On Hierarchical Composite Endpoints in Pediatric Cancer Supportive Care: Illustrative Examples From Two Multi‐Center Phase‐III Randomized Clinical Trials

Pediatric supportive care clinical trials often involve multiple clinically important outcomes, complicating trial interpretation. Hierarchical composite endpoints (HCEs) provide a framework to integrate key outcomes according to clinical importance. We performed post hoc analyses using HCE in two randomized trials conducted by the Children's Oncology Group. We found that HCE can be more sensitive overall endpoints for detecting treatment benefit as well as found that HCE can support harmonized trial conclusions in the presence of intervention benefits and harms. These analyses illustrate the potential of HCE to improve the interpretability of complex pediatric supportive care trials and support consideration of their prospective use.

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Publication Details

Journal
Pediatric Blood & Cancer
Published
2026-07-25
DOI
https://doi.org/10.1002/1545-5017.70593
Primary Topic
Statistical Methods in Clinical Trials
Type
article
Field-Weighted Citation Impact
0.00
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article

On Hierarchical Composite Endpoints in Pediatric Cancer Supportive Care: Illustrative Examples From Two Multi‐Center Phase‐III Randomized Clinical Trials

Willem Collier, Zachary D. Prudowsky, Etan Orgel, Christopher C. Dvorak et al.
Pediatric Blood & Cancer
Statistical Methods in Clinical Trials
article

On Hierarchical Composite Endpoints in Pediatric Cancer Supportive Care: Illustrative Examples From Two Multi‐Center Phase‐III Randomized Clinical Trials

Willem Collier, Zachary D. Prudowsky, Etan Orgel, Christopher C. Dvorak, Brian T. Fisher, Caitlin W. Elgarten, Nicole J. Ullrich, Lillian Sung, David R. Freyer, Mark Zobeck, Adam J. Esbenshade, Sarah Alexander
article en

Abstract

Pediatric supportive care clinical trials often involve multiple clinically important outcomes, complicating trial interpretation. Hierarchical composite endpoints (HCEs) provide a framework to integrate key outcomes according to clinical importance. We performed post hoc analyses using HCE in two randomized trials conducted by the Children's Oncology Group. We found that HCE can be more sensitive overall endpoints for detecting treatment benefit as well as found that HCE can support harmonized trial conclusions in the presence of intervention benefits and harms. These analyses illustrate the potential of HCE to improve the interpretability of complex pediatric supportive care trials and support consideration of their prospective use.

Pediatric Blood & Cancer
University of Southern California (US), Children's Hospital of Philadelphia (US), Children's Hospital of Los Angeles (US), University of California, San Francisco (US), Baylor College of Medicine (US), Hospital for Sick Children (CA), Monroe Carell Jr. Children's Hospital (US), Texas Children's Hospital (US), Dana-Farber/Boston Children's Cancer and Blood Disorders Center (US), University of Pennsylvania (US)
Good health and well-being
Openalex Percentile: Top 6%
Statistical Methods in Clinical Trials
DOIOpen Access PDFOpenAlex
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