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Introduction: People with haemophilia rely on specialists for their care, yet the specific dosing regimens of treatments prescribed by these specialists have not been widely studied. Aim: The objective of this study is to describe trends in clinician prescribing practices for the management of haemophilia in the United States (US). Methods: We administered surveys to members of the Hemostasis and Thrombosis Research Society via paper surveys at its in‐person annual symposia in 1999 and 2015, and an online survey in 2021. The surveys collected information on haemophilia treatments including factor dosing, inhibitor therapy and gene therapy. Results: Clinicians treating haemophilia for more than 50% of their practice time have increased from 37.5% of respondents in 1999 to 46.3% in 2021. Clinicians prescribing factor concentrates at >40 units/kg for routine bleeding events increased from 0% in 1999 to 29.3% in 2021 in haemophilia A (HA) and from 22.5% to 87.8% in haemophilia B (HB). In 2021, the clinicians reported prescribing emicizumab to treat HA patients (>89.5% paediatric, >85.7% adult) with or without inhibitors at least some of the time. Approximately 78.0% of respondents reported that they expected to recommend gene therapy at least some of time. Conclusion: These data indicate changing trends in prescribing practices among US haemophilia specialists during the past 22 years. Preference for high doses of factor (>40 units/kg) has increased during this period. Emicizumab prophylaxis has been prescribed for patients with and without HA inhibitors. Clinicians expect gene therapy to have value for some haemophilia patients. [ABSTRACT FROM AUTHOR]

Purpose: We compare the impact of hemophilia on comorbidities, joint problems, health-related quality of life (HRQoL) and health-care utilization between two age groups: 40– 49 years and ≥ 50 years. Patients and Methods: The HUGS VII study recruited persons with hemophilia A or B age ≥ 40 years. Participants completed surveys to collect data on sociodemographic and clinical characteristics, hemophilia treatment regimen, pain, joint problems, comorbidities, HRQoL, depression and anxiety, at baseline and 6-months later. Clinical chart reviews documented hemophilic severity and treatment. Results: The sample includes 69 males, 65.2% aged ≥ 50 years, 75.4% with hemophilia A. Individuals ≥ 50 years were more likely to have mild or moderate hemophilia (68.9% vs 41.7%, P = 0.03) than those 40– 49 years old. Among persons with mild/moderate hemophilia, those ≥ 50 years old reported a higher rate of joint pain (83.9% vs 70.0%, P = 0.34 at baseline, 91.3% vs 57.1%, P = 0.06 at follow up) or range of motion limitation (73.3% vs 60.0%, P = 0.43 at baseline, 73.9% vs 28.6%, P = 0.04 at follow up) than the younger group. Compared to the younger group, the older group reported fewer emergency room visits (4.5% vs 21.7%, P = 0.03), and physical therapy visits (15.9% vs 43.5%, P = 0.01) at baseline. The sample depression rate was 85.7%, but the differences among the age groups were not significant. The mean covariate-adjusted EQ-5D index score was lower in older persons (0.77 vs 0.89, P = 0.02). Conclusion: Older persons with hemophilia in this sample are over-represented by individuals with mild/moderate disease, potentially due to premature death among those with severe disease. Although this group included a larger proportion of individuals with mild disease than the younger group, they experienced lower quality of life, more comorbidities both of aging and of hemophilic arthropathy, and lower rates of health-care utilization. [ABSTRACT FROM AUTHOR]

