Household material hardship as a barrier to accessing sickle cell disease curative therapy

Abstract Introduction: Sickle cell disease (SCD) is a highly morbid and life-threatening condition that affects 100,000 Americans, resulting in chronic complications, poor quality of life, and early death. Gene therapy offers a compelling opportunity for possible cure and is commercially approved for patients ≥12 years with open clinical trials in younger patients. However, its resource- and time-intensive nature may limit equitable access for patients. We aimed to identify family-reported modifiable barriers to receipt of commercial gene therapy that can inform future intervention development to improve access and outcomes. Methods: We conducted a cross-sectional, single-center study of children with SCD aged 2 to <18 years receiving care at Boston Children's Hospital. Foreign national patients and those with history of bone marrow transplant or gene therapy were excluded. Parents/guardians completed a 54-item quantitative survey to evaluate associations between modifiable social risk factors and family-reported ability to pursue curative SCD therapy for their child. Survey domains included demographics, household structure, household material hardship (HMH—food, housing, utility, or transportation insecurity), income, primary language, participation in governmental means-tested programs, and distance to hospital. The primary exposure was HMH, modeled as both binary present (at least 1 domain) vs absent (0 domains), as well as an ordinal exposure (0-4 domains). The primary outcome was family-reported ability to access a hypothetical curative therapy modeled on gene therapy, described in a clinical vignette, measured by agreement with the statement “My child would be able to obtain this therapy with my current resources and social support” on a 5-point Likert scale (strongly disagree to strongly agree). Responses were dichotomized as agree/strongly agree vs all other responses. Clinical characteristics including disease genotype were abstracted from the medical record. Characteristics of the cohort were summarized with descriptive statistics. Logistic regression assessed the association between HMH and family-reported ability to obtain gene therapy, adjusting for key covariates (age, genotype, travel time to hospital, and language). Results: Among 106 eligible patients approached, 100 (94%) consented to participation of whom 96 (96%) completed the survey in 4 languages, and 82 (85%) were evaluable for the primary outcome. Children were identified as Black (60%), Hispanic (26%), publicly insured (71%), and living in a single-parent household (50%) with English as the primary language (68%). At the household-level, 54% (n=44) of children lived in households defined as low-income (<200% Federal Poverty Level); 60% (n=49) lived with at least one HMH domain over the prior 12-months, including utility (44%), food (40%), housing (39%) and transportation (13%) insecurities. Thirty-seven (45%) children were exposed to HMH in ≥2 domains. A majority of parents reported means-tested program participation (n=48, 59%)—including Supplemental Nutrition Assistance Program (SNAP) (n=33, 40%), subsidized housing assistance (n=20, 24%), and energy assistance programs (n=11, 13%)—in addition to engagement with hospital social work (n=63, 77%). Forty percent (n=33) of parents reported an ability to obtain curative SCD therapy for their child. In univariate analysis, HMH was the only variable significantly associated with family-reported ability to access curative therapy. Upon adjustment for key covariates, HMH-exposure was independently associated with 95% lower odds of family-reported ability to obtain curative therapy (95% CI 77-99%, p < 0.001). In exploratory analysis of HMH as an ordinal exposure, each domain increase was associated with 35% lower odds of family-reported ability to obtain curative SCD therapy (95% CI 8-56%, p = 0.02). Conclusion: Gene therapy offers an opportunity for potential cure for children with SCD, but only 40% of families at a quaternary care center report they would be able to access this resource- and time-intensive treatment. Notably, nearly two-thirds of children live in households with HMH, despite means-tested benefit participation and active social work engagement, and HMH-exposure was an independent negative predictor of family-reported ability to access curative therapy. These data identify an immediate opportunity to evaluate HMH-targeted interventions to improve access to and receipt of SCD gene therapy.