Dingli David, Rizio Avery A, Broderick Lynne, LaGasse Kaitlin, Rucker Sloan, Carty Michelle K, Burton Elise, Gordon Shaquilla, Yen Glorian P, Paulose Jincy, Geevarghese Anumaxine, Lee Soyon
Mayo Clinic, Rochester, MN, USA.
QualityMetric, An IQVIA Business, 1301 Atwood Avenue, Suite 216E, Johnston, RI, 02919, USA.
Ann Hematol. 2024 Dec;103(12):5213-5227. doi: 10.1007/s00277-024-06088-x. Epub 2024 Nov 18.
This study describes the health-related quality of life (HRQoL) and symptom-specific functional impairment of patients with paroxysmal nocturnal hemoglobinuria (PNH) in a real-world setting. US-based adults with PNH treated with a parenterally administered complement inhibitor (PACI) for ≥ 6 months completed an online, cross-sectional, observational survey; a subset of patients also participated in semi-structured qualitative interviews. The survey included the PROMIS 29 + 2 Profile v2.1 (PROMIS 29 + 2) to measure HRQoL. The FACIT-Fatigue, Neuro-QOL Item Bank v2.0 Cognitive Function Short Form, and PROMIS Item Bank v1.0 Dyspnea Functional Limitations 10a Short Form measured symptom-specific functional impairment. For each patient with PNH who completed the online survey, 3 age- and sex-matched adults from the general population (GP) also completed the survey. The HRQoL and functional impairment of the PNH sample were compared to that of the GP sample. The association between HRQoL/functional impairment and fatigue severity for the PNH sample was also investigated. Compared to the age- and sex-matched GP sample, patients treated with PACIs for PNH had significantly worse HRQoL and greater functional impairment for all measured domains (p < 0.05). Within the PNH sample, statistically significant associations (p < 0.05) were observed between fatigue severity and HRQoL/functional impairment for all outcomes except the PROMIS 29 + 2 Sleep Disturbance domain. Interview participants described fatigue-related impairments in their physical, social, and cognitive functioning. Despite receiving treatment for PNH, patients experienced deficits in HRQoL and functional impairment, suggesting that opportunities to improve patient-relevant outcomes through treatment should be identified.
本研究描述了阵发性夜间血红蛋白尿（PNH）患者在真实世界中的健康相关生活质量（HRQoL）和症状特异性功能损害。接受肠外补体抑制剂（PACI）治疗≥6个月的美国成年PNH患者完成了一项在线横断面观察性调查；部分患者还参与了半结构化定性访谈。该调查包括PROMIS 29 + 2简表v2.1（PROMIS 29 + 2）以测量HRQoL。FACIT疲劳量表、神经QOL项目库v2.0认知功能简表以及PROMIS项目库v1.0呼吸困难功能受限10a简表用于测量症状特异性功能损害。对于每一位完成在线调查的PNH患者，还邀请了3名来自普通人群（GP）且年龄和性别匹配的成年人完成该调查。将PNH样本的HRQoL和功能损害与GP样本进行比较。还研究了PNH样本中HRQoL/功能损害与疲劳严重程度之间的关联。与年龄和性别匹配的GP样本相比，接受PACI治疗的PNH患者在所有测量领域的HRQoL均显著更差，功能损害更大（p < 0.05）。在PNH样本中，除PROMIS 29 + 2睡眠障碍领域外，所有结局的疲劳严重程度与HRQoL/功能损害之间均观察到统计学显著关联（p < 0.05）。访谈参与者描述了疲劳对其身体、社交和认知功能的损害。尽管接受了PNH治疗，但患者仍存在HRQoL缺陷和功能损害，这表明应确定通过治疗改善与患者相关结局的机会。
