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Ipilimumab plus decitabine for patients with MDS or AML in posttransplant or transplant-naïve settings

Journal Title: 
Blood
Primary Author: 
Garcia JS
Author(s): 
Garcia JS, Flamand Y, Penter L, Keng M, Tomlinson BK, Mendez LM, Koller P, Cullen N, Arihara Y, Pfaff K, Wolff JO, Brunner AM, Galinsky I, Bashey A, Antin JH, Cutler C, Ho V, Jonas BA, Luskin MR, Wadleigh M, Winer ES, Savell A, Leonard R, Robertson T, , Davids MS, Streicher H, Rodig SJ, Ritz J, Wu CJ, DeAngelo DJ, Neuberg D, Stone RM, Soiffer RJ
Original Publication Date: 
Thursday, April 13, 2023

Two articles in this week’s issue focus on the use of ipilimumab and decitabine for patients with myelodysplasia (MDS) and acute myeloid leukemia (AML) before and after hematopoietic stem cell transplantation (HSCT) for high-risk disease. In the first article, Garcia et al report on the results of a phase 1 trial of the combination in 54 patients, demonstrating overall response rate of 52% in patients who are HSCT-naïve and 20% in patients post-HSCT; responses are usually short-lived. In the second article, Penter and colleagues characterize gene expression responses to therapy and conclude that decitabine acts directly to clear leukemic cells while ipilimumab acts on infiltrating lymphocytes in marrow and extramedullary sites. Responses are determined by leukemic cell burden and by the frequency and phenotype of infiltrating lymphocytes. Increasing bone marrow regulatory T cells is identified as a potential contributor to checkpoint inhibitor escape.

Bone Marrow Disease(s): 
  • myelodysplastic syndromes (MDS)
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