1. Primary prevention of stroke in children with sickle cell anemia in sub-Saharan Africa: rationale and design of phase III randomized clinical trial
- Author
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Muktar H. Aliyu, Brittany Covert Greene, Aisha Amal Galadanci, Binta J. Wudil, Holly Cassell, Shehu U. Abdullahi, Edwin Trevathan, Safiya Gambo, Hauwa Aminu, Halima Bello-Manga, Muhammed S. Abba, Fenella J. Kirkham, Adetola A. Kassim, Lawal Haliru, Djamila L. Ghafuri, Najibah A. Galadanci, Aisha B. Musa, Musa A. Tabari, Awwal Gambo, Lori C. Jordan, Surayya Sanusi, Awwal Musa Borodo, Kathleen Neville, Aliyu Tijjani Gaya, Michael R. DeBaun, Abdu Hamisu Dambatta, and Mark Rodeghier
- Subjects
Relative risk reduction ,Male ,Pediatrics ,medicine.medical_specialty ,Blood transfusion ,medicine.medical_treatment ,Anemia, Sickle Cell ,Article ,law.invention ,03 medical and health sciences ,0302 clinical medicine ,Randomized controlled trial ,law ,medicine ,Humans ,Stroke ,Africa South of the Sahara ,business.industry ,Hematology ,medicine.disease ,Sickle cell anemia ,Acute chest syndrome ,Clinical trial ,Oncology ,030220 oncology & carcinogenesis ,Pediatrics, Perinatology and Child Health ,Transfusion therapy ,Female ,business ,030215 immunology - Abstract
BACKGROUND: Strokes in children with sickle cell anemia (SCA) are associated with significant morbidity and premature death. Primary stroke prevention in children with SCA involves screening for abnormal transcranial Doppler (TCD) velocity coupled with regular blood transfusion therapy for children with abnormal velocities, for at least one year. However, in Africa, where the majority of children with SCA live, regular blood transfusions are not feasible due to inadequate supply of safe blood, cost, and the reluctance of caregivers to accept transfusion therapy for their children. OBJECTIVES: We describe the Primary Prevention of Stroke in Children with Sickle Cell Disease in Nigeria Trial [Stroke Prevention in Nigeria (SPRING) trial, NCT02560935], a three-center partially-blinded randomized controlled Phase III clinical trial to 1) determine the efficacy of moderate fixed-dose (20 mg/kg/day) versus low fixed-dose (10 mg/kg/day) hydroxyurea therapy for primary stroke prevention; 2) determine the efficacy of moderate fixed-dose hydroxyurea for decreasing the incidence of SCA-related hospitalization (pain, acute chest syndrome, infection, other) compared to low fixed-dose hydroxyurea. DESIGN/METHODS: We will test the primary hypothesis that there will be a 66% relative risk reduction of strokes in children with SCA and abnormal TCD measurements, randomly allocated, for a minimum of three years to receive moderate fixed-dose versus low fixed-dose hydroxyurea (total n=220). RESULTS: The results of this trial will advance the care of children with SCA in Nigeria and sub-Saharan Africa, while improving research capacity for future studies to prevent strokes in children with SCA.
- Published
- 2020