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Multimodality Therapy
Source: Childhood Rhabdomyosarcoma Treatment (PDQ®)–Health Professional Version, National Cancer Institute.
Source updated: April 11, 2025 · Captured 2026-09-09.
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Context: Treatment Option Overview for Childhood Rhabdomyosarcoma
All children with rhabdomyosarcoma require multimodality therapy with systemic chemotherapy, in conjunction with either surgery, radiation therapy (RT), or both modalities to maximize local tumor control.[1-3] Surgical resection is performed before chemotherapy if it will not result in disfigurement, functional compromise, or organ dysfunction. If this is not possible, only an initial biopsy is performed.
Low-risk Group I (complete tumor resection, about 15% of patients) patients are treated with multiagent chemotherapy after surgical resection. Group II patients typically require chemotherapy and local tumor bed irradiation (about 20% of patients). Most patients (about 50%) have Group III (gross residual) disease.[4] After initial chemotherapy, Group III patients receive definitive RT for local control of the primary tumor. Some patients with initially unresected tumors may undergo delayed primary excision after induction chemotherapy to remove residual tumor before the initiation of RT. This is appropriate only if the delayed excision is deemed feasible with acceptable functional and cosmetic outcome and if a grossly complete resection is anticipated. If a delayed primary excision results in complete resection or microscopic residual disease, a modest (15%–30%) reduction in RT could be utilized.[5] Patients with Group IV disease (about 15%) receive chemotherapy and RT to the primary tumor and metastatic disease sites when feasible.
RT is given to clinically suspicious lymph nodes (detected by palpation or imaging) unless the suspicious lymph nodes are biopsied and shown to be free of rhabdomyosarcoma. RT is also administered to lymph node basins where a sentinel lymph node biopsy has identified microscopic disease.[5]
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The discussion of treatment options for children with rhabdomyosarcoma is divided into the following sections:
Surgery (local control management).
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RT (local control management).
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Surgery and RT by primary site of disease (local control management).
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Chemotherapy.
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Rhabdomyosarcoma treatment options used by the Children's Oncology Group (COG) and by groups in Europe (as exemplified by trials from the Soft Tissue Sarcoma Committee of the COG [COG-STS], the Intergroup Rhabdomyosarcoma Study Group [IRSG], the International Society of Pediatric Oncology Malignant Mesenchymal Tumor [MMT] Group, and the European Paediatric Soft Tissue Sarcoma Study Group [EpSSG]) differ in management and overall treatment philosophy, as noted below:[2]
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The primary objective of the COG-STS, after the initial surgical resection or biopsy and induction chemotherapy, has been to use additional local control therapy, predominantly with RT or surgical resection when appropriate. Event-free survival is the target end point, attempting to avoid relapse and subsequent salvage therapy.[3]
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In the MMT trials, the main objective has been to reduce the use of local therapies using initial front-line chemotherapy, followed by second-line therapy in the presence of poor response. Subsequent surgical resection is preferred over RT, which is used only after incomplete resection, documented regional lymph node involvement, or a poor clinical response to initial chemotherapy. This approach is designed to avoid major surgical procedures and long-term damaging effects from RT. Some patients have been spared aggressive local therapy, which may reduce the potential for morbidities associated with such therapy.[1-3]The MMT Group approach led to an overall survival (OS) rate of 71% in the European MMT89 study, compared with an OS rate of 84% in the IRS-IV study. Similarly, EFS rates at 5 years were 57% in the MMT89 study versus 78% in the IRS-IV study. Differences in outcomes were most striking for patients with extremity and head and neck nonparameningeal tumors. Failure-free survival was lower for patients with bladder/prostate primary tumors who did not receive RT as part of their initial treatment, but there was no difference in OS between the two strategies for these patients.[6] The overall impression is that survival for most patient subsets is superior with the use of early local therapy, including RT.[1-3]
The EpSSG RMS-2005 (NCT00379457) study reported comprehensive outcome data for 1,733 children and adolescents with nonmetastatic rhabdomyosarcoma. These patients were enrolled in two phase III randomized trials for high-risk patients and observational trials for low-risk, standard-risk, and very-high risk patients. Eighty percent of children with localized rhabdomyosarcoma were long-term survivors. This study established the standard of care across EpSSG countries, including the following:[7]
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The EpSSG RMS-2005 (NCT00379457) study reported comprehensive outcome data for 1,733 children and adolescents with nonmetastatic rhabdomyosarcoma. These patients were enrolled in two phase III randomized trials for high-risk patients and observational trials for low-risk, standard-risk, and very-high risk patients. Eighty percent of children with localized rhabdomyosarcoma were long-term survivors. This study established the standard of care across EpSSG countries, including the following:[7]
A 22-week vincristine/dactinomycin regimen for patients with low-risk rhabdomyosarcoma.
The EpSSG RMS-2005 (NCT00379457) study reported comprehensive outcome data for 1,733 children and adolescents with nonmetastatic rhabdomyosarcoma. These patients were enrolled in two phase III randomized trials for high-risk patients and observational trials for low-risk, standard-risk, and very-high risk patients. Eighty percent of children with localized rhabdomyosarcoma were long-term survivors. This study established the standard of care across EpSSG countries, including the following:[7]
The reduction of the cumulative ifosfamide dose for patients with standard-risk disease.
The EpSSG RMS-2005 (NCT00379457) study reported comprehensive outcome data for 1,733 children and adolescents with nonmetastatic rhabdomyosarcoma. These patients were enrolled in two phase III randomized trials for high-risk patients and observational trials for low-risk, standard-risk, and very-high risk patients. Eighty percent of children with localized rhabdomyosarcoma were long-term survivors. This study established the standard of care across EpSSG countries, including the following:[7]
The omission of doxorubicin and the addition of maintenance chemotherapy for patients with high-risk disease.
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