Rare, chronic diseases such as hemophilia and other congenital coagulation disorders require coordinated delivery of services for optimal outcomes. Hemophilia Treatment Centers (HTCs) are specialized, multidisciplinary health-care centers providing team-based care to meet the physical, psychosocial, and emotional needs of people with hemophilia (PWH) and may serve as a model for other rare coagulation disorders. Health-care purchasers, as well as the general medical community, may not appreciate the breadth and quality of services provided by HTCs. They exemplify the acculturalization and actualization of integrated care by providing comprehensive diagnostic and treatment services that reduce morbidity, mortality, avoidable emergency room visits, hospitalizations, and overall costs, while promoting a longer lifespan and improved patient functioning and outcomes. This is accomplished by a team-based approach relying upon a shared decision-making model to effectively prevent complications and manage symptoms in PWH, who are dependent on high-cost treatments. This article provides a concise yet comprehensive description of the core components of an HTC and the regional and national networks in the United States, which together achieve their incomparable value for all stakeholders. [ABSTRACT FROM AUTHOR]

Background: Hemophilia is the most common inherited severe bleeding disorder. Although the most frequent complication of repeated hemorrhages is a crippling joint disease that begins in childhood, the extent of resultant joint functional impairment varies widely within the hemophilia population. Purpose: The goal of this exploratory analysis was to examine a national database that collects information on boys with hemophilia, an X-linked severe congenital bleeding disorder, to determine characteristics associated with increased risk of developing limitations in physical functioning as an outcome of recurrent hemorrhages. Methods: A standard set of data is collected annually at ∼130 U.S. comprehensive hemophilia treatment centers (HTCs) in a voluntary surveillance program called the Universal Data Collection (UDC) program. Fifteen potential predictors for poor outcomes of physical functioning related to bleeding were examined for boys (aged ≤18 years) from 1998 to 2008. Bivariate and multivariate analyses of these predictors performed in 2009 examined associations with self-reported limitation of activities, absenteeism from work or school, and reliance on assistive devices for ambulation and mobility. Results: Multiple characteristics of underlying hemophilia severity and disease chronicity (in particular, increasing age, presence of joint bleeding, and inhibitor antibodies) were independently associated with increased risk of limitations of physical function. Nonwhite race/ethnicity was associated with each of the poorer functional outcomes in bivariate analyses. After controlling for the potential confounding effects of the multiple population characteristics on race, only African-American race was independently associated with activity restrictions, and African-American and Asian/Pacific Island ethnicity with absenteeism. With the exception of indicators of underlying disease severity, only obesity and medical insurance coverage with Medicaid rather than commercial insurance were independently associated with multiple poor outcomes. Conclusions: Interventions focused on eliminating inhibitors, improving outcomes for African-American children with hemophilia, and maintaining healthy body weight are warranted. In addition, strategies are needed to assure adequate insurance coverage for all people with hemophilia to eliminate economic barriers to optimal functional outcomes. [ABSTRACT FROM AUTHOR]

Background: The benefits of a high school diploma are well documented. Studies indicate that people with hemophilia have lower than average academic achievement, particularly if they have >12 bleeding episodes annually. Purpose: This study compares the high school graduation rate of men with hemophilia to that of the U.S. population of men. Methods: Data were obtained from the Universal Data Collection Program, a surveillance project conducted by approximately 130 hemophilia treatment centers in the nation. Data from 7842 men aged ≥18 years were evaluated to determine high school graduation status and were analyzed by race/ethnicity and severity of hemophilia. These data were collected between 1998 and 2008, and analysis was conducted in 2009. Results: Men with hemophilia A had higher or similar high school graduation rates across all racial/ethnic groups and all levels of hemophilia severity, compared with U.S. men of the same age. Graduation rates for black and Hispanic men with hemophilia B were higher or similar to rates of U.S. men, but rates for whites were lower, especially among those with moderate and mild disease. However, when graduation rates were controlled for areas where Amish populations reside, differences in graduation rates for whites disappeared. Conclusions: In this study, participants obtained hemophilia care at comprehensive hemophilia treatment centers. This multidisciplinary, family-centered care emphasizes prevention of complications, encourages medically supervised disease management, and facilitates psychosocial development. The care aims to maximize the affected child''s participation in school. This care approach may partially explain the higher-than-expected high school graduation rates among the study population, which is affected by a rare, chronic, and potentially debilitating disorder. [Copyright &y& Elsevier]