Patient satisfaction with US Hemophilia Treatment Center Care, Teams and Services: The First National Survey.

INTRODUCTION: Patient satisfaction with health care is a key quality metric, associated with adherence and better outcomes. However, satisfaction with US Hemophilia Treatment Centers (HTC) is unknown. AIM: To assess patient satisfaction with US Hemophilia Treatment Centers. METHODS: A nationally uniform survey was conducted using the US HTC Network's regional infrastructure. Satisfaction with multidisciplinary team members, services and care processes was assessed. The anonymous survey, in English and Spanish, was disseminated to 28 289 households. Data were aggregated using 4 standard US Census regions. RESULTS: 5006 individuals (17.7%) who obtained care from 133 (96.4%) of 138 HTCs in 2014 responded. Satisfaction with overall HTC care at 'always' or 'usually' (A/U) levels ranged 94.2%-97.9% regardless of patient gender, age, race, ethnicity, language, diagnosis, severity, region or frequency of HTC contact. A/U satisfaction with HTC haematologist, nurse, social worker or physical therapist, individually, ranged 95.1%-97.3% nationally. A/U satisfaction with three HTC services was 89.5%-96.9% and 94.9%-98.0% for five HTC care processes nationally. Regional satisfaction at A/U levels was at least 87.0%. Nationally, 26.4% and 21.2% rated insurance and language, respectively, as A/U problems in getting needed HTC services. CONCLUSION: Patient satisfaction with US Hemophilia Treatment Center care, multi-disciplinary teams, services and processes was consistently high, documenting the value patients place on HTCs. The successful survey administration demonstrates the capability of the Network's regional infrastructure. Access to the US HTC Network is particularly critical to ongoing health in this new era of novel and gene therapies.

Don't Push Your Luck! Educational Family Board (Not Bored) Game for School-Age Children Living with Chronic Conditions.

PURPOSE: Children who are living with chronic conditions may be supported in self-care through enjoyable active learning and family social processes. This research focused on development and evaluation of "Don't Push Your Luck!", an educational board game designed to inspire family discussion about chronic conditions, and help affected children learn about self-care choices and consequences. DESIGN AND METHODS: Mixed-method research was conducted with families from one outpatient Cystic Fibrosis Clinic and four Hemophilia Treatment Centres in Canada and United States (N=72). In phase I, board game prototype and questionnaires were refined with affected boys, siblings, and parents living with hemophilia (n=11), compared with families living with cystic fibrosis (n=11). In phase II, final board game was evaluated with families living with hemophilia (n=50). Data collection included pre-post-game questionnaires on decision-making and Haemo-QoL Index©, and post-game enjoyment. Analysis included descriptive statistics, inferential statistics (non-parametric), and qualitative themes. RESULTS: Findings revealed this game was an enjoyable and effective resource to engage families in self-care discussions. Key themes included communication, being involved, knowing, decisions and consequences, and being connected. Qualitative and quantitative findings aligned. Statistical significance suggests the game enhanced family engagement to support decision-making skills, as parents identified that the game helped them talk about important topics, and children gained insight regarding family supports and self-care responsibility. CONCLUSIONS: This board game was an effective, developmentally appropriate family resource to facilitate engagement and conversation about everyday life experiences in preparation for self-care. PRACTICE IMPLICATIONS: There is promising potential to extend this educational family board game intervention with a greater range of school-age children and families living with chronic conditions.

Men with severe hemophilia in the United States: birth cohort analysis of a large national database.

The availability of longitudinal data collected prospectively from 1998 to 2011 at federally funded US hemophilia treatment centers provided an opportunity to construct a descriptive analysis of how outcomes of men with severe hemophilia have been altered by the incremental advances and setbacks in hemophilia care in the last 50 years in the United States. This surveillance collaboration with the US Centers for Disease Control and Prevention assembled the largest uniformly examined population with severe hemophilia (n = 4899 men with severe factor VIII and IX deficiency). To address the heterogeneity of this population, 4 successive birth cohorts, differentially affected by eras of hemophilia care, were examined separately in regard to demographics, complications of hemophilia and its treatment, and mortality. Severely affected men in each birth cohort were compared also with the corresponding mild hemophilia birth cohorts (n = 2587 men total) to control for outcomes that might be attributable to aging and environment independent of severely defective hemostasis. The analysis demonstrates improving access to standard of care therapy, correlating the proportion of men on prophylactic factor replacement and reduced bleeding frequency for the youngest men. Frequent bleeding persisted in one third to one half of men across all ages, however, and the disability gap between severe and mild hemophilia did not narrow. The greatest cause of death was liver failure, but attempted anti-hepatitis C virus therapy and cure were low. The study suggests a continued need for national surveillance to monitor and inform hemophilia interventions and outcomes.

