Comparable outcomes of patients eligible vs ineligible for SWOG leukemia studies

Abstract

Patients may be deemed ineligible for a clinical trial for reasons that do not directly impact efficacy or safety. We identified reasons for ineligibility and compared outcomes of ineligible with eligible patients treated on Southwest Oncology Group (SWOG) Leukemia Committee protocols. Patients enrolled in SWOG phase 2, 2/3, or 3 protocols open since 2005 were analyzed for eligibility status, reasons for ineligibility, baseline characteristics, Eastern Cooperative Oncology Group (ECOG) performance status (PS), serious adverse events (SAEs), complete remission (CR) status, and overall survival. A total of 2361 patients were enrolled in the 13 included studies. Of these, 247 (10%) were deemed ineligible; 78 were excluded from analyses, and 169 were included. Of the 169 included in analyses, 60% (101/169) were excluded due to missing baseline documentation. Baseline characteristics comparing ineligible to eligible patients were similar, with the exception of ECOG PS for S0325 (P = .02) and S0530 (P = .002). In multivariable analyses, neither the proportion of patients with ECOG PS ≥ 2 (P = .12) nor the rate of grade 5 SAEs (P = .62) differed between groups. There was no difference in survival between eligible and ineligible patients (P = .25), and CR rates were similar, with the exception of S0325 (P < .001) and S0703 (P = .004). The findings of this study suggest that nonessential eligibility criteria can be less restrictive, thus expanding patient enrollment and avoiding protocol deviations. The clinical trials included in this study were registered at www.clincialtrials.gov as #NCT00085709, #NCT00066794, #NCT00070499, #NCT00109837, #NCT00093418, #NCT00492856, #NCT00337168, #NCT00352365, #NCT00658814, #NCT00792948, #NCT00945815, #NCT00840177, and #NCT01522976.

Conflict of interest statement

Conflict-of-interest disclosure: M.A.S. serves on Celgene’s Advisory Board. A.A. provides consulting services for Pfizer. S.M. provides consulting services for, receives research funding and honoraria from, and serves on the speakers bureau for Novartis, Takeda, Celgene, Pfizer, and Bristol Myers Squibb. S.C. provides consulting services for and receives research funding from Novartis and Celgene. H.P.E. serves on the speakers bureau for Celgene, Incyte, Jazz, and Novartis; provides consulting services for Amgen, Celgene, Daiichi Sankyo, Jazz, ImmunoGen, Incyte, MacroGen, Novartis, Ono, Pfizer, Seattle Genetics, Sunesis, and Millennium/Takeda; receives research funding from Agios, Amgen, Juno, Astellas, Celator, Daiichi Sankyo, ImmunoGen, Janssen, Millennium/Takeda, and Seattle Genetics; is the Chair of the Data and Safety Monitoring Committee for Glycomimetics; and is the Chair of Celgene’s Scientific Steering Committee. The remaining authors declare no competing financial interests.

© 2018 by The American Society of Hematology.

Figures

Graphical abstract

Figure 1.

OS by eligibility status.