Bone marrow harvesting from paediatric patients undergoing haematopoietic stem cell gene therapy

Francesca Tucci, Marta Frittoli, Federica Barzaghi, Valeria Calbi, Maddalena Migliavacca, Francesca Ferrua, Francesca Fumagalli, Laura Lorioli, Laura Castagnaro, Marcella Facchini, Claudia Fossati, Stefano Zancan, Paola Massariello, Michele Manfredini, Giulia Consiglieri, Daniele Canarutto, Salvatore Recupero, Francesco Calzatini, Miriam Casiraghi, Silvia DarinGigliola Antonioli, Roberto Miniero, Rossana Fiori, Paolo Silvani, Matilde Zambelli, Sarah Marktel, Salvatore Gattillo, Raffaella Milani, Luca Santoleri, Fabio Ciceri, Alessandra Biffi, Maria Pia Cicalese, Maria Ester Bernardo, Alessandro Aiuti

Research output: Contribution to journalArticlepeer-review


Collection of an adequate amount of autologous haematopoietic stem progenitor cells (HSPC) is required for ex vivo manipulation and successful engraftment for certain inherited disorders. Fifty-seven paediatric patients (age 0.5–11.4 years) underwent a bone marrow harvest for the purpose of HSPC gene therapy (GT), including adenosine deaminase-severe combined immunodeficiency (ADA-SCID), Wiskott–Aldrich syndrome (WAS) and metachromatic leukodystrophy (MLD) patients. Total nucleated cells and the percentage and absolute counts of CD34+ cells were calculated at defined steps of the procedure (harvest, CD34+ cell purification, transduction with the gene transfer vector and infusion of the medicinal product). A minimum CD34+ cell dose for infusion was 2 × 106/kg, with an optimal target at 5–10 × 106/kg. Median volume of bone marrow harvested was 34.2 ml/kg (range 14.2–56.6). The number of CD34+ cells collected correlated inversely with weight and age in all patients and particularly in the MLD children group. All patients reached the minimum target dose for infusion: median dose of CD34+ cells/kg infused was 10.3 × 106/kg (3.7–25.9), with no difference among the three groups. Bone marrow harvest of volumes > 30 ml/kg in infants and children with ADA-SCID, WAS and MLD is well tolerated and allows obtaining an adequate dose of a medicinal product for HSPC-GT.

Original languageEnglish
Pages (from-to)1995-2003
Number of pages9
JournalBone Marrow Transplantation
Issue number12
Publication statusPublished - Dec 1 2019

ASJC Scopus subject areas

  • Hematology
  • Transplantation


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