Safety and efficacy of plerixafor dose escalation for the mobilization of CD34⁺ hematopoietic progenitor cells in patients with sickle cell disease: interim results.

ABSTRACT: Gene therapy for sickle cell disease is limited by the yield of hematopoietic progenitor cells that can be harvested for transduction or gene editing. We therefore performed a phase I dose-escalation study of the hematopoietic progenitor cell mobilizing agent plerixafor to evaluate the efficacy and safety of standard dosing on peripheral blood CD34⁺ cell mobilization. Of 15 patients enrolled to date, only one was chronically transfused and ten were on hydroxyurea. Of eight patients who achieved a CD34⁺ cell concentration >30 cells/μL, six were on hydroxyurea. There was no clear dose response to increasing plerixafor dosage. There was a low rate of serious adverse events; two patients developed vaso-occlusive crises, at the doses of 80 μg/kg and 240 μg/kg. Hydroxyurea

SUBMITTER: Boulad F 

PROVIDER: S-EPMC5927989 | biostudies-literature | 2018 May

REPOSITORIES: biostudies-literature