Comparative effectiveness and safety of rituximab and caplacizumab for immuno-mediated Thrombotic Thrombocytopenic Purpura: an overview of systematic reviews

IF 3.4 3区医学 Q1 HEMATOLOGY

Thrombosis research Pub Date : 2026-07-01 Epub Date: 2026-06-24 DOI:10.1016/j.thromres.2026.109767

Mayra Carvalho-Ribeiro, Camila Peña, Thaís D.R. Nóbrega, Marília Berlofa Visacri, Fernanda A. Orsi

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引用次数: 0

Abstract

Background

Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a life-threatening disorder caused by anti-ADAMTS13 (A Disintegrin And Metalloproteinase with a ThromboSpondin type 1 motif, member 13) antibodies. Although therapeutic plasma exchange (TPE) and corticotherapy have long been the standard of care (SOC), rituximab and caplacizumab are increasingly incorporated into clinical practice. Given that these drugs significantly increase the costs of iTTP treatment, synthesizing the available evidence is important for coverage decisions in resource-limited settings.

Objectives

To summarize and critically appraise systematic reviews with meta-analyses (SRMAs) evaluating effectiveness and safety of rituximab and caplacizumab for iTTP treatment.

Methods

PubMed, EMBASE, the Cochrane Library, and LILACS were searched up to August 2025. Two reviewers screened citations, with disagreements resolved by a third reviewer. Data extraction was performed by one reviewer and verified by another. Findings were synthesized narratively.

Results

Seven SRMAs were included: two evaluated rituximab and five caplacizumab. SRMAs of rituximab included non-randomized studies and reported reductions in mortality and relapse when added to SOC, although exacerbation and safety were not assessed. SRMAs of caplacizumab included two randomized and nine non-randomized studies. Mortality reduction with caplacizumab was not demonstrated in randomized trials, although real-world data suggested a survival benefit. Randomized trials showed reduced risk of exacerbation and increased risk of bleeding, but no consistent effect on relapse. All SRMAs were rated as critically low methodological quality.

Conclusion

Although SRMAs suggest rituximab and caplacizumab may improve clinical response and survival, with more consistent evidence favoring rituximab, their critically low methodological quality highlights limitations in the current evidence base.

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利妥昔单抗和卡普拉珠单抗治疗免疫介导的血栓性血小板减少性紫癜的比较有效性和安全性：系统综述

背景：免疫介导的血栓性血小板减少性紫癜（iTTP）是一种由抗adamts13（一种带有血小板反应蛋白1型基板的崩解素和金属蛋白酶，成员13）抗体引起的危及生命的疾病。虽然治疗性血浆置换（TPE）和皮质疗法长期以来一直是标准护理（SOC），但利妥昔单抗和卡普拉珠单抗越来越多地纳入临床实践。鉴于这些药物显著增加了iTTP治疗的费用，综合现有证据对于在资源有限的情况下做出覆盖决策非常重要。目的：通过荟萃分析（srma）对利妥昔单抗和卡普拉单抗治疗iTTP的有效性和安全性进行总结和批判性评价。方法：检索截至2025年8月的PubMed、EMBASE、Cochrane Library和LILACS数据库。两名审稿人筛选引文，分歧由第三名审稿人解决。数据提取由一位审稿人执行，并由另一位审稿人进行验证。研究结果以叙述的方式综合。结果：纳入7个srma: 2个评估利妥昔单抗，5个评估卡普单抗。利妥昔单抗的srma包括非随机研究，并报告了当加入SOC时死亡率和复发率降低，尽管没有评估恶化和安全性。卡普拉珠单抗的srma包括2个随机研究和9个非随机研究。随机试验并未证明卡帕珠单抗可降低死亡率，但实际数据表明可提高生存期。随机试验显示急性加重风险降低，出血风险增加，但对复发没有一致的影响。所有srma都被评为极低的方法学质量。结论：尽管srma提示利妥昔单抗和卡普拉珠单抗可以改善临床反应和生存率，有更一致的证据支持利妥昔单抗，但其极低的方法学质量突出了当前证据基础的局限性。

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来源期刊

Thrombosis research 医学-外周血管病

CiteScore

14.60

自引率

4.00%

发文量

364

审稿时长

31 days

期刊介绍： Thrombosis Research is an international journal dedicated to the swift dissemination of new information on thrombosis, hemostasis, and vascular biology, aimed at advancing both science and clinical care. The journal publishes peer-reviewed original research, reviews, editorials, opinions, and critiques, covering both basic and clinical studies. Priority is given to research that promises novel approaches in the diagnosis, therapy, prognosis, and prevention of thrombotic and hemorrhagic diseases.