Real-world evidence of pegcetacoplan in patients with paroxysmal nocturnal haemoglobinuria: A nationwide Italian study

IF 3.8 2区医学 Q1 HEMATOLOGY

British Journal of Haematology Pub Date : 2025-02-22 DOI:10.1111/bjh.20025

Elisabetta Metafuni, Filippo Achille Brioschi, Andrea Patriarca, Claudia Leoni, Giorgia Battipaglia, Daniela Carlino, Annalisa Condorelli, Valeria Di Giacomo, Sarah Marktel, Maria Antonietta Marzilli, Maurizio Miglino, Esther Natalie Oliva, Antonella Sau, Alessandra Ricco, Grazia Sanpaolo, Annarita Trolese, Anna Paola Iori, Simona Sica, Wilma Barcellini, Bruno Fattizzo

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Abstract

In this study, we collected real-world evidence on the use of pegcetacoplan among 22 Italian patients with paroxysmal nocturnal haemoglobinuria showing suboptimal response to anti-C5 treatments eculizumab and ravulizumab. Most patients exhibited a complete or good response as per the criteria of the European Bone Marrow Transplant group (Risitano et al. Front Immunol 2019) and median haemoglobin improvement from baseline was +3.6 g/dL. During the 6-month follow-up, 27% of patients displayed a breakthrough haemolytic event mainly managed with supportive treatment. No thromboses occurred.

Abstract Image

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pegcetacoplan对阵发性夜间血红蛋白尿患者的实际疗效：一项意大利全国性研究。

在这项研究中，我们收集了22名意大利发作性夜间血红蛋白尿患者使用pegcetacoplan的真实证据，这些患者对抗c5治疗eculizumab和ravulizumab的反应不佳。根据欧洲骨髓移植组的标准，大多数患者表现出完全或良好的反应（Risitano等）。与基线相比，血红蛋白改善的中位数为+3.6 g/dL。在6个月的随访中，27%的患者表现出突破性的溶血事件，主要是通过支持治疗来控制的。未发生血栓形成。

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

British Journal of Haematology 医学-血液学

CiteScore

8.60

自引率

4.60%

发文量

565

审稿时长

1 months

期刊介绍： The British Journal of Haematology publishes original research papers in clinical, laboratory and experimental haematology. The Journal also features annotations, reviews, short reports, images in haematology and Letters to the Editor.