M. Ullman

Assessing the costs for clinical care of patients with high-responding factor VIII and IX inhibitors.

Summary.  The costs of haemophilia‐related care and the impact of unusually expensive, or outlier, patients on these costs have been explored in numerous European, American and Canadian studies during the last decade. In particular, antibodies that neutralize infused factor VIII or IX (high‐responding inhibitors) make treatment responses, and thereby costs, much less predictable. There is little debate that the health care costs of haemophilic patients with high‐responding inhibitors are routinely higher and more variable than those of non‐inhibitor patients. However, the extent to which this is attributable to the few outlier inhibitor patients whose expenditures tend to skew the data is not as clear. To compare the variation and range in health care expenditures among patients with inhibitors and those without, we reviewed data originally gathered during a 24‐month period in 1995–1997 from a prospectively created cohort as part of a broader cost and utilization study conducted at a large haemophilia treatment centre. We conclude that although the use of outpatient factor replacement products was not significantly greater or more expensive among inhibitor patients, their hospital‐related costs greatly increased overall expenditures. Among our study population, the overall costs associated with inhibitor patients are not only higher in absolute monetary terms, but also in terms of the degree of variation. This variation was demonstrated by: (i) the extremely wide range of costs over an extended timeframe among individual inhibitor patients when compared with those without inhibitors, and (ii) the much larger year‐to‐year variation in costs among the inhibitor group.

Quality of life in haemophilia A: Hemophilia Utilization Group Study Va (HUGS‐Va)

Summary.  This study describes health‐related quality of life (HRQoL) of persons with haemophilia A in the United States (US) and determines associations between self‐reported joint pain, motion limitation and clinically evaluated joint range of motion (ROM), and between HRQoL and ROM. As part of a 2‐year cohort study, we collected baseline HRQoL using the SF‐12 (adults) and PedsQL (children), along with self‐ratings of joint pain and motion limitation, in persons with factor VIII deficiency recruited from six Haemophilia Treatment Centres (HTCs) in geographically diverse regions of the US. Clinically measured joint ROM measurements were collected from medical charts of a subset of participants. Adults (N = 156, mean age: 33.5 ± 12.6 years) had mean physical and mental component scores of 43.4 ± 10.7 and 50.9 ± 10.1, respectively. Children (N = 164, mean age: 9.7 ± 4.5 years) had mean total PedsQL, physical functioning, and psychosocial health scores of 85.9 ± 13.8, 89.5 ± 15.2, and 84.1 ± 15.3, respectively. Persons with more severe haemophilia and higher self‐reported joint pain and motion limitation had poorer scores, particularly in the physical aspects of HRQoL. In adults, significant correlations (P < 0.01) were found between ROM measures and both self‐reported measures. Except among those with severe disease, children and adults with haemophilia have HRQoL scores comparable with those of the healthy US population. The physical aspects of HRQoL in both adults and children with haemophilia A in the US decrease with increasing severity of illness. However, scores for mental aspects of HRQoL do not differ between severity groups. These findings are comparable with those from studies in European and Canadian haemophilia populations.

Prevalence of conditions associated with human immunodeficiency and hepatits virus infections among persons with haemophilia, 2001-2003

Summary.  Before the mid‐1980s, haemophilia often was unknowingly treated with contaminated plasma products, resulting in high rates of human immunodeficiency virus (HIV‐1), hepatitis C virus (HCV) and hepatitis B virus (HBV) infections. To estimate the impact of these infections, a new cohort was established. All HCV‐seropositive patients, age 13–88 years, at 52 comprehensive haemophilia treatment centres were eligible. Cross‐sectional data collected during April 2001 to January 2004 (median June 2002) were analysed. Plasma HIV‐1 and HCV RNA were quantified by polymerase chain reaction. Highly active antiretroviral therapy (HAART) was defined as use of at least three recommended medications. Among 2069 participants, 620 (30%) had HIV‐1. Of 1955 with known HBV status, 814 (42%) had resolved HBV and 90 (4.6%) were HBV carriers. Although 80% of the HIV‐1‐positive participants had ≥200 CD4+ cells μL−1, only 59% were on HAART. HIV‐1 RNA was undetectable in 23% of those not taking antiretroviral medications. Most (72%) participants had received no anti‐HCV therapy. HCV RNA was detected less frequently (59%) among participants treated with standard interferon plus ribavirin (P = 0.0001) and more frequently among HIV‐1‐positive than HIV‐1‐negative participants (85% vs. 70%, P < 0.0001). HIV‐1‐positive participants were more likely to have pancytopenia and subclinical hepatic abnormalities, as well as persistent jaundice, hepatomegaly, splenomegaly and ascites. HAART recipients did not differ from HIV‐negative participants in the prevalence of ascites. The clinical abnormalities were more prevalent with older age but were not confounded by HBV status or self‐reported alcohol consumption. Eleven participants presented with or previously had hepatocellular carcinoma or non‐Hodgkin lymphoma. Although prospective analysis is needed, our data reveal the scale of hepatic and haematological disease that is likely to manifest in the adult haemophilic population during the coming years unless most of them are successfully treated for HIV‐1, HCV or both.

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

Abstract Objective: To examine the direct and indirect costs of hemophilia care among persons with hemophilia A in the US. Methods: Observational data were obtained from HUGS-Va, a multi-center study from six federally supported hemophilia treatment centers (HTCs). Eligible individuals completed a standardized initial questionnaire and were followed regularly for 2 years to obtain information on work or school absenteeism, time spent arranging hemophilia care, and unpaid hemophilia-related support from caregivers. Data from 1-year healthcare utilization records and 2-year clotting factor dispensing records measured direct medical costs. Indirect costs were imputed using the human capital approach, which uses wages as a proxy measure of work time output. Results: A total of 222 patients with complete data were included in the analysis. Two-thirds had severe hemophilia and the mean age was 21.1 years. The use of prophylaxis in severe hemophilia patients is associated with statistically significant reduction in the numbers of emergency department (ED) visits and bleeding episodes compared with those who were treated episodically. From the societal perspective, mild hemophilia costs

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

59,101 (median:

Physical activity and health outcomes in persons with haemophilia B

7519) annually per person,

Young adults with hemophilia in the U.S.: demographics, comorbidities, and health status

84,363 (median:

Association of overweight and obesity with the use of self and home-based infusion therapy among haemophilic men

61,837) for moderate hemophilia,

An inventory of healthy weight practices in federally funded haemophilia treatment centres in the United States.

201,471 (median:

Prophylaxis use among males with haemophilia B in the United States

143,431) for severe hemophilia using episodic treatment, and