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Pigneux, A.1; Quesnel, B.2; Thomas, X.3; Legrand, O.4; de Botton, S.5; Recher, C.6; Chantepie, S.7; Hunault-Berger, M.8; Abi Nehme, S.9; Frattini, M.10; Tosolini, A.10; Marion-Gallois, R.9; Wang, J. J.10; Reitan, J. F.11; Boissel, N.12

doi: 10.1097/01.HS9.0000562460.12241.94
Poster Session II: Acute myeloid leukemia - Clinical

1CHU de Bordeaux, Service d'Hématologie Clinique et Thérapie Cellulaire, Hôpital du Haut Lévêque, Bordeaux

2Department of Hematology, CHU de Lille, Lille

3Centre Hospitalier Lyon Sud, Service Hématologie G, Pierre Benite Cedex

4APHP, Hôpital Saint-Antoine, Paris

5Institut Gustave Roussy, Villejuif Cedex

6Service d'Hématologie, CHU de Toulouse, Institut Universitaire du Cancer de Toulouse Oncopole, Université de Toulouse III Paul Sabatier, Toulouse

7CHU de Caen, Caen

8CHU d'Angers, Angers Cedex, France

9Celgene International, Boudry, Switzerland

10Celgene Corporation, Summit

11RJM Group LLC, Crown Point, United States

12Hématologie Adulte, Hôpital Saint-Louis, Paris, France

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Isocitrate dehydrogenase 2 (IDH2) mutations are linked to DNA hypermethylation, altered gene expression, and blocked differentiation of hematopoietic progenitor cells, which may trigger myeloid malignancies. Between 9% and 15% of newly diagnosed acute myeloid leukemia (AML) cases are associated with IDH2 mutations. Mutant IDH2 (mIDH2) inhibitors have recently entered clinical development and may be a promising treatment strategy for patients with mIDH2 AML. However, treatment patterns and outcomes in patients with relapsed or refractory (R/R) mIDH2 AML are ill-defined, which precludes an accurate assessment of the clinical benefit provided by mIDH2 inhibitors.

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The primary objective of this study was to describe treatment patterns and associated outcomes in patients with mIDH2 R/R AML.

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This was a retrospective, multicenter, medical chart review study, collecting data from 104 patients with mIDH2 R/R AML in France. Patients aged ≥18 years were eligible if they had been hospitalized for mIDH2 R/R AML between September 2011 and September 2016. Patients treated with the mIDH2 inhibitor enasidenib were excluded. Demographic data, disease characteristics, treatments received after diagnosis of R/R AML, response assessments, and date of death were extracted from medical charts. Overall survival (OS) was estimated by means of Kaplan-Meier curves.

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Overall, 104 patients were selected from 9 French sites and 103 patients were included in the analysis. 70% of patients had received 1 line of therapy prior to baseline while 30% of patients had >1 line of therapy before baseline. Median age was 65 years and 57% of patients were male.

A total of 23 patients received best supportive care (BSC) after baseline. For the remaining 80 patients who received ≥1 line of therapy after baseline, the most common regimen types were low-intensity chemotherapy, used in 38 patients (mainly 5-azacytidine), followed by intensive chemotherapy regimens, used in 26 patients (mainly 7+3 and cytarabine-containing regimens). Best post-baseline response was assessed in 94 patients. Among these patients, 28 (30%) were responders: 21 patients had a complete response (CR), 5 had a partial response, 1 patient had a CR with incomplete hematologic recovery, and 1 patient had CR with incomplete platelet recovery. The remaining 66 patients (70%) were non-responders; among these patients, 9 had stable disease and 27 had progressive disease.

At the cutoff date for this study (November 13, 2018), 72% of patients had died, while 28% of patients were censored as still alive at the time of last information. Median OS from baseline was 7.20 months (95% confidence interval [CI] 4.30-10.71) (Figure).



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Patients with mIDH2 R/R AML receiving standard of care had a median OS of 7.20 months. While this result cannot be directly compared with the single-arm study of enasidenib, the only currently approved IDH2 inhibitor for use in R/R AML, (median OS 8.8 months) due to potential differences in patient population, it provides a much-needed description of actual treatment patterns and associated outcomes in patients with mIDH2 R/R AML.

Copyright © 2019 The Authors. Published by Wolters Kluwer Health Inc., on behalf of the European Hematology Association.