A critical evaluation of caplacizumab for the treatment of acquired thrombotic thrombocytopenic purpura.
Expert Rev Hematol
; 13(11): 1153-1164, 2020 11.
Article
en En
| MEDLINE
| ID: mdl-32876503
ABSTRACT
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.Palabras clave
Texto completo:
1
Colección:
01-internacional
Base de datos:
MEDLINE
Contexto en salud:
6_ODS3_enfermedades_notrasmisibles
Problema de salud:
6_haemoglobinopathies_hemolytic_anaemias
/
6_malnutrition_nutritional_deficiencies
/
6_other_blood_disorders
Asunto principal:
Púrpura Trombocitopénica Trombótica
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Factor de von Willebrand
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Terapia Molecular Dirigida
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Fibrinolíticos
/
Anticuerpos de Dominio Único
Tipo de estudio:
Clinical_trials
Límite:
Humans
Idioma:
En
Revista:
Expert Rev Hematol
Asunto de la revista:
HEMATOLOGIA
Año:
2020
Tipo del documento:
Article
País de afiliación:
España