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

Improvements in hemophilia care over the last several decades might lead to expectations of a near-normal quality of life for young adults with hemophilia. However, few published reports specifically examine health status indicators in this population. To remedy this knowledge gap, we examined the impact of hemophilia on physical and social functioning and quality of life among a national US cohort of 141 young men with hemophilia aged 18-34 years of age who received care at 10 geographically diverse, federally funded hemophilia treatment centers in 11 states between 2005 and 2013 and enrolled in the Hemophilia Utilization Group Studies. Indicators studied included educational achievement, employment status, insurance, health-related quality of life, and prevalence of the following comorbidities: pain, range of motion limitation, overweight/obesity, and viral status. The cohort was analyzed to compare those aged 18-24 to those aged 25-34 years. When compared to the general US adult population, this nationally representative cohort of young US adults with hemophilia experienced significant health and social burdens: more liver disease, joint damage, joint pain, and unemployment as well as lower high-school graduation rates. Nearly half were overweight or obese. Conversely, this cohort had higher levels of health insurance and equivalent mental health scores. While attention has typically focused on newborns, children, adolescents, and increasingly, on older persons with hemophilia, our findings suggest that a specific focus on young adults is warranted to determine the most effective interventions to improve health and functioning for this apparently vulnerable age group.

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

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 $59,101 (median: $7519) annually per person, $84,363 (median: $61,837) for moderate hemophilia, $201,471 (median: $143,431) for severe hemophilia using episodic treatment, and $301,392 (median: $286,198) for severe hemophilia receiving prophylaxis. Clotting factor contributed from 54% of total costs in mild hemophilia to a maximum of 94% for patients with severe hemophilia receiving prophylaxis. CONCLUSION: Hemophilia is a costly disorder not only because of its high medical expenses, but also due to the high indirect costs incurred.

Evidence for the transmission of parvovirus B19 in patients with bleeding disorders treated with plasma-derived factor concentrates in the era of nucleic acid test screening.

BACKGROUND: Parvovirus B19 (B19V) is a small, nonenveloped virus that typically causes a benign flu-like illness that occurs most frequently in childhood. The virus is resistant to current viral inactivation steps used in the manufacture of antihemophilic factor concentrates and B19V transmission through these products has been documented. Since 2000, B19V nucleic acid test (NAT) screening of plasma pools has been implemented to further decrease the viral burden in these products, but no study has examined populations using these products to assess the impact of the screening on B19V transmission. STUDY DESIGN AND METHODS: Blood specimens obtained from participants of a surveillance system established in federally supported specialized bleeding disorders clinics were used in a B19V seroprevalence study. RESULTS: A total of 1643 specimens from 1043 participants age 2 to 7 years born after B19V NAT screening was implemented were tested. Age-specific prevalence rates were generally higher for subjects exposed to either plasma-derived products alone or in combination with other products compared to subjects with no exposure to antihemophilic products. Overall, compared to participants unexposed to blood or blood products, those exposed to plasma-derived products alone were 1.7 times more likely to have antibodies to B19V (p = 0.002). CONCLUSION: These results are consistent with continued B19V transmission through plasma-derived factor concentrates. Effective viral inactivation and detection processes are needed to protect users of these products from infection with B19V or other new or emerging viruses.

Advances in hemophilia care: report of two symposia at the Hemophilia 2010 World Congress.

