Costs and impact of disease in adults with sickle cell disease: a pilot study.

ABSTRACT: We assessed the feasibility to estimate illness burden in adults with SCD, investigated factors associated with health-related quality of life (HRQoL), and estimated societal burden. We recruited 32 participants and collected data on fatigue, HRQoL, and work productivity and activity impairment via patient survey. Health care utilization was abstracted for the 12 months before enrollment using medical chart review. Mean age was 36.7 years; 84.4% of participants had hemoglobin SS or Sßthal0 disease, and 81.3% reported chronic pain (experiencing pain on ≥3 days per week in the past 6 months). Mean EQ-5D-3L visual analogue scale score was 63.4 and the index score was 0.79. The mean fatigue score was 57.9. Higher fatigue score was correlated with lower EQ-5D index score (correlation coefficient r = -0.35; P = .049) and Adult Sickle Cell Quality of Life Measurement Information System (ASCQ-Me) scores, including pain (r = -0.47; P = .006), sleep (r = -0.38; P = .03), and emotion scores (r = -0.79; P < .0001). The number of hospitalizations was negatively correlated with HRQoL (all P < .05). Patients who reported chronic pain had significantly lower mean ASCQ-Me sleep scores (48.3 vs 57.1; P = .04) and EQ-5D index scores (0.72 vs 0.89; P = .002) than those without chronic pain. Mean estimated annual per person costs were $51 779 (median, $36 366) for total costs, $7619 ($0) for indirect costs (estimated from lost earnings of participants), and $44 160 ($31 873) for medical costs. Fatigue, SCD complications, hospitalization, and chronic pain negatively affected HRQoL. This sample experienced a high economic burden, largely from outpatient doctor visits.

Building the foundation for a community-generated national research blueprint for inherited bleeding disorders: research priorities in health services; diversity, equity, and inclusion; and implementation science.

BACKGROUND: The National Hemophilia Foundation (NHF) conducted extensive all-stakeholder inherited bleeding disorder (BD) community consultations to inform a blueprint for future research. Sustaining and expanding the specialized and comprehensive Hemophilia Treatment Center care model, to better serve all people with inherited BDs (PWIBD), and increasing equitable access to optimal health emerged as top priorities. RESEARCH DESIGN AND METHODS: NHF, with the American Thrombosis and Hemostasis Network (ATHN), convened multidisciplinary expert working groups (WG) to distill priority research initiatives from consultation findings. WG5 was charged with prioritizing health services research (HSR); diversity, equity, and inclusion (DEI); and implementation science (IS) research initiatives to advance community-identified priorities. RESULTS: WG5 identified multiple priority research themes and initiatives essential to capitalizing on this potential. Formative studies using qualitative and mixed methods approaches should be conducted to characterize issues and meaningfully investigate interventions. Investment in HSR, DEI and IS education, training, and workforce development are vital. CONCLUSIONS: An enormous amount of work is required in the areas of HSR, DEI, and IS, which have received inadequate attention in inherited BDs. This research has great potential to evolve the experiences of PWIBD, deliver transformational community-based care, and advance health equity.

Research into how people get their health care, called health services research, is important to understand if care is being delivered equitably and efficiently. This research figures out how to provide the best care at the lowest cost and finds out if everyone gets equally good care. Diversity and inclusion research focuses on whether all marginalized and minoritized populations (such as a given social standing, race, ethnicity, sex, gender identity, sexuality, age, income, disability status, language, culture, faith, geographic location, or country of birth) receive equitable care. This includes checking whether different populations are all getting the care they need and looking for ways to improve the care. Implementation science studies how to make a potential improvement work in the real world. The improvement could be a new way to diagnose or treat a health condition, a better way to deliver health care or do research, or a strategy to remove barriers preventing specific populations from getting the best available care. The National Hemophilia Foundation focuses on improving the lives of all people with bleeding disorders (BD). They brought BDs doctors, nurses, physical therapists, social workers, professors, and government and industry partners together with people and families living with BDs to discuss research in the areas described above. The group came up with important future research questions to address racism and other biases, and other changes to policies, procedures, and practices to make BD care equitable, efficient, and effective.

Sickle cell disease among Latinx in California.

INTRODUCTION: After African Americans, Latinx are the second largest population affected by Sickle Cell Disease (SCD) in the U.S. However, research has largely ignored how this devastating rare blood disorder specifically affects Latinx nationwide. METHODS: This study compared demographics, genotypes, primary insurance, and health care utilization among Latinx and non-Latinx Californians living with SCD, using data from the California SCD Data Collection Program (2016-2018) and newborn screening cases 2000-2017. RESULTS: Stemming from 6,837 SCD patients, 501(7%) were Latinx. Latinx with SCD (Lx-SCD) were statistically significantly younger than non-Latinx (NLx-SCD) counterparts. Within both groups, females predominated, with 70% being insured by Medicaid. Mean Emergency Department encounters were statistically significantly lower among Lx-SCD adults. DISCUSSION: Lx-SCD differ in age, genotype, and Emergency Department utilization, when compared to NLx-SCD counterparts in California. Latinx are now the largest racial and/or ethnic group in the US, and their presence in SCD population is expected to grow. Therefore, their specific demographic, genotypic, and health care utilization characteristics merit attention to inform policies and programs that will improve their health.

Economic Burden of Illness among Persons with Hemophilia B from HUGS Vb: Examining the Association of Severity and Treatment Regimens with Costs and Annual Bleed Rates.

OBJECTIVES: To determine US societal burden of illness, including direct and indirect costs and annual bleed rate (ABR), for persons with hemophilia B (HB), a rare and debilitating genetic disorder, and to examine associations of hemophilia severity and treatment regimens with costs and ABR. METHODS: From 2009 to 2014, the Hemophilia Utilization Group Studies Part Vb collected prospective data from 10 US hemophilia treatment centers. Participants with HB completed initial surveys on sociodemographic characteristics, clinical characteristics, and treatment patterns. During the 2-year follow-up, participants reported bleeding episodes, work absenteeism, and caregiver time quarterly. These data were used to calculate ABR and indirect costs. Direct costs were calculated using 1-year clinical chart records and 2-year dispensing records. RESULTS: Of the 148 participants, 112 with complete medical records and one or more follow-up survey were included. Total mean annual per-person costs were $85,852 (median $20,160) for mild/moderate HB, $198,733 (median $147,891) for severe HB, and $140,240 (median $63,617) for all participants without inhibitors (P < 0.0001). Mean ABR for participants with severe HB on prophylaxis (5.5 ± 7.9 bleeds/y) was almost half that of those treated episodically. Clotting factor and indirect costs accounted for 85% and 9% of total costs, respectively. Compared with episodic treatment, prophylaxis use was associated with 2.5-fold higher clotting factor costs (P < 0.01), low but significantly more missed parental workdays (P < 0.0001) and clinician (P < 0.001) or nursing visits (P < 0.0001), less part-time employment and unemployment, and lower hospitalizations costs (P = 0.17) and ABR (P < 0.0001). CONCLUSIONS: HB is associated with high economic burden, primarily because of clotting factor costs. Nevertheless, prophylaxis treatment leads to clinical benefits and may reduce other nonfactor costs.

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.

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.

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.