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Rhabdomyosarcoma

A plain-English summary of the published research on Rhabdomyosarcoma, reviewed and approved by our editors — not a hand-curated clinical overview.

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

Reviewed Jun 2026 · OncoForge editorial · How we review →

AI extractedhuman reviewedsources checkedretractions suppressed· last updated Jun 2026

Evidence at a glanceHuman trial / meta-analysisMixed results⚠ Studies disagree
72 published studies that name Rhabdomyosarcoma14 human studies approved & graded (trial, observational, or meta-analysis)288 human clinical studies in the Rhabdomyosarcoma corpus2265 source documents in the Rhabdomyosarcoma corpus

last checked June 19, 2026

Why this grade?

Human trial / meta-analysis — Includes human trial or meta-analysis evidence.

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.

Guideline / FDA options - context-specific
  • Vincristine
  • Pembrolizumab
Studied, not standard - investigational
  • surgery
  • regional lymph node evaluation
  • proton beam therapy
  • whole abdominopelvic radiotherapy (WAP-RT)
  • VAC (vincristine + actinomycin D + cyclophosphamide)
  • IVA
  • Pediatric chemotherapy protocols (PP; e.g., VAC, IVA)
  • radiotherapy
  • surgery and brachytherapy
  • vinorelbine + cyclophosphamide
  • celecoxib + thalidomide + etoposide + cyclophosphamide
  • systemic therapies (early phase studies)
  • systemic chemotherapy
  • chemotherapy (unspecified regimens)
  • anthracycline-based regimens
  • anthracycline + ifosfamide; vincristine + anthracycline + ifosfamide; vincristine + anthracycline + cyclophosphamide; etoposide + ifosfamide
  • chemotherapy
  • ablation techniques
  • vaginal cuff brachytherapy
  • external beam radiation therapy
  • Cisplatin
  • Topotecan
  • Actinomycin D
  • Cyclophosphamide
  • Doxorubicin
  • Etoposide
  • Genistein
  • Ifosfamide

Read the guidelines

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

Treatment map: 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.

31
Interventions
0
Standard of care
14
Tested in people
6
Lab / animal
9
Named in lit.
5
Classes
Standard of care (0) Guideline option (2) Tested in people (14) Lab / animal only (6) Named in the literature (9)

Tested in people, by trial phase: Phase II ×2 · phase not reported ×12

Clinical evidence
Preclinical evidence
Standard of care
Guideline option
Tested in people
Lab / animal only
Named in the literature
Surgery & procedures
—
—
3
—
3
Radiotherapy
—
—
2
—
2
Chemotherapy
—
—
7
2
4
Immunotherapy
—
1
—
—
—
Other
—
1
2
4
—

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 (2)
Immunotherapy
Pembrolizumab
FDA-approved for this cancer.
—Guideline option
Other
Vincristine
FDA-approved for this cancer.
—Guideline option
Investigational & adjunct compounds — detail (29)
Meta-analysis (12)
ablation techniquescelecoxib + thalidomide + etoposide + cyclophosphamide· Recurrent or later-lineIVAPediatric chemotherapy protocols (PP; e.g., VAC, IVA)proton beam therapyradiotherapysurgerysurgery and brachytherapy· Recurrent or later-linesystemic chemotherapysystemic therapies (early phase studies)· Recurrent or later-lineVAC (vincristine + actinomycin D + cyclophosphamide)vinorelbine + cyclophosphamide
Named in the literature
regional lymph node evaluationwhole abdominopelvic radiotherapy (WAP-RT)chemotherapy (unspecified regimens)· Neoadjuvant (before surgery)anthracycline-based regimens· Neoadjuvant (before surgery)anthracycline + ifosfamide; vincristine + anthracycline + ifosfamide; vincristine + anthracycline + cyclophosphamide; etoposide + ifosfamide· Neoadjuvant (before surgery)chemotherapy· Recurrent or later-linevaginal cuff brachytherapy· Adjuvant (after surgery)external beam radiation therapysurgery· First-line (advanced disease)

"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
Rhabdomyosarcoma is a cancer arising from primitive mesenchymal cells and is the commonest soft tissue sarcoma in children; temporal bone/middle-ear tumours in children often mimic chronic otitis media, causing delayed diagnosis and advanced disease at presentation. [1][2][3]
Survival
Pediatric rhabdomyosarcoma survival now exceeds 70%; however about one third of children relapse or have refractory disease, and patients with metastatic disease experience progressive disease or relapse in 60-70% of cases. [4][5][6][7]
Standard treatment
Surgery is the cornerstone of pediatric rhabdomyosarcoma management and is used within multimodal approaches that include radiotherapy and chemotherapy; for temporal bone rhabdomyosarcoma in children, chemotherapy combined with radiotherapy is identified as the standard approach. [8][2][9][3]
Key test
PAX/FOXO1 fusion (FOXO1 fusion) status is a critical biomarker used for risk stratification at diagnosis. [10][11]
Biggest challenge
Late presentation and a high rate of relapse/refractory disease are the main clinical problems — outcomes after relapse are generally poor. [3][5][6][7]

Ask about 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

Survival by stage

StageSurvivalNotes
Primary localized pleomorphic RMS3-year overall survival 60.6%; 5-year overall survival 57.7%; 3-year disease-free survival 40.6%; 5-year disease-free survival 35.8%; median time to recurrence 4.31 months among those who recurred. [12]

Risk factors

  • ▲ increases riskRASopathies (mosaic RASopathies, Costello syndrome) — Reported very high SIRs for rhabdomyosarcoma (mosaic RASopathies SIR=800; Costello syndrome SIR=1240) [13][14]
  • ▲ increases riskLynch syndrome (germline MSH2 mutation) — Rhabdomyosarcoma accounted for about 10% of sarcomas in Lynch syndrome patients; many sarcomas showed dMMR or MSI [15]
  • ▲ increases riskPathogenic germline variants in cancer predisposition genes (in FOXO1 fusion-positive ARMS series) — Identified in 26/191 (13.6%) patients [16]

Biomarkers

  • PAX/FOXO1 fusion statusActionableRisk stratification at diagnosis [10][11]
  • Somatic RAS–MAPK mutations · Genomic characterization (present in ~30% of fusion-negative cases) [13]
  • AFP, β-hCG, carcinoembryonic antigen (CEA) · Typically not elevated in rhabdomyosarcoma (not useful as tumor markers) [2]
  • Myogenin and desmin (immunohistochemistry) · Diagnostic immunohistochemical markers reported positive in cases (e.g., paratesticular RMS) [2]
  • Deficient MMR / microsatellite instability (dMMR/MSI) · Reported in many sarcomas in Lynch syndrome patients [15]
  • TP53 and RB1 alterations · Common non-specific genomic alterations reported in pleomorphic RMS [12][17]
  • HPV testing (with cervical cytology)ActionableUsed in cervical cancer screening to reduce incidence [18][19]

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

Overview11 points

Key figures

Survival & outcomes
OutcomeValue95% CI
screening interval; every 3 years with cervical cytology alone (ages 21–29)3—
screening interval; every 5 years with hrHPV testing alone or cotesting (ages 30–65)5—
Source quotes
  • “The USPSTF recommends screening for cervical cancer every 3 years with cervical cytology alone in women aged 21 to 29 years.”
  • “The USPSTF recommends screening every 3 years with cervical cytology alone, every 5 years with hrHPV testing alone, or every 5 years with hrHPV testing in combination with cytology (cotesting) in women aged 30 to 65 years.”
  • Sources describe rhabdomyosarcoma as the commonest soft tissue sarcoma in children. [1][6][9]3 sources
  • Approximately one third of children with rhabdomyosarcoma relapse or have refractory disease. [5][6]
  • The USPSTF recommends screening for cervical cancer every 3 years with cervical cytology alone in women aged 21 to 29 years and recommends screening every 3 years with cervical cytology alone, every 5 years with hrHPV testing alone, or every 5 years with hrHPV testing in combination with cytology (cotesting) in women aged 30 to 65 years. [20]
  • Rhabdomyosarcoma (RMS) originates from primitive mesenchymal cells. [2]
  • Temporal bone and middle-ear RMS in children often mimics chronic otitis media, leading to delayed diagnosis and advanced disease at presentation. [3]
  • Rhabdomyosarcoma is classified into embryonal and alveolar subtypes, with alveolar cases more frequently harboring specific PAX-FKHR fusions and having a worse prognosis than embryonal cases. [10]
  • Pleomorphic rhabdomyosarcoma (P-RMS) is considered a separate WHO entity from pediatric embryonal or alveolar RMS and mainly affects adults in their sixth to seventh decades. [12]
  • Rhabdomyosarcoma is listed among rare histologies of cervical cancer. [21]
  • Accurate histological identification using specific markers is essential for optimal patient management. [21]
  • Cervical cancer screening programs have been associated with marked reductions in cervical cancer incidence in communities where they have been introduced. [22]
  • Human papillomavirus (HPV) infection was identified as a crucial causal factor for cervical cancer. [18]
Epidemiology13 points

Key figures

Survival & outcomes
OutcomeValue95% CI
estimated deaths in US (2012)4220—
estimated worldwide deaths per year275000—
mortality 1975 (US)5.55—
mortality 2008 (US)2.38—
5-year survival (Korea, 2007–2011)80.1%—
Source quotes
  • “The ACS estimates that there will be 12,170 new cases of cervical cancer in the United States in 2012, with 4,220 deaths from the disease (2).”
  • “Cervical cancer is much more common worldwide, particularly in countries without screening programs, with an estimated 530,000 new cases of the disease and 275,000 resultant deaths each year (3, 4).”
  • “Mortality from the disease has undergone a similar decrease from 5.55 per 100,000 women in 1975 to 2.38 per 100,000 women in 2008 (1).”
  • “according to the National Cancer Registry statistics in 2012, the survival rate of cervical cancer was high compared to other cancers with the 5-year survival rate of 80.1% from 2007 to 2011 [1].”
  • RMS has been reported to account for approximately 3-6.5% of pediatric tumors (reports: approximately 3%, approximately 6.5%, and approximately 5% in different reviews). [2][4][9]3 sources
  • A systematic literature review of mosaic RASopathies found all reported RMS cases (n=7) were urogenital with the highest SIR for RMS (SIR=800; 95% CI 300–1648); a Costello syndrome review reported a statistically significant SIR for RMS (SIR=1240). [13][14]
  • The American Cancer Society estimated 12,170 new cases of cervical cancer and 4,220 deaths in the United States in 2012. [22]
  • Cervical cancer is estimated worldwide to cause about 530,000 new cases and 275,000 deaths each year. [22]
  • Incidence of cervical cancer in the United States decreased from 14.8 per 100,000 women in 1975 to 6.6 per 100,000 women by 2008. [22]
  • Mortality from cervical cancer in the United States decreased from 5.55 per 100,000 women in 1975 to 2.38 per 100,000 women in 2008. [22]
  • In Korea, the 5-year survival rate for cervical cancer from 2007 to 2011 was reported as 80.1%. [18]
  • At least one-third of patients with localized rhabdomyosarcoma and 60-70% of patients with metastatic rhabdomyosarcoma experience progressive disease or relapse. [7]
  • RMS has been reported to represent over 50% of pediatric soft tissue sarcomas while comprising less than 5% of adult sarcomas. [4]
  • The incidence of RMS follows a bimodal age distribution with an initial peak between ages 2 and 5 and a second peak in adolescents under 20 years old. [2]
  • The head and neck region is a common primary site, reported as about 35% of RMS primary locations. [4]
  • Childhood cancer treatment cost studies in sub-Saharan Africa included one report that specifically listed rhabdomyosarcoma. [23]
  • Pleomorphic rhabdomyosarcoma is very rare, with an incidence estimated at approximately 0.545 cases per million per year; in a multicenter series of adults with primary localized P-RMS the median age at surgery was 60 years (IQR 52–72). [12]
Staging & risk3 points
  • For the initial staging of younger rhabdomyosarcoma patients, whole-body FDG-PET/CT or whole-body FDG-PET/MR imaging along with diagnostic chest CT are recommended; brain imaging with MRI may be indicated. [24]
  • TNM staging in one systematic review was based on the Intergroup Rhabdomyosarcoma Study Group (IRSG) staging system for RMS. [4]
  • NCCN clinical practice guidelines for cervical cancer focus on early-stage disease because it occurs more frequently in the United States. [25]
Standard management12 points
  • Sources describe surgery as the cornerstone of pediatric rhabdomyosarcoma management, with radiotherapy and chemotherapy used within multimodal treatment approaches. [8][2][9]3 sources
  • Sources note that comprehensive treatment strategies combining surgery, radiotherapy, and chemotherapy are being explored. [4][9]
  • Treatment approaches for relapsed or refractory rhabdomyosarcoma include combinations of systemic therapies and local therapies directed at tumour sites. [5][9]
  • Guideline recommendations include regional lymph node evaluation for patients with paratesticular rhabdomyosarcoma who are more than 10 years old and for extremity rhabdomyosarcoma. [8]
  • The guideline panel suggests resection of residual metastatic disease after chemotherapy, resection of relapsed disease, and using the least invasive approach appropriate for patients presenting with obstruction. [8]
  • For temporal bone rhabdomyosarcoma in children, chemotherapy combined with radiotherapy has been identified as the standard treatment approach in a systematic review. [3]
  • Surgery combined with brachytherapy as local therapy for children and young people with relapsed/refractory rhabdomyosarcoma has been evaluated; reported outcomes show reasonable benefits in selected patients but are based on small sample sizes and often unclear reporting. [5]
  • Fine needle aspiration cytology (FNAC) has been reported as useful for preoperative cytological identification of paratesticular tumours, which can include rhabdomyosarcoma among malignant paratesticular lesions. [26]
  • For localized pleomorphic rhabdomyosarcoma, surgery often combined with radiotherapy is considered the standard approach, while the role and benefit of (neo)adjuvant chemotherapy for this subtype are poorly understood and have not been addressed in prospective studies. [12]
  • For early-stage cervical cancer, primary treatment options include either surgery or radiotherapy. [22]
  • The NCCN Guidelines for Cervical Cancer Screening were discontinued and the panel endorsed adopting guidelines from other organizations to avoid duplication of effort. [27]
  • The American Brachytherapy Society made recommendations for the use of adjuvant vaginal cuff brachytherapy after hysterectomy, including applicator selection, insertion techniques, target volume definition, and dose fractionation. [28]
Treatments & compounds studied21 treatments

