This is a single-arm, mechanistic clinical trial to measure predictors of senescence and the in vivo survival of transfused red blood cells (RBCs) in individuals with sickle cell disease (SCD) receiving chronic transfusion therapy (CTT). Chronic transfusion in patients with SCD is a common treatment. The efficacy of RBC transfusion therapy to treat or prevent complications of SCD may be hampered by variable survival of the transfused donor RBC. The overall aim is to see how long RBC survive in SCD patients who are chronically transfused. When a study participant has a regular blood transfusion the researchers will label a small portion of the RBCs that are transfused with biotin. The participant will return at Day 1, weekly for 3 months and monthly for 3 months to measure how long those RBCs survive.
| Marianne Yee, MD | |
| 404-785-6190 | |
| [email protected] |
> 6 Years
Phase 1
Interventional
All
Drug
HbSS or HbSβ0 thalassemia SCD
receiving CTT for ≥3 months prior to enrollment
anticipated cessation of CTT in the next ≤2 months
concurrent hydroxyurea therapy
automated RBC exchange therapy within 3 months prior to enrollment or anticipated within the next 3 months
delayed hemolytic transfusion reaction in the past 3 months
consuming high-dose biotin or raw egg supplements
current pregnancy