[Acquired thrombotic-thrombocytopenic purpura - patient management with the advent of novel therapeutic agents].

History: A 42-year-old female patient presented with pyelonephritis. Two days later she complained of hematomas and petechia of the extremities.
Diagnosis/clinical Findings: Acquired thrombotic thrombocytopenic purpura was diagnosed. Laboratory result showed a thrombotic microangiopathy and thrombocytopenia of 8 × 10E9/l. ADAMTS13-activity was reported to be reduced and inhibitory antibodies to be present.
Therapy and Course: The patient was placed on a therapy with plasma exchange, high-dose steroids, and caplacizumab based on a positive PLASMIC score. Once the diagnosis was confirmed by diminished ADAMTS13-activity and the presence of autoantibodies, rituximab was added. The patient responded well but showed persistently elevated levels of ADAMTS13-autoantibodies, which lead to an extension of Caplacizumab treatment to 58 days.
Discussion: Caplacizumab represents a novel effective treatment option for patients with acquired thrombocytopenic purpura. Time to treatment response as well as the risk of relapse were shown to be significantly reduced. However, persistent autoimmune activity can be demonstrated in a significant portion of the patients after the end of treatment putting them at risk for relapses. Thus, careful and frequent surveillance is required.
Competing Interests: PTB: Honorare (Vorträge, Advisory Board, Reiseunterstützung) Pfizer, Sanofi Genzyme, Ablynx, AlexionLAV: Honorare (Sanofi Genzyme)FG: Honorare (Advisory Board) Vifor
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