Psychosocial Department, Emma Children's Hospital/Academic Medical Center, Amsterdam, The Netherlands.
Department of Pediatric-Hematology, Hemophilia Comprehensive Care Treatment Center, Academic Medical Center, Emma Children's Hospital, Amsterdam, The Netherlands.
Haemophilia. 2018 May;24(3):395-404. doi: 10.1111/hae.13420. Epub 2018 Feb 8.
This study assesses health-related quality of life (HRQOL), and variables associated with HRQOL, in children and adolescents with haemophilia and congenital bleeding disorders (CBD) in the Netherlands.
Patients <18 years with CBD under treatment at the Hemophilia Comprehensive Care Center of the Academic Medical Center were included. Participants completed generic HRQOL questionnaires (TAPQOL 0-5 years; PedsQL 6-18 years). Differences and effect sizes in HRQOL compared to healthy peers, and between hemophilia severity groups, were tested using Mann Whitney U-tests. Multivariate regression analyses were performed to assess variables associated with HRQOL.
Data of 145 patients (81%) were analyzed (N = 32 with severe haemophilia). Children (0-12 years) show no significant impairments in HRQOL compared to healthy peers. Adolescent boys (13-18 years) with CBD report a slightly higher HRQOL on the total and emotional functioning scales than healthy peers (small-moderate effect sizes). In contrast, adolescent girls experience lower HRQOL on total, social functioning and psychosocial health scales compared to healthy peers (moderate effect sizes). No differences between severity groups were found in HRQOL, but more problem behaviour was found in young boys (0-5 years) with severe haemophilia. Male gender, participation in sports and school attendance are positively associated with HRQOL. Parental country of birth, type of treatment and number of bleeds are not associated with HRQOL.
Continuing monitoring HRQOL in daily clinical practice for children with CBD is important, since possible influencing psychosocial factors can change over time, with special focus on adolescent girls, sports participation and school absence.
本研究评估了荷兰患有血友病和先天性出血性疾病（CBD）的儿童和青少年的健康相关生活质量（HRQOL）及其与 HRQOL 相关的变量。
纳入在学术医学中心血友病综合护理中心接受治疗的 CBD 且年龄＜18 岁的患者。参与者完成了通用 HRQOL 问卷（TAPQOL 0-5 岁；PedsQL 6-18 岁）。使用 Mann Whitney U 检验比较 HRQOL 与健康同龄人之间的差异和效应大小，并比较不同血友病严重程度组之间的差异和效应大小。进行多元回归分析以评估与 HRQOL 相关的变量。
分析了 145 名患者（81%）的数据（N=32 名严重血友病患者）。与健康同龄人相比，0-12 岁的儿童 HRQOL 无明显受损。患有 CBD 的 13-18 岁青少年男孩在总评分和情绪功能评分方面报告的 HRQOL 略高于健康同龄人（中-小效应大小）。相比之下，患有 CBD 的青少年女孩在总评分、社会功能和心理社会健康评分方面的 HRQOL 低于健康同龄人（中-大效应大小）。在 HRQOL 方面，严重程度组之间无差异，但严重血友病的年轻男孩（0-5 岁）表现出更多的问题行为。男性、参与体育运动和上学与 HRQOL 呈正相关。父母的原籍国、治疗类型和出血次数与 HRQOL 无关。
在日常临床实践中继续监测 CBD 患儿的 HRQOL 很重要，因为随着时间的推移，可能影响心理社会的因素会发生变化，特别关注青少年女孩、运动参与和缺课。
