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EnglishSuccessful 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 preand post-plerixafor administration. The procedure was well-tolerated and the target cell dose of ≥8.106 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.
| Data di pubblicazione: | 2017 |
| Titolo: | Granulocyte–colony stimulating factor plus plerixafor inpatients with –thalassemia major results in the effective mobilization of primitiveCD34+ cells with specific gene expression profile |
| Autori: | |
| Citazione: | Elena, B., Rita, B., Contino, F., Rosalia Di, S., Anna, M., Aldo, F., et al. (2017). Granulocyte–colony stimulating factor plus plerixafor inpatients with –thalassemia major results in the effective mobilization of primitiveCD34+ cells with specific gene expression profile. THALASSEMIA REPORTS, 7(7), 11-17. |
| Rivista: | |
| Digital Object Identifier (DOI): | 10.4081/thal.2017.6392 |
| 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 preand post-plerixafor administration. The procedure was well-tolerated and the target cell dose of ≥8.106 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. |
| URL: | http://www.pagepressjournals.org/index.php/thal/ |
| Settore Scientifico Disciplinare: | Settore BIO/18 - Genetica Settore MED/15 - Malattie Del Sangue |
| Appare nelle tipologie: | 1.01 Articolo in rivista |
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