Stem cell transplant for children with sickle cell anemia: parent and patient interest

Abstract

We describe adolescents' and parents' interest in hematopoietic stem cell transplant (HSCT) as a cure for sickle cell disease (SCD) and factors associated with increased interest. We administered a 40 question survey to assess the interest in HSCT in parents and adolescents with HBSS or HBSβ(0) thalassemia. The survey tool assessed factors that may influence interest in HSCT including demographic data, disease severity, views on prognosis, and health-related quality of life (HRQOL). All participants were given a handout on the risks and benefits of an HSCT before completing the survey. One hundred twenty-nine parents and 59 adolescents completed the survey. Forty-five percent of parents (54 of 119) would likely have their child undergo HSCT, and 35% of adolescents (19 of 55) would likely undergo HSCT if it was recommended by their hematologist. Parents of adolescents, as well as adolescent patients with better HRQOL, were more interested in HSCT. Prior exchange transfusion was associated with increased interest in HSCT (62% [23 of 37] versus 38% [29 of 76]; P = .02). The majority of parents believe their child's SCD will get better (66%; [80 of 122]), will not likely prevent their child from achieving life goals (83%; [100 of 121]), and will not shorten their child's lifespan (86%; [102 of 119]). There is strong parent and adolescent interest in HSCT as a cure for SCD. It is concerning that few parents and adolescents believe SCD will negatively impact their prognosis. Education on the potential long-term sequelae of SCD is needed when considering the role for HSCT.