Real-world clinical outcomes and treatment response in complement inhibitor experienced and naïve PNH patients prescribed pegcetacoplan in Europe and Canada.

IF 2 4区医学 Q3 HEMATOLOGY

Hematology Pub Date : 2026-12-01 Epub Date: 2026-01-02 DOI:10.1080/16078454.2025.2608510

Jens Panse, Juan Carlos Vallejo Llamas, Koo Wilson, Zalmai Hakimi, Barbara Czech, Brianne Kerr, Sam Williamson, Yasmin Taylor, Niall Hatchell, Christopher J Patriquin

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引用次数: 0

Abstract

Objectives: This study investigated outcomes and response to pegcetacoplan in complement inhibitor naïve (CI_naïve) and experienced (CI_exp) PNH patients.

Methods: Data were drawn from the Adelphi PNH Disease Specific Programme™, a cross-sectional survey of physicians and their patients in Canada, France, Germany, Italy, Spain, and the UK between December 2023 and April 2024. Physicians reported patient characteristics and clinical outcomes, which were used to define pegcetacoplan response. Results were stratified by prior CI prescription. Analyses were descriptive.

Results: Forty-eight physicians reported on 63 patients (CI_naïve:11; CI_exp:52). Median (IQR) age was 45.0 (33.0-55.0) years, 60.3% were male. At treatment initiation versus at survey for CI_naïve and CI_exp, respectively, median (IQR) haemoglobin levels (g/dL) were 8.0 (5.0-9.0) versus 12.0 (11.2-12.3), and 9.0 (8.0-9.2) versus 11.0 (10.2-12.0); lactate dehydrogenase levels (U/L) were 500 (350-1625) versus 250 (175-525), and 470 (290-588) versus 265 (190-379); absolute reticulocyte counts (x10⁹ /L) were 220 (151-250) versus 110 (95-140), and 212 (134-281) versus 120 (105-228). At pegcetacoplan initiation, 4.4% of patients met criteria of good-to-complete response versus 79.5% at survey.

Conclusions: Pegcetacoplan proved effective for CI_naïve and CI_exp patients in a real-world setting, with numerically improved clinical outcomes and high response rate.

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在欧洲和加拿大，补体抑制剂经历和naïve PNH患者服用pegcetacoplan的实际临床结果和治疗反应。

目的：本研究调查补体抑制剂naïve （CInaïve）和经验（CIexp） PNH患者对pegcetacoplan的疗效和反应。方法：数据来自Adelphi PNH疾病特异性计划™，该计划是一项横断面调查，于2023年12月至2024年4月期间对加拿大、法国、德国、意大利、西班牙和英国的医生及其患者进行调查。医生报告了患者的特征和临床结果，用于定义pegcetacoplan反应。结果按既往CI处方分层。分析是描述性的。结果：48名医生报告63例患者（CInaïve:11例；CIexp:52例）。中位（IQR）年龄为45.0（33.0 ~ 55.0）岁，男性占60.3%。治疗开始时与CInaïve和CIexp调查时相比，中位（IQR）血红蛋白水平（g/dL）分别为8.0（5.0-9.0）和12.0(11.2-12.3),9.0（8.0-9.2）和11.0 (10.2-12.0)；乳酸脱氢酶水平（U/L）分别为500（350-1625）和250(175-525),470（290-588）和265 (190-379)；绝对网织红细胞计数（x109 /L）分别为220（151-250）和110(95-140),212（134-281）和120（105-228）。在pegcetacoplan开始治疗时，4.4%的患者符合良好至完全缓解标准，而调查时为79.5%。结论：在现实世界中，Pegcetacoplan被证明对CInaïve和CIexp患者有效，具有数值改善的临床结果和高有效率。

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

Hematology 医学-血液学

CiteScore

2.60

自引率

5.30%

发文量

140

审稿时长

3 months

期刊介绍： Hematology is an international journal publishing original and review articles in the field of general hematology, including oncology, pathology, biology, clinical research and epidemiology. Of the fixed sections, annotations are accepted on any general or scientific field: technical annotations covering current laboratory practice in general hematology, blood transfusion and clinical trials, and current clinical practice reviews the consensus driven areas of care and management.