Chemotherapy

  • VAC (vincristine + actinomycin D + cyclophosphamide): VAC (vincristine, actinomycin D, cyclophosphamide) chemotherapy is used in pediatric paratesticular spindle cell rhabdomyosarcoma and is part of Children's Oncology Group (COG) protocols. [2]
  • IVA: IVA is listed among pediatric chemotherapy protocols used for rhabdomyosarcoma. [29]
    5-year overall survival (PP vs non-PP) 56.7 vs 39.5%, p=0.042 vs non-PP
    Source quote
    • “Compared with non-PP, PP was associated with improved survival in adults with RMS (5-year OS of 56.7% vs 39.5% in a non-metastatic cohort, multivariate analysis, p=0.042).”
  • Pediatric chemotherapy protocols (PP; e.g., VAC, IVA): Treatment of adults with pediatric chemotherapy protocols (e.g., VAC, IVA) was associated with improved survival compared with non-pediatric protocols in adults with rhabdomyosarcoma. [29]
    5-year overall survival (PP vs non-PP) 56.7 vs 39.5%, p=0.042 vs non-PP
    Source quote
    • “Compared with non-PP, PP was associated with improved survival in adults with RMS (5-year OS of 56.7% vs 39.5% in a non-metastatic cohort, multivariate analysis, p=0.042).”
  • vinorelbine + cyclophosphamide: A metronomic regimen of vinorelbine and cyclophosphamide was studied in a randomized trial in patients aged 6 months to 21 years with nonmetastatic high-risk rhabdomyosarcoma who were in complete remission or had minimal imaging abnormalities at the end of standard treatment. [30]
  • celecoxib + thalidomide + etoposide + cyclophosphamide: Recurrent or later-lineA metronomic regimen including daily oral celecoxib and daily oral thalidomide with alternating oral etoposide and oral cyclophosphamide was studied in children and adolescents with refractory or progressive extracranial solid tumours after at least two prior chemotherapy lines. [30]
  • systemic chemotherapy: Systemic chemotherapy is described as a component of multimodal rhabdomyosarcoma treatment. [9]
    LC at 1 year 96% (95% CI 0.91–1.01)LC at 2 years 93% (95% CI 0.86–1)LC at 3 years 78% (95% CI 0.71–0.85)LC at 4 years 85% (95% CI 0.78–0.92)LC at 5 years 84% (95% CI 0.74–0.95)PFS at 1 year 82% (95% CI 0.72–0.92)PFS at 2 years 73% (95% CI 0.61–0.84)PFS at 3 years 63% (95% CI 0.47–0.79)PFS at 4 years 64% (95% CI 0.54–0.74)PFS at 5 years 76% (95% CI 0.59–0.94)OS at 1 year 93% (95% CI 0.86–1)OS at 2 years 85% (95% CI 0.76–0.95)OS at 3 years 80% (95% CI 0.63–0.96)OS at 4 years 71% (95% CI 0.62–0.8)OS at 5 years 82% (95% CI 0.71–0.94)
    Source quotes
    • “In our systematic review, the LC incidence at 1, 2, 3, 4, and 5 years in these studies were 96% (95% CI 0.91–1.01), 93% (95% CI 0.86–1.00), 78% (95% CI 0.71–0.85, I2 = 0%), 85% (95% CI 0.78–0.92), and 84% (95% CI 0.74–0.95, I2 = 91.1%), respectively (Fig. 2) [13–23].”
    • “In our systematic review, the LC incidence at 1, 2, 3, 4, and 5 years in these studies were 96% (95% CI 0.91–1.01), 93% (95% CI 0.86–1.00), 78% (95% CI 0.71–0.85, I2 = 0%), 85% (95% CI 0.78–0.92), and 84% (95% CI 0.74–0.95, I2 = 91.1%), respectively (Fig. 2) [13–23].”
    • “In our systematic review, the LC incidence at 1, 2, 3, 4, and 5 years in these studies were 96% (95% CI 0.91–1.01), 93% (95% CI 0.86–1.00), 78% (95% CI 0.71–0.85, I2 = 0%), 85% (95% CI 0.78–0.92), and 84% (95% CI 0.74–0.95, I2 = 91.1%), respectively (Fig. 2) [13–23].”
    • “In our systematic review, the LC incidence at 1, 2, 3, 4, and 5 years in these studies were 96% (95% CI 0.91–1.01), 93% (95% CI 0.86–1.00), 78% (95% CI 0.71–0.85, I2 = 0%), 85% (95% CI 0.78–0.92), and 84% (95% CI 0.74–0.95, I2 = 91.1%), respectively (Fig. 2) [13–23].”
    • “In our systematic review, the LC incidence at 1, 2, 3, 4, and 5 years in these studies were 96% (95% CI 0.91–1.01), 93% (95% CI 0.86–1.00), 78% (95% CI 0.71–0.85, I2 = 0%), 85% (95% CI 0.78–0.92), and 84% (95% CI 0.74–0.95, I2 = 91.1%), respectively (Fig. 2) [13–23].”
    • “The PFS rate at 1, 2, 3, 4, and 5 years in these studies were 82% (95% CI 0.72–0.92), 73% (95% CI 0.61–0.84), 63% (95% CI 0.47–0.79, I2 = 74.1%), 64% (95% CI 0.54–0.74), and 76% (95% CI 0.59–0.94, I2 = 94.6%), respectively (Fig. 3) [13–21, 23].”
    • “The PFS rate at 1, 2, 3, 4, and 5 years in these studies were 82% (95% CI 0.72–0.92), 73% (95% CI 0.61–0.84), 63% (95% CI 0.47–0.79, I2 = 74.1%), 64% (95% CI 0.54–0.74), and 76% (95% CI 0.59–0.94, I2 = 94.6%), respectively (Fig. 3) [13–21, 23].”
    • “The PFS rate at 1, 2, 3, 4, and 5 years in these studies were 82% (95% CI 0.72–0.92), 73% (95% CI 0.61–0.84), 63% (95% CI 0.47–0.79, I2 = 74.1%), 64% (95% CI 0.54–0.74), and 76% (95% CI 0.59–0.94, I2 = 94.6%), respectively (Fig. 3) [13–21, 23].”
    • “The PFS rate at 1, 2, 3, 4, and 5 years in these studies were 82% (95% CI 0.72–0.92), 73% (95% CI 0.61–0.84), 63% (95% CI 0.47–0.79, I2 = 74.1%), 64% (95% CI 0.54–0.74), and 76% (95% CI 0.59–0.94, I2 = 94.6%), respectively (Fig. 3) [13–21, 23].”
    • “The PFS rate at 1, 2, 3, 4, and 5 years in these studies were 82% (95% CI 0.72–0.92), 73% (95% CI 0.61–0.84), 63% (95% CI 0.47–0.79, I2 = 74.1%), 64% (95% CI 0.54–0.74), and 76% (95% CI 0.59–0.94, I2 = 94.6%), respectively (Fig. 3) [13–21, 23].”
    • “As shown in Fig. 4, after undergoing PBT for 1, 2, 3, 4, and 5 years, the OS rates for RMS were 93% (95% CI 0.86–1.00), 85% (95% CI 0.76–0.95), 80% (95% CI 0.63–0.96, I2 = 84.9%), 71% (95% CI 0.62–0.80), and 82% (95% CI 0.71–0.94, I2 = 92.2%), respectively [13–23].”
    • “As shown in Fig. 4, after undergoing PBT for 1, 2, 3, 4, and 5 years, the OS rates for RMS were 93% (95% CI 0.86–1.00), 85% (95% CI 0.76–0.95), 80% (95% CI 0.63–0.96, I2 = 84.9%), 71% (95% CI 0.62–0.80), and 82% (95% CI 0.71–0.94, I2 = 92.2%), respectively [13–23].”
    • “As shown in Fig. 4, after undergoing PBT for 1, 2, 3, 4, and 5 years, the OS rates for RMS were 93% (95% CI 0.86–1.00), 85% (95% CI 0.76–0.95), 80% (95% CI 0.63–0.96, I2 = 84.9%), 71% (95% CI 0.62–0.80), and 82% (95% CI 0.71–0.94, I2 = 92.2%), respectively [13–23].”
    • “As shown in Fig. 4, after undergoing PBT for 1, 2, 3, 4, and 5 years, the OS rates for RMS were 93% (95% CI 0.86–1.00), 85% (95% CI 0.76–0.95), 80% (95% CI 0.63–0.96, I2 = 84.9%), 71% (95% CI 0.62–0.80), and 82% (95% CI 0.71–0.94, I2 = 92.2%), respectively [13–23].”
    • “As shown in Fig. 4, after undergoing PBT for 1, 2, 3, 4, and 5 years, the OS rates for RMS were 93% (95% CI 0.86–1.00), 85% (95% CI 0.76–0.95), 80% (95% CI 0.63–0.96, I2 = 84.9%), 71% (95% CI 0.62–0.80), and 82% (95% CI 0.71–0.94, I2 = 92.2%), respectively [13–23].”
  • chemotherapy (unspecified regimens): Neoadjuvant (before surgery)Chemotherapy was administered to 40% of patients with primary localized pleomorphic rhabdomyosarcoma, most commonly in the neoadjuvant setting, and the 28 patients treated with neoadjuvant chemotherapy were assessable with an overall response rate of 50%. [12]
    RT effect on DFS (multivariable analysis) 0.26 (95% CI 0.14–0.48), p < 0.001RT usage 60%overall response rate to neoadjuvant chemotherapy 50%chemotherapy usage 40%
    Source quotes
    • “In multivariable analysis, younger age at surgery [hazard ratio (HR) 1.03, CI 1.01-1.05, P = 0.009], R0 surgical margins (HR 2.51, CI 1.23-5.13, P = 0.021), and RT (HR 0.26, CI 0.14-0.48, P < 0.001) remained significant (Supplementary Table S2, available at https://doi.org/10.1016/j.esmoop.2025.106044).”
    • “RT was administered in 60% of our patients, both in pre- and post-operative settings, and was significantly associated with improved DFS.”
    • “All 28 patients treated with neoadjuvant ChT were assessable for response: 12/28 (43%) achieved partial response and 2/28 (7%) had complete response (overall response rate 50%).”
    • “Thirty-seven of 93 patients (40%) received ChT, mostly in the neoadjuvant setting (28/37, 76%), with 24/28 (86%) treated using anthracycline-based regimens.”
  • anthracycline-based regimens: Neoadjuvant (before surgery)Anthracycline-based regimens were the most frequently used chemotherapy in the neoadjuvant setting for patients with primary localized pleomorphic rhabdomyosarcoma in this series. [12]
    RT effect on DFS (multivariable analysis) 0.26 (95% CI 0.14–0.48), p < 0.001RT usage 60%overall response rate to neoadjuvant chemotherapy 50%chemotherapy usage 40%
    Source quotes
    • “In multivariable analysis, younger age at surgery [hazard ratio (HR) 1.03, CI 1.01-1.05, P = 0.009], R0 surgical margins (HR 2.51, CI 1.23-5.13, P = 0.021), and RT (HR 0.26, CI 0.14-0.48, P < 0.001) remained significant (Supplementary Table S2, available at https://doi.org/10.1016/j.esmoop.2025.106044).”
    • “RT was administered in 60% of our patients, both in pre- and post-operative settings, and was significantly associated with improved DFS.”
    • “All 28 patients treated with neoadjuvant ChT were assessable for response: 12/28 (43%) achieved partial response and 2/28 (7%) had complete response (overall response rate 50%).”
    • “Thirty-seven of 93 patients (40%) received ChT, mostly in the neoadjuvant setting (28/37, 76%), with 24/28 (86%) treated using anthracycline-based regimens.”
  • anthracycline + ifosfamide; vincristine + anthracycline + ifosfamide; vincristine + anthracycline + cyclophosphamide; etoposide + ifosfamide: Neoadjuvant (before surgery)Specific neoadjuvant chemotherapy combinations reported included anthracycline with ifosfamide; vincristine with anthracycline and ifosfamide; vincristine with anthracycline and cyclophosphamide; and etoposide with ifosfamide. [12]
    RT effect on DFS (multivariable analysis) 0.26 (95% CI 0.14–0.48), p < 0.001RT usage 60%overall response rate to neoadjuvant chemotherapy 50%chemotherapy usage 40%
    Source quotes
    • “In multivariable analysis, younger age at surgery [hazard ratio (HR) 1.03, CI 1.01-1.05, P = 0.009], R0 surgical margins (HR 2.51, CI 1.23-5.13, P = 0.021), and RT (HR 0.26, CI 0.14-0.48, P < 0.001) remained significant (Supplementary Table S2, available at https://doi.org/10.1016/j.esmoop.2025.106044).”
    • “RT was administered in 60% of our patients, both in pre- and post-operative settings, and was significantly associated with improved DFS.”
    • “All 28 patients treated with neoadjuvant ChT were assessable for response: 12/28 (43%) achieved partial response and 2/28 (7%) had complete response (overall response rate 50%).”
    • “Thirty-seven of 93 patients (40%) received ChT, mostly in the neoadjuvant setting (28/37, 76%), with 24/28 (86%) treated using anthracycline-based regimens.”
  • chemotherapy: Recurrent or later-lineChemotherapy is incorporated into the multidisciplinary management of relapsed rhabdomyosarcoma alongside local control measures. [7]

