van Balen Erna C, O'Mahony Brian, Cnossen Marjon H, Dolan Gerard, Blanchette Victor S, Fischer Kathelijn, Gue Deborah, O'Hara Jamie, Iorio Alfonso, Jackson Shannon, Konkle Barbara A, Nugent Diane J, Coffin Donna, Skinner Mark W, Smit Cees, Srivastava Alok, van Eenennaam Fred, van der Bom Johanna G, Gouw Samantha C
Department of Clinical Epidemiology Leiden University Medical Center Leiden The Netherlands.
Irish Haemophilia Society Trinity College Dublin Ireland.
Res Pract Thromb Haemost. 2021 Mar 6;5(4):e12488. doi: 10.1002/rth2.12488. eCollection 2021 May.
Patient-relevant health outcomes for persons with hemophilia should be identified and prioritized to optimize and individualize care for persons with hemophilia. Therefore, an international group of persons with hemophilia and multidisciplinary health care providers set out to identify a globally applicable standard set of health outcomes relevant to all individuals with hemophilia.
A systematic literature search was performed to identify possible health outcomes and risk adjustment variables. Persons with hemophilia and multidisciplinary health care providers were involved in an iterative nominal consensus process to select the most important health outcomes and risk adjustment variables for persons with hemophilia. Recommendations were made for outcome measurement instruments.
Persons with hemophilia were defined as all men and women with an X-linked inherited bleeding disorder caused by a deficiency of coagulation factor VIII or IX with plasma activity levels <40 IU/dL. We recommend collecting the following 10 health outcomes at least annually, if applicable: (i) cure, (ii) impact of disease on life expectancy, (iii) ability to engage in normal daily activities, (iv) severe bleeding episodes, (v) number of days lost from school or work, (vi) chronic pain, (vii) disease and treatment complications, (viii) sustainability of physical functioning, (ix) social functioning, and (x) mental health. Validated clinical as well as patient-reported outcome measurement instruments were endorsed. Demographic factors, baseline clinical factors, and treatment factors were identified as risk-adjustment variables.
A consensus-based international set of health outcomes relevant to all persons with hemophilia, and corresponding measurement instruments, was identified for use in clinical care to facilitate harmonized longitudinal monitoring and comparison of outcomes.
应确定与血友病患者相关的健康结局并确定其优先级，以优化血友病患者的护理并实现个性化护理。因此，一个由血友病患者和多学科医疗服务提供者组成的国际小组着手确定一套适用于全球所有血友病患者的健康结局标准。
进行了系统的文献检索，以确定可能的健康结局和风险调整变量。血友病患者和多学科医疗服务提供者参与了一个反复的名义小组共识过程，以选择血友病患者最重要的健康结局和风险调整变量。针对结局测量工具提出了建议。
血友病患者定义为所有患有X连锁遗传性出血性疾病的男性和女性，这些疾病由凝血因子VIII或IX缺乏引起，血浆活性水平<40 IU/dL。我们建议在适用的情况下，至少每年收集以下10项健康结局：（i）治愈情况，（ii）疾病对预期寿命的影响，（iii）参与正常日常活动的能力，（iv）严重出血事件，（v）因病或工作缺勤天数，（vi）慢性疼痛，（vii）疾病和治疗并发症，（viii）身体功能的可持续性，（ix）社会功能，以及（x）心理健康。认可了经过验证有效的临床以及患者报告的结局测量工具。人口统计学因素、基线临床因素和治疗因素被确定为风险调整变量。
确定了一套基于共识的、适用于所有血友病患者的国际健康结局标准以及相应的测量工具，用于临床护理，以促进结局的统一纵向监测和比较。
