Current Attitudes of Parents and Patients towards Hematopoietic Stem Cell Transplantation for Sickle Cell Anemia

Background: Sickle cell anemia(SCA) is a chronic hemolytic anemia  with high morbidity and premature mortality. Currently, the only available cure for SCA is allogeneic hematopoietic stem cell transplantation (HSCT).  Transplant-associated risks are one of many barriers to the widespread use of HSCT for SCA.  Over 20 years ago, Kodish et al reported on parent attitudes towards HSCT for their children with SCA.  Since the risks of matched sibling donor HSCT for SCA are now better defined and community awareness of HSCT as a therapeutic option has increased, parent and patient perceptions of HSCT may have changed.

Methods:  To assess current attitudes toward HSCT, we conducted a survey of parents of children with SCA (Hgb SS or S-B⁰ thal) and teenagers with SCA by using questions based on the standard reference gamble paradigm and by comparing survey responses between African (A) and African American parents (AA) and between parents and their adolescent children. SCA severity was assessed based on their self-reported history of sickle cell disease related complications and hospitalizations.    

Results: 89 parents and adolescents (54 parents, 3 patients and 16 parent-adolescent dyads) were surveyed after a discussion of the process and outcomes of HSCT and its attendant risks. 72% of respondents were willing to accept > 5% risk of mortality.  57% of respondents were willing to accept a risk of 10% or more GVHD.  20/89 (22%) respondents were unwilling to accept any risk of mortality or GVHD.   Risk averseness did not differ amongst respondents of A or AA origin. There was no correlation between self reported SCA severity and acceptance of higher mortality or GVHD risk.  56% of respondents were willing to accept infertility post BMT regardless of SCA severity or country of origin.  Of those who were willing to accept at least a 5% risk of mortality, 75% were willing to accept a GVHD risk of at least 10%. 

Conclusions: These data suggest that the majority of parents and adolescents are willing to accept the current morbidity and mortality risks associated with matched sibling HSCT to achieve a cure of SCA regardless of disease severity.  Although attitudes towards HSCT for SCA have changed over the last 2 decades, there are still significant numbers of parents and adolescents who are unwilling to accept any risk of mortality or GVHD in order to cure their SCA.