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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.

Rhabdomyosarcoma (RMS) has two main types: embryonal (~70%, younger children, RAS-pathway mutations, favourable) and alveolar (~25%, adolescents, PAX3- or PAX7-FOXO1 fusion in ~80%, unfavourable). Fusion status has replaced histology in risk stratification since fusion-negative alveolar RMS behaves like embryonal. Sites range from orbit and parameningeal head and neck to genitourinary and extremity; risk groups combine site, size, nodal status, metastases, age and fusion status.

Therapy is VAC (vincristine, actinomycin D, cyclophosphamide) in North America or IVA (ifosfamide) in Europe, with local control by surgery and/or radiotherapy at week ~13. Key trial results: adding irinotecan (VAC/VI, ARST0531) did not improve outcomes but reduced cyclophosphamide exposure; maintenance vinorelbine-cyclophosphamide after standard therapy improved survival in high-risk localised disease (EpSSG RMS 2005, Lancet Oncol 2019); temsirolimus added to chemotherapy improved event-free survival in intermediate-risk disease (ARST1431, reported 2024). Metastatic disease (especially bone/marrow, age >10) has survival under 30% and is the target of the FaR-RMS international trial. Relapse is usually fatal outside low-risk cases.

State of the art today

  • Fusion status has replaced histology in risk stratification in both COG and European protocols.
  • Metastatic disease remains the unsolved problem; international FaR-RMS trial pools patients across continents.
Show survival figures (2)

Averages across everyone diagnosed, often years ago. Your stage, subtype, age, fitness and the treatment you receive matter more than the average, and the numbers are improving quickly.

  • Maintenance chemotherapy (EpSSG RMS 2005) is the first survival gain in decades for high-risk localised disease.
  • Temsirolimus is the first targeted agent to improve event-free survival in a randomised RMS trial (ARST1431).
Who it affects
Show survival figures (1)

Averages across everyone diagnosed, often years ago. Your stage, subtype, age, fitness and the treatment you receive matter more than the average, and the numbers are improving quickly.

  • The most common soft-tissue sarcoma of childhood (~350 cases per year in the US, ~3% of childhood cancers); survival ~70% overall, >90% low risk, <30% metastatic.

Where the cases are

Cases by country · GLOBOCAN 2022
All countries →

No country-level case numbers. This cancer is not mapped to a GLOBOCAN site.

Standard of care

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Low risk (embryonal, favourable site, complete resection)

VA ± reduced cyclophosphamide for 22-24 weeks; radiotherapy for microscopic residual disease.

COG ARST0331 / EpSSG RMS 2005
VincristineDactinomycin (actinomycin D)Cyclophosphamide
Intermediate risk

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.

COG ARST1431; EpSSG RMS 2005
VincristineDactinomycin (actinomycin D)CyclophosphamideIfosfamideIrinotecan (and liposomal irinotecan)VinorelbineTemsirolimusIMRT / IGRT (modern external beam)
High risk (metastatic)

Intensive multi-agent chemotherapy (VAC/IE, vincristine-irinotecan windows) with radiotherapy to primary and metastases; maintenance; FaR-RMS trial questions.

COG ARST0431; FaR-RMS
VincristineIrinotecan (and liposomal irinotecan)IfosfamideEtoposideDoxorubicinIMRT / IGRT (modern external beam)
Relapsed

Vinorelbine-cyclophosphamide, irinotecan-temozolomide, surgery/RT; trials (mTOR, FGFR4, CDK4/6, IGF-1R, B7-H3 CAR-T).

VinorelbineCyclophosphamideIrinotecan (and liposomal irinotecan)TemozolomideTemsirolimus

Subtypes & biomarkers

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Subtypes
  • Embryonal RMS (botryoid and spindle cell variants)
  • Alveolar RMS, PAX3/PAX7-FOXO1 fusion-positive
  • Fusion-negative alveolar RMS (behaves as embryonal)
  • Spindle cell / sclerosing RMS (MYOD1-mutant, aggressive; VGLL2-fused infantile, indolent)
  • Pleomorphic RMS (adults)
Biomarkers clinicians test
  • PAX3/PAX7-FOXO1 fusion status (FISH/RT-PCR)
  • Site (favourable: orbit, non-parameningeal head and neck, GU non-bladder/prostate)
  • IRS group and TNM stage
  • Nodal status (PET-CT, biopsy for extremity/paratesticular)
  • MYOD1 L122R (poor)
  • Age (>10 years, <1 year unfavourable)

Target prevalence in this cancer

History

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  1. 1972Intergroup Rhabdomyosarcoma Study (IRS-I) begins

    Establishes VAC and cooperative-group model.

