A critical evaluation of caplacizumab for the treatment of acquired thrombotic thrombocytopenic purpura

6533b827fe1ef96bd1286690

RESEARCH PRODUCT

A critical evaluation of caplacizumab for the treatment of acquired thrombotic thrombocytopenic purpura

Miguel Fernández-zarzoso Inés Gómez-seguí Javier De La Rubia

subject

Thrombotic microangiopathy Exacerbation viruses medicine.medical_treatment ADAMTS13 Protein Disease Bioinformatics Autoantigens 03 medical and health sciences Platelet Adhesiveness 0302 clinical medicine Fibrinolytic Agents Protein Domains Crotalid Venoms von Willebrand Factor medicine Humans Immunologic Factors Multicenter Studies as Topic Lectins C-Type Molecular Targeted Therapy Drug Approval Clinical Trials as Topic Acquired Thrombotic Thrombocytopenic Purpura Plasma Exchange Purpura Thrombotic Thrombocytopenic business.industry Standard treatment fungi Immunosuppression Drugs Investigational Hematology Aptamers Nucleotide Single-Domain Antibodies biochemical phenomena metabolism and nutrition medicine.disease Combined Modality Therapy Recombinant Proteins ADAMTS13 Acetylcysteine Treatment Outcome 030220 oncology & carcinogenesis Drug Therapy Combination Caplacizumab business Immunosuppressive Agents 030215 immunology

description

Introduction: Acquired thrombotic thrombocytopenic purpura (aTTP) is a thrombotic microangiopathy caused by inhibitory autoantibodies against ADAMTS13 protein. Until recently, the combination of plasma exchange (PEX) and immunosuppression has been the standard front-line treatment in this disorder. However, aTTP-related mortality, refractoriness, and relapse are still a matter of concern. Areas covered: The better understanding of the pathophysiological mechanisms of aTTP has allowed substantial improvements in the diagnosis and treatment of this disease. Recently, the novel anti-VWF nanobody caplacizumab has been approved for acute episodes of aTTP. Caplacizumab is capable to block the adhesion of platelets to VWF, therefore inhibiting microthrombi formation in the ADAMTS13-deficient circulation. In this review, the characteristics of caplacizumab together with the available data of its efficacy and safety in the clinical setting will be analyzed. Besides, the current scenario of aTTP treatment will be provided, including the role of other innovative drugs. Expert opinion: With no doubt, caplacizumab is going to change the way we treat aTTP. In combination with standard treatment, caplacizumab can help to significantly reduce aTTP-related mortality and morbidity and could spare potential long-term consequences by minimizing the risk of exacerbation.

year	journal	country	edition	language
2020-10-12	Expert Review of Hematology

https://doi.org/10.1080/17474086.2020.1819230