Patient services: Development of the PedsQL Sickle Cell Disease Module

确定镰状细胞病儿童的健康相关生活质量（HRQL）1）提供了一个 更广泛的方法来评估镰状细胞病及其治疗对儿童健康的影响，以及2） 可以改善对这些孩子的照顾。既往对镰状手术成人和儿童HRQL的研究 细胞疾病的研究利用了HRQL的一般措施，并显示患有 这种疾病。然而，使用这种通用措施可能会降低检测特定疾病的能力， 健康问题以及检测临床状态随时间变化的能力。因为没有 儿科疾病特异性HRQL问卷调查镰状细胞病的儿童，这项先前的工作可能会限制 我们有能力评估这些儿童特有的其他方面的福祉，如慢性病， 疼痛、药物作用和住院治疗。理想情况下，通用和疾病特异性工具 应该用来衡量HRQL。PedsQL是最广泛使用的HRQL的通用措施， 有伴随疾病特定模块的儿童。该提案旨在制定和验证一项 疾病特异性HRQL模块，用于镰状细胞病儿童。这背后的假设是 PedsQL镰状细胞病模块将成为衡量镰状细胞病影响的有效工具。 从孩子和父母的角度来看待细胞疾病和治疗。这里提出的项目将奠定 这是检验这一假设的基础。该提案的具体目标包括以下四个阶段： 工具开发，如下所示：确定代表疾病特异性HRQL项目， 镰状细胞病儿童的身体、社会、情感和认知健康， PedsQL镰状细胞病模块，包含发现与儿童相关的HRQL领域 并确定PedsQL的飞行员标准评分和心理测量学特性 镰状细胞病HRQL模块。该项目将导致一个可靠和有效的措施HRQL的 镰状细胞病儿童，可用于促进结果研究，并提供 替代方法来衡量这些儿童的临床试验中的治疗效果。最终目标是 在临床实践中使用这一工具，以改善镰状细胞病儿童的健康和福祉。 这份针对特定疾病的问卷将免费提供给所有学术研究人员。

The determination of health-related quality of life (HRQL) for children with sickle cell disease 1) provides a broader method to assess the impact of sickle cell disease and its treatments on children's health and 2) may improve the care provided to these children. Prior studies of the HRQL of adults and children with sickle cell disease have utilized generic measures of HRQL and shown the poor HRQL of those who suffer from this disease. However, use of such generic measures likely reduce the ability to detect disease-specific health issues as well as the ability to detect changes in clinical status over time. Because there are no pediatric disease-specific HRQL questionnaires for children with sickle cell disease, this prior work may limit our ability to evaluate additional aspects of well being that are specific to these children, such as chronic pain, effect of medications and hospitalizations. Ideally, both a generic and disease-specific instrument should be used to measure HRQL. The PedsQL¿ is the most widely used generic measure of HRQL in children that has companion disease-specific modules. This proposal seeks to develop and validate a disease-specific HRQL module for use in children with sickle cell disease. The underlying hypothesis of this work is that the PedsQL Sickle Cell Disease module will be an effective tool to measure the impact of sickle cell disease and treatment from the perspective of the child and parent. The project proposed here will lay the foundation for testing this hypothesis. The specific aims of this proposal encompass the four phases of isntrument development and are as follows: to identify disease-specific HRQL items representing the physical, social, emotional and cognitive well being of children with sickle cell disease, to construct a PedsQL Sickle Cell Disease Module that incorporates those HRQL areas found to be relevant to children with sickle cell disease and to determine pilot standard scores and psychometric properties for the PedsQL Sickle Cell Disease HRQL module. This project will result in a reliable and valid measure of HRQL of children with sickle cell disease that can be utilized to facilitate outcomes research as well as provide an alternative method to measure the effect of therapies in clinical trials for these children. The ultimate goal is to use this tool in clinical practice to improve the health and well being of children with sickle cell disease. This disease-specific questionnaire will be made available without fees for all academic researchers.