Quality of Life Children With Sickle Cell Disease

描述（由申请人提供）： 该研究者是一名儿科血液学家，其长期目标是成为镰状细胞病健康结局的独立临床研究者，主要关注健康相关生活质量（QOL）研究。该项目的总体目标是，不仅要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，而且要增加健康相关生活质量工具在镰状细胞病儿童中的适用性。在血液学，镰状细胞病和健康相关QOL领域的成熟研究者的指导下，候选人提出了一个量身定制的职业发展计划，以解决上述长期目标。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QOL研究领域的内容知识，2）增加与QOL研究和镰状细胞病健康结果研究相关的方法技能，3）有效地将研究想法和发现传达给同事，患者，家庭和公众，（4）有效地管理候选人的研究生涯。本申请旨在验证一种常用的健康相关QOL通用指标，并将其与另一种广泛使用的通用指标进行比较。此外，还将检查显性卒中儿童的健康相关QOL，并与无症状卒中且无脑梗死儿童的健康相关QOL进行比较。将确定家庭因素对儿童健康相关QOL的影响。对这些儿童健康相关生活质量的评估将为扩大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疗法（如羟基脲）和新的高风险实验性治疗（如替代供体骨髓移植）的效用。

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.