Measuring Red Blood Cell Lifespan in Adults with Sickle Cell Disease and Other Inherited Blood Disorders Using Biotin Labeling

What this Trial Is About

Researchers are studying how long red blood cells RBCs survive in people with sickle cell disease SCD and other inherited blood disorders, such as thalassemia. The study aims to compare RBC lifespan in those undergoing disease modifying treatments or those who have been cured with bone marrow transplants. This research will help understand how treatments affect RBC survival and the degree of correction needed to reverse symptoms in these conditions. Participants will have their own RBCs labeled with a vitamin called biotin in the lab, which sticks to the RBCs surface. These biotin-labeled RBCs are then infused back into the participants bloodstream. The study will track the lifespan of these cells by drawing smaller blood samples regularly, every 2 to 4 weeks, for up to 20 weeks or until labeled cells are no longer detected. The study includes patients before and after disease modifying therapy or hematopoietic stem cell transplantation HSCT. During the study, participants will undergo physical exams, blood and urine tests, and blood draws to measure the percentage of biotin-labeled RBCs. Researchers will monitor RBC survival over time and relate it to blood markers like reticulocyte count and hemoglobin levels. Participants will be followed for up to 20 weeks, with more frequent blood tests early on. The main outcome is the average number of days biotin-labeled RBCs remain detectable, helping to understand treatment effects on blood cell lifespan.

Determination of Red Cell Survival in Sickle Cell Disease and Other Hemoglobinopathies Using Biotin Labeling