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

Introduction The Paediatric Haemophilia Activities List (pedHAL) assesses self-reported limitations in activities and participation in children with haemophilia. Aim To assess longitudinal changes, child-parent agreement and to identify which pedHAL domains yielded most information in boys with access to early prophylaxis. Methods 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. Results 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.' Conclusion 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.