need for earlier diagnosis and more standardized treatment approaches.

Studied with: vincristine, actinomycin D, cyclophosphamide.

Key findings
  • Imaging showed a large solid-cystic pelvic mass.
  • Histopathology and immunohistochemical markers (desmin, myogenin, WT1) confirmed ovarian rhabdomyosarcoma.
  • Recurrence occurred within 3 months after surgical resection.
  • VAC chemotherapy was given after early relapse.
Limitations: Single-patient case report.; No control group.; No quantitative treatment outcome data reported.; Cannot determine effectiveness of VAC from this report alone.; Focus is diagnostic and descriptive rather than evaluative..

Describes a rare ovarian cancer case and subsequent chemotherapy, but does not evaluate a compound's anticancer effect in a comparative way.

AI summary of the abstract, human-reviewed · Jun 2026. Describes what this study reported, not medical advice. View on PubMed

Human · observationalMixed resultsLimited evidenceTier 3 · early humann = 6

Ovarian Rhabdomyosarcoma in Children

Cureus · Jun 2025 · retrospective analysis

ovarian rhabdomyosarcoma

This retrospective case series reviewed six pediatric patients with ovarian rhabdomyosarcoma treated over a 25-year period at a national cancer center in Peru. Most tumors had embryonal histology, four patients presented with distant metastases (stage/group IV), none tested positive for fusion genes, and all received chemotherapy plus surgery with efforts to preserve fertility when appropriate. Three patients received abdominopelvic radiotherapy (2,400 cGy in 16 sessions); one low-risk patient survived up to 94 months and the authors report multimodal treatment produced survival exceeding 87 months in some cases.

Reported effects: number of patients 6, n=6 · abdominopelvic radiotherapy total dose 2400, n=3 · +3 more

Key findings
  • Six female patients aged between five months and 13 years were included.
  • Four patients were classified as clinical stage/group IV due to distant metastases; two were low-risk.
  • The majority had embryonal histology and none tested positive for fusion genes.
  • All patients underwent chemotherapy and surgery; surgical approaches emphasized fertility preservation and varied by disease extent.
  • Three patients received intensity-modulated abdominopelvic radiotherapy at a total dose of 2,400 cGy delivered in 16 sessions for peritoneal sarcomatosis (two for persistent disease after chemotherapy and surgery, one for high-risk histology).
  • One low-risk patient achieved a survival of up to 94 months.
  • Authors conclude ovarian rhabdomyosarcoma is rare, presents with nonspecific clinical/radiologic features, immunohistochemistry is essential for diagnosis, and multimodal treatment can achieve long-term survival, including in metastatic cases.
Limitations: Very small sample size (n=6).; Retrospective, single-center design increases risk of selection and reporting bias.; Heterogeneous disease stages and treatments among patients limit generalizability.; Limited outcome detail reported for individual patients (only one specific long-term survival reported).; No control or comparison group and no standardized prospective follow-up reported.; Molecular/genetic testing beyond fusion-gene negativity is not described in the cohort..

AI summary of the abstract, human-reviewed · Jun 2026. Describes what this study reported, not medical advice. View on PubMed · Full text

Case reportMechanismInconclusiveLimited evidenceTier 3 · early humann = 1

DICER1-mutated rhabdomyosarcoma of the ovary with teratoid features

Genes, chromosomes & cancer · Dec 2023 · case report

ovarian rhabdomyosarcomaembryonal rhabdomyosarcomabotryoid rhabdomyosarcoma

This is a case report of a 14-year-old girl with an ovarian DICER1-mutated rhabdomyosarcoma displaying teratoid features. Pathology showed a predominant high-grade spindle cell rhabdomyosarcoma with botryoid features, cartilage islets, teratoid glands and neuroectodermal rosettes, and sequencing identified two DICER1 mutations. The sarcomatous component had a complex genetic profile while the teratoid component was diploid and neither showed 12p abnormality. The authors state molecular testing is necessary to confirm diagnosis and further studies are needed to clarify nosology and therapy.

Key findings
  • Reported a case of DICER1-mutated rhabdomyosarcoma of the ovary in a 14 years old girl with interspersed mature teratoid glands, neuroectodermal rosettes and immature blastematous-like tubes.
  • Morphology: sarcomatous component predominated and corresponded to a high grade spindle cell rhabdomyosarcoma with botryoid features; islets of cartilage were present and the sarcomatous proliferation encased the teratoid glands forming cambium layer-like arrangements.
  • Immunohistochemistry: sarcoma cells were Myogenin and MYOD1 positive; neuroectodermal rosettes expressed SALL4 along with cytokeratins and EMA and were negative for Inhibin; immature blastematous-like tubes were negative for SALL4 and Inhibin.
  • Molecular findings: whole RNA- and targeted DNA-sequencing revealed two DICER1 mutations: exon 26: c.5113G>A: p.(Glu1705Lys) and exon 12: c.1642C>T: p.(Gln548X).
  • Genomic profile: the sarcomatous component harbored a complex genetic profile while the teratoid component was diploid; none displayed abnormality of 12p.
  • Authors conclude that molecular testing is necessary to confirm diagnosis of DICER1-mutated sarcomas and that further studies are required to clarify classification and therapeutic strategies.
Limitations: Single-patient case report (n=1), limiting generalizability.; Descriptive pathology/genetics only — no treatment, clinical course, or outcomes reported.; No functional studies to demonstrate the biological impact of the reported DICER1 mutations.; Short or unspecified follow-up and absence of broader cohort or control comparisons..

