# Rhabdomyosarcoma

Source: https://onco.cc/cancers/rhabdomyosarcoma/  
OnCo record `rhabdomyosarcoma` (Cancer). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

A childhood soft-tissue sarcoma, a 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.

## Summary

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.

## Fields

- Kind: Cancer
- Last checked: 2026-09-08
- Tags: gap-fill; paediatric; sarcoma
- Group: paediatric
- Burden: 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.
- 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: 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)

## Sections of this record

The page is a hub with ten sections in reading order; large sections have their own page. The same plan as JSON: https://onco.cc/api/v1/cancers/rhabdomyosarcoma/sections.json

- Overview (on the hub): The TL;DR, the family this cancer belongs to, the organ, who gets it and what the state of the art is. https://onco.cc/cancers/rhabdomyosarcoma/#overview [4 state-of-the-art points]
- What it is (on the hub): Anatomy, the subtypes and how they differ, how it is staged, and where advanced disease spreads. https://onco.cc/cancers/rhabdomyosarcoma/#what-it-is [5 subtypes]
- Finding it (on the hub): How it shows itself, how it is confirmed, what screening exists, and the biomarkers clinicians test for. https://onco.cc/cancers/rhabdomyosarcoma/#finding-it [6 biomarkers]
- Treating it (on the hub): The standard of care by setting, the medicines, surgery and radiotherapy named in it, and the regimens behind them. https://onco.cc/cancers/rhabdomyosarcoma/#treating-it [4 settings, 1 regimen, 4 decisions with options]
- Evidence (on the hub): Trials recruiting now, the landmark trials, the key papers and what they mean, the latest literature, and the milestones year by year. https://onco.cc/cancers/rhabdomyosarcoma/#evidence [3 trials, 1 key paper, 6 milestones]
- The science (on the hub): The molecular landscape: the targets and how often each appears, the pathways, the mechanics stages and the preclinical models. https://onco.cc/cancers/rhabdomyosarcoma/#science [9 targets, 2 pathways]
- Where you are (own page): Cases by country, the UK and NHS pathway and other country lenses, and the expert centres with trials on record. https://onco.cc/cancers/rhabdomyosarcoma/where-you-are/ [1 centre]
- Living with it (on the hub): The decisions you may face, the aids that walk through them, the warnings on record, the first sixty days and the questions to ask. https://onco.cc/cancers/rhabdomyosarcoma/#living-with-it [17 questions, 6 red cards]
- What is coming (own page): Everything in development, the open problems and what is being done about them, the roadmaps, and what changed on this record. https://onco.cc/cancers/rhabdomyosarcoma/coming/ [11 medicines, 3 trials, 4 open problems]
- Data (own page): Every connected record, the notes, the JSON, Markdown and RDF twins, and where the record came from and when it was checked. https://onco.cc/cancers/rhabdomyosarcoma/data/ [58 connected records]

## Standard of care

- Low risk (embryonal, favourable site, complete resection): VA ± reduced cyclophosphamide for 22-24 weeks; radiotherapy for microscopic residual disease. ([Vincristine](https://onco.cc/drugs/vincristine/), [Dactinomycin (actinomycin D)](https://onco.cc/drugs/dactinomycin/), [Cyclophosphamide](https://onco.cc/drugs/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. ([Vincristine](https://onco.cc/drugs/vincristine/), [Dactinomycin (actinomycin D)](https://onco.cc/drugs/dactinomycin/), [Cyclophosphamide](https://onco.cc/drugs/cyclophosphamide/), [Ifosfamide](https://onco.cc/drugs/ifosfamide/), [Irinotecan (and liposomal irinotecan)](https://onco.cc/drugs/irinotecan/), [Vinorelbine](https://onco.cc/drugs/vinorelbine/), [Temsirolimus](https://onco.cc/drugs/temsirolimus/), [IMRT / IGRT (modern external beam)](https://onco.cc/technologies/imrt-igrt/))
- High risk (metastatic): Intensive multi-agent chemotherapy (VAC/IE, vincristine-irinotecan windows) with radiotherapy to primary and metastases; maintenance; FaR-RMS trial questions. ([Vincristine](https://onco.cc/drugs/vincristine/), [Irinotecan (and liposomal irinotecan)](https://onco.cc/drugs/irinotecan/), [Ifosfamide](https://onco.cc/drugs/ifosfamide/), [Etoposide](https://onco.cc/drugs/etoposide/), [Doxorubicin](https://onco.cc/drugs/doxorubicin/), [IMRT / IGRT (modern external beam)](https://onco.cc/technologies/imrt-igrt/))
- Relapsed: Vinorelbine-cyclophosphamide, irinotecan-temozolomide, surgery/RT; trials (mTOR, FGFR4, CDK4/6, IGF-1R, B7-H3 CAR-T). ([Vinorelbine](https://onco.cc/drugs/vinorelbine/), [Cyclophosphamide](https://onco.cc/drugs/cyclophosphamide/), [Irinotecan (and liposomal irinotecan)](https://onco.cc/drugs/irinotecan/), [Temozolomide](https://onco.cc/drugs/temozolomide/), [Temsirolimus](https://onco.cc/drugs/temsirolimus/))

