Clinical profile, humanistic and economic burden of paroxysmal nocturnal hemoglobinuria in patients treated with C5 inhibitors
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Authors
Gandhi, Shreyans
Radsak, Markus P.
Ueda, Yasutaka
Balp, Maria‐Magdalena
Wiyani, Anggie
Somenzi, Olivier
Snellman, Josefin
Taylor, Yasmin
Simons, Alice
Panse, Jens
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2026
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Article
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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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eJHaem
Volume
7
Issue
1