The first 60 days: Rhabdomyosarcoma
A childhood soft-tissue cancer of muscle-like cells found anywhere from the eye socket to the bladder. Most children are cured with chemotherapy, surgery and radiation, and a fusion gene (PAX-FOXO1) now decides how intensively to treat. Below, week by week, is what OnCo's record of Rhabdomyosarcoma says about the first two months: the order is typical, the timing is yours to ask about. Sections appear only where the record has something to say. Orientation, not medical advice.
What happens now
Staging tests establish exactly what and where the cancer is. Everything else follows from the answers.
Ask which of these were tested and what the results were; see the report reader for what each value means.
These specialties appear in the standard of care for this cancer. In most centres they meet weekly as a tumour board to agree each plan; you can ask when yours was discussed and what was decided.
- SurgeonNamed in the standard of care for: Low risk (embryonal, favourable site, complete resection), Intermediate risk, Relapsed.
- Medical oncologistNamed in the standard of care for: Low risk (embryonal, favourable site, complete resection), Intermediate risk, High risk (metastatic), Relapsed.
- Clinical oncologist (radiotherapy)Named in the standard of care for: Low risk (embryonal, favourable site, complete resection), Intermediate risk, High risk (metastatic), Relapsed.
- Transplant and cell therapy teamNamed in the standard of care for: Low risk (embryonal, favourable site, complete resection), Intermediate risk, High risk (metastatic), Relapsed.
- Palliative and supportive care teamNamed in the standard of care for: High risk (metastatic).
A second opinion from a centre that treats many similar cases is normal, not rude; the standard of care tells you what to compare it against.
Each row is a setting from the standard of care, in the order it usually arises. Not all will apply to you; your stage and biomarkers decide which do. The guideline grade, where recorded, says how strong the evidence is.
VA ± reduced cyclophosphamide for 22-24 weeks; radiotherapy for microscopic residual disease.
VAC (or VAC/VI) 42 weeks with radiotherapy at week 13 (COG); IVA with maintenance vinorelbine-cyclophosphamide 6 months (EpSSG); temsirolimus-VAC/VI per ARST1431 where adopted.
Intensive multi-agent chemotherapy (VAC/IE, vincristine-irinotecan windows) with radiotherapy to primary and metastases; maintenance; FaR-RMS trial questions.
- 4.Relapsed
Vinorelbine-cyclophosphamide, irinotecan-temozolomide, surgery/RT; trials (mTOR, FGFR4, CDK4/6, IGF-1R, B7-H3 CAR-T).
Generated from this cancer's standard of care, biomarkers and open problems. Take the group that matches where you are. To tick, add your own and print, open the one-page appointment sheet.
Newly diagnosed
- What is my exact diagnosis, stage, and grade, and which tests established them?Everything else follows from an accurate stage and subtype.
- Which biomarkers have been tested on my tumour (for example PAX3/PAX7-FOXO1 fusion status, Site, IRS group and TNM stage, Nodal status, MYOD1 L122R), and what were the results?These results decide eligibility for targeted therapy, immunotherapy, and trials.
- Which subtype is my cancer, and does that change the recommended treatment?Recognised subtypes for this cancer include Embryonal RMS, Alveolar RMS, PAX3/PAX7-FOXO1 fusion-positive, Fusion-negative alveolar RMS.
- Is germline (inherited) genetic testing recommended for me or my family?Inherited variants can change treatment and matter for relatives.
Low risk (embryonal, favourable site, complete resection)
- For my situation (low risk (embryonal, favourable site, complete resection)), which of the standard options do you recommend and why?Guideline options include: VA ± reduced cyclophosphamide for 22-24 weeks; radiotherapy for microscopic residual disease.
- Am I a candidate for Vincristine, Dactinomycin (actinomycin D), Cyclophosphamide, and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.
Intermediate risk
- For my situation (intermediate risk), which of the standard options do you recommend and why?Guideline options include: VAC (or VAC/VI) 42 weeks with radiotherapy at week 13 (COG); IVA with maintenance vinorelbine-cyclophosphamide 6 months (EpSSG); temsirolimus-VAC/VI per ARST1431 where adopted.
