Case Report: Zanubrutinib for refractory immune thrombotic thrombocytopenic purpura
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Published on: December 7, 2014
Adrián Dante De Angelis1,2, Annatina Schnegg-Kaufmann1,3, Elisavet Moutzouri1,4
1Department of Hematology and Central Hematological Laboratory, Inselspital, Bern University Hospital, University of Bern, Bern, Switzerland.
Abstract:
Immune-mediated thrombotic thrombocytopenic purpura (iTTP) results from severe ADAMTS13 deficiency and may relapse despite modern therapy, with survival exceeding 95% in contemporary cohorts. We report the case of a 43-year-old patient with multiple clinical and ADAMTS13 relapses over 15 years, who developed intolerance or had incomplete responses to several immunosuppressive agents, including anti-CD20 and anti-CD38 therapies. Splenectomy was considered high risk due to significant cardiac comorbidity. Off-label zanubrutinib, a second-generation Bruton tyrosine kinase (BTK) inhibitor licensed locally for chronic lymphocytic leukemia (CLL) was initiated after the sixth relapse, which was a clinical one, simultaneously with plasma exchange (PEX) and corticosteroids. Following initiation, ADAMTS13 activity rose, platelet counts normalized, and the patient has remained in clinical remission for 20 months under zanubrutinib. This case illustrates the feasibility of BTK inhibition in selected, treatment-limited iTTP.