Clinical Trial Decision Making in Pediatric Sickle Cell Disease: A Qualitative Study of Perceived Benefits and Barriers to Participation

Clinical trial research forms the foundation for advancing treatments; yet, children with sickle cell disease (SCD) are currently underrepresented. This qualitative study examines decision-making processes of youth with SCD and their caregivers regarding enrollment in clinical trial research. A subsample of participants from a study of clinical trial decision making among youth with health disparity conditions, 23 caregivers and 29 children/teens/young adults with SCD (age, 10 to 29 y), indicated whether or not they would participate in hypothetical medical and psychosocial clinical trials and prioritized barriers and benefits to participation via card sort and semistructured interviews. Audio recordings were transcribed and coded for themes. Participants reported that concerns of potential harm most affected their decision. Secondary factors were potential benefit, manageable study demands, and trust in the medical staff. Caregivers weighed potential harm more heavily than their children. Young children were more likely to endorse potential benefit. Overall, participants stated they would be willing to participate in research if the potential benefit outweighs potential harm and unmanageable study demands. To optimize recruitment, results suggest addressing potential harm first while highlighting potential benefits, creating manageable study demands, and endorsing the future benefits of research to the sickle cell community.