Pegcetacoplan in paroxysmal nocturnal haemoglobinuria: Its use, its clinical effectiveness, and its influence on health‐related quality of life and productivity

Author:

Wilson Koo1,Rich Carly1,Hakimi Zalmai1,Horneff Regina1,Fishman Jesse2,Mellor Jennifer3,Earl Lucy3,Taylor Yasmin3,Simons Alice3,Conyers Joe3,Mulherin Brian4,Majerus Elaine5,Röth Alexander6

Affiliation:

1. Sobi Stockholm Sweden

2. Apellis Pharmaceuticals Waltham Massachusetts USA

3. Adelphi Real World Cheshire UK

4. Hematology Oncology of Indiana Indianapolis Indiana USA

5. Department of Medicine Washington University St. Louis Missouri USA

6. Department of Hematology and Stem Cell Transplantation University Hospital Essen, West German Cancer Center, University of Duisburg‐Essen Essen Germany

Abstract

AbstractObjectivesTo describe real‐world use/effectiveness of pegcetacoplan (PEG) in paroxysmal nocturnal haemoglobinuria (PNH).MethodsData were drawn from the Adelphi PNH Disease Specific Programme™, a cross‐sectional survey conducted in France, Italy, Germany, Spain and the United States from January to November 2022. Patients had a confirmed PNH diagnosis and received PEG for ≥1 month. Physicians reported patient characteristics, treatment use/satisfaction and their perception of patients' fatigue and health‐related quality of life (HRQoL). Patients reported treatment satisfaction and completed questionnaires assessing fatigue, HRQoL and productivity. Descriptive statistics were reported.ResultsOverall, 14 physicians provided data for 61 patients who had received 1080 mg/dose PEG for 1.3–14.8 months. At data collection compared to PEG initiation: haemoglobin was 2.5 g/dL higher on average; proportion of patients with lactate dehydrogenase (LDH) ≥1.5 × upper limit of normal was reduced by 27.4%; physician‐perceived fatigue was lower and HRQoL better. Physician‐ and patient‐reported treatment satisfaction was high for >90% of patients. Physicians and patients were more satisfied with PEG than previously prescribed C5 complement inhibitors. Mean work impairment and activity impairment in the 7 days prior to data collection were 32.9% and 22.4%, respectively.ConclusionsThese real‐world data support the effectiveness of PEG through positive effects on haemoglobin, LDH, fatigue and HRQoL.

Funder

Apellis Pharmaceuticals

Swedish Orphan Biovitrum

Publisher

Wiley

Subject

Hematology,General Medicine

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