journal article Open Access Apr 14, 2025

Barriers to Hematopoietic Stem Cell Transplant Among Caregivers and Adolescents With Sickle Cell Disease

View at Publisher Save 10.1002/pbc.31698
Abstract
ABSTRACT

Background
Hematopoietic stem cell transplantation (HSCT) is a curative treatment for sickle cell disease (SCD). However, barriers exist that prevent access to this treatment modality.


Methods
An IRB‐approved mixed‐method survey of barriers to HSCT in SCD was given to parents, caregivers, and adolescents obtaining care at the Children's of Alabama/University of Alabama at Birmingham Comprehensive Pediatric Sickle Cell Clinic.


Results
We performed 103 surveys; 97 surveys (94%) were completed by a parent/guardian/caregiver, and 6 (6%) were completed by the patient. The most common SCD complication reported was three or more yearly pain crises (43%). Most (57%) respondents heard about HSCT from their healthcare provider. The most important concern with HSCT identified by respondents (67%) was the possibility of end‐organ damage with the potential to worsen over time. The greatest barrier for respondents (77%) was a 5% chance of death with HSCT. Other barriers identified include the risk of reversible or permanent organ damage (73%), HSCT failure (54%), and infertility (42%).


Conclusion
Respondents identified barriers to HSCT; however, they are not insurmountable. Provider education regarding the benefits and potential risks of HSCT with a clear explanation of mitigation strategies can improve acceptance of HSCT among the SCD population.
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