FACIT-fatigue score and treatment quality assessment for paroxysmal nocturnal hemoglobinuria patients treated with Eculizumab in Türkiye

@article{ozdemir2026facitfatigue,
  title = {{FACIT-fatigue score and treatment quality assessment for paroxysmal nocturnal hemoglobinuria patients treated with Eculizumab in Türkiye}},
  author = {Zehra Narlı Özdemir and Fatma Keklik Karadağ and Nuray Gül Acar and Merve Aydoğan and Filiz Yavaşoğlu and Fatma Temiz and Ajda Güneş and Gülşah Akyol and Haşim Atakan Erol and Özgür Mehtap and Vahap Okan and Merve Kakçı and İsmail Can Kendir and Turhan Köksal and Utku Iltar and Ramazan Esen and Gülten Korkmaz and Mesut Ayer and Esin Oğuz and Ozan Salim and Anıl Tombak and Gülsüm Özet and Mehmet Sönmez and Nil Güler and Mehmet Yilmaz and Mehmet Ali Özcan and Ali Ünal and Tülin Tuğlular and Selami Koçak Toprak and Birol Güvenç and Mustafa Nuri Yenerel and Osman İlhan and Güray Saydam and Fahri Şahin},
  journal = {PLOS One},
  year = {2026},
  month = sep,
  doi = {10.1371/journal.pone.0355720},
  url = {https://doi.org/10.1371/journal.pone.0355720},
  note = {CC BY 4.0},
}

TY  - JOUR
TI  - FACIT-fatigue score and treatment quality assessment for paroxysmal nocturnal hemoglobinuria patients treated with Eculizumab in Türkiye
AU  - Zehra Narlı Özdemir
AU  - Fatma Keklik Karadağ
AU  - Nuray Gül Acar
AU  - Merve Aydoğan
AU  - Filiz Yavaşoğlu
AU  - Fatma Temiz
AU  - Ajda Güneş
AU  - Gülşah Akyol
AU  - Haşim Atakan Erol
AU  - Özgür Mehtap
AU  - Vahap Okan
AU  - Merve Kakçı
AU  - İsmail Can Kendir
AU  - Turhan Köksal
AU  - Utku Iltar
AU  - Ramazan Esen
AU  - Gülten Korkmaz
AU  - Mesut Ayer
AU  - Esin Oğuz
AU  - Ozan Salim
AU  - Anıl Tombak
AU  - Gülsüm Özet
AU  - Mehmet Sönmez
AU  - Nil Güler
AU  - Mehmet Yilmaz
AU  - Mehmet Ali Özcan
AU  - Ali Ünal
AU  - Tülin Tuğlular
AU  - Selami Koçak Toprak
AU  - Birol Güvenç
AU  - Mustafa Nuri Yenerel
AU  - Osman İlhan
AU  - Güray Saydam
AU  - Fahri Şahin
PY  - 2026
DA  - 2026/09/11
JO  - PLOS One
DO  - 10.1371/journal.pone.0355720
UR  - https://doi.org/10.1371/journal.pone.0355720
AB  - 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.
SN  - 1932-6203
ER  -