Parent and Guardian Knowledge of Hematopoietic Cell Transplantation As a Treatment Option for Sickle Cell Disease

Introduction: Beginning early in childhood, patients with sickle cell disease (SCD) are at risk of life-threatening and debilitating health events. Despite the high morbidity and mortality of this disease, hematopoietic cell transplantation (HCT), the only curative therapy for SCD, remains underutilized. Between 2008 and 2012, an average of 76 HCTs per year were performed on patients with SCD in the United States (US), a remarkably low rate considering that nearly 100,000 people in the US are living with this disease and approximately 1,000 infants are born with SCD in the US each year. Multiple factors may contribute to the low rates of HCT in this population. However, there is a paucity of data concerning the level of awareness of HCT among parents and guardians of children with SCD, an important factor to consider in the assessment of HCT utilization rates.

Objectives: To identify the proportion of parents and guardians of children with SCD who are aware of HCT as a treatment option, and to evaluate the relationship between patient demographics and parental/guardian awareness of this treatment option.

Methods: A 25-item paper-based survey was developed to assess parent/guardian awareness of HCT, based upon feedback from leaders in SCD community health organizations and pediatric hematologists. The survey was administered to parents/guardians of pediatric patients with SCD during routine outpatient appointments with their child's hematologist and at SCD community events sponsored by participating clinical sites. The survey was anonymous, and a $5.00 gift card was provided to all participants who completed the survey. Participants were recruited from four clinical centers across three cities in the Midwestern United States. All parents/legal guardians of children (<18 years of age) with SCD, who have not undergone HCT, were eligible to participate. Multivariable logistic regressions were performed to assess the relationship between the following variables and parent/guardian awareness of HCT as a treatment option for SCD: accrual site, patient age, number of full siblings, parent/guardian education level, parent/guardian relationship to patient, duration of custody, and SCD genotype.

Results: 229 respondents (95% biological parent, 1% adoptive parent, 4% legal guardian) have participated in the study to date. 95% of all eligible parents/guardians agreed to participate. 82% of study respondents indicated that they have heard of HCT as a treatment option for SCD (N=187). 53% indicated that their child's physician has talked to them about HCT at some point in the past (N=120). On average, patients were 2.84 years old when the parent/guardian first heard about HCT (N=178, 95% CI: 2.31-3.37); patients were 3.08 years old on average when the child's physician first mentioned HCT (N=113, 95% CI: 2.42-3.75). 76% of respondents indicated that they are aware that HCT can potentially cure SCD (N=172). Higher parent/guardian education level and SS genotype were associated with having heard about HCT as a treatment option. SS genotype was associated with having been informed about HCT by their child's physician. Having been informed about HCT by their child's physician was associated with parent/guardian awareness that HCT is a potentially curative treatment.

Conclusions: The majority of parents/guardians of children with SCD enrolled in this study have heard of HCT and are aware that HCT is a potentially curative treatment. In this population, non-physician sources of information and parent/guardian education level play an important role in awareness of this treatment modality. Physician-family discussion about HCT plays an important role specifically in parent/guardian awareness of the curative potential of this treatment. These data underscore the importance of physicians discussing HCT with families, particularly those who have limited resources to research treatment options on their own.