Haemophilia Utilization Group Study – Part Va (HUGS Va): design, methods and baseline data

Abstract

Summary. To describe the study design, procedures and baseline characteristics of the Haemophilia Utilization Group Study -Part Va (HUGS Va), a US multi-center observational study evaluating the cost of care and burden of illness in persons with factor VIII deficiency. Patients with factor VIII level £30%, age 2-64 years, receiving treatment at one of six federally supported haemophilia treatment centres (HTCs) were enrolled in the study. Participants completed an initial interview including questions on socio-demographical characteristics, health insurance status, co-morbidities, access to care, haemophilia treatment regimen, factor utilization, self-reported joint pain and motion limitation and health-related quality of life. A periodic follow-up survey collected data regarding time lost from usual activities, disability days, health care utilization and outcomes of care. HTC clinicians documented participants' baseline clinical characteristics and pharmacy dispensing records for 2 years.Between July 2005 and July 2007, 329 participants were enrolled. Average age was 9.7 years for children and 33.5 years for adults; two-thirds had severe haemophilia. The distributions of age, marital status, education level and barriers to haemophilia care were relatively consistent across haemophilic severity categories. Differences were found in participants' employment status, insurance status and income. Overall, children with haemophilia had quality of life scores comparable to healthy counterparts. Adults had significantly lower physical functioning than the general US population. As one of the largest economic studies of haemophilia care, HUGS Va will provide detailed information regarding the burden of illness and health care utilization in the US haemophilia A population.

Burden of illness: direct and indirect costs among persons with hemophilia A in the United States

“…The study methods and population baseline characteristics have previously been reported 17 . Persons with hemophilia A were eligible for this study if they met these inclusion criteria: (1) aged 2-64 years as of the date of initial interview; (2) factor VIII level 30%, with or without a history of inhibitor; (3) received at least 90% of their hemophilia care at an HTC; (4) obtained HTC care within 2 years prior to study enrollment; and (5) were English speaking.…”

“…The Hemophilia Utilization Group Study part-Va (HUGS-Va) is a multi-center, prospective cohort study designed to examine the burden of illness among persons with hemophilia A at six federally-supported hemophilia treatment centers (HTCs) in the US 17 . The regionalized HTCs, a multi-disciplinary, team-based care delivery model, have been implemented in the US for several decades with goals to prevent orthopedic complications and to maximize the physical and psychological functioning and socioeconomic benefits for this rare disease population 18 .…”

Longitudinal Changes in Health-Related Quality of Life for Chronic Diseases: An Example in Hemophilia A

“…Details of the study methods and inclusion criteria have previously been reported. 6 , 7 Informed consent and data were collected at initial interview from participant/parent selfor proxy-report and patient chart reviews completed by healthcare providers. Information regarding clinical aspects of the disease, such as treatment regimen, arthropathy and comorbidities, as well as HrQoL and the economic consequences of having hemophilia A, were collected.…”

“…[6][7][8][9][10] The physical component summary (PCS) and mental component summary (MCS) scores were used in this analysis. 11 HrQoL of participants aged 2-17 years was assessed by parent-proxy using the PedsQL™ 4.0 generic core scales, 12 a HrQoL instrument developed in the US for children and adolescents.…”

Self-Reported Barriers to Hemophilia Care in People with Factor VIII Deficiency

“…The details of study design, methodology, and baseline data have been described elsewhere. 13 Briefly, data were collected prospectively among people with hemophilia A who received care at one of six HTCs located in California (2 centers), Colorado, Indiana, Massachusetts, and Texas. These sites were selected because they are geographically diverse and representative of HTCs in the U.S., located in populous states, thus serving relatively large hemophilia populations allowing for adequate subject enrollment, and had research personnel willing to participate and conduct IRB review.…”