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Thalassemia Reports
Volume 7
Issue 1
10.4081/thal.2017.6392
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Article

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

by
Elena Baiamonte
1,*,
Rita Barone
1,
Flavia Contino
2,
Rosalia Di Stefano
1,
Anna Marfia
3,
Aldo Filosa
4,
Emanuela D'Angelo
5,
Salvatore Feo
2,
Santina Acuto
1 and
Aurelio Maggio
1
1
Elena Baiamonte, Campus of Hematology F. and P. Cutino, Villa Sofia-Cervello Hospital, viaTrabucco 180, 90146 Palermo, Italy
2
Department of Biological, Chemical and Pharmaceutical Sciences and Technologies and ATeN Center, University of Palermo, Palermo, Italy
3
Division of Hematology, Villa Sofia-Cervello Hospital, 90146 Palermo, Italy
4
Center for Anemias, A. Cardarelli Hospital, Napoli, Italy
5
Pediatric Clinic De Marchi, Policlinico Hospital, Milan, Italy
*
Author to whom correspondence should be addressed.
Thalass. Rep. 2017, 7(1), 6392; https://doi.org/10.4081/thal.2017.6392
Submission received: 15 November 2016 / Revised: 2 March 2017 / Accepted: 20 April 2017 / Published: 26 May 2017
Versions Notes

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.
Keywords: β-thalassemia; CD34 cells expression profiling; G-CSF plerixafor mobilization; gene therapy β-thalassemia; CD34 cells expression profiling; G-CSF plerixafor mobilization; gene therapy

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MDPI and ACS Style

Baiamonte, E.; Barone, R.; Contino, F.; Di Stefano, R.; Marfia, A.; Filosa, A.; D'Angelo, E.; Feo, S.; Acuto, S.; Maggio, A. 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. Thalass. Rep. 2017, 7, 6392. https://doi.org/10.4081/thal.2017.6392

AMA Style

Baiamonte E, Barone R, Contino F, Di Stefano R, Marfia A, Filosa A, D'Angelo E, Feo S, Acuto S, Maggio A. 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. Thalassemia Reports. 2017; 7(1):6392. https://doi.org/10.4081/thal.2017.6392

Chicago/Turabian Style

Baiamonte, Elena, Rita Barone, Flavia Contino, Rosalia Di Stefano, Anna Marfia, Aldo Filosa, Emanuela D'Angelo, Salvatore Feo, Santina Acuto, and Aurelio Maggio. 2017. "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" Thalassemia Reports 7, no. 1: 6392. https://doi.org/10.4081/thal.2017.6392

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