Radiotherapy

  • proton beam therapy: Proton beam therapy was judged by the SARO panel to have a strongly convincing body of evidence (Level A) for children and a moderately convincing body of evidence (Level B) for adolescents and young adults (<39 years). [31][9]
    adjusted OR for secondary malignancies (PBT vs IMRT) 0.31, p <0.0001 vs IMRTgrade 5 3 toxicity and unplanned hospitalizations (PBT+chemo vs photons+chemo) 11.5 vs 27.6%, p <0.01 vs photonsLC at 1 year 96% (95% CI 0.91–1.01)LC at 2 years 93% (95% CI 0.86–1)LC at 3 years 78% (95% CI 0.71–0.85)LC at 4 years 85% (95% CI 0.78–0.92)LC at 5 years 84% (95% CI 0.74–0.95)PFS at 1 year 82% (95% CI 0.72–0.92)PFS at 2 years 73% (95% CI 0.61–0.84)PFS at 3 years 63% (95% CI 0.47–0.79)PFS at 4 years 64% (95% CI 0.54–0.74)PFS at 5 years 76% (95% CI 0.59–0.94)OS at 1 year 93% (95% CI 0.86–1)OS at 2 years 85% (95% CI 0.76–0.95)OS at 3 years 80% (95% CI 0.63–0.96)OS at 4 years 71% (95% CI 0.62–0.8)OS at 5 years 82% (95% CI 0.71–0.94)
    Source quotes
    • “Out of 450,373 patients, 33.5% underwent 3-dimensional conformal radiotherapy (3DCRT), 65.2% received intensity-modulated radiotherapy (IMRT), and 1.3% were treated with PBT. Over a median follow-up of 5.1 years, no significant difference in secondary cancer risk was found between IMRT and 3DCRT (adjusted odds ratio [OR]=1.00, p=0.75). However, patients treated with PBT demonstrated a significantly lower risk of developing secondary malignancies compared to those receiving IMRT (adjusted OR=0.31, p<0.0001), supporting the theoretical benefit of PBT in minimizing second cancer formation.”
    • “Across different cancer types, proton therapy concurrently with chemotherapy reduces grade 5 3 toxicity and unplanned hospitalizations when compared to photons (11.5% vs. 27.6%, p<0.01).”
    • “In our systematic review, the LC incidence at 1, 2, 3, 4, and 5 years in these studies were 96% (95% CI 0.91–1.01), 93% (95% CI 0.86–1.00), 78% (95% CI 0.71–0.85, I2 = 0%), 85% (95% CI 0.78–0.92), and 84% (95% CI 0.74–0.95, I2 = 91.1%), respectively (Fig. 2) [13–23].”
    • “In our systematic review, the LC incidence at 1, 2, 3, 4, and 5 years in these studies were 96% (95% CI 0.91–1.01), 93% (95% CI 0.86–1.00), 78% (95% CI 0.71–0.85, I2 = 0%), 85% (95% CI 0.78–0.92), and 84% (95% CI 0.74–0.95, I2 = 91.1%), respectively (Fig. 2) [13–23].”
    • “In our systematic review, the LC incidence at 1, 2, 3, 4, and 5 years in these studies were 96% (95% CI 0.91–1.01), 93% (95% CI 0.86–1.00), 78% (95% CI 0.71–0.85, I2 = 0%), 85% (95% CI 0.78–0.92), and 84% (95% CI 0.74–0.95, I2 = 91.1%), respectively (Fig. 2) [13–23].”
    • “In our systematic review, the LC incidence at 1, 2, 3, 4, and 5 years in these studies were 96% (95% CI 0.91–1.01), 93% (95% CI 0.86–1.00), 78% (95% CI 0.71–0.85, I2 = 0%), 85% (95% CI 0.78–0.92), and 84% (95% CI 0.74–0.95, I2 = 91.1%), respectively (Fig. 2) [13–23].”
    • “In our systematic review, the LC incidence at 1, 2, 3, 4, and 5 years in these studies were 96% (95% CI 0.91–1.01), 93% (95% CI 0.86–1.00), 78% (95% CI 0.71–0.85, I2 = 0%), 85% (95% CI 0.78–0.92), and 84% (95% CI 0.74–0.95, I2 = 91.1%), respectively (Fig. 2) [13–23].”
    • “The PFS rate at 1, 2, 3, 4, and 5 years in these studies were 82% (95% CI 0.72–0.92), 73% (95% CI 0.61–0.84), 63% (95% CI 0.47–0.79, I2 = 74.1%), 64% (95% CI 0.54–0.74), and 76% (95% CI 0.59–0.94, I2 = 94.6%), respectively (Fig. 3) [13–21, 23].”
    • “The PFS rate at 1, 2, 3, 4, and 5 years in these studies were 82% (95% CI 0.72–0.92), 73% (95% CI 0.61–0.84), 63% (95% CI 0.47–0.79, I2 = 74.1%), 64% (95% CI 0.54–0.74), and 76% (95% CI 0.59–0.94, I2 = 94.6%), respectively (Fig. 3) [13–21, 23].”
    • “The PFS rate at 1, 2, 3, 4, and 5 years in these studies were 82% (95% CI 0.72–0.92), 73% (95% CI 0.61–0.84), 63% (95% CI 0.47–0.79, I2 = 74.1%), 64% (95% CI 0.54–0.74), and 76% (95% CI 0.59–0.94, I2 = 94.6%), respectively (Fig. 3) [13–21, 23].”
    • “The PFS rate at 1, 2, 3, 4, and 5 years in these studies were 82% (95% CI 0.72–0.92), 73% (95% CI 0.61–0.84), 63% (95% CI 0.47–0.79, I2 = 74.1%), 64% (95% CI 0.54–0.74), and 76% (95% CI 0.59–0.94, I2 = 94.6%), respectively (Fig. 3) [13–21, 23].”
    • “The PFS rate at 1, 2, 3, 4, and 5 years in these studies were 82% (95% CI 0.72–0.92), 73% (95% CI 0.61–0.84), 63% (95% CI 0.47–0.79, I2 = 74.1%), 64% (95% CI 0.54–0.74), and 76% (95% CI 0.59–0.94, I2 = 94.6%), respectively (Fig. 3) [13–21, 23].”
    • “As shown in Fig. 4, after undergoing PBT for 1, 2, 3, 4, and 5 years, the OS rates for RMS were 93% (95% CI 0.86–1.00), 85% (95% CI 0.76–0.95), 80% (95% CI 0.63–0.96, I2 = 84.9%), 71% (95% CI 0.62–0.80), and 82% (95% CI 0.71–0.94, I2 = 92.2%), respectively [13–23].”
    • “As shown in Fig. 4, after undergoing PBT for 1, 2, 3, 4, and 5 years, the OS rates for RMS were 93% (95% CI 0.86–1.00), 85% (95% CI 0.76–0.95), 80% (95% CI 0.63–0.96, I2 = 84.9%), 71% (95% CI 0.62–0.80), and 82% (95% CI 0.71–0.94, I2 = 92.2%), respectively [13–23].”
    • “As shown in Fig. 4, after undergoing PBT for 1, 2, 3, 4, and 5 years, the OS rates for RMS were 93% (95% CI 0.86–1.00), 85% (95% CI 0.76–0.95), 80% (95% CI 0.63–0.96, I2 = 84.9%), 71% (95% CI 0.62–0.80), and 82% (95% CI 0.71–0.94, I2 = 92.2%), respectively [13–23].”
    • “As shown in Fig. 4, after undergoing PBT for 1, 2, 3, 4, and 5 years, the OS rates for RMS were 93% (95% CI 0.86–1.00), 85% (95% CI 0.76–0.95), 80% (95% CI 0.63–0.96, I2 = 84.9%), 71% (95% CI 0.62–0.80), and 82% (95% CI 0.71–0.94, I2 = 92.2%), respectively [13–23].”
    • “As shown in Fig. 4, after undergoing PBT for 1, 2, 3, 4, and 5 years, the OS rates for RMS were 93% (95% CI 0.86–1.00), 85% (95% CI 0.76–0.95), 80% (95% CI 0.63–0.96, I2 = 84.9%), 71% (95% CI 0.62–0.80), and 82% (95% CI 0.71–0.94, I2 = 92.2%), respectively [13–23].”
  • whole abdominopelvic radiotherapy (WAP-RT): Consensus-based delineation guidelines have been developed for the clinical target volume to support treatment planning for whole abdominopelvic radiotherapy (WAP-RT) in patients with peritoneal involvement. [32]
  • radiotherapy: Radiotherapy is used as a treatment modality for rhabdomyosarcoma and is applied in combination with chemotherapy in multimodal regimens. [3][12]
    RT effect on DFS (multivariable analysis) 0.26 (95% CI 0.14–0.48), p < 0.001RT usage 60%overall response rate to neoadjuvant chemotherapy 50%chemotherapy usage 40%
    Source quotes
    • “In multivariable analysis, younger age at surgery [hazard ratio (HR) 1.03, CI 1.01-1.05, P = 0.009], R0 surgical margins (HR 2.51, CI 1.23-5.13, P = 0.021), and RT (HR 0.26, CI 0.14-0.48, P < 0.001) remained significant (Supplementary Table S2, available at https://doi.org/10.1016/j.esmoop.2025.106044).”
    • “RT was administered in 60% of our patients, both in pre- and post-operative settings, and was significantly associated with improved DFS.”
    • “All 28 patients treated with neoadjuvant ChT were assessable for response: 12/28 (43%) achieved partial response and 2/28 (7%) had complete response (overall response rate 50%).”
    • “Thirty-seven of 93 patients (40%) received ChT, mostly in the neoadjuvant setting (28/37, 76%), with 24/28 (86%) treated using anthracycline-based regimens.”
  • external beam radiation therapy: External beam radiation therapy is discussed in combination with vaginal cuff brachytherapy, and suggested doses were tabulated for treatment with brachytherapy alone and in combination with external beam radiation when applicable. [28]

