Transfusion utilization during gene therapy for sickle cell disease: A single institution experience

Area of Science:

• Hematology
• Gene Therapy
• Transfusion Medicine

Background:

• Gene therapy (GT) is a key treatment for sickle cell disease (SCD).
• GT necessitates red blood cell (RBC) transfusions to suppress hemoglobin S for hematopoietic progenitor cell (HPC) collection and transplantation.
• RBC alloimmunization is common in SCD patients, requiring careful transfusion management during GT.

Purpose of the Study:

• To analyze transfusion utilization patterns in SCD patients undergoing GT.
• To inform transfusion strategies and planning for GT in SCD.

Main Methods:

• Retrospective review of transfusion records for SCD patients receiving GT.
• Categorization of transfusions into three periods: pre-HPC collection, collection to infusion, and post-infusion to independence.

Main Results:

• Nine SCD patients received GT; 44% had pre-existing RBC alloimmunization.
• Median RBC transfusion use was 38 units: 15 units pre-collection, 20 units during collection/infusion, and 6 units post-infusion.
• One patient developed alloimmunization during the pre-HPC collection phase.

Conclusions:

• Transfusion support for SCD patients undergoing GT has specific demands due to HPC mobilization requirements.
• This data aids in planning transfusion strategies and assessing feasibility for patients with alloimmunization.