Improved collection of hematopoietic stem cells and progenitors from Fanconi anemia patients for gene therapy purposes.

ABSTRACT: Difficulties in the collection of hematopoietic stem and progenitor cells (HSPCs) from Fanconi anemia (FA) patients have limited the gene therapy in this disease. We have investigated (ClinicalTrials.gov, NCT02931071) the safety and efficacy of filgrastim and plerixafor for mobilization of HSPCs and collection by leukapheresis in FA patients. Nine of eleven enrolled patients mobilized beyond the threshold level of 5 CD34⁺ cells/μL required to initiate apheresis. A median of 21.8 CD34⁺ cells/μL was reached at the peak of mobilization. Significantly, the oldest patients (15 and 16 years old) were the only ones who did not reach that threshold. A median of 4.27 million CD34⁺ cells/kg was collected in 2 or 3 aphereses. These numbers were markedly decreased to 1

SUBMITTER: Sevilla J 

PROVIDER: S-EPMC8390450 | biostudies-literature | 2021 Sep

REPOSITORIES: biostudies-literature