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Project Details

E.W. "Al" Thrasher

Status: Funded - Open


Hydroxyurea for stroke prevention in children with Sickle Cell Disease in Sub-Saharan Africa

Michael DeBaun, M.D., MPH

Summary

BACKGROUND: Approximately 150,000 children are born each year with sickle cell disease (SCD) in Nigeria, the largest burden of SCD in the world.1 Regular blood transfusion therapy remains the most effective way of preventing recurrent stroke; however, the therapy is unaffordable to most families in Africa 2-4 and is not routinely available.

GAP: The critical unanswered question is: “what HU dose maximizes therapeutic benefit in secondary stroke prevention, while minimizing toxicity in a region of the world with high rates of malaria and life threatening bacteremia?”

HYPOTHESIS: Primary Hypothesis: Moderate dose HU therapy (20 mg/kg/day) results in 80% relative risk reduction when compared to low dose HU therapy (10 mg/kg/day) for secondary stroke prevention among children 1 – 18 years of age with SCA and acute overt stroke. Secondary Hypothesis: Moderate dose HU therapy will significantly decrease the rate of all-cause hospitalizations when compared to low dose HU therapy.

METHODS: This trial is a randomized controlled trial in which participants with a new onset acute overt stroke (within 1 month of screening) will be allocated to receive moderate or low dose HU therapy in a ratio of 1:1 with follow-up for at least 24 months per participant.

RESULTS: Pending.

IMPACT: Completion of the trial will provide a targeted strategy for secondary stroke prevention in regions of the world where regular blood transfusion therapy is not routinely available, and children with strokes are left with no reasonable alternative for prevention.

Keywords:
Sickle Cell Disease, Stroke, Treatment, Human










Supervising Institution:
Vanderbilt University Medical Center

Project Location:
United States, Nigeria

Award Amount:
$375,000

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