Out of Reach: Understanding Resource Barriers to Gene Therapy for Pediatric Patients With Sickle Cell Disease.

BACKGROUND

Sickle cell disease (SCD) is a highly morbid condition resulting in poor quality of life and early death. Gene therapy offers opportunity for cure, but its resource‑ and time‑intensive nature may limit access.

PROCEDURE: We conducted a single-center, cross-sectional study to determine the proportion of families with a child with SCD who report the ability to access a hypothetical curative therapy and to identify family-reported resource barriers. A single-timepoint survey was administered to 100 families with children aged 2-17 years receiving sickle cell care at a quaternary care center. The survey queried parent-reported household characteristics and unmet basic needs in food, housing, utilities, or transportation (household material hardship [HMH]) and assessed parent-reported ability to access a hypothetical curative treatment based on a vignette. Multivariable logistic regression examined associations of HMH and disease severity with family-reported access.

RESULTS

Of the evaluable 82 families, 60% experienced HMH over the prior 12 months despite robust social work involvement (78%) and participation in means-tested benefits (59%). Forty percent of parents reported an ability to obtain curative therapy with current resources. Upon adjustment for key covariates, HMH exposure was independently associated with 95% lower odds of family-reported ability to obtain curative therapy.

CONCLUSIONS

Although gene therapy offers a potential cure for SCD, fewer than half of families reported ability to access it with current supports. HMH exposure affected nearly two-thirds of families and was independently associated with lower odds of access. As highly centralized, resource-intensive treatments expand, resource-targeted interventions are needed to ensure equitable access and receipt.