Factors Impacting Family Decisions to Pursue Transplantation for Children with Sickle Cell Disease

Background: The only cure currently available for sickle cell disease (SCD) is hematopeotic stem cell transplantation (HSCT). Barriers preventing widespread application of HSCT include limited donor pools and lack of family education about HSCT. To our knowledge, no studies have examined medical and psychosocial factors that influence families' decisions about whether or not to pursue HSCT for their child with SCD.

Methods: Surveys created by members of the Sickle Cell, Blood and Marrow Transplantation, and Psychosocial teams were distributed to family members attending an education symposium about HSCT for SCD at a large metropolitan hospital. Surveys were anonymous, optional, and approved by the Institutional Review Board; participants were entered into a gift card drawing. On a scale from 0 (“not important at all”) to 3 (“very important”), participants rated the relative importance of 17 factors that may impact the decision to pursue HSCT. 

Results: Thirty-four attendees completed surveys; 15% were parents/guardians of patients who had already had a transplant (n = 5), 73% were parents/guardians of patients who had not had a transplant (n = 25), and 12% were “other” family members (n = 4; e.g., aunt, grandmother). Items that were consistently rated important (i.e., >80% of respondents rated them “somewhat important,” “important,” or “very important”) represented multiple domains, including: HSCT-related risks (e.g., death, infertility, transplant failure, GVHD), prevention of SCD complications, medical team interactions (e.g., hematologist recommendation, trust in medical team), and psychosocial concerns (e.g., emotional strain on patient/parents, social support). Items not consistently rated important included financial strain, impact on sibling donor, child losing hair, child missing school, and religious beliefs. Mean importance ratings were highest for risk of death (M = 2.91) and prevention of SCD complications (M = 2.82) and lowest for religious beliefs (M = .68) and child losing hair (M = 1.00). See Table 1 for importance ratings of individual survey items. There were few differences between ratings of family members who had been through transplant and those who had not.

Conclusions: When considering HSCT for SCD, parents and caregivers report HSCT-related risks, interactions with medical teams, and emotional effects as most important to their decision. Results emphasize the importance of ongoing education for families, the role of the family-physician relationship, and the value of psychosocial support throughout the transplant process. Although the current study is limited by a small convenience sample that may not be representative of the large sickle cell population (i.e., self-selection to attend the symposium may indicate greater interest in pursuing transplant), these findings provide important insight into the complexity of family decisions about HSCT.