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Project targets sickle cell disease in Nigeria

Oct. 25, 2012, 10:20 AM

Researchers from the Monroe Carell Jr. Children’s Hospital at Vanderbilt and Aminu Kano Teaching Hospital in Kano, Nigeria, will work on a collaborative project to study a sickle cell disease treatment for children in Nigeria.

The National Institutes of Health (NIH) recently awarded the project a two-year, $281,490 grant (grant No. 1R21NS080639-01). The funding will be used to investigate the effectiveness of the drug hydroxyurea as a primary prevention of stroke among children with sickle cell disease, an inherited blood disorder, in Nigeria. Stroke can be a complication in patients with sickle cell disease. Hydroxyurea is often given to patients with sickle cell disease as part of a maintenance protocol to prevent pain episodes, another complication associated with the disease.

The principal investigators for the study are Michael DeBaun, M.D., MPH, vice chair of Clinical Research for Vanderbilt’s Department of Pediatrics; Lori Jordan, M.D., Ph.D., assistant professor in Neurology, and Muktar Aliyu, MBBS, MPH, Dr.PH, assistant professor of Preventive Medicine in the Vanderbilt Institute for Global Health.

Michael DeBaun, M.D., MPH

The collaboration will also include Bruce Compas, Ph.D., professor of Psychology and Human Development, and Yu Shyr, Ph.D., professor and chief in the Division of Cancer Biostatistics and Department of Biostatistics.

Nigeria has the largest burden of sickle cell disease in the world with an estimated 150,000 people living with the condition. Yet the most common therapy for the disease, recurrent blood transfusions, is not readily available. The newly funded trial could help researchers determine if hydroxyurea would prove to be an alternative treatment to frequent blood transfusion to prevent the occurrence of stroke in children at risk. Sickle cell disease is the most common cause of stroke in children.

This is both the first stroke and sickle cell disease trial supported by the NIH and will act as a precursor to a future multi-center, Phase III trial to determine if hydroxyurea can prevent a strokes in children at high risk with sickle cell disease.

To initiate the planning of the trial the Vanderbilt team recently met with the team from Kano, Nigeria, for two days in London. The first patient is expected to be enrolled in January 2013.

This project serves as an addition to the efforts of the Vanderbilt-Meharry-Matthew Walker Center of Excellence in Sickle Cell Disease, established nearly two years ago to provide comprehensive, high quality, lifelong care in a community-based setting to adults and children with sickle cell disease.

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