Targeting B-Cell-Activating Factor and Its Receptor in Immune Thrombocytopenia and Warm Autoimmune Hemolytic Anemia.

Current treatments for immune thrombocytopenia (ITP) and warm autoimmune hemolytic anemia (wAIHA), rare autoimmune diseases in which autoreactive B cells play a major role, can lead to high response rates; however, for many patients these responses are not durable or maintained after treatment discontinuation. Binding of B-cell-activating factor (BAFF) to its receptor (BAFF-R) has been shown to lead to B-cell differentiation, proliferation, and survival, with BAFF levels shown to be elevated in patients with ITP and wAIHA. Targeting the BAFF/BAFF-R pathway could address unmet needs that remain for patients with ITP and wAIHA despite available treatments.