• David O Walterhouse, Alberto S Pappo, Jane L Meza, John C Breneman, Andrea A Hayes-Jordan, David M Parham, Timothy P Cripe, James R Anderson, William H Meyer, and Douglas S Hawkins.
• David O. Walterhouse, Ann & Robert H. Lurie Children's Hospital of Chicago, Northwestern University Feinberg School of Medicine, Chicago, IL; Alberto S. Pappo, Saint Jude Children's Research Hospital, Memphis, TN; Jane L. Meza, James R. Anderson, University of Nebraska Medical Center College of Public Health, Omaha, NE; John C. Breneman, Children's Hospital Medical Center, Cincinnati; Timothy P. Cripe, Nationwide Children's Hospital, Columbus, OH; Andrea Hayes-Jordan, MD Anderson Cancer Center, Houston, TX; David M. Parham, William H. Meyer, University of Oklahoma School of Medicine, Oklahoma City, OK; Douglas S. Hawkins, Seattle Children's Hospital, Fred Hutchinson Cancer Research Center, University of Washington, Seattle, WA. d-walterhouse@northwestern.edu.
• J. Clin. Oncol. 2014 Nov 1; 32 (31): 3547-52.

PurposeIntergroup Rhabdomyosarcoma Study Group (IRSG) studies III and IV showed improved failure-free survival (FFS) rates with vincristine, dactinomycin, and cyclophosphamide (VAC; total cumulative cyclophosphamide dose, 26.4 g/m(2)) compared with vincristine and dactinomycin (VA) for patients with subset-one low-risk embryonal rhabdomyosarcoma (ERMS; stage 1/2 group I/II ERMS or stage 1 group III orbit ERMS). The objective of Children's Oncology Group ARST0331 was to reduce the length of therapy without compromising FFS for this subset of low-risk patients by using VA in combination with lower-dose cyclophosphamide (total cumulative dose, 4.8 g/m(2)) plus radiotherapy (RT).Patients And MethodsThis noninferiority prospective clinical trial enrolled newly diagnosed patients with subset-one clinical features. Therapy included four cycles of VAC followed by four cycles of VA over 22 weeks. Patients with microscopic or gross residual disease at study entry received RT.ResultsWith a median follow-up of 4.3 years, we observed 35 failures among 271 eligible patients versus 48.4 expected failures, calculated using a fixed outcome based on the FFS expected for similar patients treated on the IRSG D9602 protocol. The estimated 3-year FFS rate was 89% (95% CI, 85% to 92%), and the overall survival rate was 98% (95% CI, 95% to 99%). Patients with paratesticular tumors had the most favorable outcome. Three-year cumulative incidence rates for any local, regional, or distant failures were 7.6%, 1.5%, and 3.4%, respectively.ConclusionShorter-duration therapy that included lower-dose cyclophosphamide and RT did not compromise FFS for patients with subset-one low-risk ERMS.© 2014 by American Society of Clinical Oncology.

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