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

Haemophilia (2011), 1–8 DOI: 10.1111/j.1365-2516.2011.02595.x ORIGINAL ARTICLE Haemophilia Utilization Group Study – Part Va (HUGS Va): design, methods and baseline data Z.-Y. ZHOU,* J. WU,* J. B AKER, R. CURTIS,a A. FORSBERG,§ H. HUSZTI,– M. KOERPER,** M. LOU,* R. MILLER, K. PARISH,aa B. RISKE,§§ A. SHAPIRO, –– M. ULLMAN*** and K. JOHNSON* *University of Southern California, Los Angeles, CA; University of California, Los Angeles, CA; aFactor VIII Computing, Berkeley, CA; §UMass Memorial Hospital, Worcester, MA; –Children’s Hospital of Orange County, Orange, CA; **University of California, San Francisco, CA; Children’s Hospital, Los Angeles, CA; aaSouth Pasadena, CA; §§University of Colorado Denver, CO; ––Indiana Hemophilia and Thrombosis Center, IN; and ***University of Texas Health Sciences Center, Houston, TX, USA Summary. To describe the study design, procedures and baseline characteristics of the Haemophilia Utili- zation 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 limi- tation 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 characteris- tics and pharmacy dispensing records for 2 years. Introduction Haemophilia is a rare chronic inherited bleeding disor- der affecting approximately 400 000 persons of all races worldwide and 20 000 in the United States [1–3]. Patients with haemophilia either lack or are deficient in clotting factors VIII or IX, which places them at high risk of bleeding into the joints, muscles and other Correspondence: Kathleen A. Johnson, PharmD, MPH, PhD, Titus Family Department of Clinical Pharmacy and Pharmaceutical Economics and Policy, USC School of Pharmacy, 1985 Zonal Avenue, PSC 100, Los Angeles, CA 90033, USA. Tel.: +1 323 442 1393; fax: +1 323 442 1395; e-mail: kjohnson@usc.edu Accepted after revision 20 May 2011 2011 Blackwell Publishing Ltd 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 cate- gories. 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. Keywords: cost of illness, haemophilia, observational study, outcome, quality of life, utilization hollow organs. Repeated haemorrhage into joints, especially in persons with severe haemophilia, can lead to the development of chronic arthropathy, which causes joint pain, reduction in joint range of motion (ROM), crippling musculoskeletal deformity and dis- ability. The availability of commercially available clotting factor has transformed haemophilia from a frequently fatal to a manageable disease. Since the 1960s, clotting factor concentrate has been used widely to treat haemophilia patients episodically [4]. Episodic treat- ment, administered after a bleed has occurred, is known to slow the progression of arthropathy, but not to reverse it. Clotting factors can also be infused prophy- lactically on a regular schedule to maintain the blood factor activity above 1%, especially in persons with