Patient reported outcome measures in rare diseases: a narrative review.
Patient reported outcome measures in rare diseases: a narrative review.
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DOI：
10.1186/s13023-018-0810-x
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发表时间：
2018-04-23
影响因子：
3.7
通讯作者：
Calvert M
中科院分区：
文献类型：
--
作者：
Slade A;Isa F;Kyte D;Pankhurst T;Kerecuk L;Ferguson J;Lipkin G;Calvert M
罕见疾病可能导致患者及其家属的生活质量显著降低。确保患者的声音是临床决策的核心，是提供，评估和理解治疗干预措施有效性的关键。患者报告结果测量（PROM）用于捕获患者对其健康状况的看法，并促进我们了解这些疾病及其治疗对患者生活质量和症状的影响。本综述探讨了目前在罕见病中使用PROM的一些问题，包括患者人群少和缺乏有效的PROM。在验证新的或当前的PROM用于临床试验和研究的困难进行了讨论。该审查突出了审查中概述的一些问题的潜在解决方案，并讨论了PROM在研究和临床实践中的实施。在PROM的整个开发过程中，患者的输入（包括定性研究）对于确保适当捕获对罕见疾病患者重要的结果至关重要。鉴于罕见疾病的数量众多，每种疾病的患者数量较少，以及仪器开发的成本，可能需要创造性和务实的解决方案来开发和使用PROM。解决方案包括定性访谈，现代心理测量学和资源，如项目银行和计算机自适应测试。在罕见病研究和临床实践中使用PROM提供了改善患者护理和临床结局的潜力。
Rare diseases can lead to a significant reduction in quality of life for patients and their families. Ensuring the patients voice is central to clinical decision making is key to delivering, evaluating and understanding the efficacy of therapeutic interventions. Patient reported outcome measures (PROMs) are used to capture the patient’s views about their health status and facilitate our understanding of the impact of these diseases and their treatments on patient’s quality of life and symptoms. This review explores some of the current issues around the utilisation of PROMs in rare diseases, including small patient populations and dearth of valid PROMs. Difficulties in validating new or current PROMs for use in clinical trials and research are discussed. The review highlights potential solutions for some of the issues outlined in the review and the implementation of PROMs in research and clinical practice are discussed. Patient input throughout the development of PROMs including qualitative research is essential to ensure that outcomes that matter to people living with rare disease are appropriately captured. Given the large number of rare diseases, small numbers of patients living with each condition and the cost of instrument development, creative and pragmatic solutions to PROM development and use may be necessary. Solutions include qualitative interviews, modern psychometrics and resources such as item banking and computer adaptive testing. Use of PROMs in rare disease research and clinical practice offers the potential to improve patient care and clinical outcomes.
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影响因子：
3.7
作者：
Calvert M;Kyte D;Duffy H;Gheorghe A;Mercieca-Bebber R;Ives J;Draper H;Brundage M;Blazeby J;King M
通讯作者：
King M
影响因子：
10.3
作者：
Basch, Ethan;Jia, Xiaoyu;Schrag, Deborah
通讯作者：
Schrag, Deborah
影响因子：
17.3
作者：
Calvert, Melanie;Thwaites, Rob;Devlin, Nancy
通讯作者：
Devlin, Nancy
影响因子：
5.7
作者：
Basch, Ethan;Bennett, Antonia V.
通讯作者：
Bennett, Antonia V.
影响因子：
3.7
作者：
Bosch, Annet M.;Burlina, Alberto;Regnault, Antoine
通讯作者：
Regnault, Antoine