What does this research mean for the field?

Household material hardship affects a majority of families with pediatric sickle cell disease and is independently associated with significantly lower odds of being able to access resource-intensive curative treatments like gene therapy. Novelty: ClaimNovelty.INCREMENTAL. Consensus alignment: ConsensusAlignment.NEUTRAL.

What question did this study set out to answer?

This research aims to explore access to gene therapy among families of children with sickle cell disease and identify existing resource barriers.

July 27, 2026Pediatric Blood & Cancer

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

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Authors

CKColleen Kelly MPMorgan A. Paul MGMckenzie Griffin

Discussion

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Overview

Cross-sectional study identifies resource barriers affecting access to gene therapy in families of SCD patients, suggesting interventions for equality.

Key Points

This research aims to explore access to gene therapy among families of children with sickle cell disease and identify existing resource barriers.
Single-center, cross-sectional study design
Survey administered to 100 families of children aged 2-17 years with sickle cell disease
Multivariable logistic regression analysis of household characteristics and resource barriers
60% of families experienced household material hardship over the prior 12 months
40% of parents reported an ability to access curative therapy with current resources
HMH exposure was associated with a 95% reduction in odds of accessing the therapy

Cite This Study

Kelly et al. (2026) studied this question.

synapsesocial.com/papers/6a6700a340bca442e0d4a7b9 https://doi.org/10.1002/1545-5017.70536

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July 27, 2026Pediatric Blood & Cancer

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

View Full Paper

Ask AI

Bookmark

Authors

CKColleen Kelly MPMorgan A. Paul MGMckenzie Griffin

Discussion

Member takes

Overview

Cross-sectional study identifies resource barriers affecting access to gene therapy in families of SCD patients, suggesting interventions for equality.

Key Points

This research aims to explore access to gene therapy among families of children with sickle cell disease and identify existing resource barriers.
Single-center, cross-sectional study design
Survey administered to 100 families of children aged 2-17 years with sickle cell disease
Multivariable logistic regression analysis of household characteristics and resource barriers
60% of families experienced household material hardship over the prior 12 months
40% of parents reported an ability to access curative therapy with current resources
HMH exposure was associated with a 95% reduction in odds of accessing the therapy

Cite This Study

Kelly et al. (2026) studied this question.

synapsesocial.com/papers/6a6700a340bca442e0d4a7b9 https://doi.org/10.1002/1545-5017.70536

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