AI summary of the abstract, human-reviewed · Jun 2026. Describes what this study reported, not medical advice. View on PubMed

Animal studyInconclusiveLimited evidenceTier 2 · animaln = 1

Primary ovarian rhabdomyosarcoma in a dog

Journal of comparative pathology · Nov 2012 · case report

ovarian rhabdomyosarcomaalveolar rhabdomyosarcoma

This report describes a single 10-year-old female English pointer with an ovarian tumor and multiple abdominal nodules. Necropsy, histology and immunohistochemistry led to a diagnosis of primary ovarian alveolar rhabdomyosarcoma, with tumor cells positive for myosin, desmin, vimentin and CD10 and negative for cytokeratin, PLAP, inhibin-alpha and SMA. The tumor showed marked pleomorphism and vascular invasion and had metastasized across abdominal organs.

Key findings
  • A 10-year-old female English pointer presented with an ovarian tumour and abdominal metastases; ultrasonography showed several nodules of 1-5 cm diameter within the abdominal cavity.
  • Fine needle aspiration cytology of the nodules suggested a malignant mesenchymal tumour.
  • On necropsy the right ovary and its capsule were enlarged and white-red, friable nodular masses were distributed over the pancreas, liver, omentum, mesentery and serosae of the small and large intestines.
  • Microscopical evaluation revealed neoplastic cells with a high degree of pleomorphism and vascular invasion.
  • Immunohistochemically, the neoplastic cells expressed myosin, desmin, vimentin and CD10, but were negative for cytokeratin, placental alkaline phosphatase, inhibin-alpha and smooth muscle actin.
  • Based on these findings a diagnosis of primary ovarian alveolar rhabdomyosarcoma was made.
Limitations: Single-animal case report (n=1) limits generalizability.; Descriptive post-mortem report with no treatment or clinical outcome data beyond necropsy.; No molecular or genetic testing reported to further characterize the tumor.; Findings are limited to one dog and may not apply to other breeds or species..

AI summary of the abstract, human-reviewed · Jun 2026. Describes what this study reported, not medical advice. View on PubMed

Case reportTrialReported positiveLimited evidenceTier 3 · early humann = 2

Primary ovarian rhabdomyosarcoma in children

Pediatric surgery international · May 2008 · case reports

CyclophosphamideVincristineDoxorubicinovarian rhabdomyosarcomaalveolar rhabdomyosarcomaembryonal rhabdomyosarcoma

This report describes two children with very rare primary ovarian rhabdomyosarcoma. After complete surgical removal, both received chemotherapy with vincristine, doxorubicin, and cyclophosphamide and had a good response. Both patients were alive 8 and 9 months after surgery.

Key findings
  • Two pediatric cases of primary ovarian rhabdomyosarcoma were described.
  • Both patients underwent complete resection of the primary tumor.
  • Both received vincristine, doxorubicin, and cyclophosphamide chemotherapy.
  • The abstract reports a good response to therapy and survival at 8 and 9 months post-operatively.
Limitations: Case report with only 2 patients.; No control group.; Short follow-up.; Cannot separate the effect of surgery from chemotherapy.; No dosing details reported..

Describes management of a rare ovarian cancer in children, including chemotherapy, but does not isolate the effect of any single compound.

AI summary of the abstract, human-reviewed · Jun 2026. Describes what this study reported, not medical advice. View on PubMed

Human · observationalMixed resultsLimited evidenceTier 3 · early humann = 13

Primary ovarian rhabdomyosarcoma: a report of 13 cases

International journal of gynecological pathology : official journal of the International Society of Gynecological Pathologists · Apr 1998 · case series with literature review

ovarian rhabdomyosarcoma

This paper reports 13 cases of primary ovarian rhabdomyosarcoma in females aged 7–79 years (mean 37), describing clinical presentation, tumor size, laterality, stage, and histology. Histologically, 11 tumors were embryonal and 2 were alveolar. Follow-up was available for 11 patients: 7 died of disease between 10 days and 26 months after surgery, and 4 were alive 2 to 9 months postoperatively. The authors also reviewed an additional 10 cases from the English-language literature and discuss the differential diagnosis.

Reported effects: n 13, n=13 · mean_age 37, n=13 · +23 more

Key findings
  • Primary ovarian rhabdomyosarcomas were found in 13 patients aged 7 to 79 (mean 37) years who had reported abdominal pain and swelling.
  • Six tumors involved the right ovary, 3 involved the left, 1 involved both, and the laterality was unknown in 3 cases.
  • Four tumors were stage I, 2 were stage II, 4 were stage III, and 2 were stage IV; the stage of 1 tumor is not known.
  • The tumors ranged from 10 to 19.5 (average 16) cm in diameter.
  • Microscopically, 11 tumors were embryonal and 2 were alveolar rhabdomyosarcomas.
  • Follow-up information was available for 11 patients: 7 died of disease 10 days to 26 months postoperatively; 2 of these had stage II, 3 had stage III, and 2 had stage IV disease.
  • Four patients were alive 2 to 9 months postoperatively; 3 had stage I and 1 had stage III disease.
  • The 13 cases were reviewed alongside an additional 10 cases from the English-language literature, and the differential diagnosis is discussed.
Limitations: Small case series (n=13) limits generalizability.; Follow-up data were incomplete (available for 11 patients) and follow-up duration was short (maximum 26 months).; Retrospective, descriptive design with no control group or standardized treatment data reported in the abstract.; Prognostic factors and treatment details are not provided in the abstract, limiting interpretation of outcomes..

