What Prompts Parents of Children with Sickle Cell Disease to Consider a Curative Option in This Era of Novel Hematopoietic Cell Donor Sources and Gene Modification?

Background Hematopoietic stem cell transplantation (HCT) from matched sibling donors has been associated with excellent outcomes in patients with SCD but has been limited by the availability of HLA identical related donors and acceptability of HCT. Improved outcomes and the availability of clinical trials of HCT from haploidentical related donors and HLA matched unrelated donors and autologous hematopoietic progenitor cells modified by gene addition and gene editing has expanded the applicability of HCT for SCD. The objective of this study was to determine from the perspective of the parent about what motivates parents of a child with SCD to consider HCT/gene therapy in the setting of alternate donors and gene modification? Methods We conducted semi-structured qualitative interviews with at least one parent of a child with SCD who had scheduled a BMT consultation. Interviews were conducted pre-consultation and a second time post-consultation Audio recordings were transcribed verbatim. Data were analyzed using open and axial coding stages of grounded theory methodology. Results We interviewed 22 parents and one caregiver (18 mothers, 2 fathers, and 1 grandmother). Six participants were only available for the post-consultation interview. The median age of caregivers was 43 years (range 31 to 64 years.) Median age of the child was 13 years (range 14 months to 18 years). Three main themes relevant to the research question emerged. First, the child's quality of life, limitations placed by the disease and the concern for future potential to live independently because of SCD complications. Second, knowledge acquired by parents about curative options through multiple sources. Third, recent disease exacerbation or new SCD complication. Conclusion These results provide insight into the factors that prompt parents of children with SCD to HCT and gene therapy and have the potential to inform efforts to raise awareness about curative options for patients with SCD.