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Treating Immune-Mediated Thrombotic Thrombocytopenic Purpura with Caplacizumab

Júlia Weisinger1, Bérangère S Joly1,2,3, Agnès Veyradier1,2,3

  • 1Centre de Référence des Microangiopathies Thrombotiques (CNR-MAT), Paris, France.

Methods in Molecular Biology (Clifton, N.J.)
|March 31, 2026
PubMed

Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a life-threatening disease caused by the deficiency of the von Willebrand factor (VWF)-cleaving protease ADAMTS13. Caplacizumab is an anti-VWF nanobody blocking the interaction between VWF and platelets, thus preventing microthrombi formation and ischemic organ damage. Both clinical studies and large real-world studies proved the beneficial effects of caplacizumab in iTTP, including faster clinical response, shorter hospitalization, and improved overall survival. Caplacizumab is safe in the majority of patients, although increased bleeding risk has to be taken into account.

Keywords:
Anti-VWF nanobodyCaplacizumabTherapyThrombotic thrombocytopenic purpura

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