Myelodysplastic syndromes (MDS)
are heterogeneous in cause and manifestations but share the common features of
aberrant hematopoiesis and deteriorating cytopenias. Patients with MDS suffer
from anemia, neutropenia, and thrombocytopenia, all of which lead to outcomes
such as extreme fatigue, high rates of infections, excessive bleeding, and
shortened life span; MDS patients also are at risk of their disease
transforming into acute myeloid leukemia.
<>MDS patients are classified by
morphology and prognosis into lower-risk or higher-risk MDS categories. Only
three drugs have been approved in the United States or Europe specifically for
MDS, and second-line therapies do not exist beyond clinical trials or best supportive
care (BSC). Allogeneic hematopoietic stem cell transplant (HSCT) is the only
potentially curative therapeutic option; however, approximately three-quarters
of the MDS patient population are ineligible for HSCT due to older age and poor
overall health status. MDS patients need new treatments that are targeted at
specific patient subsets, particularly transfusion-dependent patients and the
higher-risk patients who are refractory to hypomethylating agents (HMA). Clinical
trials for such therapies should focus on improved overall survival in higher-risk
patients and improved quality of life (e.g., reduced transfusion dependence or
reduced rate of infections) in lower-risk patients.
Questions Answered in This Report:
There is a high level of unmet need for specific subsets of MDS
patients. For example, HMA-refractory higher-risk patients have no second-line
therapeutic options outside of clinical trials or BSC; lower-risk patients have
a longer expected lifespan than higher-risk patients but still suffer from significantly
reduced quality of life, requiring frequent transfusions, antibiotics, and
hospitalizations. Are there any treatments in clinical development that are
likely to address the needs of these patients? Which patient subsets have the highest
level of unmet need?
The pathology of MDS is not well understood and the disease is
heterogeneous. What are the new avenues of research and understanding of MDS
pathophysiology? What new mechanisms of action are in clinical development? Do
they offer advantages over current first-line therapies or focus on
combination-therapy opportunities? Will any of these emerging MDS therapies
launch between 2012 and 2021? In which patients will these drugs be used?
Resistance to HMAs remains an issue for higher-risk patients, and
resistance to lenalidomide remains an issue for lower-risk patients. What
therapies are being investigated for patients with refractory disease? Which of
these therapies will launch between 2012 and 2021 and in which patients will
these treatments be used?
Unmet needs in MDS are many and span a range of challenging
issues. What unmet needs are expected to remain unaddressed by mid- to
late-stage emerging therapies?
Markets covered: United States, France, Germany, Italy,
Spain, United Kingdom.
Primary research: 7 country-specific interviews with MDS,
Epidemiology: Diagnosed incidence segmented by age and
country; diagnosed prevalence segmented by morphology classification and level
of International Prognostic Scoring System (IPSS) prognostic risk.
- Morphologic classification: refractory anemia
(RA), RA with ringed sideroblasts (RARS), MDS with 5q deletion (del[5q]), refractory
cytopenia with multilineage dysplasia (RCMD), therapy-related MDS (tMDS), RA with excess blasts (RAEB), RAEB in
transformation (RAEB-T), MDS unclassified.
- IPSS classification: low, intermediate-1, intermediate-2, and
high; also lower-risk and higher-risk (categories based on the IPSS classifications).
Emerging therapies: Phase I: 8; Phase II: 15 drugs; Phase
III: 3 drugs.