Procedures & devices

  • surgery · 2 findings
    • Surgery is described as a cornerstone of treatment and is used as local therapy in multimodal management of rhabdomyosarcoma, including radical orchiectomy for paratesticular disease. [8][2][9][12]4 sources
      LC at 1 year 96% (95% CI 0.91–1.01)LC at 2 years 93% (95% CI 0.86–1)LC at 3 years 78% (95% CI 0.71–0.85)LC at 4 years 85% (95% CI 0.78–0.92)LC at 5 years 84% (95% CI 0.74–0.95)PFS at 1 year 82% (95% CI 0.72–0.92)PFS at 2 years 73% (95% CI 0.61–0.84)PFS at 3 years 63% (95% CI 0.47–0.79)PFS at 4 years 64% (95% CI 0.54–0.74)PFS at 5 years 76% (95% CI 0.59–0.94)OS at 1 year 93% (95% CI 0.86–1)OS at 2 years 85% (95% CI 0.76–0.95)OS at 3 years 80% (95% CI 0.63–0.96)OS at 4 years 71% (95% CI 0.62–0.8)OS at 5 years 82% (95% CI 0.71–0.94)RT effect on DFS (multivariable analysis) 0.26 (95% CI 0.14–0.48), p < 0.001RT usage 60%overall response rate to neoadjuvant chemotherapy 50%chemotherapy usage 40%
      Source quotes
      • “In our systematic review, the LC incidence at 1, 2, 3, 4, and 5 years in these studies were 96% (95% CI 0.91–1.01), 93% (95% CI 0.86–1.00), 78% (95% CI 0.71–0.85, I2 = 0%), 85% (95% CI 0.78–0.92), and 84% (95% CI 0.74–0.95, I2 = 91.1%), respectively (Fig. 2) [13–23].”
      • “In our systematic review, the LC incidence at 1, 2, 3, 4, and 5 years in these studies were 96% (95% CI 0.91–1.01), 93% (95% CI 0.86–1.00), 78% (95% CI 0.71–0.85, I2 = 0%), 85% (95% CI 0.78–0.92), and 84% (95% CI 0.74–0.95, I2 = 91.1%), respectively (Fig. 2) [13–23].”
      • “In our systematic review, the LC incidence at 1, 2, 3, 4, and 5 years in these studies were 96% (95% CI 0.91–1.01), 93% (95% CI 0.86–1.00), 78% (95% CI 0.71–0.85, I2 = 0%), 85% (95% CI 0.78–0.92), and 84% (95% CI 0.74–0.95, I2 = 91.1%), respectively (Fig. 2) [13–23].”
      • “In our systematic review, the LC incidence at 1, 2, 3, 4, and 5 years in these studies were 96% (95% CI 0.91–1.01), 93% (95% CI 0.86–1.00), 78% (95% CI 0.71–0.85, I2 = 0%), 85% (95% CI 0.78–0.92), and 84% (95% CI 0.74–0.95, I2 = 91.1%), respectively (Fig. 2) [13–23].”
      • “In our systematic review, the LC incidence at 1, 2, 3, 4, and 5 years in these studies were 96% (95% CI 0.91–1.01), 93% (95% CI 0.86–1.00), 78% (95% CI 0.71–0.85, I2 = 0%), 85% (95% CI 0.78–0.92), and 84% (95% CI 0.74–0.95, I2 = 91.1%), respectively (Fig. 2) [13–23].”
      • “The PFS rate at 1, 2, 3, 4, and 5 years in these studies were 82% (95% CI 0.72–0.92), 73% (95% CI 0.61–0.84), 63% (95% CI 0.47–0.79, I2 = 74.1%), 64% (95% CI 0.54–0.74), and 76% (95% CI 0.59–0.94, I2 = 94.6%), respectively (Fig. 3) [13–21, 23].”
      • “The PFS rate at 1, 2, 3, 4, and 5 years in these studies were 82% (95% CI 0.72–0.92), 73% (95% CI 0.61–0.84), 63% (95% CI 0.47–0.79, I2 = 74.1%), 64% (95% CI 0.54–0.74), and 76% (95% CI 0.59–0.94, I2 = 94.6%), respectively (Fig. 3) [13–21, 23].”
      • “The PFS rate at 1, 2, 3, 4, and 5 years in these studies were 82% (95% CI 0.72–0.92), 73% (95% CI 0.61–0.84), 63% (95% CI 0.47–0.79, I2 = 74.1%), 64% (95% CI 0.54–0.74), and 76% (95% CI 0.59–0.94, I2 = 94.6%), respectively (Fig. 3) [13–21, 23].”
      • “The PFS rate at 1, 2, 3, 4, and 5 years in these studies were 82% (95% CI 0.72–0.92), 73% (95% CI 0.61–0.84), 63% (95% CI 0.47–0.79, I2 = 74.1%), 64% (95% CI 0.54–0.74), and 76% (95% CI 0.59–0.94, I2 = 94.6%), respectively (Fig. 3) [13–21, 23].”
      • “The PFS rate at 1, 2, 3, 4, and 5 years in these studies were 82% (95% CI 0.72–0.92), 73% (95% CI 0.61–0.84), 63% (95% CI 0.47–0.79, I2 = 74.1%), 64% (95% CI 0.54–0.74), and 76% (95% CI 0.59–0.94, I2 = 94.6%), respectively (Fig. 3) [13–21, 23].”
      • “As shown in Fig. 4, after undergoing PBT for 1, 2, 3, 4, and 5 years, the OS rates for RMS were 93% (95% CI 0.86–1.00), 85% (95% CI 0.76–0.95), 80% (95% CI 0.63–0.96, I2 = 84.9%), 71% (95% CI 0.62–0.80), and 82% (95% CI 0.71–0.94, I2 = 92.2%), respectively [13–23].”
      • “As shown in Fig. 4, after undergoing PBT for 1, 2, 3, 4, and 5 years, the OS rates for RMS were 93% (95% CI 0.86–1.00), 85% (95% CI 0.76–0.95), 80% (95% CI 0.63–0.96, I2 = 84.9%), 71% (95% CI 0.62–0.80), and 82% (95% CI 0.71–0.94, I2 = 92.2%), respectively [13–23].”
      • “As shown in Fig. 4, after undergoing PBT for 1, 2, 3, 4, and 5 years, the OS rates for RMS were 93% (95% CI 0.86–1.00), 85% (95% CI 0.76–0.95), 80% (95% CI 0.63–0.96, I2 = 84.9%), 71% (95% CI 0.62–0.80), and 82% (95% CI 0.71–0.94, I2 = 92.2%), respectively [13–23].”
      • “As shown in Fig. 4, after undergoing PBT for 1, 2, 3, 4, and 5 years, the OS rates for RMS were 93% (95% CI 0.86–1.00), 85% (95% CI 0.76–0.95), 80% (95% CI 0.63–0.96, I2 = 84.9%), 71% (95% CI 0.62–0.80), and 82% (95% CI 0.71–0.94, I2 = 92.2%), respectively [13–23].”
      • “As shown in Fig. 4, after undergoing PBT for 1, 2, 3, 4, and 5 years, the OS rates for RMS were 93% (95% CI 0.86–1.00), 85% (95% CI 0.76–0.95), 80% (95% CI 0.63–0.96, I2 = 84.9%), 71% (95% CI 0.62–0.80), and 82% (95% CI 0.71–0.94, I2 = 92.2%), respectively [13–23].”
      • “In multivariable analysis, younger age at surgery [hazard ratio (HR) 1.03, CI 1.01-1.05, P = 0.009], R0 surgical margins (HR 2.51, CI 1.23-5.13, P = 0.021), and RT (HR 0.26, CI 0.14-0.48, P < 0.001) remained significant (Supplementary Table S2, available at https://doi.org/10.1016/j.esmoop.2025.106044).”
      • “RT was administered in 60% of our patients, both in pre- and post-operative settings, and was significantly associated with improved DFS.”
      • “All 28 patients treated with neoadjuvant ChT were assessable for response: 12/28 (43%) achieved partial response and 2/28 (7%) had complete response (overall response rate 50%).”
      • “Thirty-seven of 93 patients (40%) received ChT, mostly in the neoadjuvant setting (28/37, 76%), with 24/28 (86%) treated using anthracycline-based regimens.”
    • First-line (advanced disease)Surgery is identified as one of the primary treatment options for early-stage cervical cancer alongside radiotherapy. [25]
  • regional lymph node evaluation: Regional lymph node evaluation is recommended for paratesticular rhabdomyosarcoma in patients older than 10 years and for extremity rhabdomyosarcoma as part of staging/intervention. [8]
  • surgery and brachytherapy: Recurrent or later-lineSurgery combined with brachytherapy has been studied as local therapy for children and young people with relapsed or refractory rhabdomyosarcoma. [5]
  • vaginal cuff brachytherapy: Adjuvant (after surgery)Vaginal cuff brachytherapy has specific ABS recommendations for applicator selection, insertion technique, target definition, and dose fractionation in the adjuvant post-hysterectomy setting. [28]
Show 1 lab & early-research entry
  • ablation techniques: Ablation techniques (radiofrequency, microwave, cryoablation and others) have been applied in a small number of pediatric patients, including three reported cases of rhabdomyosarcoma within largely heterogeneous series. [33]
    complication rate after ablation 64%
    Source quote
    • “Eighteen of the patients (64%) experienced a complication, but only 6 (21%) of these needed treatment other than supportive care.”

