G-CSF plus AMD3100 mobilization in adult patients with beta-thalassemia results in effective mobilization of CD34+ cells with primitive signatures

ABSTRACT: Successful gene therapy for β-thalassemia requires optimal numbers of autologous gene-transduced hematopoietic progenitors stem cells (HPSC) with high repopulating capacity and mobilization is the optimal approach for achieving this goal. We performed a clinical study to investigate safety and efficacy of G-CSF+AMD3100 mobilization in four adult with β-thalassemia to reach a cell dose of ≥8x106 CD34+cells/Kg and to characterize the CD34+cell populations mobilized before and after plerixafor administration. The procedure resulted well-tolerated in all patients. Three of the four patients reached the target cell dose or more in single-apheresis collections, even one patient where a significant dose reduction of G-CSF was imposed due to early hyperleukocytosis. A significant increase i

ORGANISM(S): Homo sapiens

SUBMITTER: Flavia Contino 

PROVIDER: E-GEOD-70219 | biostudies-arrayexpress |

REPOSITORIES: biostudies-arrayexpress