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

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.

• Authors: 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
• Journal: Cancer (Cancer) Vol. 125 Issue 15 Pg. 2602-2609 (08 01 2019) ISSN: 1097-0142 [Electronic] United States
• PMID: 31067356 (Publication Type: Journal Article, Research Support, N.I.H., Extramural)
• Copyright: © 2019 American Cancer Society.

Topics

• Child
• Disease Progression
• Female
• Humans
• Male
• Recurrence
• Rhabdomyosarcoma (drug therapy, mortality)
• Risk Factors
• Survival Analysis