Peripheral blood stem and progenitor cell collection in pediatric candidates for ex vivo gene therapy: a 10-year series.

ABSTRACT: Hematopoietic stem and progenitor cell (HSPC)-based gene therapy (GT) requires the collection of a large number of cells. While bone marrow (BM) is the most common source of HSPCs in pediatric donors, the collection of autologous peripheral blood stem cells (PBSCs) is an attractive alternative for GT. We present safety and efficacy data of a 10-year cohort of 45 pediatric patients who underwent PBSC collection for backup and/or purification of CD34⁺ cells for ex vivo gene transfer. Median age was 3.7 years and median weight 15.8 kg. After mobilization with lenograstim/plerixafor (n = 41) or lenograstim alone (n = 4) and 1-3 cycles of leukapheresis, median collection was 37 × 10⁶ CD34⁺ cells/kg. The procedures were well tolerated. Patients who collect

SUBMITTER: Canarutto D 

PROVIDER: S-EPMC8390560 | biostudies-literature | 2021 Sep

REPOSITORIES: biostudies-literature