Other

  • systemic therapies (early phase studies): Recurrent or later-lineEarly phase studies of systemic therapies in pediatric relapsed/refractory rhabdomyosarcoma reported an objective response rate of 21.6% and, where reported, 70% of studies had a median progression-free survival ≤6 months. [6]
    objective response rate 21.6%proportion of studies reporting median PFS ≤6 months 70%
    Source quotes
    • “Objective response rate was 21.6%.”
    • “Where reported, 70% of studies reported a median progression-free survival ≤6 months.”
Prognosis8 points

Key figures

Survival & outcomes
OutcomeValue95% CI
pediatric RMS survival70%—
average lifespan after diagnosis (mastoid RMS)7.1 months—
Source quotes
  • “The precise selection of treatment modalities and implementation of multimodal treatment plans have significantly improved therapeutic outcomes and survival rates for RMS at various sites, with pediatric RMS survival now exceeding 70% [11, 12].”
  • “The average lifespan with rhabdomyosarcoma of the mastoid was 7.1 months after diagnosis, with 41.7% of patients alive at the time of report.”
  • Following relapse or in relapsed/refractory disease, outcomes generally remain poor with limited treatment options and a high risk of subsequent recurrence; outcomes for children who enroll on early-phase studies are poor. [7][6]
  • In an international multicenter series of 93 adults with primary localized pleomorphic RMS the 3-year and 5-year overall survival rates were reported as 60.6% and 57.7%, respectively; in the same series the 3-year and 5-year disease-free survival rates were 40.6% and 35.8%, with a median time to recurrence of 4.31 months among those who recurred. [12]
  • One review states that pediatric RMS survival now exceeds 70%. [4]
  • Rhabdomyosarcoma originating in the mastoid has a poor short-term outcome with an average lifespan of 7.1 months after diagnosis in an aggregated series. [34]
  • Reported survival for ocular RMS varies widely, from as low as 10% with unimodal treatment to over 70% with multimodal therapy. [35]
  • Paratesticular spindle cell RMS is often cited as having a favorable prognosis compared to other subtypes, although recurrence can still occur. [2]
  • Pleomorphic rhabdomyosarcoma is an aggressive and rare malignant neoplasm with a poor prognosis; in pleomorphic RMS, age at diagnosis, tumor stage, and surgical management significantly affected patient outcome while ethnicity, sex, or tumor site did not. [36]
  • Introduction of widespread screening with cervical cytology has been associated with more than a 50% decrease in cervical cancer incidence in the United States over about 30 years. [22]
Safety & interactions6 points

Key figures

Survival & outcomes
OutcomeValue95% CI
median diagnostic delay5 months—
median_latency_to_radiation_myelopathy7 months—
Source quotes
  • “Median diagnostic delay [months] 5 (range: 0.25–360, IQR 0–23.25)”
  • “The median latency period was 7 months (range, 1-29).”
  • When soft-tissue sarcomas were initially misdiagnosed as benign vascular anomalies, the median diagnostic delay was 5 months in a systematic review. [37]
  • In pediatric patients reviewed, radiation myelopathy had a median latency period of 7 months (range, 1-29), and chemotherapy appears to reduce spinal cord tolerance to radiotherapy. [38]
  • Ablation procedures in reported pediatric series had a high overall complication frequency but most complications required only supportive care. [33]
  • Certain inherited DNA repair syndromes (eg, Ataxia-telangiectasia, xeroderma pigmentosum, Bloom syndrome, Fanconi anemia, and Li-Fraumeni syndrome) are strongly associated with significant morbidity and, in some cases, mortality after radiotherapy; for individuals with these conditions radiotherapy may need to be avoided or adjusted. [31]
  • Adults treated with paediatric chemotherapy protocols had lower chemotherapy exposure and different toxicities, including more peripheral neuropathy but similar or reduced haematological toxicity compared to children. [29]
  • Across PBT studies, the incidence of acute and late toxicities was mainly grades 1 to 2; in PBT reports grade 3 acute toxicity incidence ranged 9–25% and grade 3 late toxicity incidence ranged 2.1–26%. [9]
What we don't know yet9 points
  • Study quality in reports of relapsed or refractory rhabdomyosarcoma is limited by poor or inconsistent reporting and, in some series (for surgery plus brachytherapy), potential selection bias. [5][6]
  • Few randomized clinical trials have been published comparing proton beam therapy with photon radiotherapy, and clinical use of proton beam therapy is guided largely by prospective and retrospective studies. [31]
  • The treatment of adults with rhabdomyosarcoma remains an area of unmet need, and adequate chemotherapy exposure and inclusion of disease-specific agents may be important. [29]
  • Evidence about ocular rhabdomyosarcoma from Africa is inconclusive and variability in study designs and reporting limits generalizability. [35]
  • Further studies are required to better characterize the sub-population of sarcomas that develop in patients with Lynch syndrome. [15]
  • Available data were insufficient to assess causal associations between cancer predisposition syndromes and FOXO1 fusion-positive alveolar rhabdomyosarcoma (ARMS). [16]
  • Sources indicate that for pleomorphic rhabdomyosarcoma, the contribution of chemotherapy and radiotherapy to the cure rate remains unclear. [12]
  • Large multicenter prospective trials are needed to establish the efficacy of ablation techniques for pediatric malignant or aggressive benign lesions, including the few reported rhabdomyosarcoma cases. [33]
  • New technologies for cervical cancer screening continue to evolve and so do recommendations for managing screening results. [22]
Key biomarkers10 points

Key figures

Survival & outcomes
OutcomeValue95% CI
Proportion of rhabdomyosarcoma among sarcomas in Lynch syndrome10%—
Source quotes
  • “Although undifferentiated pleomorphic sarcoma (UPS), leiomyosarcoma, and liposarcoma remain the most represented histologic subtype, a higher proportion of rhabdomyosarcoma (10 %, especially pleomorphic rhabdomyosarcoma) is reported.”
  • Sources indicate that fusion gene status (PAX/FOXO1), rather than histologic alveolar appearance alone, is a critical factor for risk stratification in rhabdomyosarcoma. [10][11]
  • The genomic profile of pleomorphic rhabdomyosarcoma is non-specific and often shows copy-number variations and alterations in tumor suppressor genes such as TP53 and RB1. [12][17]
  • In patients with FOXO1 (PAX3/7–FKHR) fusion-positive alveolar rhabdomyosarcoma who underwent germline sequencing, pathogenic or likely pathogenic variants in cancer predisposition genes were identified in 26/191 (13.6%) patients; typical cancer predisposition syndrome associations are rare but not nonexistent, and FOXO1 fusion status alone is insufficient to distinguish patients with versus without cancer predisposition syndromes. [16]
  • In a systematic review of sarcomas in Lynch syndrome, most sarcomas in patients with a germline MSH2 mutation exhibited deficient mismatch repair (dMMR) or microsatellite instability (MSI), and rhabdomyosarcoma accounted for about 10% of sarcomas in Lynch syndrome patients (especially pleomorphic rhabdomyosarcoma). [15]
  • Tumor markers such as AFP, β-hCG, and carcinoembryonic antigen are typically not elevated in rhabdomyosarcoma. [2]
  • Sources describe immunohistochemical positivity for myogenin and desmin in a reported paratesticular rhabdomyosarcoma case. [2]
  • Somatic RAS–MAPK mutations are present in approximately 30% of patients with fusion-negative rhabdomyosarcoma. [13]
  • Guidance from the American College of Physicians states clinicians should not perform HPV testing in average-risk women younger than 30 years. [19]
  • The American College of Physicians advises that clinicians may use a combination of cytology and HPV testing once every 5 years in average-risk women aged 30 years or older who prefer screening less often than every 3 years. [19]
  • The combined use of cervical cytology with HPV testing has been shown in meta-analysis to have moderate-level evidence for reducing the incidence of cervical cancer. [18]
Biology & pathways4 points
  • Sources report that germline pathogenic variants in HRAS cause Costello syndrome. [13][14]
  • Germline pathogenic variants in various RAS–MAPK pathway genes lead to a growing group of developmental disorders collectively called 'RASopathies'. [13]
  • Sources report a general down-regulation of energy production pathways in rhabdomyosarcoma. [10]
  • Sources suggest a hypoxic physiology in rhabdomyosarcoma and note involvement of anti-apoptotic processes in low-oxygen conditions. [10]

Sources

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

  1. GuidelineSoft Tissue Sarcoma, Version 2.2022, NCCN Clinical Practice Guidelines in Oncology · 2022
  2. Systematic reviewA Rare Pediatric Paratesticular Spindle Cell Rhabdomyosarcoma and Systematic Literature Review · 2026
  3. Systematic reviewRhabdomyosarcoma of the temporal bone in children - a systematic review · 2026
  4. Systematic reviewClinical features, treatment and prognosis of primary pulmonary rhabdomyosarcoma: A systemic review · 2025
  5. Systematic reviewA systematic review of combined surgery and brachytherapy approaches for children and young people with relapsed and refractory rhabdomyosarcoma (Local-REFoRMS) · 2024
  6. Systematic reviewA systematic review of early phase studies for children and young people with relapsed and refractory rhabdomyosarcoma: The REFoRMS-SR project · 2024
  7. GuidelineRelapsed rhabdomyosarcoma: treatment recommendations from the European pediatric soft tissue sarcoma study group (EpSSG) · 2025
  8. GuidelineEvidence-Based Surgical Guidelines for Treating Children With Rhabdomyosarcoma · 2025
  9. Meta-analysisEfficacy and safety of proton beam therapy for rhabdomyosarcoma: a systematic review and meta-analysis · 2023
  10. Meta-analysisDefining the gene expression signature of rhabdomyosarcoma by meta-analysis · 2006
  11. Meta-analysisFusion gene-negative alveolar rhabdomyosarcoma is clinically and molecularly indistinguishable from embryonal rhabdomyosarcoma · 2010
  12. Clinical trialInternational multicenter retrospective study on pleomorphic rhabdomyosarcoma (P-RMS), a PUSH platform study: outcome of primary localized disease · 2026
  13. Meta-analysisCancer in Multilineage Mosaic RASopathies due to Pathogenic Variants in HRAS or KRAS: A Systematic Review and Meta-analysis · 2024
  14. Meta-analysisCancer in Costello syndrome: a systematic review and meta-analysis · 2023
  15. Systematic reviewSpecial features of sarcomas developed in patients with Lynch syndrome: A systematic review · 2023
  16. Systematic reviewA systematic review of the prevalence of pathogenic or likely pathogenic germline variants in individuals with FOXO1 fusion-positive rhabdomyosarcoma · 2023
  17. Clinical trialGenome-Wide Copy-Number Landscape of Germ Cell Tumors With Synchronous Conventional and "Somatic-Type" Malignancy Components · 2026
  18. GuidelineThe Korean guideline for cervical cancer screening · 2015
  19. GuidelineCervical Cancer Screening in Average-Risk Women: Best Practice Advice From the Clinical Guidelines Committee of the American College of Physicians · 2015
  20. GuidelineScreening for Cervical Cancer: US Preventive Services Task Force Recommendation Statement · 2018
  21. GuidelineSEOM-GEICO Clinical Guidelines on cervical cancer (2023) · 2024
  22. GuidelineACOG Practice Bulletin Number 131: Screening for cervical cancer · 2012
  23. Systematic reviewFinancial costs of pediatric cancer management in Africa: systematic review · 2023
  24. GuidelineSoft tissue tumor imaging in adults: whole-body staging in sarcoma, non-malignant entities requiring special algorithms, pitfalls and special imaging aspects. Guidelines 2024 from the European Society of Musculoskeletal Radiology (ESSR) · 2025
  25. GuidelineCervical cancer · 2013
  26. Systematic reviewRole of Fine Needle Aspiration Cytology in Paratesticular Neoplasms-A Case Reports-Based Systematic Review · 2024
  27. GuidelineCervical cancer screening · 2014
  28. GuidelineAmerican Brachytherapy Society consensus guidelines for adjuvant vaginal cuff brachytherapy after hysterectomy · 2012
  29. Systematic reviewEfficacy and feasibility of treating adults with paediatric chemotherapy protocols in Ewing sarcoma, rhabdomyosarcoma and osteosarcoma - A systematic review by the Australia and New Zealand Sarcoma Association clinical practice guidelines working party · 2026
  30. Meta-analysisMetronomic chemotherapy for paediatric extracranial solid tumours: a systematic review and meta-analysis of randomised clinical trials · 2024
  31. GuidelineProton beam therapy indications in the Kingdom of Saudi Arabia: A SARO clinical practice guideline · 2025
  32. Review articleHighly conformal whole abdominopelvic radiotherapy: Consensus delineation guidelines from EpSSG and QUARTET · 2026
  33. Meta-analysisSystematic review of ablation techniques for the treatment of malignant or aggressive benign lesions in children · 2014
  34. Systematic reviewCranial Rhabdomyosarcoma Masquerading as Infectious Mastoiditis: Case Report and Literature Review · 2018
  35. Meta-analysisPrevalence, Risk Factors and Management of Ocular Rhabdomyosarcoma (ORMS) in Africa: A Systematic Review · 2025
  36. Systematic reviewPleomorphic Rhabdomyosarcoma: A Systematic Review with Outcome Analysis and Report of a Rare Abdominal Wall Lesion · 2023
  37. Systematic reviewDiagnostic pitfalls: soft-tissue sarcomas initially misdiagnosed as benign vascular anomalies-a case report and systematic review · 2025
  38. Systematic reviewPredictive Factors Associated With Radiation Myelopathy in Pediatric Patients With Cancer: A PENTEC Comprehensive Review · 2024

What supports this page

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

Guideline
62
Meta-analysis
166
Systematic review
84
Randomized trial
7
Clinical trial
41
Observational
2
Case report
325
Review
1560
Preclinical
0
Other
18

Living document — last change June 19, 2026: Cancer page updated. 5 recent updates logged.

