Development and psychometric evaluation of a novel tool for assessing patient perception and preference for haemophilia treatment (HaemoPREF)
Development and psychometric evaluation of a novel tool for assessing patient perception and preference for haemophilia treatment (HaemoPREF)
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DOI：
10.1111/hae.12432
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发表时间：
2014-09-01
期刊：
影响因子：
3.9
通讯作者：
Cappelleri, J. C.
中科院分区：
文献类型：
--
作者：
Teal, S.;Brohan, E.;Cappelleri, J. C.
预防性使用治疗对于血友病的良好结局很重要，但依从性可能不理想。为了更好地理解治疗依从性和患者对治疗的信念之间的关系，需要量化患者的治疗态度。本研究的目的是制定一个简短的、临床相关的、患者报告的结果 (PRO)，以衡量易用性和患者对血友病治疗的偏好。来自奥地利、德国、意大利、西班牙和英国的 A 型血友病男性成人完成了一份包含 40 项的调查问卷。用于项目评估的稳健统计方法，包括项目级统计、维度分析以及临床和结果专家的输入，用于为项目减少提供信息。保留的项目接受心理测量评估，包括探索性因素分析（EFA）、已知群体的有效性和内部一致性信度。 273 名患者完成了问卷调查。在这 40 个项目中，有 28 个项目根据项目级统计数据被标记为可能删除，其中 3 个项目由于临床相关性而被保留。有两个项目具有可接受的统计性能，但由于临床相关性较低而被删除。共保留13项。 EFA 制定了概念性定义的 5 因素解决方案。该调查具有可接受的已知群体有效性和内部一致性。对措辞和评分进行了改进，并添加了一项新项目来评估总体易用性，从而形成了一份包含 14 项的调查问卷 - HaemoPREF。 HaemoPREF 的初步测量特性支持该仪器评估患者对血友病治疗的看法和偏好。需要进一步的心理测量评估来检查和确认量表的测量特性。
Prophylactic use of treatment is important for good outcomes in haemophilia, yet adherence can be suboptimal. To better understand the relationship between treatment adherence and patients' beliefs about treatment there is a need to quantify patients' treatment attitudes. The aim of this study was to develop a brief, clinically relevant, patient-reported outcome (PRO) to measure ease of use and patients' preference for haemophilia treatment. A 40-item questionnaire was completed by male adults with haemophilia A from Austria, Germany, Italy, Spain and the UK. Robust statistical methods for item evaluation including item-level statistics, dimensionality analyses and input from clinical and outcomes experts were used to inform item reduction. Retained items were subjected to psychometric evaluation including exploratory factor analysis (EFA), known-groups validity and internal consistency reliability. 273 patients completed the questionnaire. Of the 40 items, 28 items were flagged for possible deletion based on item-level statistics, three of which were retained due to clinical relevance. Two items had acceptable statistical performance but were deleted based on low clinical relevance. A total of 13 items were retained. EFA produced a conceptually defined 5-factor solution. The survey had acceptable known-groups validity and internal consistency. Refinements were made to wording and scoring, and one new item was added to assess general ease of use, resulting in a 14-item questionnaire - the HaemoPREF. Preliminary measurement properties of the HaemoPREF support the instrument to evaluate patient perception and preference for haemophilia treatment. Further psychometric evaluation is required to examine and confirm the measurement properties of the scale.