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Updated: Dec 27, 2025

Use of Hematopoietic Stem Cell Transplantation to Assess the Origin of Myelodysplastic Syndrome
Published on: October 3, 2018
Prospects for Venetoclax in Myelodysplastic Syndromes
1Dana-Farber Cancer Institute, Harvard Medical School, 450 Brookline Avenue, Dana 2054, Boston, MA 02215, USA.
Abstract:
BCL-2 is an antiapoptotic protein that plays a critical role acute and chronic leukemias. Venetoclax is an orally selective BCL-2 inhibitor and BH3 mimetic approved in chronic lymphocytic leukemia and in combination with low dose cytarabine or hypomethylating agent in acute myeloid leukemia for the treatment of patients unfit for intensive chemotherapy. This article reviews the biology of BCL-2, focusing on its relationship to the myeloid microenvironment, and discusses the rationale for BCL-2 inhibition in myelodysplastic syndrome (MDS). Clinical trials testing venetoclax in MDS patients are under way. Potential biomarkers for clinical response to BCL-2 inhibition are discussed. Therapeutic opportunities for venetoclax in the therapeutic landscape of MDS are explored.
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