ArticleEJHaem2026
Clinical Profile, Humanistic and Economic Burden of Paroxysmal Nocturnal Hemoglobinuria in Patients Treated With C5 Inhibitors.
Article in EJHaem, 2026. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Who cites it
1 citing paper in PubMed.
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Authors and funding
10 authors.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Objectives: To evaluate disease burden among paroxysmal nocturnal hemoglobinuria (PNH) patients prescribed C5 inhibitors (C5i). Methods: Data were drawn from the Adelphi Real World PNH Disease Specific Programme, a cross-sectional survey of physicians and PNH patients in Australia, Canada, France, Germany, Italy, Spain, the United Kingdom, and Japan from January-December 2022. Physicians reported patient demographics, laboratory parameters, and treatments. Patients completed the EQ-5D-5L, Functional Assessment of Chronic Illness Therapy - Fatigue (FACIT-Fatigue), and Work Productivity and Activity Impairment (WPAI) questionnaire. Analyses were descriptive. Results: Overall, 81 physicians reported on 288 patients receiving C5i. Median (IQR) age was 50.0 (38.3-65.0) years, 79.2% were white, and 54.5% were male. Median (IQR) C5i duration was 1.0 (0-3.0) years; 77.4% received eculizumab, 22.6% ravulizumab. At time of survey, median (IQR) hemoglobin (Hb) was 11.0 (9.9-12.0) g/dL, 73.5% of patients had Hb < 12 g/dL. Lactate dehydrogenase was >1.5× upper limit of normal for 16.7% of patients. Mean (SD) EQ-5D-5L was 0.76 (0.20), FACIT-Fatigue was 36.1 (9.7), WPAI work impairment was 27.5% (22.3) and activity impairment was 35.3% (24.6). Conclusions: Despite C5i treatment, a notable proportion of patients remained anemic and reported impaired quality of life, indicating the need for novel, efficacious therapies.
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