Efficacy and safety of current treatments for paroxysmal nocturnal hemoglobinuria: A systematic review

Clinical Immunology Communications Pub Date : 2022-11-23 DOI:10.1016/j.clicom.2022.11.002

Yara Maria da Silva Pires , Aline de Fátima Bonetti , Jessica Telma Ciecilinsky , Astrid Wiens Souza

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Abstract

Paroxysmal nocturnal hemoglobinuria (PNH) is a non-malignant clonal disorder of the pluripotent hematopoietic stem cell. Currently, Eculizumab, Ravulizumab, and Pegcetacoplan are the approved drugs to treat PNH. In order to assess the efficacy and safety of different medications available for PNH, we performed a systematic search. The primary efficacy endpoint was the percentage change in lactate dehydrogenase, transfusion avoidance, and stabilized hemoglobin. Key secondary endpoints included the proportion of patients with breakthrough hemolysis, anemia, adverse events, number of deaths, and discontinuation of treatment. From 2526 articles retrieved from electronic databases and manual searches, a total of five studies were included in this review: 1 observational study and 4 randomized clinical trials. For all efficacy and safety endpoints, Ravulizumab and Pegcetacoplan achieved noninferiority compared with the first standardized treatment Eculizumab. The use of complement inhibition therapy can further improve hematological outcomes in PNH patients.

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目前治疗阵发性夜间血红蛋白尿的有效性和安全性：一项系统综述

阵发性夜间血红蛋白尿症（PNH）是一种多能干细胞的非恶性克隆性疾病。目前，Eculizumab、Ravulizumab和Pegcetacoplan是已批准的治疗PNH的药物。为了评估可用于PNH的不同药物的疗效和安全性，我们进行了系统搜索。主要疗效终点是乳酸脱氢酶、避免输血和稳定血红蛋白的百分比变化。关键的次要终点包括突破性溶血、贫血、不良事件、死亡人数和停止治疗的患者比例。从电子数据库和手动搜索中检索到的2526篇文章中，共有5项研究被纳入本综述：1项观察性研究和4项随机临床试验。对于所有疗效和安全性终点，与第一种标准化治疗Eculizumab相比，Ravulizumab和Pegcetacoplan实现了非劣效性。补体抑制疗法的使用可以进一步改善PNH患者的血液学结果。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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Clinical Immunology Communications

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