Introduction: Sickle cell disease (SCD) is a chronic genetic disease with high morbidity and early mortality; affecting nearly 100,000 individuals in the United States. Bone marrow transplantation, the only curative treatment, is available to less than 20% of patients, due to a number of access barriers. Gene transfer therapy (GTT) has shown to be curative in animal models, and is approved for use in humans for early-phase studies at a few centers. GTT would offer a more accessible treatment option available to all patients. It is important to understand patient perspectives on GTT to help ensure human clinical trial success. Methods: Two focus groups were conducted with younger (18–30 years) and older (≥ 31 years) adults with SCD to obtain data on patient knowledge and beliefs about GTT. Data from these two focus groups was used to develop a GTT educational brochure. A third focus group was conducted to obtain participant feedback on acceptability and feasibility of education and the brochure. Results: Most adults, especially young adults, had little knowledge about GTT and expressed fear and uncertainty about the side effects of chemotherapy (e.g., hair loss, infertility), use of a human immunodeficiency virus (HIV)-derived viral vector, and potential for cancer risk. Participants wanted full transparency in educational materials, but advised researchers not to share the vector’s relation to HIV due to cultural stigma and no HIVvirus is used for the GTT-vector. Conclusions: Older adults had more desire to participate in human clinical GTT trials than younger participants. When recruiting for trials, researchers should develop GTT educational materials that address participant lack of trust in the healthcare system, cultural beliefs, fears related to side effects, and include visual illustrations. Use of such materials will provide adults with SCD the information they need to fully evaluate GTT.

Findings support the value of depression screening and interventions to promote parent self-efficacy in managing childhood SCD pain.

Adolescents and young adults (AYA) with sickle cell disease (SCD) are at risk for poor healthrelated quality of life (HRQOL). Research suggests that vulnerability factors (e.g., disease severity) and self-management resources (e.g., disease self-efficacy) jointly impact health outcomes, including HRQOL; however, this has not been studied among AYA with SCD. This study examined the relationship between disease self-efficacy, HRQOL, and disease severity in AYA with SCD. HRQOL was positively correlated with disease self-efficacy and negatively correlated with disease severity. Disease self-efficacy and severity accounted for 35% of variance in HRQOL. Findings support the impact of disease self-efficacy on HRQOL.