Haemophilia utilization group study - Part Va (HUGS Va): design, methods and baseline data

Area of Science:

• Hematology
• Public Health
• Health Economics

Background:

• Hemophilia A, a genetic bleeding disorder, significantly impacts patients' quality of life and healthcare costs.
• Understanding the economic burden and healthcare utilization is crucial for effective management of hemophilia.
• The Haemophilia Utilization Group Study - Part Va (HUGS Va) aimed to address these knowledge gaps in the US population.

Purpose of the Study:

• To detail the study design, procedures, and baseline characteristics of the HUGS Va study.
• To evaluate the cost of care and burden of illness in individuals with factor VIII deficiency.
• To provide comprehensive data on health care utilization and outcomes in US hemophilia A patients.

Main Methods:

• A multi-center, observational study involving 329 participants aged 2-64 with factor VIII deficiency.
• Data collection included interviews, follow-up surveys, clinician documentation, and pharmacy records over two years.
• Participants were recruited from six federally supported hemophilia treatment centers (HTCs) across the US.

Main Results:

• Two-thirds of participants had severe hemophilia; average ages were 9.7 years for children and 33.5 for adults.
• Socio-demographic characteristics varied by insurance status, income, and employment, but were consistent across hemophilia severity.
• Adults reported significantly lower physical functioning compared to the general US population, while children's quality of life was comparable to healthy peers.

Conclusions:

• HUGS Va provides extensive economic data on hemophilia care in the US.
• Findings highlight disparities in employment and insurance status among hemophilia patients.
• The study underscores the significant burden of illness and healthcare utilization associated with hemophilia A.