Quality of Life Children With Sickle Cell Disease

描述(由申请人提供)： 这位研究员是一名儿科血液学家，他的长期目标是成为镰状细胞疾病健康结局的独立临床调查员，主要关注与健康相关的生活质量(QOL)研究。该项目的总体目标是，不仅了解常见疾病并发症对儿童健康状况和健康相关生活质量的影响，而且增加健康相关生活质量量表在镰状细胞疾病儿童中的适用性。在血液学、镰状细胞疾病和健康相关生活质量领域的知名研究人员的指导下，应聘者提出了一项针对上述长期目标的量身定制的职业发展计划。拟议的教学课程将提供针对候选人短期目标的理论和基本技能培训，以1)增加生活质量研究领域的内容知识，2)增加与生活质量研究和镰状细胞疾病健康结果研究相关的方法学技能，3)与同事、患者、家庭和公众有效地交流研究想法和结果，以及4)有效地管理候选人的研究生涯。该应用程序建议验证健康相关生活质量的常用通用测量方法，并将其与另一种广泛使用的通用测量方法进行比较。此外，还将检查显性中风儿童的健康相关生活质量，并将其与无症状性中风和无脑梗死者的健康相关生活质量进行比较。将确定家庭因素对儿童健康相关生活质量的影响。对这些儿童的健康相关生活质量的评估将为扩大对治疗结果的评估提供一个至关重要的工具，并有助于精确确定目前使用的治疗方法的效用，如羟基尿素和新的高风险实验性治疗，如替代捐赠者骨髓移植。

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.