Kinetics of Donor Red Blood Cell Survival in Sickle Cell Disease

About the study

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.

Study point of contact

Marianne Yee, MD
404-785-6190
Marianne.Yee@choa.org

Locations

3 United States sites

Age

> 6 Years

Genotypes

HbSS

Phase

Phase 1

Study type

Interventional

Gender

All

Interventions

Drug

participation requirements

HbSS or HbSβ0 thalassemia SCD
receiving CTT for ≥3 months prior to enrollment

participation restrictions

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

Locations

  • Atlanta, Georgia, United States, Hughes Spalding Children's Hospital, 30303 [Recruiting]
  • Atlanta, Georgia, United States, Childrens Healthcare of Atlanta, 30322 [Recruiting]
  • Atlanta, Georgia, United States, Grady Health System, 30322 [Recruiting]
Last updated 2021-11-01