Treatment patterns, health-related quality of life and adherence to prophylaxis among haemophilia A patients in the United States

Haemophilia. 2012 Sep;18(5):760-5. doi: 10.1111/j.1365-2516.2012.02813.x. Epub 2012 May 1.


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.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Age Factors
  • Child
  • Cross-Sectional Studies
  • Data Collection
  • Electronic Mail
  • Factor VIII / administration & dosage
  • Hemarthrosis / prevention & control
  • Hemophilia A / complications
  • Hemophilia A / therapy*
  • Humans
  • Male
  • Patient Compliance
  • Quality of Life
  • Self Administration
  • United States


  • Factor VIII