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- Title
Granulocyte-colony stimulating factor plus plerixafor in patients with β-thalassemia major results in the effective mobilization of primitive CD34+ cells with specific gene expression profile.
- Authors
Baiamonte, Elena; Barone, Rita; Contino, Flavia; Di Stefano, Rosalia; Marfia, Anna; Filosa, Aldo; D'Angelo, Emanuela; Feo, Salvatore; Acuto, Santina; Maggio, Aurelio
- Abstract
Successful gene therapy for β-thalassemia requires optimal numbers of autologous gene-transduced hematopoietic stem and progenitor cells (HSPCs) with high repopulating capacity. Previous studies suggested superior mobilization in these patients by the combination of granulocyte-colony stimulating factor (G-CSF) plus plerixafor over single agents. We mobilized four adult patients using G-CSF+plerixafor to assess the intra-individual variation of the circulating CD34+ cells number and subtypes pre- and post-plerixafor administration. The procedure was well-tolerated and the target cell dose of ≥8x106 CD34+ cells/kg was achieved in three of them with one apheresis procedure. The addition of plerixafor unanimously increased the number of circulating CD34+ cells, and the frequency of the most primitive CD34+ subtypes: CD34+/38- and CD34+/133+/38- as well as the in vitro clonogenic potency. Microarray analyses of CD34+ cells purified from the leukapheresis of one patient mobilized twice, with G-CSF and with G-CSF+plerixafor, highlighted in G-CSF+plerixafor-mobilized CD34+ cells, higher levels of expression genes involved in HSPC motility, homing, and cell cycles. In conclusion, G-CSF+plerixafor in β-thalassemia patients mobilizes optimal numbers of HSPCs with characteristics that suggest high capacity of engraftment after transplantation.
- Subjects
THALASSEMIA treatment; GRANULOCYTE-macrophage colony-stimulating factor; GENE therapy; CD34 antigen; LEUKAPHERESIS; GENE expression; BETA-Thalassemia; THERAPEUTICS
- Publication
Thalassemia Reports, 2017, Vol 7, Issue 1, p11
- ISSN
2039-4357
- Publication type
Article
- DOI
10.4081/thal.2017.6392