This proposal is submitted by the Cincinnati Comprehensive Sickle Cell Center, one of the first comprehensive programs in the country. The CCSCC is based in the Division of Hematology/Oncology at Children's Hospital Medical Center and its affiliated institution, the University Hospital of the University of Cincinnati College of Medicine, which provide a full range of clinical services to people affected by sickle cell disease. The proposal contains four interactive basic science projects focused on globin gene regulation (Project 1), erythropoietin receptor gene expression in early hematopoiesis (Project 2), mechanism of dehydration of sickle cells and the therapeutic potential for inhibiting this process (Project 3), and the hydration state and survival in vivo of sickle cells (Project 4). A clinical research project is focused on the treatment of sickle cell vaso-occlusive episodes employing behavioral, pharmacological, and physiological approaches (Project 5). Educational (Project 6) initiatives include the development of a Transition Program for young adults transferring from pediatric to adult medical care, and educational programs from a patient/patient perspective for patients, families, and professionals. These educational activities will be carried out in collaboration with two community agencies, the Sickle Cell Awareness Group and the Sickle Cell Parent and Family Network. The Sickle Cell Research Scholars Program (Project 7) provides an important opportunity to contribute to the development of a new generation of investigators focused on sickle cell disease. Pediatric and Adult Clinical Program (Cores A and B) provide organization of the clinical services for patients and essential support for the basic science and clinical research projects. An Administrative Core (C) provides overall fiscal and programmatic management, and serves as a focal point for Center activities, programs, and communication. The projects and cores in this proposal will be integrated with ongoing, independently funded programs of follow-up of newborn hemoglobinopathy screening, education and counseling, a Hemoglobin Diagnostic Laboratory, and other basic science research projects. With this integrated program, the Cincinnati Comprehensive Sickle Cell Center will contribute to the national effort to optimize care for those affected by sickle cell disease, and ultimately to find a cure for patients with this disorder.
