title: Treatment patterns, health-related quality of life and adherence to prophylaxis among haemophilia A patients in the United States.
abstract: Prophylaxis and adherence to prophylaxis are increasingly recognized as important factors for the health-related quality of life (HRQOL) of haemophilia patients. This study aims to assess treatment practices over time, HRQOL and adherence among severe haemophilia A patients in the US. Severe haemophilia A patients or their caregivers participated in a 2009 cross-sectional survey. HRQOL was measured using either PEDS-QL or SF-12; adherence was measured using the VERITAS-Pro. Student t-tests evaluated differences between children vs. adults and self-infusion status. A total of 117 respondents participated in the survey, capturing data for 64 adults (mean age = 37.9 years) and 53 children (mean age = 10.5 years). Although 96% of paediatric patients were currently receiving prophylaxis, only 32 (50%) adults reported receiving prophylaxis at some point in their life. Adults who have always been on prophylaxis reported better physical functioning and physical HRQOL (both P < 0.05) than adults who had not. The paediatric group reported better adherence compared to the adult group on the total scale (38 vs. 45.8, P < 0.05). Children <12 years had higher adherence than adolescents 12-18 years old (35.5 vs. 40.8; P < 0.05). Paediatric patients infused by family members showed better adherence than paediatric self-infusers (P < 0.05). This study showed different treatment patterns between paediatric and adult patients and how the patterns impacted HRQOL. It also provided the first standardized evaluation of adherence using the VERITAS-Pro in a US national sample. This study enhances understanding of treatment practices and adherence for the US haemophilia population and may offer insight into where adherence can be improved.
authors.affiliation: Indiana Hemophilia and Thrombosis Center, Indianapolis, IN, USA.
authors.forename: N
authors.initials: N
authors.lastname: Duncan
authors.forename: A
authors.initials: A
authors.lastname: Shapiro
authors.forename: X
authors.initials: X
authors.lastname: Ye
authors.forename: J
authors.initials: J
authors.lastname: Epstein
authors.forename: M P
authors.initials: MP
authors.lastname: Luo
mesh_terms: D000293:Adolescent; D000328:Adult; D000367:Age Factors; D002648:Child; D003430:Cross-Sectional Studies; D003625:Data Collection; D034742:Electronic Mail; D005169:Factor VIII / Q000008:administration & dosage; D006395:Hemarthrosis / Q000517:prevention & control; D006467:Hemophilia A / Q000150:complications / Q000628:therapy*; D006801:Humans; D008297:Male; D010349:Patient Compliance; D011788:Quality of Life; D012646:Self Administration; D014481:United States
chemical_list: D005169:Factor VIII