  2. 1993PAX3-FOXO1 fusion identified (Galili, Barr)
    Gene fusion
  3. 2010Fusion-negative alveolar RMS behaves like embryonal (Williamson, JCO)

    Leads to fusion-based risk stratification.

  4. 2019EpSSG RMS 2005: maintenance vinorelbine-cyclophosphamide improves survival (Lancet Oncol)
    VinorelbineCyclophosphamide
  5. 2019ARST0531: irinotecan does not improve outcome but spares cyclophosphamide
    Irinotecan (and liposomal irinotecan)
  6. 2024ARST1431: temsirolimus + chemotherapy improves EFS in intermediate risk
    Temsirolimus

Pipeline

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Not mapped hereIntravenous mTOR inhibitor (rapamycin analogue)
Temsirolimus · Torisel
Not mapped hereSemi-synthetic vinca alkaloid
Vinorelbine · Navelbine
CAR-T cell therapyB7-H3

Open problems

  • Metastatic alveolar RMS: survival <30% for 30 years.
  • PAX3-FOXO1 is an undrugged fusion transcription factor.
  • Local-control morbidity in young children (orbit, bladder, prostate).
  • Relapse after intermediate-risk therapy is rarely curable.

Trials

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Recruiting now (live from ClinicalTrials.gov)

Recruiting trials near you · live from ClinicalTrials.gov
Rhabdomyosarcoma
condition: Rhabdomyosarcoma
Open on ClinicalTrials.gov →

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Expert centres

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Where the expertise is

Centres linked to this cancer in OnCo

Seeking a second opinion: ask your oncologist for a referral to a high-volume centre; most accept records and pathology by mail or telehealth. In the US, use the NCI's Find a Cancer Center tool or the nonprofit Cancer Commons, which navigates options for advanced cancers at no cost.

Questions to ask

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Bring to your appointment

Questions to ask your oncologist about Rhabdomyosarcoma

Generated from this cancer's standard of care, biomarkers, and pipeline · 17 questions

Newly diagnosed

  1. What is my exact diagnosis, stage, and grade, and which tests established them?
    Why: Everything else follows from an accurate stage and subtype.
  2. 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?
    Why: These results decide eligibility for targeted therapy, immunotherapy, and trials.
  3. Which subtype is my cancer, and does that change the recommended treatment?
    Why: Recognised subtypes for this cancer include Embryonal RMS, Alveolar RMS, PAX3/PAX7-FOXO1 fusion-positive, Fusion-negative alveolar RMS.
  4. Is germline (inherited) genetic testing recommended for me or my family?
    Why: Inherited variants can change treatment and matter for relatives.

Low risk (embryonal, favourable site, complete resection)

  1. For my situation (low risk (embryonal, favourable site, complete resection)), which of the standard options do you recommend and why?
    Why: Guideline options include: VA ± reduced cyclophosphamide for 22-24 weeks; radiotherapy for microscopic residual disease.
  2. Am I a candidate for Vincristine, Dactinomycin (actinomycin D), Cyclophosphamide, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.

Intermediate risk

  1. For my situation (intermediate risk), which of the standard options do you recommend and why?
    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.
  2. Am I a candidate for Vincristine, Dactinomycin (actinomycin D), Cyclophosphamide or related drugs, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.

High risk (metastatic)

  1. For my situation (high risk (metastatic)), which of the standard options do you recommend and why?
    Why: Guideline options include: Intensive multi-agent chemotherapy (VAC/IE, vincristine-irinotecan windows) with radiotherapy to primary and metastases; maintenance; FaR-RMS trial questions.
  2. Am I a candidate for Vincristine, Irinotecan (and liposomal irinotecan), Ifosfamide or related drugs, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.

Relapsed

  1. For my situation (relapsed), which of the standard options do you recommend and why?
    Why: Guideline options include: Vinorelbine-cyclophosphamide, irinotecan-temozolomide, surgery/RT; trials (mTOR, FGFR4, CDK4/6, IGF-1R, B7-H3 CAR-T).
  2. Am I a candidate for Vinorelbine, Cyclophosphamide, Irinotecan (and liposomal irinotecan) or related drugs, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.

Any stage

  1. Are there clinical trials I could join, for example of Temsirolimus, Vinorelbine, CAR-T cell therapy, B7-H3?
    Why: Trials are how the next standard of care is set; asking early keeps options open.
  2. Would a second opinion at a high-volume centre change anything, and can you help arrange it?
    Why: Rare or high-stakes decisions benefit from a centre that treats many similar patients.
  3. What supportive care (symptom control, nutrition, exercise, mental health, financial help) is available from the start?
    Why: Supportive care improves quality of life and helps patients complete treatment.
  4. I read that “Metastatic alveolar RMS: survival <30% for 30 years”. How does that affect my plan?
    Why: Open problems are where trials and second opinions matter most.
  5. I read that “PAX3-FOXO1 is an undrugged fusion transcription factor”. How does that affect my plan?
    Why: Open problems are where trials and second opinions matter most.

