An updated systematic review and meta-analysis of Caplacizumab for immune thrombotic thrombocytopenic purpura: Insights into efficacy and safety

Abstract

This review synthesizes the current evidence to assess the efficacy and safety of caplacizumab in the treatment of iTTP, focusing on its impact on TPE requirements, time to platelet normalization, relapse prevention, and treatment-related adverse events. Thirteen studies (11 observational and 2 RCTs), involving 2956 patients with iTTP were included. The majority of included studies were of high methodological quality, indicating a low risk of bias based on the assessment tools. The analysis found that caplacizumab was associated with significant clinical benefits. It marked a reduction in daily TPE sessions (MD = −3.92, p < 0.001) and time to platelet normalization (MD = −2.44, p = 0.03). Caplacizumab also significantly reduced mortality (RR = 0.29, p < 0.001) and iTTP exacerbations (RR = 0.27, p < 0.001), leading to shorter hospital stays (MD = −5.13, p = 0.001). Caplacizumab demonstrated notable clinical benefits in managing iTTP, with significant reductions in daily TPE sessions, time to platelet normalization, mortality, and iTTP exacerbations. Promising results were also noticed for early initiation compared to delayed initiation.