Fostamatinib resulted in clinically meaningful, ongoing platelet responses and a low rate of bleeding events in 43% of patients with chronic immune thrombocytopenic purpura (ITP) with long-disease duration who had been previously randomized to placebo in phase III studies, according to the open-label extension trial Study 049 (NCT02077192).
AEs were manageable and mild to moderate in their severity. The most common AEs were diarrhea (22%), hepatic disease (17%), and hypertension (16%). Treatment-related AEs—which were all mild (25%) or moderate (27%) and resolved over time—occurred in 23 (52%) of the 44 patients. Serious bleeding occurred in 7 (28%) out of 25 patients who did not respond to treatment compared with none of the 19 responders. There were 5 (11%) discontinuations due to AEs of diarrhea, sepsis, thrombocytopenia, pneumonia, and increased transaminases.
Bussel J, Khalafallah AA, Arnold DM, et al. Fostamatinib, a spleen tyrosine kinase inhibitor, for the treatment of chronic immune thrombocytopenic purpura: safety and efficacy in patients initiating treatment in an open-label extension study 049. In: Proceedings from the 22nd Annual International Congress on Hematologic Malignancies®: Focus on Leukemias, Lymphomas and Myeloma; March 1 to 4, 2018; Hollywood, Florida.
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