The Paediatric Haemophilia Activities List (pedHAL) in routine assessment: changes over time, child-parent agreement and informative domains.

Abstract

IntroductionThe Paediatric Haemophilia Activities List (pedHAL) assesses self‐reported limitations in activities and participation in children with haemophilia.AimTo assess longitudinal changes, child‐parent agreement and to identify which pedHAL domains yielded most information in boys with access to early prophylaxis.MethodsThe 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.ResultsSeventy‐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.’ConclusionIn 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.