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

IF 7.2 2区医学 Q1 HEMATOLOGY

Blood Reviews Pub Date : 2026-04-01 Epub Date: 2025-12-20 DOI:10.1016/j.blre.2025.101359

Mohamed Yassin , Rola Ghasoub , Neda Jafari , Lulwah Al-Tourah , Anas Babiker , Muna AlRasheed

{"title":"An updated systematic review and meta-analysis of Caplacizumab for immune thrombotic thrombocytopenic purpura: Insights into efficacy and safety","authors":"Mohamed Yassin , Rola Ghasoub , Neda Jafari , Lulwah Al-Tourah , Anas Babiker , Muna AlRasheed","doi":"10.1016/j.blre.2025.101359","DOIUrl":null,"url":null,"abstract":"<div><div>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, <em>p</em> < 0.001) and time to platelet normalization (MD = −2.44, <em>p</em> = 0.03). Caplacizumab also significantly reduced mortality (RR = 0.29, <em>p</em> < 0.001) and iTTP exacerbations (RR = 0.27, p < 0.001), leading to shorter hospital stays (MD = −5.13, <em>p</em> = 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.</div></div>","PeriodicalId":56139,"journal":{"name":"Blood Reviews","volume":"76 ","pages":"Article 101359"},"PeriodicalIF":7.2000,"publicationDate":"2026-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Blood Reviews","FirstCategoryId":"3","ListUrlMain":"https://www.sciencedirect.com/science/article/pii/S0268960X25001043","RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2025/12/20 0:00:00","PubModel":"Epub","JCR":"Q1","JCRName":"HEMATOLOGY","Score":null,"Total":0}

引用次数: 0

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.

查看原文本刊更多论文

卡普拉珠单抗治疗免疫性血栓性血小板减少性紫癜的最新系统评价和荟萃分析：疗效和安全性的见解。

本综述综合了目前的证据来评估卡普拉珠单抗治疗iTTP的有效性和安全性，重点关注其对TPE要求、血小板正常化时间、复发预防和治疗相关不良事件的影响。纳入13项研究（11项观察性研究和2项随机对照试验），涉及2956例iTTP患者。大多数纳入的研究具有较高的方法学质量，表明基于评估工具的偏倚风险较低。分析发现，卡普拉珠单抗与显著的临床获益相关。它标志着每日TPE会话的减少(MD = -3.92, p

本文章由计算机程序翻译，如有差异，请以英文原文为准。

求助全文

约1分钟内获得全文求助全文

来源期刊

Blood Reviews 医学-血液学

CiteScore

13.80

自引率

1.40%

发文量

期刊介绍： Blood Reviews, a highly regarded international journal, serves as a vital information hub, offering comprehensive evaluations of clinical practices and research insights from esteemed experts. Specially commissioned, peer-reviewed articles authored by leading researchers and practitioners ensure extensive global coverage across all sub-specialties of hematology.