AI summary of the abstract, human-reviewed · Jun 2026. Describes what this study reported, not medical advice. View on PubMed

Browse all studies mentioning Ovarian Rhabdomyosarcoma →

Where the evidence is

What has been studied, and how strong it is, by topic. A dashed cell means no studies were found for that combination — a gap, not evidence of no effect. Open a row to see its studies.

CompoundHuman evidenceMechanismSafetyTrial
Cyclophosphamide—1—1
Vincristine—1—1
Actinomycin D—1——
Doxorubicin———1

Study mix

6 published studies by what they were done in. Lab and animal findings often do not carry over to people.

2 Human1 Animal3 Review/other
Reported directionReported positive1Mixed results2Inconclusive3

Compounds with reported-positive results in Ovarian Rhabdomyosarcoma

Where at least one study reported a positive result, shown with the full picture, not just the wins. Positive results are more likely to be published, and most of these are early lab or animal studies that may not translate to people. This reports what studies found, not what works.

Preclinical only: lab / animal (3)
Limitations: Case report with only 2 patients.; No control group.; Short follow-up.; Cannot separate the effect of surgery from chemotherapy.; No dosing details reported..
Cited positive studies (1)
Vincristine1 positive
Limitations: Case report with only 2 patients.; No control group.; Short follow-up.; Cannot separate the effect of surgery from chemotherapy.; No dosing details reported..
Cited positive studies (1)
Doxorubicin1 positive
Limitations: Case report with only 2 patients.; No control group.; Short follow-up.; Cannot separate the effect of surgery from chemotherapy.; No dosing details reported..
Cited positive studies (1)

Evidence at a glance: compounds studied in Ovarian Rhabdomyosarcoma

A deterministic grade of what published studies report for each: strength of evidence, the reported direction, and the largest credible effect, strongest-evidence first. This summarizes findings; it is not a claim that anything works.

CyclophosphamideInsufficient evidenceReported positive

No primary experimental studies yet.

Most authoritative study: Primary ovarian rhabdomyosarcoma: A diagnostic dilemma in an uncommon tumor

No human studies yet · No numeric effect sizes reported · All studies are small (n < 30).
VincristineInsufficient evidenceReported positive

No primary experimental studies yet.

Most authoritative study: Primary ovarian rhabdomyosarcoma: A diagnostic dilemma in an uncommon tumor

No human studies yet · No numeric effect sizes reported · All studies are small (n < 30).
Actinomycin DInsufficient evidenceInconclusive

No primary experimental studies yet.

Most authoritative study: Primary ovarian rhabdomyosarcoma: A diagnostic dilemma in an uncommon tumor

No human studies yet · No numeric effect sizes reported · Based on a single study.
DoxorubicinInsufficient evidenceReported positive

No primary experimental studies yet.

Most authoritative study: Primary ovarian rhabdomyosarcoma in children

No human studies yet · No numeric effect sizes reported · Based on a single study.

Clinical trials in Ovarian Rhabdomyosarcoma

1 ongoing · 38 completed · tracked from ClinicalTrials.gov. Recruiting is not the same as proven, and completed is not the same as positive — read the results. Not a recommendation.

Completed
8 stopped (terminated / withdrawn / suspended)

Search all trials on ClinicalTrials.gov →

Getting care & support

Nonprofit / Gov

Practical, vetted help for Ovarian Rhabdomyosarcoma — advocacy, paying for treatment, second opinions, and caregivers.

If you’re struggling emotionally, you don’t have to wait.

Advocacy & community

No dedicated organization for this specific cancer is curated yet — these general organizations can help in the meantime.

Financial help

  • PAN Foundation ↗ — Copay assistance funds by diagnosis (funds open and close as money allows). · status changes often — check the fund’s site
  • HealthWell Foundation ↗ — Copay and premium assistance funds by disease. · status changes often — check the fund’s site
  • CancerCare — financial assistance ↗ — Limited grants plus free financial counseling. · status changes often — check the fund’s site
  • Family Reach ↗ — Help with everyday living costs (rent, transport, food) during treatment. · status changes often — check the fund’s site
  • NeedyMeds ↗ — Searchable directory of drug patient-assistance and discount programs. · status changes often — check the fund’s site
What you’ll typically need to apply
  • Your diagnosis and, if you have it, the specific drug/treatment name (from your care team).
  • Insurance details — your member ID card, or a note that you're uninsured (some funds require active insurance, some don't).
  • Proof of income and household size (recent pay stubs, a tax return, or a benefits letter) — most funds are income-based.
  • Your prescriber's contact information; some programs need the clinic to submit part of the application.
  • Apply early and re-check: funds open and close as money is available, so a closed fund may reopen.

