Across the Myeloablative Spectrum: Hematopoietic Cell Transplant Conditioning Regimens for Pediatric Patients with Sickle Cell Disease

Emily Limerick; Allistair Abraham

doi:10.3390/jcm11133856

Across the Myeloablative Spectrum: Hematopoietic Cell Transplant Conditioning Regimens for Pediatric Patients with Sickle Cell Disease

J Clin Med. 2022 Jul 3;11(13):3856. doi: 10.3390/jcm11133856.

Authors

Emily Limerick¹, Allistair Abraham²

Affiliations

¹ Cellular and Molecular Therapeutics Branch, National Health, Lung, and Blood Institute, Bethesda, MD 20892, USA.
² Division of Blood and Marrow Transplantation, Children's National Hospital, Washington, DC 20010, USA.

Abstract

One out of every five hundred African American children in the United States has sickle cell disease (SCD). While multiple disease-modifying therapies are available, hematopoietic cell transplantation (HCT) remains the only curative option for children with SCD. HLA-matched sibling HCT has demonstrated excellent efficacy, but its availability remains limited; alternative donor strategies are increasingly explored. While Busulfan-Cyclophosphamide has become the most widespread conditioning regimen employed in HCT for pediatric SCD, many other regimens have been examined. This review explores different conditioning regimens across the intensity spectrum: from myeloablative to non-myeloablative. We describe survival and organ function outcomes in pediatric SCD patients who have received HCT and discuss the strengths and weaknesses of the various conditioning intensities. Finally, we posit novel directions in allogeneic HCT for SCD.

Keywords: allogeneic transplant; conditioning intensity; myeloablative conditioning; non-myeloablative conditioning; reduced intensity conditioning; sickle cell disease.

Publication types

Review

Grants and funding

This research received no external funding