Pooled evidence across studies

PubMed
  • Benign lesion counts: 1.5 patients (1–2 across studies) · (regimen unspecified)
    4 studies · 0% agree · heterogeneous28474256
  • Tumor stage distribution: 3 tumors (2–4 across studies) · (regimen unspecified)
    4 studies · 0% agree · heterogeneous9553806
  • Stage among deaths: 2 patients (2–3 across studies) · (regimen unspecified)
    3 studies · 67% agree · moderate · 1 flagged9553806
  • ASCVD incidence: HR 1.56 (0.91–2.8 across studies) · (regimen unspecified)
    3 studies · 33% agree · heterogeneous40445187
  • Tumor laterality: 2 patients (1–3 across studies) · (regimen unspecified)
    2 studies · 0% agree · heterogeneous9553806
  • Microscopic subtype: 6.5 tumors (2–11 across studies) · (regimen unspecified)
    2 studies · 0% agree · heterogeneous9553806

Numbers pooled deterministically across 65 published studies that passed our figure-accuracy checks — a quick synthesis, not a formal meta-analysis. Updated 8/13/2026.

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
1Cisplatin ChemotherapyHuman trial / meta-analysis2—
2Topotecan OtherHuman trial / meta-analysis1—
3Pembrolizumab ImmunotherapyAnimal only1—
4Vincristine OtherInsufficient evidence3—
5Actinomycin D OtherInsufficient evidence2—
6Cyclophosphamide ChemotherapyInsufficient evidence2—
7Doxorubicin ChemotherapyInsufficient evidence1—
8Etoposide OtherInsufficient evidence1—
9Genistein OtherInsufficient evidence1—
10Ifosfamide OtherInsufficient evidence1—

Ranked across 10 compounds with published studies for this cancer. Updated 8/13/2026.

Medicines & supplements studied for Rhabdomyosarcoma

PubMedFDAClinicalTrials.gov

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

Medicines · 10

CisplatinHuman trial / meta-analysisReported positive1 human

Includes human trial or meta-analysis evidence.

Largest credible effect: treatment_completion 96%, n=92 PMID 23151423 · response rates 13–77 across 9 studies

Most authoritative study: Weekly topotecan and cisplatin (TOPOCIS) as neo-adjuvant chemotherapy for locally-advanced squamous cervical carcinoma: Results of a phase II multicentric study

ChemotherapyFDA off-labelPhase 22 studiesFull profile →
TopotecanHuman trial / meta-analysisReported positive1 human

Includes human trial or meta-analysis evidence.

Largest credible effect: treatment_completion 96%, n=92 PMID 23151423 · response rates 13–77 across 9 studies

Most authoritative study: Weekly topotecan and cisplatin (TOPOCIS) as neo-adjuvant chemotherapy for locally-advanced squamous cervical carcinoma: Results of a phase II multicentric study

Based on a single study.
OtherFDA off-labelPhase 21 studyFull profile →
PembrolizumabAnimal onlyReported positive1 animal

Animal studies only — no human data.

Most authoritative study: Targeting PI3Kα increases the efficacy of anti-PD-1 antibody in cervical cancer

No human studies yet · No numeric effect sizes reported · Based on a single study.
ImmunotherapyFDA approved1 studyFull profile →
VincristineInsufficient evidenceReported positive

No primary experimental studies yet.

Largest credible effect: chemotherapy duration 10 months 10 mo, n=1 PMID 11558020 · effect sizes 2–10 across 2 studies

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

No human studies yet · Effect sizes reported in only 1 of 3 studies · All studies are small (n < 30).
OtherFDA approved3 studiesFull profile →
Actinomycin DInsufficient evidenceReported positive

No primary experimental studies yet.

Largest credible effect: chemotherapy duration 10 months 10 mo, n=1 PMID 11558020 · effect sizes 2–10 across 2 studies

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

No human studies yet · Effect sizes reported in only 1 of 2 studies · All studies are small (n < 30).
Other2 studiesFull profile →
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).
ChemotherapyFDA off-label2 studiesFull 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.
ChemotherapyFDA off-label1 studyFull profile →
EtoposideInsufficient evidenceReported positive

No primary experimental studies yet.

Largest credible effect: tumor_free_survival 24 mo, n=1 PMID 37660086

Most authoritative study: Malignant germ cell tumor of fallopian tube with rhabdomyosarcoma: a case report and literature review

No human studies yet · Based on a single study.
OtherFDA off-label1 studyFull profile →
GenisteinInsufficient evidenceReported positive

No primary experimental studies yet.

Most authoritative study: Biological activities and therapeutic potential of soy isoflavones: a focus on anticancer activity

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

No primary experimental studies yet.

Largest credible effect: chemotherapy duration 10 months 10 mo, n=1 PMID 11558020 · effect sizes 2–10 across 2 studies

Most authoritative study: Pleuropulmonary blastoma

No human studies yet · Based on a single study.
OtherFDA off-label1 studyFull profile →

What recent studies report in Rhabdomyosarcoma

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

60 studies11 human4 animal1 lab⚠ Conflicting evidenceMechanism (30)Supportive care (3)Trial (2)

Tracking 60 published studies of Rhabdomyosarcoma: 11 in humans, 4 in animals, 1 in the lab, 44 reviews/other.

Reported direction across studies: 21 positive, 7 mixed, 1 negative, 31 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 Rhabdomyosarcoma.

Compounds with studies mentioning Rhabdomyosarcoma

Cisplatin (2)Genistein (1)Cyclophosphamide (1)Actinomycin d (1)Vincristine (1)Pembrolizumab (1)Etoposide (1)Topotecan (1)
Meta-analysisTrialMixed resultsModerate evidenceTier 4 · clinical

Comparative effectiveness of immune checkpoint inhibitors in squamous vs. non-squamous cervical carcinoma: a systematic review and meta-analysis of randomized controlled trials

Translational cancer research · Jun 2026 · systematic review and meta-analysis of randomized controlled trials

metastatic cervical cancercervical squamous cell carcinoma (SCC)cervical non-squamous cell carcinoma (non-SCC)

The authors performed a systematic review and meta-analysis of four randomized controlled trials evaluating immune checkpoint inhibitors in metastatic cervical cancer, comparing outcomes in squamous versus non-squamous histologies. They report that ICI therapy improved progression-free and overall survival overall, but benefits varied by histology and by ICI class (anti-PD-1, anti-PD-L1, anti-PD-1/CTLA-4).

Reported effects: ORR anti-PD-1 in SCC vs control, p P&lt;0.001 · ORR anti-PD-1 in non-SCC vs control, p P=0.23 · +9 more

Studied with: anti-PD-1/CTLA-4 combination.

Key findings
  • Four eligible randomized controlled trials were included in the meta-analysis.
  • ICI therapy significantly improved PFS and OS in both SCC and non-SCC patients compared with the control treatment.
  • Anti-programmed cell death protein 1 (anti-PD-1) monotherapy significantly increased the ORR in SCC patients (P&lt;0.001), while no significant improvement was observed in non-SCC patients (P=0.23).
  • Subgroup analysis showed that anti-PD-1 and anti-PD-1/CTLA-4 combination therapies significantly prolonged PFS in SCC patients (both P&lt;0.001), but not in non-SCC patients (P=0.13 and P=0.83, respectively).
  • Anti-programmed cell death ligand 1 (anti-PD-L1) therapy failed to improve PFS in SCC patients (P=0.22) but significantly enhanced PFS in non-SCC patients (P&lt;0.001).
  • OS subgroup analysis revealed that anti-PD-1, anti-PD-L1, and anti-PD-1/CTLA-4 therapies all significantly prolonged OS in SCC patients.
  • In non-SCC patients, only the anti-PD-1 subgroup exhibited a significant OS benefit (P=0.006), with no statistically significant differences observed in the anti-PD-L1 and anti-PD-1/CTLA-4 subgroups (P=0.08 and P=0.83, respectively).
Limitations: Meta-analysis included only four randomized controlled trials (small number of trials).; Abstract provides no patient-level data, aggregate trial-level pooling may mask heterogeneity.; Different ICI classes and combination regimens were pooled and subgroup analyses may be underpowered.; Details on control treatments, exact effect sizes (HRs, ORs, rates), and sample sizes are not reported in the abstract..

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

ReviewReported positivePreclinical onlyTier 1 · lab

Biological activities and therapeutic potential of soy isoflavones: a focus on anticancer activity

Molecular biology reports · May 2026 · narrative review

Genisteinbreast cancerovarian cancerprostate cancergliomaneuroblastomahepatocellular carcinomalung cancerbladder cancerosteosarcomarhabdomyosarcoma

This is a narrative review of the biological activities and potential therapeutic roles of soy isoflavones (including genistein and daidzein). The authors summarize proposed anticancer mechanisms (estrogen receptor modulation, apoptosis, anti-angiogenesis, epigenetic effects, etc.) and report that in vitro and in vivo studies have shown promising results across a range of tumor types. They conclude that further research—especially studies combining isoflavones with established chemotherapeutics—is needed.

Studied with: chemotherapeutic agents.

Key findings
  • Soy isoflavones (genistein, daidzein) have estrogenic and non-estrogenic activities including anti-inflammatory, antioxidant, and immunomodulatory effects.
  • Proposed anticancer mechanisms include modulation of estrogen receptors, copper ion-dependent induction of cell death, promotion of apoptosis, inhibition of angiogenesis and metastasis, regulation of epigenetic processes, and effects on platelet function.
  • Because of estrogen receptor interactions, isoflavones have been studied particularly in hormone-dependent cancers such as breast, ovarian, and prostate cancer.
  • In vitro and in vivo studies have reported promising results in multiple malignancies (gliomas, neuroblastoma, hepatocellular carcinoma, lung and bladder cancers, osteosarcoma, rhabdomyosarcoma).
  • Authors recommend further investigation, particularly combining isoflavones with established chemotherapeutics, to evaluate potential synergy.
Limitations: This article is a narrative review and does not present new clinical trial data.; The evidence summarized is primarily preclinical (in vitro and in vivo) with no clinical trial data provided in the abstract.; Mechanistic proposals are not proven clinical effects and require further experimental and clinical validation.; Safety and efficacy in patients, optimal dosing, and interactions with standard therapies are not established in this review..

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

Human · observationalMechanismReported positiveLimited evidenceTier 3 · early humann = 11

Ovarian Sertoli-Leydig cell tumors with heterologous rhabdomyosarcoma: Clinicopathologic features and molecular analysis highlighting recurrent genetic alterations

Histopathology · Apr 2026 · case series

ovarian Sertoli-Leydig cell tumorrhabdomyosarcoma (heterologous component)ovarian neoplasms

This case series of 11 ovarian Sertoli-Leydig cell tumors with heterologous rhabdomyosarcoma describes clinicopathologic features and molecular findings. All 7 tumors analyzed by next-generation sequencing had DICER1 hotspot mutations, most (6/7) also had a second loss-of-function DICER1 mutation, and additional mutually exclusive TERT promoter or TP53 alterations were observed. Component-specific sequencing in two cases showed shared DICER1 hotspots between SLCT and RMS components, supporting a clonal origin.

Reported effects: n_cases 11, n=11 · embryonal_RMS_count 10, n=11 · +17 more

Key findings
  • We report clinicopathologic features of 11 ovarian SLCTs with heterologous RMS (positivity for desmin and myogenin);
  • Ten tumors were in keeping with embryonal RMS and 1 with pleomorphic RMS.
  • The patients showed a bimodal age distribution: seven patients (64%) were aged 33&#x2009;years or younger (mean 20) and four patients (36%) were aged 52&#x2009;years or older (mean 60).
  • All tumors were unilateral.
  • Eight of 11 cases (73%) contained other heterologous elements, including gastrointestinal-type mucinous epithelium (5 cases) and immature cartilage (3 cases).
  • Seven of 11 cases (64%) underwent next-generation sequencing analysis.
  • All tumors tested molecularly (7/7, 100%) harbored hotspot DICER1 mutations.
  • Of these, six cases (86%) also carried a second nonsense or frameshift loss-of-function DICER1 mutation.
  • In addition to DICER1 mutations, TERT c.-124C&gt;T promoter (4 cases) or TP53 mutations (3 cases) were present in all cases and were mutually exclusive.
  • Component-specific analysis in two cases revealed shared common DICER1 hotspot mutations in both the SLCT and RMS components, supporting a clonal origin.
  • In one case, a TERT promoter c.-124C&gt;T somatic mutation was present only in the RMS component; in the other case the TERT promoter mutation was found in both components while a BRAF p.V600E mutation was exclusive to the RMS component.
Limitations: Small sample size (11 cases).; Retrospective case series design without systematic clinical outcome data reported in the abstract.; Next-generation sequencing was performed in only a subset of cases (7/11).; Component-specific molecular analysis was limited to two cases.; The abstract does not report whether DICER1 mutations were somatic versus germline in all cases..