General guidance — each program sets its own eligibility. Confirm requirements on the program’s site.

Help paying for the medicines on this page

Second opinions

Caregiver support

We list only non-profit and government resources — never product sellers — and take no affiliate fees. If a link is broken or a resource doesn't meet that bar, tell us.

Heading to an appointment? Get a printable one-page summary — studied compounds, open trials, interactions, and questions to ask.
Bring this to your appointment →

Biomarkers — what to test for, and what it could unlock

PubMedFDA

A positive result may make you eligible for the linked options — it doesn’t mean a treatment will work, and not every test is standard everywhere. Confirm testing and results with your care team.

Many of these can be checked at once from a single tumor (or blood) sample — ask your team whether a comprehensive multi-gene (NGS) panel is right for you, rather than one test at a time.

Cytokeratins (AE1/AE3), EMA, PAX8, WT1differential diagnosis (Müllerian carcinoma vs pure RMS); carcinosarcoma component ID · negativePrognostic / other

Pre-screen recruiting trials for Cytokeratins (AE1/AE3), EMA, PAX8, WT1 →

Hereditary cancer gene — a germline (inherited) result can affect blood relatives. Consider genetic counseling ↗; your team can advise whether germline testing is right for you.

Desmin / myogenin / MyoD1myogenic differentiation · positivePrognostic / other

How it’s tested: MYOD1 hotspot (p.L122R)

Pre-screen recruiting trials for Desmin / myogenin / MyoD1 →

DICER1microRNA processing · occasionalPrognostic / other

How it’s tested: NGS panel

Pre-screen recruiting trials for DICER1 →

Hereditary cancer gene — a germline (inherited) result can affect blood relatives. Consider genetic counseling ↗; your team can advise whether germline testing is right for you.

FGFR4 (overexpression / activating mutations N535D/K, V550E/L)growth signaling receptor · occasionalPrognostic / other

How it’s tested: FGFR4 mutation/expression

Pre-screen recruiting trials for FGFR4 (overexpression / activating mutations N535D/K, V550E/L) →

Ki-67proliferation index · commonPrognostic / other

Pre-screen recruiting trials for Ki-67 →

MMR/MSIDNA repair · rarePrognostic / other

How it’s tested: MMR/MSI

Pre-screen recruiting trials for MMR/MSI →

Hereditary cancer gene — a germline (inherited) result can affect blood relatives. Consider genetic counseling ↗; your team can advise whether germline testing is right for you.

MYOD1 p.L122Rmyogenic TF hotspot mutation (spindle/sclerosing RMS) · rarePrognostic / other

How it’s tested: MYOD1 hotspot (p.L122R)

Pre-screen recruiting trials for MYOD1 p.L122R →

NTRK1/2/3 fusions (screen)tumor-agnostic kinase fusion · rarePrognostic / other

Pre-screen recruiting trials for NTRK1/2/3 fusions (screen) →

PAX3–FOXO1 / PAX7–FOXO1fusion oncoprotein · occasionalPrognostic / other

How it’s tested: Fusion testing (PAX3/7–FOXO1)

Pre-screen recruiting trials for PAX3–FOXO1 / PAX7–FOXO1 →

PAX5 (pitfall)B-cell TF; rare aberrant expression · rarePrognostic / other

Pre-screen recruiting trials for PAX5 (pitfall) →

PD-L1immune checkpoint · variablePrognostic / other

How it’s tested: MMR/MSI

Pre-screen recruiting trials for PD-L1 →

SMARCB1/INI1SWI/SNF complex tumor suppressor · positivePrognostic / other

Pre-screen recruiting trials for SMARCB1/INI1 →

TFCP2-related fusions (EWSR1::TFCP2 / FUS::TFCP2)fusion-driven RMS subset with ALK upregulation · rarePrognostic / other

How it’s tested: Fusion testing (PAX3/7–FOXO1)

Pre-screen recruiting trials for TFCP2-related fusions (EWSR1::TFCP2 / FUS::TFCP2) →

TMB (tumor mutational burden)immunotherapy biomarker · variablePrognostic / other

How it’s tested: MMR/MSI

Pre-screen recruiting trials for TMB (tumor mutational burden) →

Marker → medicine links are derived from this page’s published claims for Ovarian Rhabdomyosarcoma; resistance and companion-diagnostic notes appear only where an established, sourced entry exists.

