SILENT INFARCT TRANSFUSION TRIAL

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This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. The goal of this trial is to determine whether blood transfusion therapy limits strokes and/or new or progressive MRI lesions in children with pre-existing silent cerebral infarcts, while secondary objectives examine the effectiveness of this therapy to limit decline of intellectual ability and establish a risk-benefit ratio. In this multicenter randomized study with participants from 22 clinical sites, 110 children from this site will be screened over the course of 3 years. Children, age 6-12 years with sickle cell anemia or beta-thalassemia, identified through the clinical database, will be screened with a brain MRI, complete phenotypic/demographic and quality of life questionnaires, and provide a blood sample for genetic testing. Those whose brain MRIs show evidence of a silent stroke will be examined by transcranial Doppler (TCD) and undergo neurological examination by a neurologist. 10 children whose TCD and neurology exams are within normal limits will have a repeat MRI performed, followed by randomization to receive either monthly blood transfusions (n = 5) or 3 years of observational care. All patients will undergo physical exams, laboratory testing, neurological and neurocognitive testing, 2 educational sessions on sickle cell disease and silent stroke, and risk-benefit analysis. The exit evaluation includes MRI, neurology examination, and cognitive testing, while repeat TCD and cognitive assessment will be done at 12-18 months for safety and interim analysis. GCRC nursing is requested for phlebotomy and red cell preparation, and space is requested to accommodate the children during transfusions. Informatics is asked to create scannable forms for the questionnaire, provide the use of scanning equipment, and create a database for the responses. The GCRC is also requested to cover costs for blood draw and cross-match for each transfusion, along with the packed red cells for transfusion.