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Luspatercept is indicated for the treatment of adults with transfusion-dependent anemia due to very low, low and intermediate-risk myelodysplastic syndromes (MDS) with ring sideroblasts, who had an unsatisfactory response to or are ineligible for erythropoietin-based therapy. [6]
A myelodysplastic syndrome (MDS) is one of a group of cancers in which blood cells in the bone marrow do not mature, and as a result, do not develop into healthy blood cells. [3] Early on, no symptoms typically are seen. [3] Later, symptoms may include fatigue, shortness of breath, bleeding disorders, anemia, or frequent infections. [3]
The Mayo Clinic Cancer Center is one of the oldest NCI-designated cancer centers in the United States, having first been designated in 1973. [3] The main location of the Mayo Clinic is in Rochester, MN. Campuses in Arizona and Florida opened later and became part of the Mayo Clinic Cancer Center in 2003. [4] [5]
This syndrome affects bone marrow cells causing treatment-resistant anemia and myelodysplastic syndromes that may lead to acute myelogenous leukemia. Examination of the bone marrow shows characteristic changes in the megakaryocytes. They are more numerous than usual, small and mononuclear.
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Roxadustat is reported to increase VEGF, a signal protein that can activate tumor growth [8] and also is considered to cause pulmonary hypertension. [9] In phase 3 trial conducted at 29 sites in China, roxadustat treatment was found to cause hyperkalemia, i.e., increase in serum potassium, and metabolic acidosis in patients.