## State of the art

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

## 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.

## Sources

- Wikipedia: https://en.wikipedia.org/wiki/Rhabdomyosarcoma
- NCI PDQ: childhood rhabdomyosarcoma: https://www.cancer.gov/types/soft-tissue-sarcoma/patient/rhabdomyosarcoma-treatment-pdq
- EpSSG RMS 2005 maintenance (Lancet Oncol 2019): https://doi.org/10.1016/S1470-2045(19)30617-5
- FaR-RMS trial: https://clinicaltrials.gov/study/NCT04625907

## Connected records

- cancers: [Alveolar soft part sarcoma](https://onco.cc/cancers/alveolar-soft-part-sarcoma/), [Childhood cancers (all types)](https://onco.cc/cancers/childhood-cancers/), [Childhood lung and airway tumours (pleuropulmonary blastoma, tracheobronchial tumours)](https://onco.cc/cancers/pleuropulmonary-blastoma/), [Desmoplastic small round cell tumour](https://onco.cc/cancers/desmoplastic-small-round-cell-tumour/), [Ewing sarcoma](https://onco.cc/cancers/ewing-sarcoma/), [Osteosarcoma](https://onco.cc/cancers/osteosarcoma/), [Sarcomas (soft tissue, bone, GIST)](https://onco.cc/cancers/sarcoma/), [Synovial sarcoma](https://onco.cc/cancers/synovial-sarcoma/), [Vaginal cancer](https://onco.cc/cancers/vaginal/)
- technologies: [Brachytherapy](https://onco.cc/technologies/brachytherapy/), [CAR-T cell therapy](https://onco.cc/technologies/car-t/), [Cytotoxic chemotherapy](https://onco.cc/technologies/cytotoxic-chemotherapy/), [IMRT / IGRT (modern external beam)](https://onco.cc/technologies/imrt-igrt/), [Methylation classifier for sarcomas](https://onco.cc/technologies/sarcoma-methylation-classifier/), [PET/CT](https://onco.cc/technologies/pet-ct/), [Proton therapy](https://onco.cc/technologies/proton-therapy/)
- targets: [B7-H3](https://onco.cc/targets/b7h3/), [CDK4/6](https://onco.cc/targets/cdk4-6/), [FGFR2](https://onco.cc/targets/fgfr2/), [FOXO1](https://onco.cc/targets/foxo1/), [MYOD1](https://onco.cc/targets/myod1/), [PAX3](https://onco.cc/targets/pax3/), [PAX7](https://onco.cc/targets/pax7/), [TP53](https://onco.cc/targets/tp53/)
- drugs: [Cobolimab](https://onco.cc/drugs/cobolimab/), [Cyclophosphamide](https://onco.cc/drugs/cyclophosphamide/), [Dactinomycin (actinomycin D)](https://onco.cc/drugs/dactinomycin/), [Doxorubicin](https://onco.cc/drugs/doxorubicin/), [Etoposide](https://onco.cc/drugs/etoposide/), [Ifosfamide](https://onco.cc/drugs/ifosfamide/), [Irinotecan (and liposomal irinotecan)](https://onco.cc/drugs/irinotecan/), [Temozolomide](https://onco.cc/drugs/temozolomide/), [Temsirolimus](https://onco.cc/drugs/temsirolimus/), [Vincristine](https://onco.cc/drugs/vincristine/), [Vinorelbine](https://onco.cc/drugs/vinorelbine/)
- companies: [Children's Oncology Group (COG)](https://onco.cc/companies/childrens-oncology-group/), [Innovative Therapies for Children with Cancer (ITCC)](https://onco.cc/companies/itcc/), [Pfizer (incl. Seagen)](https://onco.cc/companies/pfizer/)
- pathways: [PI3K / AKT / mTOR](https://onco.cc/pathways/pi3k-akt-mtor/), [RAS / RAF / MEK / ERK (MAPK)](https://onco.cc/pathways/ras-mapk/)
- terms: [FOXO1 fusion status and IRS group (rhabdomyosarcoma)](https://onco.cc/terms/foxo1-fusion-status/), [Gene fusion](https://onco.cc/terms/gene-fusion/), [Li-Fraumeni syndrome (germline TP53)](https://onco.cc/terms/li-fraumeni/)
- trials: [Childhood Cancer Survivor Study (CCSS)](https://onco.cc/trials/ccss/), [MASCT-I Combined With Doxorubicin and Ifosfamide for First-line Treatment of Advanced Soft Tissue Sarcoma](https://onco.cc/trials/nct06277154/), [Study Of Palbociclib Combined With Chemotherapy In Pediatric Patients With Recurrent/Refractory Solid Tumors](https://onco.cc/trials/nct03709680/)
- key papers: [Vinorelbine and continuous low-dose cyclophosphamide as maintenance chemotherapy in patients with high-risk rhabdomyosarcoma (RMS 2005): a multicentre, open-label, randomised, phase 3 trial](https://onco.cc/key-papers/paper-bisogno-lancet-oncol/)
- collections: [Alex's Lemonade Stand Foundation (ALSF)](https://onco.cc/collections/alexs-lemonade-stand/), [St. Baldrick's Foundation](https://onco.cc/collections/st-baldricks/)
- institutions: [Nationwide Children's Hospital](https://onco.cc/institutions/nationwide-childrens/)

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JSON: https://onco.cc/api/v1/entities/rhabdomyosarcoma.json