OBJECTIVES: Hemophilia A is a rare, inherited bleeding disorder in which the affected individual lacks or has limited production of coagulation factor VIII (FVIII), resulting in the inability of the blood to clot normally. Treatment typically involves life-long replacement of FVIII through intravenous infusions to stop or prevent spontaneous or traumatic bleeds. However, FVIII storage and stability remain a constant challenge. Obtaining patients’ and caregivers’ perspectives is critical to disease management, particularly with rare conditions. A web-based survey was developed to assess hemophilia A patients’ and caregivers’ preferences around FVIII storage and stability attributes.
METHODS: Individuals with hemophilia A and caregivers of minor children with hemophilia A from local and national hemophilia support groups in the United States and Canada were invited to complete a web-based survey assessing patients’ FVIII ordering, usage, and storage routines.
RESULTS: Of the 145 individuals who responded to the survey invitation, 101 individuals (67% caregivers [82% female]) completed the survey (70%). Nearly half (48%) of respondents ordered FVIII monthly, and 8% reported often having FVIII vials expire before their use. Challenges to storing FVIII included refrigeration when traveling (28%) and carrying an insulated tote bag (27%). More than half (54%) preferred a new FVIII product with longer storage at room temperature. The majority (80%) indicated preference for a FVIII product that could be stored at a higher room temperature for longer durations, primarily for ease in traveling, keeping more factor at home, and having easy access when needed.
CONCLUSIONS: This web-based study provides a unique perspective from patients and caregivers on treatment attributes that are important to this population. There is a need for an improved FVIII product that offers more convenience and faster access to patients in daily life and while traveling. Additionally, flexibility in storage potentially could reduce the frequency of FVIII wastage.
