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

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.