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

Daniele Canarutto, Francesca Tucci, Salvatore Gattillo, Matilde Zambelli, Valeria Calbi, Bernhard Gentner, Francesca Ferrua, Sarah Marktel, Maddalena Migliavacca, Federica Barzaghi, Giulia Consiglieri, Vera Gallo, Francesca Fumagalli, Paola Massariello, Cristina Parisi, Gianluca Viarengo, Elena Albertazzi, Paolo Silvani, Raffaella Milani, Luca SantoleriFabio Ciceri, Maria Pia Cicalese, Maria Ester Bernardo, Alessandro Aiuti

Research output: Contribution to journalArticlepeer-review


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 collected ≥7 and ≥13 × 106 CD34+ cells/kg in the first cycle had pre-apheresis circulating counts of at ≥42 and ≥86 CD34+ cells/μL, respectively. Weight-adjusted CD34+ cell yield was positively correlated with peripheral CD34+ cell counts and influenced by female gender, disease, and drug dosage. All patients received a GT product above the minimum target, ranging from 4 to 30.9 × 106 CD34+ cells/kg. Pediatric PBSC collection compares well to BM harvest in terms of CD34+ cell yields for the purpose of GT, with a favorable safety profile.

Original languageEnglish
Pages (from-to)76-83
Number of pages8
JournalMolecular Therapy - Methods and Clinical Development
Publication statusPublished - Sep 10 2021


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