This finding is a call to action for worldwide access to this treatment.
Sickle cell disease (SCD) is prevalent (1.5% of live births) in sub-Saharan countries, yet limited research has been done in this region due to lack a basic clinical care. Stroke, either silent or overt, is a major cause of morbidity and mortality in children with SCD and transcranial Doppler (TCD) screening is a standard of care to identify those at risk.
In a prospective, open-label, phase 2 trial researchers addressed whether treatment with hydroxyurea at maximal tolerated dose (MTD) can reduce the risk of stroke in children with SCD in Tanzania. The investigators also focused on building infrastructure to perform TCD for stroke screening at local institutions.
The study enrolled 196 children with SCD aged 2 to 16 years. All participants u…
Reviewing Author
DisclosuresConsultant/Advisory BoardGenentech
Grant/Research SupportX4 Pharma; Pfizer; Health Resources and Services Administration; American Thrombosis and Hemostasis Network/CDC; Carver College of Medicine
Leadership Positions in Professional SocietiesInternational Society on Thrombosis and Haemostasis (Finance Committee Member); American Society of Hematology Clinical Research Translational Institute
DisclosuresConsultant/Advisory BoardGenentech
Grant/Research SupportX4 Pharma; Pfizer; Health Resources and Services Administration; American Thrombosis and Hemostasis Network/CDC; Carver College of Medicine
Leadership Positions in Professional SocietiesInternational Society on Thrombosis and Haemostasis (Finance Committee Member); American Society of Hematology Clinical Research Translational Institute