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

Cancer Medicine

Ollivier Legrand¹  Andrew H. Wei²  Alessandra Tosolini³  Mark G. Frattini³  Jixian J. Wang³  Roland Marion‐Gallois³  Mathilde Hunault‐Berger⁴  Arnaud Pigneux⁵  Bruno Quesnel⁶  Sylvain Chantepie⁷  Christian Recher⁸  Salem A. Nehme⁹  Xavier Thomas^1,10  Muhaimen Siddiqui^1,11  Chris Cameron^1,11  Stéphane deBotton^1,12  Nicolas Boissel^1,13  Eytan M. Stein^1,14  Brian Hutton^1,15  Gary Milkovich^1,16  Joseph M. Brandwein^1,17 

[1] APHP Hôpital Saint‐Antoine Paris France;Alfred Hospital and Monash University Melbourne Australia;Bristol Myers Squibb Summit New Jersey USA;CHU Angers Angers France;CHU Bordeaux Hôpital du Haut Lévêque Bordeaux France;CHU Lille Lille France;CHU de Caen Caen France;CHU de Toulouse Université de Toulouse III Paul Sabatier Toulouse France;Celgene International, a Bristol Myers Squibb Company Boudry Switzerland;Centre Hospitalier Lyon Sud Pierre Bénite France;Cornerstone Research Group Burlington Ontario Canada;Departement d'hematologie Institut Gustave Roussy Villejuif France;Hôpital Saint‐Louis Paris France;Memorial Sloan Kettering Cancer Center New York New York USA;Ottawa Hospital Research Institute Ottawa Ontario Canada;RJM Group LLC Crown Point Indiana USA;University of Alberta Edmonton Alberta Canada;

关键词: acute myeloid leukemia;    enasidenib;    IDH2 mutations;    overall survival;    standard of care;   

DOI  :  10.1002/cam4.4182

来源: DOAJ

【 摘 要 】

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.

【 授权许可】

Unknown