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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

“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)

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

For each medicine above, search manufacturer and nonprofit programs at medicineassistancetool.org.

Questions to ask your oncologist

  1. Has my tumor been tested for Desmin / myogenin / MyoD1, and would the result open up targeted treatments or trials?
  2. Has my tumor been tested for PAX3–FOXO1 / PAX7–FOXO1, and would the result open up targeted treatments or trials?
  3. Has my tumor been tested for DICER1, and would the result open up targeted treatments or trials?
  4. Of the open trials I found (for example NCT03755739), am I eligible for any — here or at a larger cancer center?
  5. Which RMS subtype do I have (embryonal, alveolar, spindle/sclerosing, pleomorphic) and how does it change treatment?
  6. Is fertility-sparing surgery possible and safe in my case?
  7. Which chemo backbone is recommended for my age and subtype?
  8. Do I need radiation for margin-positive or nodal disease?
  9. Are there trials matched to my tumor’s drivers (e.g., PAX–FOXO1, PI3K/AKT/mTOR, MSI)?
  10. Can we send tissue for RNA fusion panel and DNA NGS now, and again at progression to capture targets (e.g., TFCP2, FGFR4, ALK)?
  11. Do we have enough banked tumor (FFPE + fresh-frozen) and a recent biopsy to qualify for molecular trials?
  12. What is the plan if there’s inadequate response after 2–3 cycles—what are our pre-agreed switch criteria?
  13. Am I a candidate for neoadjuvant therapy to improve the chance of an R0 resection?
  14. If disease is limited, could metastasectomy, ablation, or SBRT consolidate a systemic response?
  15. Would anthracycline cardioprotection (dexrazoxane) help preserve intensity without compromising efficacy?
  16. Should we involve a sarcoma specialty center or enroll via a national consortium (SARC, EORTC, NCI)?
  17. Are there basket trials for my biomarkers (FGFR4, ALK, NTRK, BET/BRD4, YAP/TEAD, MDM2)?
  18. Could we combine targeted agents with RT or chemo on a protocol to blunt resistance feedback (e.g., PI3K/mTOR + MEK)?
  19. What’s our VTE prevention plan and symptom-triggered pathway for urgent evaluation?
  20. How will we monitor for cardiac, renal, and neurotoxicity, and what dose-modification rules protect outcomes?
  21. Do I qualify for compassionate use / expanded access if a matched trial isn’t available?
  22. Is there a role for ctDNA as an adjunct to imaging for trend-tracking in my case?