Abstract | OBJECTIVES: To assess the clinical, humanistic and economic burden of paroxysmal nocturnal haemoglobinuria (PNH) among C5 inhibitor (C5i)-treated patients with PNH. METHODS: This was a web-based, cross-sectional survey (01FEB2021-31MAR2021) of adults with PNH treated with eculizumab (France, Germany, United Kingdom) or ravulizumab (Germany). Self-reported outcomes included: patient characteristics; patient-reported symptoms; and standardised patient-reported outcomes (e.g. Functional Assessment of Chronic Illness Therapy [FACIT]- Fatigue, European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 [EORTC QLQ-C30]). RESULTS: Among 71 included patients, 98.6% were C5i-treated for ≥3 months (88.7% ≥12 months); among those with self-reported haemoglobin (Hb) levels (n = 63), most (85.7%) were anaemic (defined as ≤12.0 g/dL). Fatigue was the most common symptom at both diagnosis (73.2%) and survey time (63.4%); there were no statistically significant differences in symptom prevalence between treatment subgroups ( eculizumab vs. ravulizumab). Total FACIT- Fatigue and EORTC QLQ-C30 scores were substantially lower than European general population references, but there were no statistically significant differences between treatment subgroups. Hb-level subgroups (<10.5 g/dL vs. ≥10.5 d/dL) followed similar trends for all measures, with few significant subgroup differences. CONCLUSIONS: Results suggest that there remains a considerable burden and unmet need among C5i-treated patients with PNH that requires improved therapies.
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Authors | Jens Panse, Flore Sicre de Fontbrune, Pascale Burmester, Maria Piggin, Joana E Matos, Halley Costantino, Koo Wilson, Zalmai Hakimi, Jameel Nazir, Renaud Desgraz, Jesse Fishman, Emmelie Persson, Austin Kulasekararaj |
Journal | European journal of haematology
(Eur J Haematol)
Vol. 109
Issue 4
Pg. 351-363
(Oct 2022)
ISSN: 1600-0609 [Electronic] England |
PMID | 35746830
(Publication Type: Journal Article)
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Copyright | © 2022 The Authors. European Journal of Haematology published by John Wiley & Sons Ltd. |
Topics |
- Adult
- Cost of Illness
- Cross-Sectional Studies
- Fatigue
(drug therapy, epidemiology, etiology)
- Germany
(epidemiology)
- Hemoglobinuria, Paroxysmal
(complications, diagnosis, drug therapy)
- Humans
- Patient Reported Outcome Measures
- Quality of Life
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