Improved survival with enasidenib versus standard of care in relapsed/refractory acute myeloid leukemia associated with IDH2 mutations using historical data and propensity score matching analysis Journal Article


Authors: de Botton, S.; Brandwein, J. M.; Wei, A. H.; Pigneux, A.; Quesnel, B.; Thomas, X.; Legrand, O.; Recher, C.; Chantepie, S.; Hunault-Berger, M.; Boissel, N.; Nehme, S. A.; Frattini, M. G.; Tosolini, A.; Marion-Gallois, R.; Wang, J. J.; Cameron, C.; Siddiqui, M.; Hutton, B.; Milkovich, G.; Stein, E. M.
Article Title: Improved survival with enasidenib versus standard of care in relapsed/refractory acute myeloid leukemia associated with IDH2 mutations using historical data and propensity score matching analysis
Abstract: Background: The present study evaluated the relative survival benefits associated with enasidenib and current standard of care (SoC) therapies for patients with relapsed/refractory (R/R) acute myeloid leukemia (AML) and an isocitrate dehydrogenase 2 (IDH2) mutation who are ineligible for hematopoietic stem cell transplantation (HSCT). Methods: Propensity score matching (PSM) analysis compared survival outcomes observed with enasidenib 100 mg daily in the phase I/II AG221-C-001 trial and SoC outcomes obtained from a real-world chart review of patients in France. Results: Before matching, enasidenib (n = 195) was associated with numerically improved overall survival (OS) relative to SoC (n = 80; hazard ratio [HR], 0.82; 95% confidence interval [CI], 0.61–1.11). After matching and adjusting for covariates (n = 78 per group), mortality risk was significantly lower with enasidenib than with SoC (HR, 0.67; 95% CI, 0.47–0.97). The median OS was 9.26 months for enasidenib (95% CI, 7.72–13.24) and 4.76 months for SoC (95% CI, 3.81–8.21). Results remained robust across all sensitivity analyses conducted. Conclusions: PSM analyses indicate that enasidenib significantly prolongs survival relative to SoC among patients with R/R AML and an IDH2 mutation who are ineligible for HSCT. Future prospective studies are needed to validate these findings using other data sources and to assess the comparative efficacy of enasidenib for other treatment outcomes. © 2021 The Authors. Cancer Medicine published by John Wiley & Sons Ltd.
Keywords: overall survival; standard of care; acute myeloid leukemia; idh2 mutations; enasidenib
Journal Title: Cancer Medicine
Volume: 10
Issue: 18
ISSN: 2045-7634
Publisher: Wiley Blackwell  
Date Published: 2021-09-01
Start Page: 6336
End Page: 6343
Language: English
DOI: 10.1002/cam4.4182
PUBMED: 34427990
PROVIDER: scopus
PMCID: PMC8446562
DOI/URL:
Notes: Article -- Export Date: 1 October 2021 -- Source: Scopus
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  1. Eytan Moshe Stein
    204 Stein