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Updated: May 9, 2025

A Precision Medicine Tool for Measurement and Monitoring of Hemoglobin S in Sickle Cell Disease Patients Receiving Transfusion Therapy
Alloimmunization as a barrier to gene therapy in sickle cell disease
Hannah R Abrams1, Nabiha H Saifee2,3, William S Miller4
1Fred Hutchinson Cancer Center, University of Washington, Seattle, Washington, USA.
None:
Alloimmunization is prevalent in patients with sickle cell disease (SCD) and can be a barrier to gene therapy (GT) due to the necessary transfusion support for successful stem cell collection and infusion. We estimate that standard-of-care GT for an adult with SCD will require an average of 35-45 units of red blood cells over a 6-month period. Institutions should actively plan for these transfusion needs and share information to inform national consensus policies on the management of alloimmunization during GT.
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