Impact of modernizing eligibility criteria on enrollment and representation in acute myeloid leukemia clinical trials

作者：Andrew Hantel, Yating Wang, Angel M. Cronin, Irum Khan, Ivy Abraham, Ann‐Kathrin Eisfeld, Anand Patel, W Stock, Sarah Monick, Thomas Walsh, Erin Gallagher, Marlise R. Luskin, Ana Maria Avila Rodriguez, Carlos Galvez, Peter Doukas, Jessica K. Altman, Madelyn Burkart, Amani Erra, Maryam Zia, Melissa L. Larson, Ami Dave, Stephanie Tsai, Ahmed Aleem, Nepheli Raptis, Christopher S. Lathan, Hajime Uno, Daniel J. DeAngelo, Gregory A. Abel · 发表于：Blood · 年份：2025 · DOI：10.1182/blood.2025030741 · 被引用次数：3 · 研究领域：Ethics in Clinical Research、Health Systems, Economic Evaluations, Quality of Life、Meta-analysis and systematic reviews

ABSTRACT: Clinical trial eligibility criteria select a target population and reduce anticipated risks for participants but may unnecessarily limit participation both overall and differently across demographic groups. We previously abstracted eligibility criteria for 190 phase 2/3 acute myeloid leukemia (AML) trials and used US Food and Drug Administration and professional society guidance on modernizing criteria to develop alternative, safety-based eligibility criteria for each trial. In this analysis, these trial- and safety-based eligibility criteria sets were applied to a retrospective cohort of 2226 newly diagnosed patients across 8 hospitals to assess the impact on eligibility. Eligibility proportions increased from a median of 47.9% with trial-based criteria to 84.2% with safety-based criteria (median difference, 30.0%; P< .001); excluding age criteria, the increase was 11.5% (P< .001). Non-Hispanic (NH) Asian, NH Black, NH White, and Hispanic patients were eligible for median proportions of 41.1%, 44.0%, 47.9%, and 50.0%, respectively, with trial-based criteria, increasing by 27.9% to 31.6% when using safety-based criteria (within-group changes, all P< .001; between-group changes, all P> .05). Excluding age criteria, increases were between 10.0% and 11.9%. Moving from trial- to safety-based criteria decreased the proportion of trials with significant eligibility differences between NH White and NH Asian (-11.1%), NH Black (-4.2%), and Hispanic (-12.1%) patients. Criter...