According to a study published in Leukemia & Lymphoma, patients with newly diagnosed acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) may have higher complete remission (CR) rates when treated on study compared with patients treated off study.

Researchers at the University of Washington in Seattle and the Fred Hutchinson Cancer Research Center in Seattle, Washington, conducted a retrospective analysis comparing outcomes of patients with newly diagnosed or relapsed/refractory AML or high-risk MDS who were treated on or off study.

Through their institutional database, the researchers identified and analyzed 397 induction attempts in 365 patients (160 newly diagnosed and 237 relapsed/refractory) between January 1, 2008, and November 15, 2015. The median follow-up was 16 months. In most cases, no differences in baseline characteristics were identified between patients treated on or off study; however, one exception was higher treatment-related mortality scores in patients treated off study for medical reasons compared with those treated on study.

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For newly diagnosed patients, CR rates were higher for patients on study than for patients off study, both when analyzed using stringent criteria (CR without measurable residual disease [MRD]: 61% vs 35%, P =.001) and when analyzed using broader criteria (CR with or without MRD or with neutropenia or thrombocytopenia [CR/CRi]: 86% vs 64%, P =.002). For patients treated off study for medical reasons, remission rates were much lower (CR without MRD: 30%; CR/CRi: 61%). In contrast, no differences were found in remission rates among relapsed/refractory patients treated on or off study (CR without MRD: 26% vs 22%, P =.41; CR/CRi: 50% vs 44%, P =.42).

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The authors suggested that “RR studies should include more liberal eligibility criteria,” and that medically ineligible patients could be included in clinical trials “when evaluating new treatments.”


1. Buckley SA, Percival ME, Othus M, et al. A comparison of patients with acute myeloid leukemia and high-risk myelodysplastic syndrome treated on versus off study [published online October 2, 2018]. Leuk Lymphoma. doi: 10.1080/10428194.2018.1516036