Appointment dossier — Ovarian Rhabdomyosarcoma
Bring this to your appointment. It summarizes what published studies report — it is not medical advice and does not say anything works. Decisions are yours and your care team’s.
Compounds studied in Ovarian Rhabdomyosarcoma
- Cyclophosphamide — Review evidence · 1 positive · PMID 18004572, 41474586
- Doxorubicin — Review evidence · 1 positive · PMID 18004572
- Vincristine — Review evidence · 1 positive · PMID 18004572, 41474586
- Actinomycin D — Review evidence · 0 positive · PMID 41474586
“Positive” means a study reported a positive result — most are early lab/animal work that may not translate to people.
Guideline-backed standard of care
Aim for complete macroscopic resection when feasible, Unilateral salpingo-oophorectomy often required, Nodal evaluation considered, especially for alveolar RMS given higher nodal risk, Fertility-sparing approaches only in highly selected early cases with multidisciplinary input, Plan en-bloc resection to avoid capsular rupture or tumor spill, Mark close/deep margins with clips to guide adjuvant radiation planning, If an unplanned (‘whoops’) resection occurred, restage and consider re-excision to achieve R0 before RT, In bulky disease, consider neoadjuvant chemotherapy to downstage before definitive surgery, For oligometastatic disease, discuss metastasectomy or ablation (lung/liver) in tumor board after systemic control, Coordinate ureteral stents or bowel resection with peri-chemo timing to minimize infectious complications, Consider for positive margins, nodal involvement, or unresectable/local recurrence, Pelvic RT planning must balance organ tolerance and prior surgeries, SBRT, Use IMRT/VMAT to spare bowel, bladder, rectum, and ovaries/uterus when organ preservation matters, Post-op RT, Spine/bone mets: consider SBRT for pain control and local control, Time RT around systemic therapy to minimize overlapping toxicities (e, Lung mets: SBRT or wedge resection discussed case-by-case after systemic response, VAC-based regimens (vincristine, actinomycin, cyclophosphamide) standard in pediatric/AYA RMS, Adult options, Platinum/taxane regimens, Clinical trial enrollment strongly encouraged due to rarity, Risk-adapted intensity with early response assessment (typically after 2–3 cycles), Consider VAC/IVA variants or VDC/IE-style intensity in AYA/fit adults when tolerated—individualize to comorbidity and goals, Anthracycline cardioprotection (dexrazoxane) and dose-capping strategies, Ifosfamide protocols: ensure mesna, aggressive hydration, and CNS/renal monitoring (encephalopathy, proximal tubulopathy), Maintenance concepts (e.g., low-dose alkylator/vinca), Anti-angiogenic TKIs (e, VAC (vincristine/actinomycin/cyclophosphamide) (pediatric/AYA), Doxorubicin/Ifosfamide variants (adult STS), Gemcitabine/Docetaxel (recurrent/palliative), VDC/IE (vincristine/doxorubicin/cyclophosphamide ↔ ifosfamide/etoposide) (high-risk/AYA-fit adult), IVA (ifosfamide/vincristine/actinomycin) (neoadjuvant/anthracycline-avoidant), VIT (vincristine/irinotecan/temozolomide) (relapsed/chemo-pretreated), High-dose Ifosfamide (salvage), Doxorubicin/Dacarbazine (± Ifosfamide) (adult STS legacy), Oral maintenance (vinorelbine + low-dose cyclophosphamide) (post-response/investigational), MSI-H/dMMR (rare), IGF1R/PI3K/AKT/mTOR: pathway alterations support trial eligibility, ctDNA/NGS, NTRK fusion (rare): TRK inhibitors (tumor-agnostic), TFCP2-fusion subset: ALK overexpression—consider ALK-focused trials and keratin-positive RMS recognition, PAX–FOXO1 epigenetic dependency: BET/BRD4 inhibition trials and transcriptional-complex disruption strategies, YAP/TEAD (Hippo dysregulation): early-phase TEAD inhibitor programs for resistance/stemness biology, p53–MDM2 axis: MDM2 antagonists in development—consider when TP53 wild-type and MDM2-high, DDR targeting (PARP/ATR) as radiosensitizers/combos in refractory disease—trial contexts only.
The established options for Ovarian Rhabdomyosarcoma — ask which apply to your case. Investigational options appear in the compounds list above.
