Health-related quality of life in haemophilia patients with inhibitors and their caregivers.

Data on the health-related quality of life (HRQoL) of congenital haemophilia patients with inhibitors (CHwI) and their caregivers are limited. To understand the association between patient demo-graphics/clinical characteristics with HRQoL among CHwI patients and caregivers, a survey was developed to assess HRQoL with haemophilia-specific QoL questionnaires (HAEMO-QoL/HAEM-A-QoL). In the cross-sectional study, paper-pencil questionnaires were mailed to 261 US CHwI patients/caregivers in July 2010. Descriptive analyses were performed to characterize HRQoL by age and to identify drivers of impairment, from both patient/caregiver perspectives. HRQoL scores were transformed on a scale of 0-100, with higher scores indicating higher impairment in HRQoL. Ninety-seven respondents completed the HRQoL assessment. HRQoL impairment was higher in adult patients. In children ages 8-16 years, mean HAEMO-QoL total score was 33.8 (SD = 15.5), and 35.0 (SD = 16.1) in children ages 4-7 years; for adult patients the mean HAEM-A-QoL total score was 42.2 (SD = 14.8). Adults reported highest impairment in the 'sports/leisure' subscale (Mean = 62.5, SD = 18.7), whereas patients 8-16 years reported highest impairment in the 'physical health' subscale (Mean = 50.8, SD = 30.5).Caregivers of patients ages 4-7 years reported greatest impairment within the 'family' subscale (Mean = 55.6, SD = 19.4). Caregivers were ''considerably/very much'' bothered by their child's inhibitors and reported higher QoL impairment for their child than parents who were not bothered. Within ChwI patients, HRQoL impairments increased with age and existed across a range of physical/psychosocial domains. In addition, caregiver burden also affected the perceived HRQoL of paediatric CHwI patients. Additional research is considered necessary to further understand the support caregivers need while caring for children with CHwI.