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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.
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.
| 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): | http://dx.doi.org/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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