Go deeper

Questions to ask your oncologist

  • Which RMS subtype do I have (embryonal, alveolar, spindle/sclerosing, pleomorphic) and how does it change treatment?
  • Is fertility-sparing surgery possible and safe in my case?
  • Which chemo backbone is recommended for my age and subtype?
  • Do I need radiation for margin-positive or nodal disease?
  • Are there trials matched to my tumor’s drivers (e.g., PAX–FOXO1, PI3K/AKT/mTOR, MSI)?
  • Can we send tissue for RNA fusion panel and DNA NGS now, and again at progression to capture targets (e.g., TFCP2, FGFR4, ALK)?
  • Do we have enough banked tumor (FFPE + fresh-frozen) and a recent biopsy to qualify for molecular trials?
  • What is the plan if there’s inadequate response after 2–3 cycles—what are our pre-agreed switch criteria?
  • Am I a candidate for neoadjuvant therapy to improve the chance of an R0 resection?
  • If disease is limited, could metastasectomy, ablation, or SBRT consolidate a systemic response?
  • Would anthracycline cardioprotection (dexrazoxane) help preserve intensity without compromising efficacy?
  • Should we involve a sarcoma specialty center or enroll via a national consortium (SARC, EORTC, NCI)?
  • Are there basket trials for my biomarkers (FGFR4, ALK, NTRK, BET/BRD4, YAP/TEAD, MDM2)?
  • Could we combine targeted agents with RT or chemo on a protocol to blunt resistance feedback (e.g., PI3K/mTOR + MEK)?
  • What’s our VTE prevention plan and symptom-triggered pathway for urgent evaluation?
  • How will we monitor for cardiac, renal, and neurotoxicity, and what dose-modification rules protect outcomes?
  • Do I qualify for compassionate use / expanded access if a matched trial isn’t available?
  • Is there a role for ctDNA as an adjunct to imaging for trend-tracking in my case?

Find a trial

Search active and completed trials at these registries:

Citations

  1. WHO Classification of Tumors: Soft Tissue and Bone (RMS chapters)

    WHO/IARC

    • Subtypes and diagnostic criteria
    • Pathology markers and fusions

  2. PDQ® Childhood Rhabdomyosarcoma Treatment

    NCI

    • VAC-based regimens; RMS biology and staging concepts

  3. ESMO–EURACAN–GENTURIS Clinical Practice Guidelines: Soft Tissue and Visceral Sarcomas

    ESMO

    • Adult STS backbones (doxorubicin/ifosfamide; gemcitabine/docetaxel)
    • Radiation/local control principles

  4. Gynecologic tract rhabdomyosarcoma: clinicopathologic updates

    Peer-reviewed reviews/case series

    • Rarity and ovary-specific presentations
    • Patterns of spread and management nuances

  5. Ovarian Rhabdomyosarcoma in Children - PMC

    PubMed Central / Authors: M.G. Pérez et al.

    • Subtypes (embryonal most common, alveolar, pleomorphic, spindle cell/sclerosing)
    • Biomarkers (desmin, MyoD1, myogenin positive; negative for pancytokeratin, etc.)
    • Pathology (rare, from stromal fibroblasts/endometriotic stroma)
    • Treatment (surgery, chemotherapy like VAC, selective radiotherapy)
    • Prognosis (aggressive, multimodal improves outcomes)
    • Overview and rarity

  6. Molecular diagnostics in the management of rhabdomyosarcoma

    PubMed Central / Authors: M. Arnold et al.

    • Biomarkers (MYOD1 mutations, PAX3/7-FOXO1 fusions, RAS pathway mutations, NCOA2/VGLL2 fusions)
    • Subtypes and fusion-driven biology

  7. Embryonal rhabdomyosarcoma of the uterine corpus: a clinicopathologic study

    ScienceDirect / Authors: J.A. Bennett et al.

    • Biomarkers (desmin positive, myogenin and MyoD1 often focal positive)

  8. Myogenin is a Specific Marker for Rhabdomyosarcoma: An Immunohistochemical Study

    ResearchGate / Authors: G. Kumar et al.

    • Biomarkers (myogenin specific for RMS, desmin, MYOD1 define RMS)

  9. Rhabdomyosarcoma - BINASSS

    BINASSS / Authors: Various (review)

    • Biomarkers (desmin, myogenin, MyoD1 positive; spindle cell RMS fusions)

  10. Embryonal Rhabdomyosarcoma of the Ovary and Fallopian Tube: Rare Neoplasms Associated With Germline and Somatic DICER1 Mutations

    ResearchGate / Authors: W.G. McCluggage et al.

    • Biomarkers (DICER1 germline/somatic mutations in gynecologic RMS; some cases negative for desmin/myogenin/MyoD1 but atypical)

  11. Biological Role and Clinical Implications of MYOD1 L122R Mutation in Rhabdomyosarcoma

    MDPI / Authors: I. Szymczak et al.

    • Biomarkers (MYOD1 p.L122R mutation in spindle/sclerosing RMS, aggressive subset)

  12. Improving Individualized Rhabdomyosarcoma Prognosis Prediction: A Multimodal Machine Learning Approach

    medRxiv / Authors: L. Elouan et al.

    • Biomarkers (PAX3/PAX7-FOXO1 fusions as key prognostic markers)

  13. The Emerging Role and Clinical Significance of PI3K-Akt-mTOR in Rhabdomyosarcoma

    PubMed Central / Authors: Various (2025 Review)

    • Pathways (PI3K-Akt-mTOR hyperactivation, proliferation, survival, resistance)
    • Resistance (therapy pressure and network redundancy)

  14. Molecular Targets in Alveolar Rhabdomyosarcoma

    PubMed Central / Authors: Various

    • Pathways (PAX-FOXO1 transcriptional repression, interconnected mechanisms)

  15. Molecular and Cellular Biology of Rhabdomyosarcoma

    Wiley Online Library / Authors: Various (2015, updated references)

    • Pathways (FGF signaling, IGF1-R-PI3K-mTOR, ERK/MAPK)
    • Resistance (apoptosis prevention, cross-activation)

