Quality of Life Children With Sickle Cell Disease

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DESCRIPTION (provided by applicant): This investigator is a pediatric hematologist whose long-term objective is to become an independent clinical investigator in health outcomes in sickle cell disease with a primary focus on the study of health-related quality of life (QOL). The overall goal of this project is, not only to understand the impact of common disease complications on the health status and health-related QOL of the child, but also to increase the applicability of health-related QOL instruments in children with sickle cell disease. Under the mentorship of well established investigators in the fields of hematology, sickle cell disease, and health-related QOL, the candidate proposes a tailored career development program that addresses the long-term objective above. The proposed didactic coursework will provide training in theory and fundamental skills specific to the candidate's short-term goals to 1) increase content knowledge in the QOL research field, 2) increase pertinent methodological skills related to QOL research and the study of health outcomes in sickle cell disease, 3) effectively communicate research ideas and findings to colleagues, patients, families and lay public, and 4) effectively manage the candidate's research career. This application proposes to validate a commonly used generic measure of health-related QOL and compare it to another widely used generic measure. In addition, the health-related QOL of children with overt stroke will be examined and compared to the health-related QOL of those with silent stroke and no cerebral infarction. The impact of family factors on the health-related QOL of the child will be determined. The assessment of health-related quality of life for these children will provide a critically important tool for the expanded assessment of treatment outcomes and contribute to a refined determination of the utilities of currently used therapies such as hydroxyurea and new, high-risk experimental treatment, such as alternative donor bone marrow transplantation.