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Risk-based Treatment for Patients With First Relapse or Progression of Rhabdomyosarcoma: A Report From the Children's Oncology Group

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

Cancer. 2019 Aug 1;125(15):2602-2609.

PMID: 31067356

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.

Chemicals Related in the Paper:

Catalog Number	Product Name	Structure	CAS Number	Price
AP27314972	Tirapazamine		27314-97-2	Price

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