Disease severity impacts plerixafor-mobilized stem cell collection in patients with sickle cell disease

Abstract

Recent studies suggest that plerixafor mobilization and apheresis in patients with sickle cell disease (SCD) is safe and can allow collection of sufficient CD341 hematopoietic stem cell (HSC) collection for clinical gene therapy applications. However, the quantities of plerixafor-mobilized CD341 cells vary between different SCD patients for unknown reasons. Twenty-three participants with SCD underwent plerixafor mobilization followed by apheresis, processing, and HSC enrichment under a phase 1 safety and efficacy study conducted at 2 institutions. Linear regression or Spearman's correlation test was used to assess the relationships between various hematologic and clinical parameters with total CD341 cells/kg collected. Median CD341 cells/kg after 2 or fewer mobilization and apheresis cycles was 4.0 3 106 (range, 1.5-12.0). Similar to what is observed generally, CD341 yield correlated negatively with age (P