FACIT-fatigue score and treatment quality assessment for paroxysmal nocturnal hemoglobinuria patients treated with Eculizumab in Türkiye
PloS one, cilt.21, sa.9, 2026 (SCI-Expanded, Scopus)
- Yayın Türü: Makale / Tam Makale
- Cilt numarası: 21 Sayı: 9
- Basım Tarihi: 2026
- Doi Numarası: 10.1371/journal.pone.0355720
- Dergi Adı: PloS one
- Derginin Tarandığı İndeksler: Science Citation Index Expanded (SCI-EXPANDED), Scopus, L'Année philologique, Aerospace Database, BIOSIS, Chemical Abstracts Core, EMBASE, Index Islamicus, Linguistic Bibliography, MEDLINE, Psycinfo, zbMATH, Directory of Open Access Journals, Zoological Record, Academic Search Ultimate (EBSCO), Natural Science Collection (ProQuest), Biological Science Database (ProQuest), Biomedical Reference Collection: Corporate Edition (EBSCO), Health Research Premium Collection (ProQuest), Materials Science & Engineering Collection (ProQuest), Pharma Collection (ProQuest), Technology Collection (ProQuest)
- Açık Arşiv Koleksiyonu: AVESİS Açık Erişim Koleksiyonu
- Kocaeli Üniversitesi Adresli: Evet
Özet
OBJECTIVE: Paroxysmal nocturnal hemoglobinuria (PNH) is an ultra-rare acquired clonal abnormality that makes hematopoietic cells highly vulnerable to complement-mediated destruction. Fatigue is one of the most commonly reported unresolved symptoms by patients with PNH. In this study, the fatigue status of PNH patients receiving eculizumab treatment and the factors affecting fatigue were evaluated using a quality-of-life questionnaire. MATERIALS AND METHODS: The study included 18 centers from Türkiye. The quality of life of PNH patients receiving eculizumab treatment was assessed through face-to-face surveys using the Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) scale. RESULTS: A total of 104 PNH patients treated with eculizumab were included in the study, of whom 51 (49%) were female and 53 (51%) were male. The mean (±SD) age of the patients was 48.04 (±16.16) years at the time of the survey. The mean (±SD) FACIT-Fatigue score was 32.63 (±11.79). A total of 39 (37.5%) patients had a fatigue score of 30 or below, which was defined as severe fatigue. Patients with severe fatigue had lower hemoglobin levels both at diagnosis and at the time of the survey [8.05 g/dL vs. 9.15 g/dL (p = 0.017) and 10.09 g/dL vs. 11.38 g/dL (p = 0.006), respectively]. The mean (±SD) duration of additional medication use was longer in the severe fatigue group compared to patients with a fatigue score >30 [97.92(±51.98) months vs. 75.94 (±50.55) months, p = 0.039]. In the multivariate analysis, the independent variables 'female gender,' 'presence of comorbidities,' 'duration of additional medication,' and 'initial hemoglobin level' were statistically significantly associated with severe fatigue. The female gender increased the likelihood of severe fatigue by 3.3 times, while the presence of comorbidities increased it by 7.1 times. CONCLUSION: In this study, which evaluated the fatigue status of PNH patients at various stages of eculizumab treatment in a real-life setting, more than one-third of the patients reported severe fatigue. Female patients experienced severe fatigue more than male patients, and anemia was not the sole factor contributing to severe fatigue in PNH patients. Comorbidities and medications related to these conditions also played a significant role in their quality of life.