The World Federation of Hemophilia (WFH) 2010 World Congress held in Buenos Aires, Argentina, in July 2010, attracted more than 4,300 participants from 106 countries. This report summarizes two symposia held during the congress. The first, titled "Emerging Co-Morbidities in the Aging Hemophilia Population: Healthcare Challenges and Treatment Opportunities," chaired by Gerry Dolan, MD, and Jussara Almeida Cruz, MD, examined the co-morbidities experienced by the aging hemophilic patient population, such as cardiovascular disease, cancer, arthritis, osteoporosis, hypertension, and obesity. In addition, Bayer's products in preclinical and clinical development were reviewed, including a novel factor VIIa variant and a long-acting factor VIII molecule, i.e., one that has undergone site-specific PEGylation (attachment of polyethylene glycol [PEG] polymer chains to another molecule). The other symposium, titled "Practical Steps to Making Better Care for Hemophilia Patients a Reality," chaired by Carmen Altisent, MD, and Cesar Guerrero, RN, reviewed the steps that hemophilia caregivers can take to improve the care of their patients. Issues such as the treatment of hemarthroses, the role of the research nurse, and the management of pediatric patients transitioning to adulthood were discussed.

Insurance, home therapy, and prophylaxis in U.S. youth with severe hemophilia.

BACKGROUND: Home infusion therapy, particularly on a prophylactic regimen, is linked with reduced morbidity among youth with severe hemophilia. However, the association of insurance coverage with these home therapies is unknown. PURPOSE: This study explores the connections among insurance, home infusion therapy, and prophylaxis treatment in a nationwide cohort of 3380 boys and young men (aged 2 to 20 years) with severe hemophilia. These youth obtained care at one of 129 federally supported hemophilia treatment centers (HTCs), and enrolled in the CDC's bleeding disorder surveillance project. METHODS: Multiple regression was used to analyze the independent association among risk factors, including insurance, and both home infusion and prophylaxis. Data were obtained between January 1, 2008, and December 31, 2010, and analyzed in 2011. RESULTS: Ninety percent used home therapy and 78% a prophylaxis regimen. Only 2% were uninsured. Health insurance was significantly associated with prophylaxis, but not with home therapy. Lower prophylaxis utilization rates were independently associated with having Medicaid, "other," and no insurance as compared to having private insurance. Race, age, inhibitor status, and HTC utilization were also independently associated with both home therapy and prophylaxis. CONCLUSIONS: Youth with severe hemophilia who annually obtain care within the U.S. HTC network had a high level of health insurance, home therapy, and prophylaxis. Exploration of factors associated with insurance coverage and yearly HTC utilization, and interventions to optimize home infusion and prophylaxis among youth of African-American and "other" race/ethnic backgrounds are warranted.

Self-reported barriers to hemophilia care in people with factor VIII deficiency.

BACKGROUND: In 1975, a national network of hemophilia treatment centers (HTCs) was created to increase access to healthcare services for individuals with hemophilia. Studies demonstrate that care in HTCs improves outcomes and reduces costs. PURPOSE: The objective of the study was to assess the association of demographic, insurance, and clinical characteristics with self-reported barriers to HTC utilization. METHODS: Data were collected from six HTCs from 2005 through 2007. Adult participants and parents of children aged <18 years were interviewed. Barriers were assessed by asking whether it was difficult to obtain care in the past 12 months. Chi-square test and logistic regression were used to assess factors associated with self-reported barriers to care. All analyses were performed in 2010-2011. RESULTS: Data for 327 participants (50% adult, 64% severe hemophilia) were analyzed in 2010-2011. Most participants/parents did not report barriers to HTC utilization. However, 46 participants/parents (14%) reported one to six barriers, and 23 reported one barrier. Most frequently reported barriers were "distance to the clinic" for children (44%) and "insurance coverage" for adults (40%). Factors significantly associated with self-reported barriers were: lower income (<$20,000; OR=3.11, 95% CI=1.14-8.45), difficulty finding insurance or obtaining full-year coverage (OR=5.71, 95% CI=2.63-12.41), and decreased state Medicaid coverage for low-income, non-elderly individuals (OR=0.93, 95% CI=0.89-0.98). CONCLUSIONS: This study indicates that, although few people with hemophilia have barriers to care at HTCs, those with lower income, difficulty finding or maintaining adequate insurance coverage, or living in states with lower Medicaid generosity are more likely to report barriers. Identifying and resolving such barriers may improve care access and patient-reported outcomes.

