Red Cell Half Life Determination in Patients With and Without Sickle Cell Disease

Summary

Participation Requirements

Description

The mean potential lifespan (MPL) of RBCs can be quantified by either a population or cohort study. Such studies have corroborated the MPL of healthy donor (HD) RBCs to be approximately 115 days while RBCs from subjects with sickle cell disease (SCD) have a much more variable but consistently shorte...

The mean potential lifespan (MPL) of RBCs can be quantified by either a population or cohort study. Such studies have corroborated the MPL of healthy donor (HD) RBCs to be approximately 115 days while RBCs from subjects with sickle cell disease (SCD) have a much more variable but consistently shorter MPL of approximately 32 days. Allogeneic hematopoietic stem cell transplant (HSCT) is utilized as a curative therapy for the treatment of severe SCD resulting in, among other pathophysiological advantages, restoration of a HD RBC phenotype. This study will evaluate the population methodology (biotin-labeled RBCs) to determine MPL in subjects with SCD compared to patients who have successfully undergone allogeneic BMT, subjects with sickle cell trait, and healthy donors without SCD. Data generated will be used to determine the utility of performing a population study of RBC lifespan in gene therapy treated patients to ultimately target the percentage of transferred globin gene needed to reverse SCD.

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