TY - JOUR
T1 - Fostamatinib for the treatment of warm antibody autoimmune hemolytic anemia
T2 - Phase 2, multicenter, open-label study
AU - Kuter, David J.
AU - Rogers, Kerry A.
AU - Boxer, Michael A.
AU - Choi, Michael
AU - Agajanian, Richy
AU - Arnold, Donald M.
AU - Broome, Catherine M.
AU - Field, Joshua J.
AU - Murakhovskaya, Irina
AU - Numerof, Robert
AU - Tong, Sandra
N1 - Publisher Copyright:
© 2022 The Authors. American Journal of Hematology published by Wiley Periodicals LLC.
PY - 2022/6/1
Y1 - 2022/6/1
N2 - Patients with relapsed warm antibody autoimmune hemolytic anemia (wAIHA) have limited treatment options. Fostamatinib is a potent, orally administered spleen tyrosine kinase inhibitor approved in the United States and Europe for the treatment of adults with chronic immune thrombocytopenia (ITP). This phase 2 study evaluated the response to fostamatinib, administered at 150 mg BID orally with or without food in adults with wAIHA and active hemolysis with hemoglobin (Hgb) <10 g/dL who had failed at least one prior treatment. Hemoglobin levels and safety assessments were performed at visits every 2 weeks. The primary endpoint was Hgb >10 g/dL with an increase of ≥2 g/dL from baseline by week 24 without rescue therapy or red blood cell transfusion. Eleven of 24 (46%) patients achieved the primary endpoint. Increases in median Hgb were detected at week 2 and sustained over time. Median lactate dehydrogenase levels and reticulocyte counts generally declined over time with little change in median haptoglobin levels. The most common adverse events (AEs) were diarrhea (42%), fatigue (42%), hypertension (27%), dizziness (27%), and insomnia (23%). AEs were manageable and consistent with the fostamatinib safety database of over 3900 patients across multiple diseases (rheumatoid arthritis, B-cell lymphoma, COVID-19, and ITP). No new safety signals were detected. Fostamatinib may be a promising therapeutic option for wAIHA. A randomized, double-blind, phase 3 study is nearing completion.
AB - Patients with relapsed warm antibody autoimmune hemolytic anemia (wAIHA) have limited treatment options. Fostamatinib is a potent, orally administered spleen tyrosine kinase inhibitor approved in the United States and Europe for the treatment of adults with chronic immune thrombocytopenia (ITP). This phase 2 study evaluated the response to fostamatinib, administered at 150 mg BID orally with or without food in adults with wAIHA and active hemolysis with hemoglobin (Hgb) <10 g/dL who had failed at least one prior treatment. Hemoglobin levels and safety assessments were performed at visits every 2 weeks. The primary endpoint was Hgb >10 g/dL with an increase of ≥2 g/dL from baseline by week 24 without rescue therapy or red blood cell transfusion. Eleven of 24 (46%) patients achieved the primary endpoint. Increases in median Hgb were detected at week 2 and sustained over time. Median lactate dehydrogenase levels and reticulocyte counts generally declined over time with little change in median haptoglobin levels. The most common adverse events (AEs) were diarrhea (42%), fatigue (42%), hypertension (27%), dizziness (27%), and insomnia (23%). AEs were manageable and consistent with the fostamatinib safety database of over 3900 patients across multiple diseases (rheumatoid arthritis, B-cell lymphoma, COVID-19, and ITP). No new safety signals were detected. Fostamatinib may be a promising therapeutic option for wAIHA. A randomized, double-blind, phase 3 study is nearing completion.
UR - http://www.scopus.com/inward/record.url?scp=85125529424&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85125529424&partnerID=8YFLogxK
U2 - 10.1002/ajh.26508
DO - 10.1002/ajh.26508
M3 - Article
C2 - 35179251
AN - SCOPUS:85125529424
SN - 0361-8609
VL - 97
SP - 691
EP - 699
JO - American Journal of Hematology
JF - American Journal of Hematology
IS - 6
ER -