Outcome of patients with relapsed or refractory nonrhabdomyosarcoma soft tissue sarcomas enrolled in phase 2 cooperative group clinical trials: A report from the Children's Oncology Group

Authors

Sapna Oberoi, Department of Pediatrics and Child Health, University of Manitoba, Winnipeg, Manitoba, Canada.
Amira Qumseya, Department of Biostatistics, College of Public Health and Health Professions College of Medicine, University of Florida, Gainesville, Florida, USA.
Wei Xue, Department of Biostatistics, College of Public Health and Health Professions College of Medicine, University of Florida, Gainesville, Florida, USA.
Rajkumar Venkatramani, Division of Hematology/Oncology, Department of Pediatrics, Texas Children's Cancer Center, Texas Children's Hospital, Baylor College of Medicine, Houston, Texas, USA.
Aaron R. Weiss, Division of Pediatric Hematology-Oncology, Department of Pediatrics, Maine Medical Center, Portland, Maine, USA.

Document Type

Article

Publication Date

3-12-2024

Institution/Department

Oncology

Journal Title

Cancer

Abstract

BACKGROUND: The aim of this study was to estimate the event-free survival (EFS) of children and young adults with relapsed or refractory nonrhabdomyosarcoma soft tissue sarcoma (NRSTS) treated in nonrandomized phase 2 studies conducted by the Children's Oncology Group (COG) and predecessor groups to establish a benchmark EFS for future phase 2 NRSTS trials evaluating the activity of novel agents. METHODS: A retrospective analysis of patients with recurrent or refractory NRSTS prospectively enrolled in nonrandomized phase 2 COG and predecessor group trials between 1994 and 2015 was conducted. EFS was defined as disease progression/relapse or death and calculated via the Kaplan-Meier method. The log-rank test and relative risk regression were used to compare EFS distribution by age at enrollment, sex, race, NRSTS histology, prior lines of therapy, calendar year of trial, and type of radiographic response. RESULTS: In total, 137 patients were enrolled in 13 phase 2 trials. All trials used radiographic response rate as a primary outcome, and none of the agents used were considered active on the basis of trial-specified thresholds. The estimated median EFS and 6-month EFS of the entire study cohort was 1.5 months (95% confidence interval [CI], 1.3-1.8 months) and 19.4% (95% CI, 12.7%-26%), respectively. No difference in EFS was observed by age at enrollment, sex, race, NRSTS histology subtype, prior lines of therapies, and trial initiation year. EFS significantly differed by radiographic response. CONCLUSIONS: The EFS for children and young adults with relapsed or refractory NRSTS remains suboptimal. Established EFS can be referenced as a benchmark for future single-agent phase 2 trials incorporating potentially active novel agents in this population.

Recommended Citation

Oberoi, Sapna; Qumseya, Amira; Xue, Wei; Venkatramani, Rajkumar; and Weiss, Aaron R., "Outcome of patients with relapsed or refractory nonrhabdomyosarcoma soft tissue sarcomas enrolled in phase 2 cooperative group clinical trials: A report from the Children's Oncology Group" (2024). Maine Medical Center. 3636.
https://knowledgeconnection.mainehealth.org/mmc/3636