Van Creveldkliniek, University Medical Centre Utrecht, Utrecht University, Utrecht, The Netherlands.
Center for Child Development, Exercise and Physical Literacy, University Medical Centre Utrecht, Utrecht University, University Children's Hospital, Utrecht, The Netherlands.
Haemophilia. 2021 Nov;27(6):1062-1070. doi: 10.1111/hae.14408. Epub 2021 Sep 8.
The Haemophilia Activities List (HAL) was developed to measure activities and participation in persons with haemophilia (PWH). Shortening the questionnaire may facilitate use of the HAL.
The aim of this study was to determine which items of the HAL are redundant, to construct a shorter version of the HAL, and to determine the construct validity of the HAL .
A secondary analysis was performed on pooled data of two published studies using the HAL (seven domains, 42 items, optimum score: 100) in adults with haemophilia A/B. Data were divided into a derivation (62%) and a validation set (38%). Redundant items were identified by evaluation of: floor and ceiling effects, proportions of missing and 'not applicable' responses, inter-item correlations, component loadings in an exploratory factor analysis, internal consistency, and item-total correlations. Correlations with the SF-36 and EQ-5D-5L were used to determine construct validity of the HAL .
Data on 680 PWH were evaluated. In the derivation dataset (n = 420), median age was 30 years (range 18-80), 43% had severe haemophilia and 61% received prophylaxis. Median (IQR) HAL sum score was 65.0 (55.7-88.8). The stepwise procedure resulted in a HAL of 18 items with a median sum score of 63.3 (54.4-86.7). Construct validity was similar for the HAL and HAL in the validation dataset (n = 260).
This clinimetric study resulted in a >50% shortening of the HAL. The 18-item HAL reduces patient burden and is expected to capture the information on activities and participation. The HAL needs further validation.
血友病活动清单（HAL）旨在衡量血友病患者（PWH）的活动和参与情况。缩短问卷可能会更便于使用 HAL。
本研究旨在确定 HAL 中的哪些项目是多余的，构建一个更简短的 HAL，并确定 HAL 的结构效度。
对使用 HAL（七个领域，42 个项目，最佳得分为 100）的两项已发表研究的 pooled 数据进行二次分析，研究对象为血友病 A/B 成人患者。数据分为推导数据集（62%）和验证数据集（38%）。通过评估：地板效应和天花板效应、缺失和“不适用”响应的比例、项目间相关性、探索性因素分析中的组成负荷、内部一致性和项目总分相关性来识别冗余项目。使用 SF-36 和 EQ-5D-5L 的相关性来确定 HAL 的结构效度。
对 680 名 PWH 的数据进行了评估。在推导数据集（n=420）中，中位年龄为 30 岁（范围 18-80 岁），43%患有重度血友病，61%接受预防性治疗。HAL 总分中位数（IQR）为 65.0（55.7-88.8）。逐步过程产生了一个由 18 个项目组成的 HAL，总分中位数为 63.3（54.4-86.7）。在验证数据集（n=260）中，HAL 和 HAL 的结构效度相似。
这项临床研究导致 HAL 缩短了>50%。18 项 HAL 减少了患者负担，预计能捕捉到活动和参与情况的信息。HAL 需要进一步验证。