  16. Signaling Pathways that Overactivate Metabolism in Cancer

    JRPMS / Authors: Various

    • Pathways (PI3K/AKT/mTOR reprogramming glucose metabolism, tumor progression)

  17. Signaling Pathways in Rhabdomyosarcoma Invasion and Metastasis

    ResearchGate / Authors: Various

    • Pathways (PAX-FOXO1, IGF/RAS/MEK/ERK, PI3K/AKT/mTOR, cMET, FGFR4)
    • Resistance (parallel activation, RTK upregulation)

  18. Targeting the Hedgehog Pathway in Rhabdomyosarcoma

    MDPI / Authors: Various

    • Pathways (Hedgehog/GLI oncogenic role, stemness, proliferation)
    • Resistance (pathway bypass, limited monotherapy)

  19. Redox Biology in Rhabdomyosarcoma

    ScienceOpen / Authors: Various (2019)

    • Pathways (IGF1R, FGFR, EGFR leading to mTOR via PI3K/AKT)
    • Angiogenesis (VEGF involvement)

  20. Targeting Hedgehog and PI3K/AKT/mTOR in RMS

    Semantic Scholar / Authors: Geyer et al. (2018)

    • Pathways (PI3K/AKT/mTOR importance, regulation of HH signaling)
    • Resistance (feedback loops)

  21. PI3K/AKT/mTOR Pathway in Angiogenesis

    Frontiers / Authors: Various

    • Pathways (PI3K role in angiogenesis, normal/cancer tissues)

  22. Molecular Drivers of PAX3/7-FOXO1 Tumorigenesis

    BioMed Central / Authors: Various (2012)

    • Pathways (PAX-FOXO1 cooperating genes, target genes for tumorigenesis)

  23. Current Evidence and Directions for Intermittent Fasting During Chemotherapy

    PubMed Central / Authors: Various

    • Adjuncts (Fasting/FMD as adjunct to chemotherapy, safety, effects on nutritional status)

  24. Review of Under-Recognized Adjunctive Therapies for Cancer

    PubMed Central / Authors: Various

    • Adjuncts (Melatonin for circadian reset, sleep improvement in cancer)

  25. Review of Fasting-Mimicking Diets in Cancer Treatment

    News-Medical / Authors: Various (2024)

    • Adjuncts (Cyclic fasting/FMD role in cancer therapy, enhancing efficacy)

  26. Short-Term Fasting Synergizes with Solid Cancer Therapy

    MDPI / Authors: Various (2022)

    • Adjuncts (Fasting diets boosting antitumor immunity, synergizing with therapy)

  27. Fasting-Mimicking Diet in Patients Undergoing Active Cancer Treatment

    ClinicalTrials.gov / Authors: Various

    • Adjuncts (Pilot trial on 5-day FMD feasibility/safety during cancer treatment)

  28. Fasting Mimicking Diet May Be Safe, Effective as Adjunct to Chemo

    Cancer Network / Authors: Various (2020)

    • Adjuncts (FMD safe/effective adjunct to chemo, general oncology)

  29. Fasting and Fasting Mimicking Diets in Cancer Prevention and Therapy

    ScienceDirect / Authors: Various

    • Adjuncts (Fasting/FMD enhancing chemo, hormone therapy, immunotherapy)

  30. Fasting as Cancer Treatment: Myth or Breakthrough in Oncology

    Cureus / Authors: Various (2025)

    • Adjuncts (Fasting reducing tumor growth, enhancing chemo sensitivity)

  31. Immunomodulation after a Fasting Mimicking Diet Analyzed

    ASCO / Authors: Various

    • Adjuncts (FMD promoting CD8+ T-cell infiltration, decreasing Tregs)

  32. Therapeutic Fasting in Reducing Chemotherapy Side Effects

    MDPI / Authors: Various (2023)

    • Adjuncts (Fasting as adjunct to chemo, effects on side effects)

  33. Emergency Care for Ovarian Cancer: When to Call Your Doctor

    Not These Ovaries / Authors: Various (2025)

    • Red flags/Emergencies (Sudden chest pain, shortness of breath, fever during chemo, abdominal pain)
    • When to call (Persistent symptoms, dehydration)

  34. Ovarian Cancer Red Flags: Help Prevent Delayed Diagnosis

    PubMed / Authors: Various (2024)

    • Red flags (Unexplained abdominal lump, bloating, sterile urine samples)
    • When to call (Recurrent urinary symptoms, new IBS in >50s)

  35. Mode of Referral of Ovarian Cancer Patients

    PubMed Central / Authors: Various

    • Red flags (GPs/ED identifying symptoms for referral)

  36. Ovarian Cancer Red Flags: What to Know

    MDedge / Authors: Various (2024)

    • Red flags (Recurrent urinary symptoms, sterile urine)

  37. Rhabdomyosarcoma Stages and Risk Groups

    American Cancer Society / Authors: Various (2025)

    • Red flags (Metastasis assessment, bone marrow involvement)

  38. Referral to a Specialist for Symptoms of Ovarian Cancer

    Cancer Research UK / Authors: Various (2025)

    • Red flags (Unexplained abdominal lump, urgent referral)

  39. Ovarian Cancer Red Flags: Help Prevent Delayed Diagnosis

    ResearchGate / Authors: Various (2024)

