Nationwide examination of access to comprehensive care in sickle cell disease

Sickle cell disease (SCD) is a complex and highly morbid inherited blood disorder that affects at least 100,000 people in the US. It is associated with a 20-year reduction in life expectancy and substantial economic costs to the health care system. Research and clinical care for SCD has faced challenges from a lack of population- level registry data and a historic paucity of specialist care relative to comparator inherited diseases like hemophilia or cystic fibrosis. Recent years have seen an increased focus on the development of comprehensive sickle cell centers (CSCCs); however, we do not know how many people with SCD have access to CSCCs at a population level, nor do we know the impact of receiving care at these centers on health-related outcomes. To address these gaps in knowledge, we propose two analytic aims using national insurance claims data from Medicaid, which insures the majority of people with SCD in the US. In the first aim, we will identify three groups of people with SCD: those who A) do not receive care from a hematology provider, B) receive care from a hematology provider but not at a CSCC, and C) receive health care at a CSCC, and we will identify sociodemographic characteristics associated with not receiving care at a CSCC. The second aim will use an econometric approach called instrumental variable (IV) analysis, which is a quasi-experimental design that accounts for bias in observational data to produce estimates of causal effect. We will use IV analysis to estimate the causal effect of receiving care at a CSCC on mortality and health care utilization, using differential distance to a CSCC as an instrument. The proposed research will provide the first national description of access to comprehensive care for people with SCD and the most robust estimate of the impact of receiving comprehensive care to date. These results will help health care providers consider the role of CSCCs for people with SCD, and they will inform policymakers’ discussions about allocation of resources toward comprehensive care in SCD. This work will also provide critical training to the applicant in the form of mentorship in SCD and advanced research methodology as he pursues his goal of becoming an independent health services researcher in SCD. Project Number: 1F32HL182307-01 | Fiscal Year: 2025 | NIH Institute/Center: National Heart Lung and Blood Institute (NHLBI) | Principal Investigator: Joshua Rivenbark | Institution: UNIV OF NORTH CAROLINA CHAPEL HILL, CHAPEL HILL, NC | Award Amount: $85,744 | Activity Code: F32 | Study Section: Special Emphasis Panel[ZRG1 F18-E (20)] View on NIH RePORTER: https://reporter.nih.gov/project-details/1F32HL18230701