Pediatric hematologists/oncologists who focus on pediatric sickle cell disease (SCD) and hematopoietic stem cell transplant (HSCT) are more likely to refer children with SCD for matched sibling donor (MSD) HSCT in nearly every hypothetical clinical scenarios compared with general pediatric hematologists/oncologists, according to the results of a survey published in Pediatric Blood & Cancer.
Investigators for the Sickle Cell Transplant Advocacy and Research Alliance (STAR) study sought to understand why adoption of HSCT as a curative therapy for SCD has been slow among the pediatric hematologists/oncologists community.
To assess providers’ perceptions about MSD HSCT for children with variable SCD severity, the investigators emailed the survey to American Society of Pediatric Hematology/Oncology Clinical Forum listserv subscribers and American Society of Hematology members who self-identified as pediatric hematologists/oncologists. Of 3000 potential respondents, 195 responses were received (estimated response rate, 6.5%), and 194 responses were included in the final analysis (1 nonpediatric hematologists/oncologists was excluded).
Most respondents were women (58%) who were aged 30 to 49 years (69%). Pediatric hematologists/oncologist self-identified ethnicities were 68% Caucasian (68%), Asian/Pacific Islander (19%), African American (7%), and Hispanic (3%).
The only significant associations between pediatric hematologists/oncologist demographics and characteristics were practice focus, and the likelihood to refer to HSCT for any of the scenarios presented in the survey.
Among the 11 clinical scenarios, providers who focused on SCD and HSCT were more likely to refer an asymptomatic child, a child who had suboptimal adherence to hydroxyurea, or had never been admitted to the hospital compared with general pediatric hematologists/oncologists. The authors suggested that these differences were likely due to increased awareness of long-term effects of SCD and safety of MSD HSCT for children with SCD among SCD- and HSCT-focused providers.
Among all respondents, 87% would refer a child with β-thalassemia major while only 47% and 23% would do so for an asymptomatic child with SCD with the HbSS (P <.00001) variant or non-HbSS variant (P <.00001), respectively.
The reasons given by respondents who were somewhat or very unlikely to refer for HSCT were transplant-related mortality, chronic graft-vs-host disease in those children with a history of suboptimal hydroxyurea adherence, and poor adherence with post-HSCT medication in children with a history of suboptimal adherence to hydroxyurea.
For children with nonsickle cell anemia sickle genotypes, reasons for somewhat or very unlikely to refer for HSCT were lack of data for HSCT as a treatment option, an unfavorable risk benefit ratio, and the economic burden of HSCT on the family.
“Data from early and current studies of MSD HCST performed for patients with SCA show very high rates of survival and cure. However, this curative therapy is underutilized, and our study provides a better understanding of why providers may be hesitant to refer families for MSD HSCT,” wrote the authors.
Disclosure: Some authors have declared affiliations with or received funding from the pharmaceutical industry. Please refer to the original study for a full list of disclosures.
Meier ER, Abraham AA, Ngwube A, et al. 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. Pediatr Blood Cancer. Published online January 6, 2021. doi:10.1002/pbc.28861