What is the role of chemotherapy in the treatment of pediatric rhabdomyosarcoma (RMS)?

Updated: Mar 28, 2019
  • Author: Roshni Dasgupta, MD, MPH; Chief Editor: Eugene S Kim, MD, FACS, FAAP  more...
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Low-risk patients receive vincristine weekly for nine doses and actinomycin D with or without cyclophosphamide (VAC regimen) and granulocyte colony-stimulating factor (G-CSF) for four doses every 12 weeks (at weeks 0, 12, 24, and 36). RT is added in patients with residual localized disease.

The protocol for intermediate-risk patients includes RT and either VAC or VAC plus topotecan (according to randomized assignment) for nearly 1 year. For patients with intermediate-risk disease, VAC-VI (irinotecan) has been shown to be as effective as VAC while decreasing the dose of cyclophosphamide. A currently open intermediate-risk RMS trial is examining the use of temsirolimus with a VAC-VI backbone.

High-risk patients begin therapy with irinotecan, which is followed by VAC and RT. Newer chemotherapeutic agents and molecular therapies are frequently tried in high-risk, relapse, and disease-progression patients in the hopes of improving outcomes.

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