Children with severe hemophilia in a U.K. study had generally good self-reported health-related quality of life and physical functioning, but impairment on both measures was greater in those who reported pain.
Of 123 boys with a mean age of 12 years who were included in the multicenter SO-FIT study, 110 had hemophilia A, 25 had a past inhibitor, and 9 had a current inhibitor. Prophylactic treatment was given in 120 of the boys, Kate Khair, PhD of Great Ormond Street Hospital for Children, London, reported at the annual meeting of the European Association for Haemophilia and Allied Disorders.
In the preceding 6 months, participants experienced a median of 0 joint bleeds (range of 0-8), and a median Hemophilia Joint Health Score (HJHS) of 1 (range, 0-37). Physical function scores were good; the Haemophilia & Exercise Project questionnaire (HEP-Test-Q) mean score was 80.82, with the highest impairments seen in the domain of “endurance” (mean, 72.21), and the Paediatric Haemophilia Activities List (PedHAL) mean score was 85.13, with the highest impairments seen in the domains of “leisure activities and sports” and “lying/sitting/kneeling/standing” (mean, 82.04 and 82.97, respectively).
Health-related quality of life as assessed by the Haemo-QoL Short Form was also generally good (mean, 23.07), with the highest impairments seen in the domains of “friends” and “family” (mean, 27.08 and 26.59, respectively).
In the 27% of participants with pain, a high correlation was seen between the HEP-Test-Q and Haemo-QoL, and moderate correlation was seen between the PedHAL and Haemo-QoL, which implies that good physical functioning is related to good health-related quality of life, Dr. Khair explained.
In fact, patients who said they often or always had pain reported significantly worse subjective physical functioning in all HEP-Test-Q and PedHAL domains, as well as higher impairments in their HRQoL vs. those who said they sometimes, rarely, or never experienced pain, she said.
Interestingly, data completion by study participants was improved with use of an iPad vs. “paper and pencil” for questionnaire completion, Dr. Khair said in an interview.
“They preferred this method – it was more in keeping with how they communicate and learn at school,” she said, noting that the iPad database was set up to give real time scores, which could then be used to tailor assessments.
This is an approach that could be undertaken at home pre-clinic visits, she said.
“Gaining the views of children and young adults in patient-reported outcomes is complex, but reveals their views of their care and their lives – an area we should address more,” she added.
Dr. Khair received grant or research support from Pfizer ASPIRE.