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

This study investigated outcomes and response to pegcetacoplan in complement inhibitor naïve (CInaïve) and experienced (CIexp) PNH patients. 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. Forty-eight physicians reported on 63 patients (CInaïve:11; CIexp:52). Median (IQR) age was 45.0 (33.0–55.0) years, 60.3% were male. At treatment initiation versus at survey for CInaïve and CIexp, 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. Pegcetacoplan proved effective for CInaïve and CIexp patients in a real-world setting, with numerically improved clinical outcomes and high response rate.