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

Koo Wilson, Carly Rich, Zalmai Hakimi, Regina Horneff, Jesse Fishman, Jennifer Mellor, Lucy Earl, Yasmin Taylor, Alice Simons, Joe Conyers, Brian Mulherin, Elaine Majerus, Alexander Röth

Research output: Contribution to journalArticlepeer-review

Abstract

Objectives: To describe real-world use/effectiveness of pegcetacoplan (PEG) in paroxysmal nocturnal haemoglobinuria (PNH). Methods: Data 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. Results: Overall, 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. Conclusions: These real-world data support the effectiveness of PEG through positive effects on haemoglobin, LDH, fatigue and HRQoL.

Original languageEnglish
Pages (from-to)516-529
Number of pages14
JournalEuropean Journal of Haematology
Volume112
Issue number4
DOIs
StatePublished - Apr 2024

Keywords

  • absenteeism
  • complement inactivating agents
  • paroxysmal nocturnal haemoglobinuria
  • pegcetacoplan
  • presenteeism
  • quality of life
  • rare diseases
  • real-world evidence

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