Myelodysplastic syndromes (MDS) consists of a group of disorders that are heterogeneous in cause and manifestations but share the common features of aberrant hematopoiesis and deteriorating cytopenias. Patients with MDS may suffer from anemia, neutropenia, and thrombocytopenia, with outcomes such as extreme fatigue, high rates of infections, excessive bleeding, and shortened life span.

This report provides an overview of the MDS landscape featuring a comprehensive analysis of patient populations, current therapies and medical practices, and opportunities for emerging therapies. The report identifies a very high level of unmet need for new, disease-modifying MDS therapies and provides expert insight on potential drug targets. At this time, the only potentially curative therapy for MDS is allogeneic hematopoietic stem cell transplant (HSCT). However, interviewed experts report that approximately three-quarters of the MDS patient population is ineligible for HSCT due to advanced age and/or poor overall health status. Current treatment options for MDS are very limited and frequently ineffective. Moreover, there are no good therapeutic options for treatment-refractory patients.

Questions Answered in This Report:

  • MDS is a disorder of the elderly. What is the size of the U.S. and EU5 (France, Germany, Italy, Spain, and the United Kingdom) MDS patient population, and how will it change over the next ten years? What are the key MDS subpopulations?

  • The pathology of MDS is not well understood and the disease is heterogeneous. How are clinicians currently managing treatment of MDS patients? What are the key drivers for prescribing in MDS?

  • Resistance to hypomethylating agents (HMAs) remains an issue for higher-risk patients, and resistance to lenalidomide remains an issue for lower-risk patients. What treatments in clinical development are likely to address the needs of specific subsets of MDS patients, such as HMA-refractory patients and lower-risk patients with a significantly reduced quality of life? 

  • Unmet needs in MDS are many and span a range of challenging issues. What are the new avenues of research and understanding of MDS pathophysiology? Which emerging therapies with new mechanisms of action are in clinical development? What are the key primary and secondary clinical end points with which new therapies are evaluated? What unmet needs are expected to remain unaddressed by mid- to late-stage emerging therapies for MDS?


Markets covered: United States, France, Germany, Italy, Spain, and United Kingdom.

Primary research: Eight country-specific interviews with thought-leader hematologists.

Epidemiology: Diagnosed incident cases and diagnosed prevalent cases of MDS by country and by age. Diagnosed prevalent cases by morphology and by prognostic risk.

Emerging Therapies:  Phase II: 19; Phase III/PR: 2; coverage of select preclinical and Phase I products.

Author(s): Jing Wu, M.S., M.B.A.
Alison Isherwood, M.Res., M.Sc., Ph.D.

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