Physical functioning in boys with hemophilia in the U.S.

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.

High school completion rates among men with hemophilia.

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 >or=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.

Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia.

BACKGROUND: Effective ways to prevent arthropathy in severe hemophilia are unknown. METHODS: We randomly assigned young boys with severe hemophilia A to regular infusions of recombinant factor VIII (prophylaxis) or to an enhanced episodic infusion schedule of at least three doses totaling a minimum of 80 IU of factor VIII per kilogram of body weight at the time of a joint hemorrhage. The primary outcome was the incidence of bone or cartilage damage as detected in index joints (ankles, knees, and elbows) by radiography or magnetic resonance imaging (MRI). RESULTS: Sixty-five boys younger than 30 months of age were randomly assigned to prophylaxis (32 boys) or enhanced episodic therapy (33 boys). When the boys reached 6 years of age, 93% of those in the prophylaxis group and 55% of those in the episodic-therapy group were considered to have normal index-joint structure on MRI (P=0.006). The relative risk of MRI-detected joint damage with episodic therapy as compared with prophylaxis was 6.1 (95% confidence interval, 1.5 to 24.4). The mean annual numbers of joint and total hemorrhages were higher at study exit in the episodic-therapy group than in the prophylaxis group (P<0.001 for both comparisons). High titers of inhibitors of factor VIII developed in two boys who received prophylaxis; three boys in the episodic-therapy group had a life-threatening hemorrhage. Hospitalizations and infections associated with central-catheter placement did not differ significantly between the two groups. CONCLUSIONS: Prophylaxis with recombinant factor VIII can prevent joint damage and decrease the frequency of joint and other hemorrhages in young boys with severe hemophilia A. (ClinicalTrials.gov number, NCT00207597 [ClinicalTrials.gov].).

A model for a regional system of care to promote the health and well-being of people with rare chronic genetic disorders.

People with rare, inherited chronic health conditions, such as hemophilia, face added physical, social, emotional, and fiscal challenges beyond those that are common to more prevalent chronic conditions. In 1975, a partnership among clinicians, consumers, and government agencies created a nationwide regional health delivery system that increased access to clinical care, prevention, and research, thereby improving health outcomes for people with hemophilia in the United States. Today, more than 130 Comprehensive Hemophilia Diagnostic and Treatment Centers in 12 regions serve 70%-80% of the nation's hemophilia patients. Health care leaders and advocates for other rare, expensive, chronic disorders may find that regionalization improves survival and reduces disability among affected populations. However, diverse and stable resources are needed to sustain such a model in our profit-oriented US health care arena.

Advances in care of children with hemophilia.

Care for children with severe hemophilia has moved from pediatric hospital wards and rehabilitation services to the home, school, and community. Advances in hemophilia are due largely to the development of specialized hemophilia treatment centers, which created a system of comprehensive care and focused healthcare efforts on prevention and education. Parallel advances in coagulation resulted in identification of clotting factors VIII and IX, elucidation of the protein molecular and biochemical structures and functions, sequencing of their respective genes and transfer of the human genes for production of proteins by recombinant technology, and development of gene therapy. The tragedy of the human immunodeficiency virus and hepatitis C raised awareness in patients as well as healthcare providers of the vulnerability of blood products to viral contamination and spurred progress in science leading to viral inactivation of purified proteins. Concomitantly, physicians treating bleeding episodes in the clinic investigated pharmacokinetics and pharmacoeconomics of various strategies of clotting factor replacement. The observation that trough factor levels as low as 1 to 2% were adequate to prevent most bleeding episodes led to current prophylactic regimens that allow boys to participate fully in school and community activities while factor concentrate is infused at home on a regular schedule. Currently, children with hemophilia look forward to a normal life expectancy and excellent health-related quality of life. Physician and community partnerships through research and advocacy societies have accelerated clinical advancements as well as extension of treatment to developing countries. The future of hemophilia promises a cure with gene therapy. Given the past accomplishments in hemophilia, a long-term solution to replacement of the genetically deficient protein lies on the horizon.