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

ReviewMechanismInconclusiveLimited evidenceTier 3 · early human

DICER1 -Associated Gynecologic Neoplasms: An Update and Review

Advances in anatomic pathology · Jan 2026 · narrative review

gynecologic neoplasmsembryonal rhabdomyosarcomaSertoli-Leydig cell tumorpleuropulmonary blastoma-like peritoneal sarcomaadenosarcomagynandroblastomajuvenile granulosa cell tumorSertoli cell tumorDICER1-related Wilms-like uterine tumor

This narrative review summarizes how germline and somatic DICER1 mutations are associated with a range of benign and malignant gynecologic neoplasms and describes their shared morphologic features. The authors note that a germline loss-of-function DICER1 mutation is often followed by a somatic hotspot (second-hit) mutation in tumors, and they recommend that recognition of characteristic morphology should prompt genetic testing and surveillance for patients and families. The review proposes the term "DICER1-related primitive polyphenotypic neoplasm" to encompass the diverse histologic features of these tumors.

Key findings
  • DICER1 is crucial for microRNA biogenesis and maturation.
  • Germline DICER1 mutations are associated with increased risk of a wide range of benign and malignant neoplasms; the same tumors can also arise sporadically via somatic DICER1 mutations.
  • In syndromic patients, a germline loss-of-function DICER1 mutation is usually followed by a somatic hotspot mutation in the tumor as a second hit.
  • DICER1-associated gynecologic neoplasms most commonly include embryonal rhabdomyosarcoma and moderately to poorly differentiated Sertoli-Leydig cell tumor, with several less frequent tumor types also described.
  • DICER1-mutant gynecologic neoplasms frequently share characteristic morphology (primitive mesenchyme, fetal-type epithelium/cartilage, rhabdomyoblastic and/or neuroectodermal differentiation, osteoid formation, and anaplasia).
  • Recognition of these distinctive morphologic features should prompt consideration of DICER1-associated neoplasm and genetic testing to facilitate surveillance for patients and families.
  • The morphologic spectrum of most DICER1-mutant gynecologic neoplasms appears wider than that of any known type of sarcoma.
  • The authors propose the term "DICER1-related primitive polyphenotypic neoplasm" to better capture the diverse histologic features.
Limitations: Narrative review without description of systematic search or methods — potential selection bias in included reports.; No primary data or quantitative synthesis (no new experimental or cohort data presented).; Extent of evidence, frequency estimates, and outcomes are not quantified in the abstract..

Summarizes the association between DICER1 mutations and a spectrum of gynecologic tumors and highlights implications for pathologic recognition and genetic testing.

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

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

ReviewMechanismInconclusiveLimited evidenceTier 4 · clinical

DICER1 Syndrome: What Do We Know of the Pathogenetic Mechanisms?

Cancers · Sep 2025 · review

pleuropulmonary blastomaSertoli-Leydig cell tumorcystic nephromacervical embryonal rhabdomyosarcomacystic lung lesionsthyroid follicular nodular disease

This is a narrative review of DICER1 syndrome, a hereditary cancer predisposition caused by germline loss-of-function variants in DICER1 and characteristic second somatic hotspot mutations in the RNase IIIb domain. The review summarizes the range of associated benign and malignant tumors, describes DICER1's role as an endoribonuclease in RNA interference and microRNA processing, and concludes that the exact molecular mechanisms linking altered DICER1 function to tumorigenesis remain incompletely understood and require further research.

Key findings
  • DICER1 syndrome is a hereditary cancer predisposition syndrome with a broad phenotype including pleuropulmonary blastoma, Sertoli-Leydig cell tumor, cystic nephroma, cervical embryonal rhabdomyosarcoma, cystic lung lesions, and thyroid follicular nodular disease.
  • The syndrome is caused by loss-of-function germline variants in the DICER1 gene and DICER1-related tumors commonly have second somatic hotspot variants in the RNase IIIb domain.
  • DICER1 encodes an endoribonuclease important for RNA interference and microRNA biogenesis.
  • The review highlights gaps in knowledge about the precise molecular mechanisms by which altered DICER1 function contributes to tumorigenesis and calls for more research.
Limitations: Narrative review without original experimental or patient-level data.; Conclusions limited by existing incomplete knowledge of molecular mechanisms as noted by the authors.; Review methodology is not described in the abstract (e.g., not specified as a systematic review), so selection bias in covered literature is possible..

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

Lab · in vitroReported positivePreclinical onlyTier 1 · lab

Chrysin induces cell death and inhibits migration and invasion in squamous cervical carcinoma using a three-dimensional cell culture model

Brazilian journal of medical and biological research = Revista brasileira de pesquisas medicas e biologicas · Aug 2025

squamous cell cervical carcinomacervical cancer

This laboratory study used a 3D spheroid model of the HPV-16-positive SiHa cervical cancer cell line to test the natural flavonoid chrysin. Chrysin caused concentration-dependent reductions in cell viability and spheroid size, and inhibited spheroid migration and invasion; these effects were associated with decreased MMP-2 and VEGF production. The authors state these results support further in vivo preclinical studies.

Key findings
  • Chrysin treatment exhibited concentration-dependent cytotoxic and cytostatic effects, reducing cell proliferation and decreasing SiHa spheroid size.
  • Chrysin inhibited cell migration and invasion in spheroid assays.
  • Chrysin decreased production of MMP-2 and VEGF, which the authors link to the observed anti-migratory and anti-invasive effects.
  • Experiments were performed in a 3D spheroid culture using the SiHa (HPV-16-positive) human cervical cancer cell line.
Limitations: In vitro study only (3D cell culture); no animal or human data reported.; Single cell line (SiHa) studied, limiting generalizability across cervical cancers.; Abstract provides no quantitative dose levels, sample sizes, statistical values, or detailed methods.; Mechanistic detail limited to measured decreases in MMP-2 and VEGF; causal pathways not fully established in this report..

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

Animal studyReported positivePreclinical onlyTier 2 · animal

A humanized anaplastic lymphoma kinase (ALK)-directed antibody-drug conjugate with pyrrolobenzodiazepine payload demonstrates efficacy in ALK-expressing cancers

Nature communications · Aug 2025 · xenograft antitumor assays

neuroblastomarhabdomyosarcomacolorectal carcinomamelanomaovarian carcinomabreast carcinoma

This study tested a humanized antibody-drug conjugate called CDX0239-PBD in ALK-expressing cancer models. In cell lines, it was taken up by ALK-positive neuroblastoma cells and killed them in a way that depended on surface ALK expression. In mouse xenograft models, it produced strong antitumor activity and complete responses were maintained in several ALK-expressing cancers.

Key findings
  • ALK RNA, protein, and tumor cell surface expression was elevated in multiple pediatric and adult malignancies with minimal expression in childhood normal tissues.
  • CDX0239-PBD was internalized in ALK-expressing neuroblastoma cell lines with cell surface expression-dependent cytotoxicity.
  • CDX0239-PBD exhibited potent antitumor efficacy including maintained complete responses in ALK-expressing patient and cell line-derived neuroblastoma, fusion-positive rhabdomyosarcoma, and colorectal carcinoma xenograft models.
Limitations: Preclinical study only; no human treatment data are reported in the abstract.; Efficacy was shown in cell lines and xenograft mouse models, which may not predict clinical benefit.; No quantitative effect sizes, dosing details, or toxicity results are provided in the abstract..

The abstract describes a preclinical anticancer antibody-drug conjugate targeting ALK-expressing tumors.

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

Human · observationalSupportive careMixed resultsModerate evidenceTier 3 · early humann = 267586

Risk of atherosclerotic cardiovascular disease after cancer diagnosis: findings from 3 prospective cohort studies

Journal of the National Cancer Institute · Aug 2025 · prospective cohort study

Supportive carecervical cancerHodgkin lymphomaprostate cancerbreast cancercolorectal cancerlung cancerendometrial canceroral cavity and pharynx cancerkidney cancerovarian cancersarcomamelanomaleukemia

Researchers followed participants in three large prospective cohorts for up to 36 years to examine whether a cancer diagnosis was associated with later atherosclerotic cardiovascular disease (ASCVD). They documented 4,334 new ASCVD events among 49,603 incident cancer cases and found that cervical cancer and Hodgkin lymphoma were associated with higher ASCVD risk, prostate cancer with slightly lower risk, and that ASCVD risk trajectories over time varied by cancer type (for example, breast cancer survivors had lower ASCVD risk for the first 7.5 years, then risk increased).

Reported effects: new-onset ASCVD events among incident cancer cases 4334, n=49603 · cervical cancer HR 1.56 [1.06–2.29] · +6 more

Key findings
  • During up to 36 years of follow-up, 4,334 new-onset ASCVD events among 49,603 incident cancer cases were documented.
  • Cervical cancer was associated with increased ASCVD incidence (HR = 1.56, 95% CI = 1.06 to 2.29).
  • Hodgkin lymphoma was associated with increased ASCVD incidence (HR = 2.80, 95% CI = 1.89 to 4.15).
  • Prostate cancer was associated with lower ASCVD incidence (HR = 0.91, 95% CI = 0.85 to 0.97).
  • Breast cancer survivors experienced lower ASCVD risk during the first 7.5 years after diagnosis, but risk gradually increased afterward (Pnonlinearity = .01).
  • ASCVD risk increased over time among patients with cancers of the colorectum (P = .003), lung (P = .002), and endometrium (P = .04).
  • No statistically significant association with ASCVD risk was observed for cancers of the oral cavity and pharynx, kidney, or ovary; sarcoma; melanoma; or leukemia.
Limitations: Observational cohort design cannot establish causality.; Potential for residual confounding despite multivariable adjustment.; Cohorts consist of nurses and health professionals, which may limit generalizability to other populations.; Abstract does not report details on cancer stage or treatments, which could influence ASCVD risk..

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

ReviewMechanismInconclusiveModerate evidenceTier 4 · clinical

The Histological Spectrum of DICER1-Associated Neoplasms

Pediatric and developmental pathology : the official journal of the Society for Pediatric Pathology and the Paediatric Pathology Society · Jul 2025 · narrative review

pleuropulmonary blastomaSertoli-Leydig cell tumorcystic nephromauterine cervical embryonal rhabdomyosarcomathyroid follicular nodular disease

This narrative review summarizes the spectrum of benign and malignant tumors associated with germline heterozygous pathogenic DICER1 variants and emphasizes DICER1's role in miRNA processing and RNA interference. It highlights the most prevalent tumor types (pleuropulmonary blastoma, Sertoli-Leydig cell tumor, cystic nephroma, uterine cervical embryonal rhabdomyosarcoma, and thyroid follicular nodular disease), notes that these neoplasms are rare and often occur in children, and focuses on their histological features and molecular pathogenesis. The authors recommend that pathologists consider additional molecular testing and referral for genetic counseling and testing to facilitate earlier diagnosis.

Key findings
  • DICER1 syndrome is caused by germline heterozygous pathogenic variants in DICER1 and is essential in miRNA processing and RNA interference.
  • The syndrome is heterogeneous and includes a large variety of benign and malignant tumor types.
  • The most prevalent tumor types include pleuropulmonary blastoma, Sertoli-Leydig cell tumor, cystic nephroma, uterine cervical embryonal rhabdomyosarcoma, and thyroid follicular nodular disease.
  • These neoplasms are rare and particularly occur in the pediatric population.
  • Pathologists should be aware of the potential relationship to DICER1 syndrome to perform or suggest additional molecular pathologic analysis and refer patients and their parents for genetic counseling and testing.
  • The review emphasizes histological features of DICER1-related tumors and reflects on DICER1 molecular pathogenesis to raise awareness and facilitate earlier diagnosis.
Limitations: Review article that does not present new primary data.; Rarity of the tumor types limits the available evidence base and generalizability.; Focus is on histological features and molecular pathogenesis; the review does not provide primary clinical outcome or treatment data..

