Successful hematopoietic stem cell mobilization and apheresis collection using plerixafor alone in sickle cell patients.

ABSTRACT: Novel therapies for sickle cell disease (SCD) based on genetically engineered autologous hematopoietic stem and progenitor cells (HSPCs) are critically dependent on a safe and effective strategy for cell procurement. We sought to assess the safety and efficacy of plerixafor when used in transfused patients with SCD for HSC mobilization. Six adult patients with SCD were recruited to receive a single dose of plerixafor, tested at lower than standard (180 µg/kg) and standard (240 µg/kg) doses, followed by CD34⁺ cell monitoring in peripheral blood and apheresis collection. The procedures were safe and well-tolerated. Mobilization was successful, with higher peripheral CD34⁺ cell counts in the standard vs the low-dose group. Among our 6 donors, we improved apheresis cell c

SUBMITTER: Esrick EB 

PROVIDER: S-EPMC6177648 | biostudies-literature | 2018 Oct

REPOSITORIES: biostudies-literature