Disease prevention objectives study in children with sickle cell disease

The investigators for St. Jude Children Research Hospital has recently received a grant of $ 23 million from the federal government to launch a national study of the first drug hydroxyurea to prevent children and adolescents with sickle cell anemia .D., principal investigator of the study, funded by the National Heart, Lung, and Blood Institute. Ware chairs the St. Jude Department of Hematology and holds the Lemuel Diggs Professor of sickle cell anemia.

The effort will be the fifth St. Jude involving hydroxyurea for the treatment of children with SCA. The emphasis will be on SCA patients who have suffered strokes, but have been identified as high risk of complications from transcranial Doppler screening. The five-year study is expected to include a total of 26 medical centers and about 140 patients

The new study is called Twitch, short for TCD transition to transfusions with hydroxyurea. Patients will be randomized to receive either standard therapy to continue with monthly blood transfusions and chelation to remove the accumulation of iron resulting or to receive a daily dose of hydroxyurea and monthly phlebotomy to remove excess iron. The objective is to compare two treatments, including their ability to prevent a first stroke.