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What are the most promising new agents in myelodysplastic syndromes?

Chandhok, Namrata S.; Boddu, Prajwal C.; Gore, Steven D.; Prebet, Thomas

Current Opinion in Hematology: March 2019 - Volume 26 - Issue 2 - p 77–87
doi: 10.1097/MOH.0000000000000483
MYELOID DISEASE: Edited by Martin S. Tallman

Purpose of review Myelodysplastic syndromes (MDS) are a diverse group of clonal disorders of hematopoietic stem or progenitor cells that represent the most common class of acquired bone marrow failure syndromes in adults. Despite significant improvement in the pathologic insight into this group of disorders, therapeutic options remain limited and allogeneic hematopoietic stem-cell transplantation is the only treatment that can induce long-term remission in patients with MDS. The goals of therapy for MDS are based on disease prognostication, with a focus of minimizing transfusion dependence and preserving quality of life in low-risk groups and preventing progression of disease to acute myeloid leukemia in high-risk groups. Given the dearth of approved treatment options, there is a marked need for novel therapies across the board, and there are several novel agents currently in the pipeline.

Recent findings Among the promising agents with preclinical and early phase efficacy in higher risk MDS, apoptosis targeting with BCL-2 inhibitors have been a standout. There is also a keen interest in immunotherapy, and targeted agents (genetic, signaling pathways, bispecific antibodies, antibody-drug conjugates, and others described in this review).

Summary In this review, we will highlight some of the promising new agents currently under investigation for the management of MDS.

Smilow Cancer Center at Yale New Haven Hospital, New Haven, Connecticut, USA

Correspondence to Thomas Prebet, Division of Hematology, Oncology, Smilow Cancer Center at Yale New Haven Hospital, 35 Park Street, New Haven, CT 06511, USA. Tel: +1 203 812 9312; e-mail:

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