    • Red flags (Symptoms warranting investigations, peritoneal signs)

  40. Ovarian Cancer Red Flags

    Target Ovarian Cancer / Authors: Various

    • Red flags (Sterile urine, new IBS >50s, abdominal girth increase)

  41. NCCN Guidelines for Patients: Ovarian Cancer

    NCCN / Authors: Various

    • Red flags/Emergencies (Abnormal blood counts, health issues signaling problems)

  42. Ovarian Cancer Staging

    OCRA / Authors: Various

    • Red flags (High-grade atypical cells, metastasis likelihood)

  43. Rhabdomyosarcoma: Current Therapy, Challenges, and Future Directions

    PubMed Central / Authors: Various

    • Complications (Surgical side effects, orbital RMS blindness)
    • Pathways/Adjuncts (General multimodal therapy)

  44. Molecular Review of Ovarian Rhabdomyosarcoma in Children

    PubMed / Authors: M.G. Pérez et al. (2025)

    • Overview (Rare, presents in young children, favorable site)

  45. Current and Future Treatment Strategies for Rhabdomyosarcoma

    Frontiers / Authors: Various

    • Adjuncts (Targeted therapies, multi-modality frontline)

  46. The Emerging Role of PI3K-Akt-mTOR in Rhabdomyosarcoma

    MDPI / Authors: Various

    • Pathways (PI3K-Akt-mTOR role, targeted therapies)

  47. Molecular Review of Rhabdomyosarcoma

    MalaCards / Authors: Various

    • Pathways (Molecular drivers in RMS)

  48. Case Report: Uterine Embryonal Rhabdomyosarcoma

    Frontiers / Authors: Various (2025)

    • Symptoms/Complications (Prolonged diagnostic process in gynecologic RMS)

  49. Signs and Symptoms of Rhabdomyosarcoma

    American Cancer Society / Authors: Various (2025)

    • Red flags (Lump/swelling, abdominal pain, dyspnea)

  50. Diagnosis and Treatment of Rhabdomyosarcomas

    Via Medica / Authors: Łomiak et al. (2023)

    • Symptoms (Head/neck, urogenital, abdominal presentations)

  51. Rhabdomyosarcoma: Symptoms, Prognosis & Treatment

    Cleveland Clinic / Authors: Various

    • Red flags (Nosebleeds, vomiting, lumps, similar to less serious conditions)

  52. Molecular Diagnostics in the Management of Rhabdomyosarcoma

    PubMed Central / Authors: M. Arnold et al.

    • Pathways (PAX3/7-FOXO1 fusions, FGFR4, MET, Hippo)
    • Resistance (Not explicitly, but network implications)

  53. Biological Role and Clinical Implications of MYOD1 L122R Mutation in Rhabdomyosarcoma

    MDPI / Authors: I. Szymczak et al.

    • Pathways (Myogenic transcriptional program, MYOD1 role in differentiation block, MYC-like activity)

  54. Childhood Rhabdomyosarcoma Treatment (PDQ®)

    National Cancer Institute / Authors: Various

    • Adjuncts (Chemotherapy regimens like VAC, radiation techniques, surgery)
    • Red flags/Emergencies (Metastatic complications, recurrence management)
    • Ovarian applicability (Genitourinary sites, conservative surgery)

  55. Evolving Classification of Rhabdomyosarcoma - PMC

    PubMed Central / Authors: Various (2023)

    • Subtypes (embryonal, alveolar, spindle/sclerosing, pleomorphic)
    • IHC (desmin+, MyoD1+, myogenin+)
    • Molecular (PAX3/7-FOXO1 fusions, MYOD1 mutations, TFCP2 fusions)

  56. Embryonal rhabdomyosarcoma - Pathology Outlines

    Pathology Outlines / Authors: Various (2025)

    • Histology (small round/spindle cells, rhabdomyoblastic differentiation)
    • IHC (desmin+, myogenin+, MyoD1+; negative cytokeratin/EMA)
    • Molecular (no FOXO1 fusion in embryonal; high Ki-67)

  57. Fusion-driven Spindle Cell Rhabdomyosarcomas of Bone and Soft Tissue

    Modern Pathology / Authors: Various (2023)

    • Molecular (TFCP2 fusions with ALK expression, desmin/MyoD1+)
    • Subtypes (spindle cell RMS)
    • IHC (ALK+, keratin+ in TFCP2 cases)

  58. Identification of FGFR4-activating Mutations in Human Rhabdomyosarcomas

    PubMed / Authors: Various (2009)

    • Molecular (FGFR4 mutations N535/V550 activating, frequent in alveolar RMS)
    • Translational (oncogene function, trial targets)

  59. Embryonal Rhabdomyosarcoma of the Uterine Corpus: Molecular Analysis Highlighting DICER1 Association

    ScienceDirect / Authors: J.A. Bennett et al. (2023)

    • Molecular (DICER1 alterations in gynecologic RMS)
    • IHC (variable myogenin/MyoD1)
    • Clinical (re-biopsy, trial triage)

