Flood Emuella, Pocoski Jennifer, Michaels Lisa A, Bell Jill A, Valluri Satish, Sasanè Rahul
Oxford Outcomes, an ICON plc. Company, Bethesda, MD, USA.
Eur J Haematol. 2014 Jun;93 Suppl 75:9-18. doi: 10.1111/ejh.12328.
INTRODUCTION/AIMS: Haemophilia and its treatment have a significant impact on patients' lives. The study objectives were to understand the impacts of haemophilia and its treatment from the patient perspective and to inform the development of comprehensive health-related quality-of-life (HRQL) conceptual models to illustrate these impacts.
The study included two phases. Phase I involved a review of literature published from 1995 to 2010, qualitative analysis of six patient (N = 31) and three healthcare provider (N = 15) focus group transcripts, and interviews with two experts to inform draft conceptual models of mild/moderate and severe haemophilia. Phase II involved interviews with 20 haemophilia patients and qualitative analysis of transcripts to confirm the concepts and structure of the conceptual models.
The literature search resulted in 66 publications assessing HRQL, four of which were qualitative studies on the impact of haemophilia from the patient perspective. Results from Phase I indicated that acute bleeding events result in pain, swelling, bruising and restricted joint movement; repeated joint bleeds result in chronic symptoms, such as pain and arthropathy. Acute bleeds cause interruptions in daily activities and interfere with work/school. Patients have fears about having bleeds, which can affect their participation in activities, such as sports or crowded events. Patients also expressed feelings of depression, frustration, isolation and embarrassment. Results of Phase II corroborated findings from Phase I.
The conceptual models illustrate the substantial impact of haemophilia and its treatments on patients' lives and can help inform clinical study design and the selection of endpoints to assess treatment benefit.
引言/目的：血友病及其治疗对患者的生活有重大影响。本研究的目的是从患者角度了解血友病及其治疗的影响，并为制定全面的健康相关生活质量（HRQL）概念模型提供信息，以阐明这些影响。
该研究包括两个阶段。第一阶段包括对1995年至2010年发表的文献进行综述，对六个患者焦点小组记录（N = 31）和三个医疗服务提供者焦点小组记录（N = 15）进行定性分析，以及对两位专家进行访谈，以形成轻度/中度和重度血友病的概念模型草案。第二阶段包括对20名血友病患者进行访谈，并对访谈记录进行定性分析，以确认概念模型的概念和结构。
文献检索得到66篇评估HRQL的出版物，其中四篇是从患者角度对血友病影响的定性研究。第一阶段的结果表明，急性出血事件会导致疼痛、肿胀、瘀伤和关节活动受限；反复的关节出血会导致慢性症状，如疼痛和关节病。急性出血会导致日常活动中断，并干扰工作/学习。患者担心出血，这会影响他们参与体育或拥挤活动等。患者还表达了抑郁、沮丧、孤立和尴尬的情绪。第二阶段的结果证实了第一阶段的发现。
这些概念模型阐明了血友病及其治疗对患者生活的重大影响，并有助于为临床研究设计和评估治疗益处的终点选择提供信息。
