Paediatric haematologists’ attitudes regarding haematopoietic cell transplantation as treatment for sickle cell disease

Abstract

Summary Beginning early in childhood, patients with sickle cell disease [SCD; a group of genetic haemoglobin disorders characterized by the sickle or HbS mutation (HBB E7V)] are at risk of life‐threatening and debilitating health events. Despite the high morbidity and mortality of this disease, haematopoietic cell transplantation (HCT), a curative therapy for SCD, remains underutilized. A variety of factors, including the limited availability of suitable donors, play a role in this trend, but do not fully explain the low frequency with which this therapy is employed. The objective of this study was to identify paediatric haematologists’ attitudes about HCT as a treatment option for SCD, and to describe the impact of these attitudes on their practices of discussing HCT with families of children affected by this disease. A nationwide survey of paediatric haematologists in the United States was conducted between February and May 2016. Two hundred and eighty‐seven surveys were included in the final analysis (response rate 20%). On average, respondents reported informing 42% of families about HCT as a treatment option (N = 248, 95% confidence interval: 38–46). Clinician attitudes about the cost and safety of HCT were associated with practices of discussing this therapy with families. These findings suggest that clinician attitudes and referral practices may play a role in the underutilization of this therapy in the SCD population.

Hematopoietic stem cell transplant referral patterns for children with sickle cell disease vary among pediatric hematologist/oncologists’ practice focus: A Sickle Cell Transplant Advocacy and Research Alliance (STAR) study

“…28 In 2016, a survey was sent to PHO physicians throughout the United States with the goal of evaluating provider characteristics and the timing of HSCT discussions with families of children with SCD, as well as family characteristics affecting the decision to discuss HSCT and clinician beliefs about safety and efficacy of HSCT. 33 Compared to 74% of respondents in our survey, only 45% of respondents routinely discussed HLA typing of full siblings. Similar to our findings, no provider demographics were associated with being more likely to discuss or refer for HSCT, and clinicians were concerned about previous history of suboptimal adherence with treatment regimens and the risk benefit ratio of HSCT compared to SCA severity.…”

“…HSCT methods and efficacy have improved in the almost 10 years since the survey was distributed; our knowledge of SCD and its complications has also improved, with SCI now recognized as the most common cause of neurologic damage 28 . In 2016, a survey was sent to PHO physicians throughout the United States with the goal of evaluating provider characteristics and the timing of HSCT discussions with families of children with SCD, as well as family characteristics affecting the decision to discuss HSCT and clinician beliefs about safety and efficacy of HSCT 33 . Compared to 74% of respondents in our survey, only 45% of respondents routinely discussed HLA typing of full siblings.…”