Van Creveldkliniek, University Medical Center Utrecht, Utrecht University, Utrecht, The Netherlands.
Center for Child Development, Exercise and Physical Literacy, University Medical Center Utrecht, Utrecht University, University Children's Hospital, Utrecht, The Netherlands.
Haemophilia. 2019 Nov;25(6):953-959. doi: 10.1111/hae.13835. Epub 2019 Aug 16.
The Paediatric Haemophilia Activities List (pedHAL) assesses self-reported limitations in activities and participation in children with haemophilia.
To assess longitudinal changes, child-parent agreement and to identify which pedHAL domains yielded most information in boys with access to early prophylaxis.
The pedHAL (53 items, 7 domains, optimum 100) was completed annually at the Van Creveldkliniek by boys aged 4-18 years with moderate/severe haemophilia and their parents. Development of the pedHAL in relation to bleeds, changes per domain over 3-5 years, child-parent agreement (% difference child-parent≤|5|) per domain and domain scores (limitations defined as ≤ 95) were determined.
Seventy-three patients and their parents (92% severe haemophilia, median age 13.1 years [range 5.4;18.0]) completed ≥1 pedHAL. Median (IQR) pedHAL sum score was 99.5 (95.2;100.0) for children and 99.6 (95.8;100.0) for parents. If patients scored >95 and had no joint and/or muscle bleed, 90.9% of the patients scored >95 at the next assessment. The median change in sum score was 0.0 for both the 3- and 5-year interval. Child-parent agreement varied between domains from 92% ('self-care') to 71% ('sitting/kneeling/standing'). Most limitations were reported in the domains 'sitting/kneeling/standing', 'functions of the legs' and 'leisure activities and sports.'
In routine clinical practice in Dutch children on prophylaxis, pedHAL scores were high and remained stable in 3-5 years at group level. In individual patients without joint and/or muscle bleeds, pedHAL scores remained high after 1 year. Child-parent agreement was not optimal which indicated that both child report and parent proxy should be reported.
儿科血友病活动清单（pedHAL）评估血友病儿童的活动受限和参与情况。
评估纵向变化、患儿与家长的一致性，并确定哪些 pedHAL 领域在接受早期预防治疗的男孩中提供最多信息。
在 Van Creveldkliniek，年龄在 4-18 岁、患有中重度血友病的男孩及其家长每年使用 pedHAL（共 53 项，分为 7 个领域，最佳值为 100）进行自评。确定 pedHAL 的发展与出血的关系、3-5 年内各领域的变化、各领域的患儿与家长的一致性（%差异患儿-家长≤|5|）以及领域得分（定义为≤95 的为受限）。
73 名患儿及其家长（92%为重度血友病，中位年龄 13.1 岁[范围 5.4-18.0]）完成了至少 1 次 pedHAL。患儿的 pedHAL 总分中位数（IQR）为 99.5（95.2-100.0），家长的为 99.6（95.8-100.0）。如果患儿评分>95 且无关节和/或肌肉出血，90.9%的患儿在下一次评估时评分>95。3 年和 5 年间隔的总分中位数变化均为 0.0。患儿与家长的一致性在各领域存在差异，从 92%（“自我护理”）到 71%（“坐/跪/站”）。大多数限制发生在“坐/跪/站”、“腿部功能”和“休闲活动和运动”等领域。
在荷兰接受预防治疗的儿童常规临床实践中，pedHAL 评分较高，在 3-5 年内保持稳定。在没有关节和/或肌肉出血的个别患儿中，1 年后 pedHAL 评分仍较高。患儿与家长的一致性不理想，这表明应同时报告患儿自评和家长代评。
