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Advancing drug development in myelodysplastic syndromes

Journal Title: 
Blood Advances
Primary Author: 
Mina A
Author(s): 
Mina A, McGraw KL, Cunningham L, Kim N, Jen EY, Calvo KR, Ehrlich LA, Aplan PD, Garcia-Manero G, Foran JM, Garcia JS, Zeidan AM, DeZern AE, Komrokji R, Sekeres MA, Scott B, Buckstein R, Tinsley-Vance S, Verma A, Wroblewski T, Pavletic S, Norsworthy K
Original Publication Date: 
Tuesday, March 11, 2025
Bone Marrow Disease(s): 

Myelodysplastic syndromes/neoplasms (MDSs) are heterogeneous stem cell malignancies characterized by poor prognosis and no curative therapies outside of allogeneic hematopoietic stem cell transplantation. Despite some recent approvals by the US Food and Drug Administration, (eg, luspatercept, ivosidenib, decitabine/cedazuridine, and imetelstat), there has been little progress in the development of truly transformative therapies for the treatment of patients with MDS. Challenges to advancing drug development in MDS are multifold but may be grouped into specific categories, including criteria for risk stratification and eligibility, response definitions, time-to-event end points, transfusion end points, functional assessments, and biomarker development. Strategies to address these challenges and optimize future clinical trial design for patients with MDS are presented here.