ACCEPTABILITY OF IN UTERO HEMATOPOIETIC CELL TRANSPLANTATION FOR SICKLE CELL DISEASE

Purpose: In Utero Hematopoietic Cell Transplantation (IUHCT) is an experimental treatment that has curative potential for Sickle Cell Disease (SCD) but carries a risk of fetal demise. Using a reference-gamble paradigm, we sought to understand under what conditions parents of children with SCD and young adults (YA) with SCD would consider fetal intervention.

Methods: Following description of the procedure, including a 5% fixed risk of fetal demise, participants were randomized to consider a hypothetical cure rate (20%, 40%, or 70%). Subsequently, cure rate was either increased or decreased by set increments depending on the previous answer to reveal the lowest acceptable cure rate. Participants also completed the Pediatric Research Participation Questionnaire (PRPQ) to assess attitudes toward clinical trials.  Finally, to study the role of omission bias in decision making for IUHCT, participants completed a 6-item omission scale. 

Results: Overall, 74 of 79 (94%) participants were willing to consider IUHCT, and 52 (66%) participants accepted IUHCT at a cure rate of 40%, the estimated rate of therapeutic mixed chimerism. There were no significant differences between parents and YA. Individuals with higher scores on the PRPQ perceived benefits scale were more likely to participate at lower cure rates (OR 1.08, p=0.007) and individuals with higher omission scale scores (tendency toward omission bias) were less likely to participate at lower cure rates (OR 0.83, p=0.04).  Demographics and SCD severity were not significantly associated with acceptability of IUHCT.

Conclusion: This study suggests that the majority of parents and YA would consider IUHCT in a future pregnancy under expected therapeutic conditions.