Risk-based treatment for patients with first relapse or progression of rhabdomyosarcoma: A report from the Children's Oncology Group

Leo Mascarenhas*, Elizabeth R. Lyden, Philip P. Breitfeld, David O. Walterhouse, Sarah S. Donaldson, David A. Rodeberg, David M. Parham, James R. Anderson, William H. Meyer, Douglas S. Hawkins

*Corresponding author for this work

Research output: Contribution to journalArticle

4 Scopus citations

Abstract

Background: The purpose of this study was to evaluate risk and response–based multi-agent therapy for patients with rhabdomyosarcoma (RMS) at first relapse. Methods: Patients with RMS and measurable disease at first relapse with unfavorable-risk (UR) features were randomized to a 6-week phase 2 window with 1 of 2 treatment schedules of irinotecan with vincristine (VI) (previously reported). Those with at least a partial response to VI continued to receive 44 weeks of multi-agent chemotherapy including the assigned VI regimen. UR patients who did not have measurable disease at study entry, did not have a radiographic response after the VI window, or declined VI window therapy received 31 weeks of multi-agent chemotherapy including tirapazamine (TPZ) at weeks 1, 4, 10, 19, and 28. Favorable-risk (FR) patients received 31 weeks of the same multi-agent chemotherapy without VI and TPZ. Results: One hundred thirty-six eligible patients were enrolled. For 61 patients not responding to VI, the 3-year failure-free survival (FFS) and overall survival (OS) rates were 17% (95% confidence interval [CI], 8%-29%) and 24% (13%-37%), respectively. For 30 UR patients not treated with VI, the 3-year FFS and OS rates were 21% (8%-37%) and 39% (20%-57%), respectively. FR patients had 3-year FFS and OS rates of 79% (47%-93%) and 84% (50%-96%), respectively. There were no unexpected toxicities. Conclusions: Patients with UR RMS at first relapse or disease progression have a poor prognosis when they are treated with this multi-agent therapy, whereas FR patients have a higher chance of being cured with second-line therapy.

Original languageEnglish (US)
Pages (from-to)2602-2609
Number of pages8
JournalCancer
Volume125
Issue number15
DOIs
StatePublished - Aug 1 2019

Keywords

  • recurrent
  • refractory
  • relapse
  • rhabdomyosarcoma
  • treatment

ASJC Scopus subject areas

  • Oncology
  • Cancer Research

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    Mascarenhas, L., Lyden, E. R., Breitfeld, P. P., Walterhouse, D. O., Donaldson, S. S., Rodeberg, D. A., Parham, D. M., Anderson, J. R., Meyer, W. H., & Hawkins, D. S. (2019). Risk-based treatment for patients with first relapse or progression of rhabdomyosarcoma: A report from the Children's Oncology Group. Cancer, 125(15), 2602-2609. https://doi.org/10.1002/cncr.32122