Ratnayake Irushi, Ahern Susannah, Ruseckaite Rasa
Department of Epidemiology and Preventive Medicine, Monash University, Clayton, Victoria, Australia.
Department of Epidemiology and Preventive Medicine, Monash University, Clayton, Victoria, Australia
BMJ Open. 2020 Oct 1;10(10):e033867. doi: 10.1136/bmjopen-2019-033867.
To determine patient-reported outcome measures (PROMs) which may be suitable for incorporation into the Australian Cystic Fibrosis Data Registry (ACFDR) by identifying PROMs administered in adult and paediatric cystic fibrosis (CF) populations in the last decade.
We searched MEDLINE, EMBASE, Scopus, CINAHL, PsycINFO and Cochrane Library databases for studies published between January 2009 and February 2019 describing the use of PROMs to measure health-related quality of life (HRQoL) in adult and paediatric patients with CF. Validation studies, observational studies and qualitative studies were included. The search was conducted on 13 February 2019. The COnsensus-based Standards for the selection of health Measurement INstruments Risk of Bias Checklist was used to assess the methodological quality of included studies.
Twenty-seven different PROMs were identified. The most commonly used PROMs were designed specifically for CF. Equal numbers of studies were conducted on adult (32%, n=31), paediatric (35%, n=34) and both (27%, n=26) populations. No PROMs were used within a clinical registry setting previously. The two most widely used PROMs, the Cystic Fibrosis Questionnaire-Revised (CFQ-R) and the Cystic Fibrosis Quality of Life Questionnaire (CFQoL), demonstrated good psychometric properties and acceptability in English-speaking populations.
We found that although PROMs are widely used in CF, there is a lack of reporting on the efficacy of methods and timepoints of administration. We identified the CFQ-R and CFQoL as the most suitable for incorporation in the ACFDR as they captured significant effects of CF on HRQoL and were reliable and valid in CF populations. These PROMs will be used in a further qualitative study assessing patients' with CF and clinicians' perspectives toward the acceptability and feasibility of incorporating a PROM in the ACFDR.
CRD42019126931.
通过识别过去十年中在成人和儿童囊性纤维化（CF）人群中使用的患者报告结局量表（PROMs），确定可能适合纳入澳大利亚囊性纤维化数据登记处（ACFDR）的量表。
我们检索了MEDLINE、EMBASE、Scopus、CINAHL、PsycINFO和Cochrane图书馆数据库，以查找2009年1月至2019年2月发表的描述使用PROMs测量成人和儿童CF患者健康相关生活质量（HRQoL）的研究。纳入验证研究、观察性研究和定性研究。检索于2019年2月13日进行。使用基于共识的健康测量工具选择标准偏倚风险清单来评估纳入研究的方法学质量。
识别出27种不同的PROMs。最常用的PROMs是专门为CF设计的。针对成人（32%，n = 31）、儿童（35%，n = 34）以及两者（27%，n = 26）人群进行的研究数量相等。以前在临床登记环境中未使用过PROMs。使用最广泛的两种PROMs，即修订版囊性纤维化问卷（CFQ-R）和囊性纤维化生活质量问卷（CFQoL），在英语人群中显示出良好的心理测量特性和可接受性。
我们发现，尽管PROMs在CF中广泛使用，但缺乏关于方法有效性和给药时间点的报告。我们确定CFQ-R和CFQoL最适合纳入ACFDR，因为它们捕捉到了CF对HRQoL的显著影响，并且在CF人群中可靠且有效。这些PROMs将用于进一步的定性研究，评估CF患者和临床医生对将PROM纳入ACFDR的可接受性和可行性的看法。
PROSPERO注册号：CRD42019126931。