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

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

ReviewInconclusiveLimited evidenceTier 3 · early human

Atypical Pelvic Tumors in Children

Cancers · Feb 2025 · literature review and case series (selected cases presented)

sacrococcygeal teratomaovarian teratomarhabdomyosarcomaEwing sarcomacervical cancersmall cell neuroendocrine carcinoma of the ovaryEwing sarcoma/primitive neuroectodermal tumor (ES/PNET) of the ovarydiffuse large B-cell lymphoma of the ovariesovarian Sertoli-Leydig cell tumor (SLCT)neuroblastomaplexiform neurofibromaRosai-Dorfman disease

The authors review selected atypical pelvic tumors seen in children and present their own cases, focusing on imaging (MRI) characteristics. They describe a variety of reproductive-system and nervous-system tumors (including rare ovarian and testicular neoplasms, lymphomas, neuroblastoma, plexiform neurofibroma, and Rosai-Dorfman disease). The study sought radiological features that could help radiologists reach correct diagnoses but emphasizes that MRI cannot be interpreted alone and must be combined with clinical, syndromic and laboratory information.

Key findings
  • Selected atypical pelvic tumors in children are presented, many arising in the reproductive system (examples listed include cervical cancer, ovarian small cell neuroendocrine carcinoma, ES/PNET of the ovary, ovarian DLBCL, and ovarian SLCT associated with DICER1 syndrome).
  • Tumors originating from the nervous system discussed include neuroblastoma and plexiform neurofibroma (both NF1-associated and not associated with NF1).
  • Rosai-Dorfman disease involving pelvic and inguinal lymph nodes is presented as an additional differential diagnosis.
  • The authors aimed to identify radiological (MRI) features to guide radiologists toward correct diagnosis, but state that MR images must be interpreted alongside clinical picture, comorbidities/syndromes, and laboratory results.
Limitations: Study presents selected cases and a literature review rather than a systematic or comprehensive series; potential selection bias.; No sample size, quantitative diagnostic accuracy, or outcome data are reported in the abstract.; Imaging findings are descriptive and the abstract does not report validation of MRI features against definitive diagnoses or standardized criteria..

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

Browse all studies mentioning 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
Cisplatin1——1
Topotecan1——1
Vincristine—1—1
Actinomycin D—1——
Cyclophosphamide—1—1
Doxorubicin———1
Etoposide————
Genistein—1——
Ifosfamide————
Pembrolizumab—1——

Study mix

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

14 Human4 Animal1 Lab53 Review/other
Reported directionReported positive24Mixed results9Reported negative2Inconclusive37

Compounds with reported-positive results in 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.

Human evidence

Cisplatin2 positive1 human
Limitations: Single-patient case report — no control group and limited generalizability.; No dosing amounts/administration details for chemotherapy provided in the abstract.; Follow-up limited to 24 months as reported; longer-term outcomes beyond that are not reported in the abstract.; Outcomes from a single case cannot establish efficacy or safety of the treatment.; Single-arm Phase II design with no randomized or concurrent control group; Relatively short median follow-up (18 months) for survival outcomes.
Cited positive studies (2)
Topotecan1 positive1 human
Limitations: Single-arm Phase II design with no randomized or concurrent control group; Relatively short median follow-up (18 months) for survival outcomes; Adjuvant therapy was given to 55% of patients, which may confound interpretation of longer-term outcomes; Sample size (n=92) limits precision of survival and subgroup estimates; Endpoints are primarily response and short-term outcomes rather than long-term randomized efficacy.
Cited positive studies (1)
Preclinical only: lab / animal (8)
Vincristine2 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.; Single-patient case report (n=1) limits generalizability..
Cited positive studies (2)
Genistein1 positive
Limitations: This article is a narrative review and does not present new clinical trial data.; The evidence summarized is primarily preclinical (in vitro and in vivo) with no clinical trial data provided in the abstract.; Mechanistic proposals are not proven clinical effects and require further experimental and clinical validation.; Safety and efficacy in patients, optimal dosing, and interactions with standard therapies are not established in this review..
Cited positive studies (1)
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)
Actinomycin D1 positive
Limitations: Single-patient case report (n=1) limits generalizability.; No control or comparison group, so causality between interventions and outcome cannot be established.; No specific chemotherapy dose amounts provided in the abstract.; Short-to-moderate follow-up (two years) for long-term outcomes is limited..
Cited positive studies (1)
Pembrolizumab1 positive1 animal
Limitations: Predominantly preclinical evidence: in vitro assays and mouse xenograft (CDX/PDX) models; no controlled clinical trial data presented.; Human evidence limited to correlative tissue analyses and a single patient case report; results from one patient cannot establish efficacy.; Abstract does not report sample sizes, dosing, or statistical effect sizes for findings.; Safety, toxicity, and tolerability of PI3Kα inhibitor ± anti-PD-1 not reported in abstract.; Potential selection bias and lack of randomized comparison in described experiments (not specified in abstract)..
Cited positive studies (1)
Etoposide1 positive
Limitations: Single-patient case report — no control group and limited generalizability.; No dosing amounts/administration details for chemotherapy provided in the abstract.; Follow-up limited to 24 months as reported; longer-term outcomes beyond that are not reported in the abstract.; Outcomes from a single case cannot establish efficacy or safety of the treatment..
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)
Ifosfamide1 positive
Limitations: Single-patient case report (n=1) limits generalizability.; No control or comparison group, so causality between interventions and outcome cannot be established.; No specific chemotherapy dose amounts provided in the abstract.; Short-to-moderate follow-up (two years) for long-term outcomes is limited..
Cited positive studies (1)

Evidence at a glance: compounds studied in 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.

CisplatinHuman trial / meta-analysisReported positive1 human

Includes human trial or meta-analysis evidence.

Largest credible effect: treatment_completion 96%, n=92 PMID 23151423 · response rates 13–77 across 9 studies

Most authoritative study: Weekly topotecan and cisplatin (TOPOCIS) as neo-adjuvant chemotherapy for locally-advanced squamous cervical carcinoma: Results of a phase II multicentric study

TopotecanHuman trial / meta-analysisReported positive1 human

Includes human trial or meta-analysis evidence.

Largest credible effect: treatment_completion 96%, n=92 PMID 23151423 · response rates 13–77 across 9 studies

Most authoritative study: Weekly topotecan and cisplatin (TOPOCIS) as neo-adjuvant chemotherapy for locally-advanced squamous cervical carcinoma: Results of a phase II multicentric study

Based on a single study.
PembrolizumabAnimal onlyReported positive1 animal

Animal studies only — no human data.

Most authoritative study: Targeting PI3Kα increases the efficacy of anti-PD-1 antibody in cervical cancer

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

No primary experimental studies yet.

Largest credible effect: chemotherapy duration 10 months 10 mo, n=1 PMID 11558020 · effect sizes 2–10 across 2 studies

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

No human studies yet · Effect sizes reported in only 1 of 3 studies · All studies are small (n < 30).
Actinomycin DInsufficient evidenceReported positive

No primary experimental studies yet.

Largest credible effect: chemotherapy duration 10 months 10 mo, n=1 PMID 11558020 · effect sizes 2–10 across 2 studies

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

No human studies yet · Effect sizes reported in only 1 of 2 studies · All studies are small (n < 30).
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).
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.
EtoposideInsufficient evidenceReported positive

No primary experimental studies yet.

Largest credible effect: tumor_free_survival 24 mo, n=1 PMID 37660086

Most authoritative study: Malignant germ cell tumor of fallopian tube with rhabdomyosarcoma: a case report and literature review

No human studies yet · Based on a single study.
GenisteinInsufficient evidenceReported positive

No primary experimental studies yet.

Most authoritative study: Biological activities and therapeutic potential of soy isoflavones: a focus on anticancer activity

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

No primary experimental studies yet.

Largest credible effect: chemotherapy duration 10 months 10 mo, n=1 PMID 11558020 · effect sizes 2–10 across 2 studies

Most authoritative study: Pleuropulmonary blastoma

No human studies yet · Based on a single study.

What the research shows for Rhabdomyosarcoma

A plain-language summary of the reviewed studies OncoForge tracks for Rhabdomyosarcoma. It reports what those studies described, not a claim that any compound or therapy helps or harms Rhabdomyosarcoma. Most of this evidence is early, and findings often conflict.

  • Several narrative reviews summarize diagnosis, classification, and treatment approaches for pediatric and gynecologic rhabdomyosarcomas but do not present new controlled clinical-trial data.
  • Small case reports and a tiny case series describe patients with primary ovarian or pediatric rhabdomyosarcoma treated with surgery followed by combination chemotherapy (examples include regimens using vincristine, cyclophosphamide, and doxorubicin); these reports are observational with very small patient numbers.
  • One retrospective series of six pediatric ovarian rhabdomyosarcoma patients described tumor histology and stage at presentation (most embryonal; multiple patients with distant metastases) but provided limited, heterogeneous details on treatment and outcomes.
  • A preclinical animal study of an experimental antibody–drug conjugate (CDX0239‑PBD) showed activity in ALK-expressing cancer cell lines and produced tumor responses in mouse xenograft models that included rhabdomyosarcoma.
  • Overall the findings across the sources are heterogeneous and consist mainly of reviews, case reports/series, and preclinical work rather than controlled clinical trials.

Compounds studied in Rhabdomyosarcoma

Cyclophosphamide2 studies
In the provided reports cyclophosphamide was used as part of combination chemotherapy regimens (for example with vincristine ± doxorubicin or in VAC-type regimens) for rare ovarian and pediatric rhabdomyosarcomas; the evidence comes from case reports and small case series with limited follow-up.
Vincristine2 studies
Vincristine appears in multiple case reports/series as part of combination chemotherapy (including VAC-type or vincristine+doxorubicin+cyclophosphamide regimens) for ovarian and pediatric rhabdomyosarcoma; the data are observational and limited to very small numbers of patients.
Doxorubicin1 study
Doxorubicin was reported in a small case series combined with vincristine and cyclophosphamide after surgery in two children with primary ovarian rhabdomyosarcoma (both alive at 8 and 9 months post‑surgery in that report); this evidence is limited to a tiny number of cases with short follow-up.

Supportive & alternative options discussed

  • Exercise / prehabilitation: Also discussed as a supportive option for maintaining fitness and quality of life in cancer care; not evaluated in the studies summarized here.
  • Mind–body (MBSR / CBT): Also discussed as a supportive option for coping and symptom management in cancer care; not evaluated in the studies summarized here.
  • Acupuncture: Also discussed as a supportive option for symptom relief in some cancer settings; not evaluated in the studies summarized here.
  • Ketogenic / metabolic therapy: Also discussed by some as a complementary dietary approach in oncology contexts; not evaluated in the studies summarized here.

What we don’t know yet

  • Do the chemotherapy regimens reported in case reports improve long-term survival or disease-free intervals compared with other approaches? (no randomized or comparative trial data provided)
  • What are the optimal drugs, doses, and schedules for different rhabdomyosarcoma presentations (e.g., ovarian vs. other primary sites)?
  • How generalizable are outcomes from single-case reports and very small series to broader patient populations, including different ages and disease stages?
  • What are the long-term toxicities and late effects after the reported combination chemotherapies in these patients? (follow-up in reports is short)
  • Can the preclinical activity of experimental agents such as the antibody–drug conjugate translate to safety and benefit in humans with rhabdomyosarcoma? (no clinical data reported)
  • Are there predictive biomarkers that identify which patients might respond to specific systemic agents? (not established in the provided reports)
The available evidence in these reports is early and limited—dominated by narrative reviews, isolated case reports/small case series, and preclinical models—so it does not establish the safety or efficacy of specific agents for rhabdomyosarcoma.

Clinical trials in Rhabdomyosarcoma

33 ongoing · 70 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
14 stopped (terminated / withdrawn / suspended)

Search all trials on ClinicalTrials.gov →

Getting care & support

Nonprofit / Gov

Practical, vetted help for 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

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