FAQs

Is CA-125 useful in ovarian rhabdomyosarcoma?
  • Generally no. CA-125 tracks epithelial ovarian carcinoma; RMS lacks reliable serum markers. Monitoring relies on symptoms and imaging.
Which chemo backbones are typical?
  • Pediatric/AYA: VAC-based regimens (vincristine, actinomycin, cyclophosphamide) with risk-adapted additions. Adults: anthracycline/ifosfamide or gemcitabine/docetaxel are common STS backbones; choices individualize to histology, stage, and tolerance.
Is fertility-sparing surgery possible?
  • Occasionally in strictly unilateral, early disease with careful staging and pediatric/AYA considerations. Most adults require oophorectomy; decisions are individualized.
Does immunotherapy help?
  • RMS responsiveness to single-agent PD-1 is limited; benefit is more likely with MSI-H/dMMR (rare) or in trials using combinations.
Should we pursue genetic testing?
  • Yes—tumor NGS for fusions (e.g., PAX–FOXO1) and other alterations; consider germline testing if syndromic features (e.g., DICER1) or family history.
How often should imaging be done during treatment?
  • Commonly every 6–12 weeks during active therapy, faster if symptoms change or trial protocol dictates. Use the same modalities over time to compare response consistently.
Is ctDNA useful for RMS follow-up?
  • Sensitivity is limited in sarcomas; bespoke assays can help in select cases but imaging remains the primary tool. Consider ctDNA as adjunctive, not decisive.
When should we re-biopsy?
  • At progression or when biology may change management—e.g., to confirm RMS component in carcinosarcoma recurrence, to look for targetable fusions/mutations, or to enroll on a molecular trial.
What trial keywords should we search for adults with RMS?
  • Adult rhabdomyosarcoma; fusion-positive PAX3/7–FOXO1; TFCP2 fusion; FGFR4; ALK; YAP/TEAD; BET/BRD4; MDM2; sarcoma basket; immunotherapy combinations.
When do we change therapy for lack of response?
  • If clear radiologic progression, symptomatic worsening, or failure to achieve disease control after an adequate trial (usually 2–3 cycles) and adherence confirmed.
Is there a role for radiation?
  • Yes—local control (post-op margins, unresectable primaries), palliation of painful bone mets, and control of oligoprogression. Coordinate fields with systemic therapy timing.
Can local therapies help liver or bone lesions?
  • In selected cases, ablation/embolization (liver) or stereotactic/targeted radiation (bone/spine) can add control or palliation. Consider case-by-case in tumor board.
How do we manage high VTE risk?
  • Low threshold to evaluate unilateral leg swelling or chest symptoms. Prophylaxis is individualized; balance bleeding risk, surgery timing, and any anti-angiogenic agents.
What about anti-angiogenic agents (e.g., pazopanib) in adult RMS?
  • Evidence is stronger in non-RMS STS; RMS data are limited. Consider mainly in refractory settings or trials; watch wound-healing and hypertension risks.
How do we protect the heart with anthracyclines?
  • Baseline and periodic ECHO/MUGA; manage CV risk factors; consider dexrazoxane when cumulative doses rise or in higher-risk patients per guidelines.
How do we prevent chemo delays from neutropenia?
  • Primary G-CSF prophylaxis for high-risk regimens, prompt fever workups, and growth-factor support per regimen risk and prior cycle history.
What labs and symptoms should we track at home?
  • CBC/CMP per cycle; monitor fever, SOB, chest/leg pain, new neuro deficits, urine output, RUQ pain/jaundice, escalating bone/spine pain, and port changes.
Any special considerations with ureteral stents?
  • Watch for flank pain, fever, or reduced output (possible obstruction/infection). Coordinate stent exchanges around chemo cycles when feasible.
How does having a carcinosarcoma with RMS differentiation change management?
  • It confirms a biphasic tumor; systemic therapy is often guided by the dominant/aggressive component and stage. Re-sampling at progression can clarify the biology driving relapse.
Are off-label or repurposed agents worth exploring?
  • Discuss risks/benefits with the team. Prioritize agents with plausible biology and safety, and consider enrolling in trials to access combinations with oversight.
What supportive care moves the needle the most?
  • Early nutrition/hydration planning, aggressive antiemetics/mucositis care, neuropathy prevention tactics, DVT awareness, physical conditioning, and symptom-triggered rapid access.
What signals possible spinal cord compression?
  • New back pain with leg weakness/numbness, gait changes, or bladder/bowel dysfunction—this is an emergency; contact the team or go to the ED immediately.

What we don't know yet

  • No randomized trials specific to primary ovarian RMS.
  • Heterogeneous histology across small case series.
  • Limited prospective data on adult RMS regimens in ovarian site.
  • Unclear role of immunotherapy outside biomarker-selected trials.
  • Comparative effectiveness of pediatric VAC-like vs adult STS backbones in adults with ovarian RMS.
  • Optimal sequencing of surgery ↔ neoadjuvant/adjuvant therapy to maximize R0 rates.
  • Defined indications/dosing for pelvic RT and nodal fields specific to ovarian RMS.
  • Prospective value of ctDNA/MRD assays for response-adapted switches.
  • Real-world outcomes of ALK/FGFR4/BET/TEAD/MDM2 targeted approaches in RMS subsets.
  • Best criteria for oligometastatic consolidation (SBRT/ablation/metastasectomy) in potential cure pathways.
  • Fertility-sparing oncologic safety data in very early unilateral disease.