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Everything relevant

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Direct links plus the targets, companies, and technologies of this cancer's products.

technologies

12
Allogeneic stem cell transplantationAutologous stem cell transplant (high-dose therapy)BrachytherapyCAR-T cell therapyCardio-oncologyCytotoxic chemotherapyIMRT / IGRT (modern external beam)Isolated limb perfusion and infusionPET/CTProton therapySmall-molecule kinase inhibitorsTopoisomerase-I inhibitors (and ADC payloads)

targets

5
B7-H3CDK4/6FGFR2KMT2A (MLL) rearrangementTP53

drugs

10
Not mapped hereAlkylating agent (oxazaphosphorine prodrug)
Cyclophosphamide · Cytoxan / Endoxan
Not mapped hereActinomycin antibiotic (transcription inhibitor)
Dactinomycin (actinomycin D) · Cosmegen
ApprovedCytotoxic chemotherapy (anthracycline)
Doxorubicin · Adriamycin
Not mapped hereTopoisomerase II inhibitor (podophyllotoxin derivative)
Etoposide · VePesid / Etopophos / Toposar
ApprovedCytotoxic chemotherapy (alkylating agent)
Ifosfamide · Ifex
Not mapped hereTopoisomerase I inhibitor (camptothecin prodrug)
Irinotecan (and liposomal irinotecan) · Camptosar / Onivyde
Not mapped hereOral alkylating chemotherapy
Temozolomide · Temodar
Not mapped hereIntravenous mTOR inhibitor (rapamycin analogue)
Temsirolimus · Torisel
Not mapped hereVinca alkaloid (microtubule inhibitor)
Vincristine · Oncovin / Marqibo
Not mapped hereSemi-synthetic vinca alkaloid
Vinorelbine · Navelbine

companies

2
IpsenPfizer (incl. Seagen)

institutions

2
Children's Oncology Group (COG)Nationwide Children's Hospital

pathways

2
PI3K / AKT / mTORRAS / RAF / MEK / ERK (MAPK)

terms

2
Gene fusionLi-Fraumeni syndrome (germline TP53)

collections

2
Alex's Lemonade Stand Foundation (ALSF)St. Baldrick's Foundation

Latest papers

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Latest papers · live from Europe PMC
Open in Europe PMC

Query for this cancer: (TITLE:"Rhabdomyosarcoma" OR ABSTRACT:"Rhabdomyosarcoma") AND (treatment OR therapy OR trial OR survival OR diagnosis). Results are unfiltered search hits about Rhabdomyosarcoma, not a curated reading list.

Connected

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Pages like this

not linked directly; found by shared links

technologies

6
BrachytherapyCAR-T cell therapyCytotoxic chemotherapyIMRT / IGRT (modern external beam)PET/CTProton therapy

targets

4
B7-H3CDK4/6FGFR2TP53

drugs

10
Not mapped hereAlkylating agent (oxazaphosphorine prodrug)
Cyclophosphamide · Cytoxan / Endoxan
Not mapped hereActinomycin antibiotic (transcription inhibitor)
Dactinomycin (actinomycin D) · Cosmegen
ApprovedCytotoxic chemotherapy (anthracycline)
Doxorubicin · Adriamycin
Not mapped hereTopoisomerase II inhibitor (podophyllotoxin derivative)
Etoposide · VePesid / Etopophos / Toposar
ApprovedCytotoxic chemotherapy (alkylating agent)
Ifosfamide · Ifex
Not mapped hereTopoisomerase I inhibitor (camptothecin prodrug)
Irinotecan (and liposomal irinotecan) · Camptosar / Onivyde
Not mapped hereOral alkylating chemotherapy
Temozolomide · Temodar
Not mapped hereIntravenous mTOR inhibitor (rapamycin analogue)
Temsirolimus · Torisel
Not mapped hereVinca alkaloid (microtubule inhibitor)
Vincristine · Oncovin / Marqibo
Not mapped hereSemi-synthetic vinca alkaloid
Vinorelbine · Navelbine

companies

1
Pfizer (incl. Seagen)

institutions

2
Children's Oncology Group (COG)Nationwide Children's Hospital

pathways

2
PI3K / AKT / mTORRAS / RAF / MEK / ERK (MAPK)

terms

2
Gene fusionLi-Fraumeni syndrome (germline TP53)

collections

2
Alex's Lemonade Stand Foundation (ALSF)St. Baldrick's Foundation