Open recruiting trials (18)
- NCT03755739 · Phase 2 / Phase 3 — Trans-Artery/Intra-Tumor Infusion of Checkpoint Inhibitors Plus Chemodrug for Immunotherapy of Advanced Solid Tumors (China)
- NCT01174121 · Phase 2 — Immunotherapy Using Tumor Infiltrating Lymphocytes for Patients With Metastatic Cancer (United States)
- NCT06386146 · Phase 1 / Phase 2 — JAB-30355 in Patients With Advanced Solid Tumors Harboring TP53 Y220C Mutation (United States)
- NCT04851119 · Phase 1 / Phase 2 — Tegavivint for the Treatment of Recurrent or Refractory Solid Tumors, Including Lymphomas and Desmoid Tumors (United States)
- NCT06771219 · Phase 1 — SLV-154 Treatment of Metastatic Solid Tumors (United States)
- NCT06666270 · Phase 1 — Study of SYN818 for the Treatment of Advanced or Metastatic Solid Tumors (China)
- NCT05606692 · Phase 4 — Influences of Propofol and Sevoflurane Anesthesia in Ovarian Cancer (Anesthetics) (Taiwan)
- NCT06836128 · Phase 3 — The Combined Effect of N-Acetyl Cysteine and Metformin in Polycystic Ovary Syndrome Patients (Egypt)
- NCT06343870 · Phase 3 — Estradiol and Testosterone Subdermal Implants for Menopause Treatment (ESTIME) (Brazil)
- NCT06161025 · Phase 2 / Phase 3 — A Study of Raludotatug Deruxtecan (R-DXd) in Subjects With Platinum-resistant, High-grade Ovarian, Primary Peritoneal, or Fallopian Tube Cancer (United States)
- NCT07023484 · Phase 2 — Personalized Timing of Interval Debulking Surgery in Advanced Ovarian Cancer (China)
- NCT07050771 · Phase 2 — Comprehensive Multimodal Prehabilitation Alone or With Neoadjuvant Therapy Before Major Cancer Surgery (United States)
- NCT06014528 · Phase 2 — IN10018 in Combination With Pegylated Liposomal Doxorubicin (PLD) vs. Placebo in Combination With PLD for the Treatment of Platinum-resistant Recurrent Ovarian Cancer (China)
- NCT06971744 · Phase 2 — Autophagy Maintenance (AUTOMAIN) (United States)
- NCT07371104 · Phase 1 — Bioequivalence Study of DWZ2501 in Patients With Advanced BRCA-mutated High-grade Ovarian Cancer (South Korea)
- NCT07489287 · Phase 1 — GB-5267 for the Treatment Platinum-Resistant Ovarian, Peritoneal, or Fallopian Tube Cancer (United States)
- NCT04847063 · Phase 1 — Individual Response to Hyperthermic Intraperitoneal Chemotherapy (HIPEC) Treatment of Peritoneal Carcinomatosis From Peritoneal Mesothelioma or Atypical Mesothelial Proliferation or From Ovarian, Colorectal, or Appendiceal Histologies (United States)
- NCT07403201 · Early Phase 1 — The Effect of Therapy Combination in PCOS With Dlbs 3233 (Indonesia)
Most-relevant first: trials that name Ovarian Rhabdomyosarcoma, then broader trials you may still qualify for. Eligibility is decided by each trial's team — bring these NCT numbers to your appointment.
Financial help to look into
- PAN Foundation — Copay assistance funds by diagnosis (funds open and close as money allows). https://www.panfoundation.org/
- HealthWell Foundation — Copay and premium assistance funds by disease. https://www.healthwellfoundation.org/
- CancerCare — financial assistance — Limited grants plus free financial counseling. https://www.cancercare.org/financial
- Family Reach — Help with everyday living costs (rent, transport, food) during treatment. https://familyreach.org/
- NeedyMeds — Searchable directory of drug patient-assistance and discount programs. https://www.needymeds.org/
For each medicine above, search manufacturer and nonprofit programs at medicineassistancetool.org.
Questions to ask your oncologist
- Has my tumor been tested for Desmin / myogenin / MyoD1, and would the result open up targeted treatments or trials?
- Has my tumor been tested for PAX3–FOXO1 / PAX7–FOXO1, and would the result open up targeted treatments or trials?
- Has my tumor been tested for DICER1, and would the result open up targeted treatments or trials?
- Of the open trials I found (for example NCT03755739), am I eligible for any — here or at a larger cancer center?
- Which RMS subtype do I have (embryonal, alveolar, spindle/sclerosing, pleomorphic) and how does it change treatment?
- Is fertility-sparing surgery possible and safe in my case?
- Which chemo backbone is recommended for my age and subtype?
- Do I need radiation for margin-positive or nodal disease?
- Are there trials matched to my tumor’s drivers (e.g., PAX–FOXO1, PI3K/AKT/mTOR, MSI)?
- Can we send tissue for RNA fusion panel and DNA NGS now, and again at progression to capture targets (e.g., TFCP2, FGFR4, ALK)?
- Do we have enough banked tumor (FFPE + fresh-frozen) and a recent biopsy to qualify for molecular trials?
- What is the plan if there’s inadequate response after 2–3 cycles—what are our pre-agreed switch criteria?
- Am I a candidate for neoadjuvant therapy to improve the chance of an R0 resection?
- If disease is limited, could metastasectomy, ablation, or SBRT consolidate a systemic response?
- Would anthracycline cardioprotection (dexrazoxane) help preserve intensity without compromising efficacy?
- Should we involve a sarcoma specialty center or enroll via a national consortium (SARC, EORTC, NCI)?
- Are there basket trials for my biomarkers (FGFR4, ALK, NTRK, BET/BRD4, YAP/TEAD, MDM2)?
- Could we combine targeted agents with RT or chemo on a protocol to blunt resistance feedback (e.g., PI3K/mTOR + MEK)?
- What’s our VTE prevention plan and symptom-triggered pathway for urgent evaluation?
- How will we monitor for cardiac, renal, and neurotoxicity, and what dose-modification rules protect outcomes?
- Do I qualify for compassionate use / expanded access if a matched trial isn’t available?
- Is there a role for ctDNA as an adjunct to imaging for trend-tracking in my case?