Although combination therapies have improved outcomes in rhabdomyosarcoma (RMS) patients, prognosis remains poor for those who relapse, emphasizing a need for new targeted therapies. The multi-tyrosine kinase inhibitor (TKI) regorafenib targets several kinases associated with RMS, including VEGFR, FGFR, and PDGFR, and has demonstrated promising preclinical antitumor activity in Ewing sarcoma (EWS), neuroblastoma, and RMS models. In this phase I trial, Casanova and colleagues establish a maximum tolerated dose for regorafenib and evaluate the efficacy of regorafenib combined with vincristine and irinotecan in pediatric patients with recurrent/refractory RMS, EWS, neuroblastoma, and Wilms tumor. Treatment was well tolerated, with less than 20% of patients experiencing grade 3/4 adverse events. The authors report an overall response rate of 48% and a disease control rate of 86%, with 2 of 21 of patients achieving complete response, 8 patients demonstrating partial response, and 7 patients maintaining stable disease. Median progression-free survival was 7.0 months,...
Skip Nav Destination
Article navigation
1 November 2023
Highlights|
November 01 2023
Selected Articles from This Issue
Online ISSN: 1557-3265
Print ISSN: 1078-0432
©2023 American Association for Cancer Research
2023
American Association for Cancer Research
Clin Cancer Res (2023) 29 (21): 4315.
- Views Icon Views
- Share Icon Share
-
Tools Icon
Tools
- Search Site
-
Article Versions Icon
Versions
- Version of Record November 1 2023
Citation
Selected Articles from This Issue. Clin Cancer Res 1 November 2023; 29 (21): 4315. https://doi.org/10.1158/1078-0432.CCR-29-21-HI
Download citation file:
Sign in
Don't already have an account? Register
Client Account
You could not be signed in. Please check your email address / username and password and try again.
Could not validate captcha. Please try again.
Pay-Per-View 24-Hour Access
$50.00
93
Views
Citing articles via
Advertisement