Knowledge to date on secondary malignancy following hematopoietic cell transplantation for sickle cell disease.

ABSTRACT: Allogeneic hematopoietic cell transplantation, gene therapy, and gene editing offer a potential cure for sickle cell disease (SCD). Unfortunately, myelodysplastic syndrome and acute myeloid leukemia development have been higher than expected after graft rejection following nonmyeloablative conditioning and lentivirus-based gene therapy employing myeloablative busulfan for SCD. Somatic mutations discovered in 2 of 76 patients who rejected their grafts were identified at baseline at much lower levels. While a whole-genome sequencing analysis reported no difference between patients with SCD and controls, a study including whole-exome sequencing revealed a higher prevalence of clonal hematopoiesis in individuals with SCD compared with controls. Genetic risk factors for myeloid malignancy devel

SUBMITTER: Fitzhugh CD 

PROVIDER: S-EPMC9820448 | biostudies-literature | 2022 Dec

REPOSITORIES: biostudies-literature