Caplacizumab for relapsing thrombotic thrombocytopenic purpura.

BACKGROUND: Thrombotic thrombocytopenic purpura (TTP) is an ultra-rare disorder in childhood and belongs to the microangiopathic hemolytic anemias (MAHA) and the thrombotic microangiopathies (TMA). In the acquired form, autoantibodies against ADAMTS13 inhibit cleaving of von Willebrand factor (vWF) multimers, thereby promoting their interaction with thrombocytes, causing TMA and MAHA. A recently introduced nanobody, caplacizumab, inhibits the binding of platelets to vWF. CASE-DIAGNOSIS/TREATMENT: During a first episode, a 10-year-old girl was admitted for TTP. Plasma exchange (PE) and immunosuppressive therapy with corticosteroids and mycophenolate mofetil were initiated. The course was complicated by catheter-associated septicemia and a very slow hematological and clinical recovery. Platelet count became normal at day 40 after admission and treatment initiation. Three years later, the child presented again with TTP. During this second episode, caplacizumab was introduced together with PE and immunosuppressive therapy within 4 days after admission. With this regimen, platelet count normalized within 3 days of treatment, and PE treatment could be stopped after a total of 14 days. The child could be discharged and caplacizumab was continued on an outpatient basis until day 30 after initiation. Adverse events during the use of caplacizumab were not encountered.
CONCLUSIONS: Caplacizumab treatment was safe and effective in a child with relapsing, autoantibody-mediated TTP. With respect to this potentially life-threatening condition, the add-on use of caplacizumab represents a novel option to reduce morbidity and mortality and improve quality of life in children and adolescents with TTP.