Safety and efficacy of danicopan in patients with paroxysmal nocturnal hemoglobinuria: a systematic review and meta-analysis.

IF 2.3 4区医学 Q2 HEMATOLOGY

Expert Review of Hematology Pub Date : 2024-11-01 Epub Date: 2024-10-28 DOI:10.1080/17474086.2024.2422558

Emir Muvaffak, Muhammed Edib Mokresh, Abdullah Varda, Mahmoud Lakmoush, Merve Kabasakal Ilter

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引用次数: 0

Abstract

Introduction: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare disease characterized by complement-mediated intravascular hemolysis (IVH). Current treatments like Eculizumab and Ravulizumab have limitations, with many patients still requiring transfusions. This study aimed to investigate the safety and efficacy of the new emergent treatment called Danicopan.

Methods: We systematically searched five electronic databases - Epistemonikos, Web of Science, Medline, Scopus, and ClinicalTrials - to ensure comprehensive coverage. The systematic review was conducted following the PRISMA guidelines, ensuring methodological rigor.

Results: Four studies were eligible for inclusion, all of them were multicenter trials with 79 patients studied. Treatment with Danicopan led to a notable improvement in hemoglobin levels and a decrease in reticulocyte counts. However, LDH levels did not significantly change after treatment. Additionally, there was a significant increase in GPI-deficient erythrocytes but not in GPI-deficient granulocytes. Total and direct bilirubin levels showed significant differences between treatment groups, and there was an improvement in FACIT scores from baseline.

Conclusions: Our systematic review and meta-analysis support the potential of Danicopan as a viable therapeutic option for PNH patients. The targeted inhibition of factor D within the complement system by Danicopan demonstrates both safety and efficacy in managing PNH, as evidenced by our findings.

Registration: This paper was registered with the PROSPERO database (CRD42024499375).

查看原文本刊更多论文

阵发性夜间血红蛋白尿患者服用达尼可潘的安全性和疗效：系统综述和荟萃分析。

简介阵发性夜间血红蛋白尿症（PNH）是一种以补体介导的血管内溶血（IVH）为特征的罕见疾病。目前的治疗方法如Eculizumab和Ravulizumab存在局限性，许多患者仍然需要输血。本研究旨在探讨一种名为达尼单抗的新型紧急治疗方法的安全性和有效性：我们系统地检索了五个电子数据库--Epistemonikos、Web of Science、Medline、Scopus 和 ClinicalTrials--以确保全面覆盖。系统性综述按照 PRISMA 指南进行，以确保方法的严谨性：有四项研究符合纳入条件，均为多中心试验，共研究了 79 名患者。达尼可潘治疗后，血红蛋白水平明显改善，网织红细胞计数下降。此外，GPI 缺乏的红细胞显著增加，但 GPI 缺乏的粒细胞没有增加。总胆红素和直接胆红素水平在治疗组之间存在显著差异，FACIT评分较基线有所提高：我们的系统综述和荟萃分析支持将达尼高潘作为治疗 PNH 患者的可行方案。我们的研究结果表明，达尼可潘在补体系统内对因子 D 的靶向抑制在治疗 PNH 方面既安全又有效：本文已在 PROSPERO 数据库注册（CRD42024499375）。

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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来源期刊

Expert Review of Hematology HEMATOLOGY-

CiteScore

4.70

自引率

3.60%

发文量

审稿时长

6-12 weeks

期刊介绍： Advanced molecular research techniques have transformed hematology in recent years. With improved understanding of hematologic diseases, we now have the opportunity to research and evaluate new biological therapies, new drugs and drug combinations, new treatment schedules and novel approaches including stem cell transplantation. We can also expect proteomics, molecular genetics and biomarker research to facilitate new diagnostic approaches and the identification of appropriate therapies. Further advances in our knowledge regarding the formation and function of blood cells and blood-forming tissues should ensue, and it will be a major challenge for hematologists to adopt these new paradigms and develop integrated strategies to define the best possible patient care. Expert Review of Hematology (1747-4086) puts these advances in context and explores how they will translate directly into clinical practice.