Pediatric hematologists may need to rethink how they address hematopoietic cell transplantation (HCT) with patients with sickle cell disease (SCD), according to research published in the British Journal of Haematology. The study results suggested that clinicians’ attitudes and referral practices may play a role in the underutilization of this therapy by the SCD pediatric population.

Researchers conducted a nationwide survey of pediatric hematologists in the United States between February and May 2016. A 37-question survey was emailed to 1149 members of the American Society of Pediatric Hematology/Oncology (ASPHO) and program coordinators at 70 pediatric hematology and oncology fellowship programs.

Related Articles

The final analysis included 287 surveys, and the respondents reported they had informed 42% of families of children with SCD who were seen at their practice about HCT as a treatment option. Factors that were significantly associated with informing families about HCT included the clinician having personally performed HCT, the practice comprising a higher proportion of patients with SCD, and the practice being community-based.

Though 93% of respondents felt there is adequate evidence to support use of HCT to treat pediatric SCD, some respondents cited issues involving HCT safety, with 23% agreeing that the cure rate after HCT is too low, 30% agreeing that risk for graft-versus-host disease is too high, and 31% agreeing that mortality rate is too high to justify the use of HCT. A smaller percentage of clinicians also cited the cost of HCT as being a limiting factor; clinicians who felt HCT was too costly were less likely to discuss HCT as a treatment option.

The researchers suggested that all families of patients with SCD be informed of HCT as a curative treatment option within the first year of establishing care with a hematologist, as it is paramount that patients and families understand all possible treatment options prior to enrolling in clinical trials for new agents or gene therapy. In addition, they recommended every full sibling of affected patients be tested to establish whether they are a human leukocyte antigen match.

Disclosures: Some authors have declared affiliations with the pharmaceutical industry. Please refer to the original study for a full list of disclosures.

Reference

  1. Stallings AM, Majhail NS, Nowacki AS, et al. Paediatric haematologists’ attitudes regarding haematopoietic cell transplantation as treatment for sickle cell disease [published online November 12, 2019]. Br J Haematol. doi:10.1111/bjh.16248