CMML is a Rare and Aggressive Cancer of the blood.
CMML affects approximately 1,500 – 3,000 new patients per year in the United States. The cancer is considered a clonal disease which begins with one or more mutations to the DNA of bone marrow stem cell that multiplies uncontrollably. The change interferes with normal blood cell production, including red and white blood cells and platelets.
CMML patients often present with anemia, but can also experience low platelet counts leading to bleeding and abnormal white cell counts leading to infections, an enlarged spleen, bone marrow fibrosis and other symptoms. CMML patients have a shortened life expectancy and approximately 15-30 percent of patients progress to develop acute myeloid leukemia (AML).
The World Health Organization (WHO) reclassified CMML in 2008 into a separate disease from a therapeutic perspective. Prior to reclassification, CMML accounted for 10 percent of all myelodysplastic syndrome (MDS) cases for which hypomethylating agents (HMAs) have been approved. Treatment options are limited and include: blood transfusions; HMAs for non-proliferative forms; hydroxyurea for palliating symptoms related to massive splenomegaly and controlling elevated blood counts; and erythropoiesis stimulating agents for anemia.