- Am I a candidate for Vincristine, Dactinomycin (actinomycin D), Cyclophosphamide or related drugs, and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.
High risk (metastatic)
- For my situation (high risk (metastatic)), which of the standard options do you recommend and why?Guideline options include: Intensive multi-agent chemotherapy (VAC/IE, vincristine-irinotecan windows) with radiotherapy to primary and metastases; maintenance; FaR-RMS trial questions.
- Am I a candidate for Vincristine, Irinotecan (and liposomal irinotecan), Ifosfamide or related drugs, and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.
Relapsed
- For my situation (relapsed), which of the standard options do you recommend and why?Guideline options include: Vinorelbine-cyclophosphamide, irinotecan-temozolomide, surgery/RT; trials (mTOR, FGFR4, CDK4/6, IGF-1R, B7-H3 CAR-T).
- Am I a candidate for Vinorelbine, Cyclophosphamide, Irinotecan (and liposomal irinotecan) or related drugs, and what side effects should I expect?Knowing the expected toxicities helps you plan work, family, and supportive care.
Any stage
- Are there clinical trials I could join, for example of Temsirolimus, Vinorelbine, CAR-T cell therapy, B7-H3?Trials are how the next standard of care is set; asking early keeps options open.
- Would a second opinion at a high-volume centre change anything, and can you help arrange it?Rare or high-stakes decisions benefit from a centre that treats many similar patients.
- What supportive care (symptom control, nutrition, exercise, mental health, financial help) is available from the start?Supportive care improves quality of life and helps patients complete treatment.
- I read that “Metastatic alveolar RMS: survival <30% for 30 years”. How does that affect my plan?Open problems are where trials and second opinions matter most.
- I read that “PAX3-FOXO1 is an undrugged fusion transcription factor”. How does that affect my plan?Open problems are where trials and second opinions matter most.
Trials open now for this cancer in OnCo, largest phase first. Joining a trial is a decision like any other: ask what the comparison arm is, whether a placebo is used, and what happens if you leave. The cancer page searches ClinicalTrials.gov live for more.
- MASCT-I Combined With Doxorubicin and Ifosfamide for First-line Treatment of Advanced Soft Tissue SarcomaPhase 2 · recruiting · NCT06277154A Phase II Study Evaluating the Safety and Efficacy of MASCT-I Combined With Doxorubicin and Ifosfamide for First-line Treatment in Patients With Advanced Soft Tissue Sarcoma
- Study Of Palbociclib Combined With Chemotherapy In Pediatric Patients With Recurrent/Refractory Solid TumorsPhase 2 · active · NCT03709680PHASE 1/2 STUDY TO EVALUATE PALBOCICLIB (IBRANCE®) IN COMBINATION WITH IRINOTECAN AND TEMOZOLOMIDE OR IN COMBINATION WITH TOPOTECAN AND CYCLOPHOSPHAMIDE IN PEDIATRIC PATIENTS WITH RECURRENT OR REFRACTORY SOLID TUMORS
- Childhood Cancer Survivor Study (CCSS)Phase observational · active · NCT01120353Retrospective cohort with prospective follow-up of five-year survivors of childhood cancer diagnosed 1970-1999 at 31 North American centres, with sibling controls
Much of what helps in the first weeks is free if you know to ask: testing, helplines, rides and lodging, second opinions, trial travel.
- Rhabdomyosarcoma: the full pageA childhood soft-tissue cancer of muscle-like cells found anywhere from the eye socket to the bladder. Most children are cured with chemotherapy, surgery and radiation, and a fusion gene (PAX-FOXO1) now decides how intensively to treat.
- One-page appointment sheetYour questions, the words you may hear, what to bring, and space for the answers. Print it.
- Treatment sequencingWhich treatment tends to follow which, line by line.
- NavigatorStandard of care for your stage, what you have tried, and trials near you.
- Li-Fraumeni syndrome (germline TP53): Li-Fraumeni syndrome is an inherited fault in the TP53 gene giving a lifetime cancer risk near 100% in women and ~75% in men, with sarcomas, breast cancer, brain tumours, adrenal cancer and leukaemias often in childhood.
- Gene fusion: A gene fusion is two genes broken and joined together, creating a hybrid protein that can drive cancer.
Every term links to the glossary.