Capturing daily assessments and home treatment of congenital hemophilia with inhibitors: design, disposition, and implications of the Dosing Observational Study in Hemophilia (DOSE)

OBJECTIVE: The daily recordings of treatment by patients with congenital hemophilia with inhibitors and their caregivers were assessed as part of the Dosing Observational Study in Hemophilia (DOSE) to understand the patterns of bypassing agent use and health-related quality of life. METHODS: Frequently bleeding patients prescribed recombinant activated factor VII as first-line therapy were eligible. Participants recorded daily paper diaries for at least 90 days and until at least four bleeding episodes had occurred. Web-based entry was optional. Assessment included bleeding status, work or school day status, bleeding episode, treatment, impact on planned activities, and health-related quality of life. RESULTS: Diaries were completed by 18 adults and 19 caregivers (21 children). A total of 4063 diary days and 194 bleeding episodes over 491 bleed days were recorded. A small proportion of diary days were bleed days (8.2%) or treatment days (8.2%). Half the bleed days were not planned work or school days for patients (53%) or caregivers (48%). An exact agreement was observed between electronic and paper records for 93% of the reviewed health-related quality of life measurements. CONCLUSION: Daily diary completion by patients and caregivers is feasible and provides insight into the impact of congenital hemophilia with inhibitors on daily activities and overall quality of life. Positive participation and completion rates were supported by frequent patient contact made by independent patient support liaison personnel.