Allogeneic hematopoietic cell transplantation (HCT) is the only curative option for sickle cell disease (SCD) patients but its use is significantly limited by the lack of compatible donors. Gene therapy using autologous hematopoietic stem cell (HSC) transplantation represents an alternative approach but requires the collection of significant numbers of cells. Severe adverse events have been observed in SCD patients after mobilization using the standard agent granulocyte colony-stimulating factor (G-CSF), and bone marrow harvesting is also associated with side effects. The use of a single administration of plerixafor has been suggested as an alternative mobilization strategy in SCD but may not mobilize sufficient number of HSC. In this study, our primary objective is to assess if up to two injections of plerixafor (starting dose level: 240 µg/kg/dose) are safe in SCD patients and can recruit enough blood stem cells needed for future gene therapy.
The objective of this study is to investigate if up to two injections of plerixafor represent a safe and effective strategy to mobilize adequate numbers of CD34+ hematopoietic stem progenitor cells (HSPC) for autologous hematopoietic cell transplantation (HCT) in sickle cell disease (SCD) patients