Patient services: Development of the PedsQL Sickle Cell Disease Module

镰状细胞病儿童健康相关生活质量(HRQL)的测定提供了一个 评估镰状细胞病及其治疗对儿童健康的影响的更广泛的方法和2) 可能会改善向这些儿童提供的护理。成人和儿童镰刀患者HRQL的研究现状 细胞疾病使用了HRQL的通用测量方法，并显示出患有这种疾病的人的HRQL较差 这种病。然而，使用这种通用方法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 镰状细胞病儿童的儿科疾病特异性HRQL问卷，这项先前的工作可能会限制 我们评估这些儿童特有的幸福感的其他方面的能力，例如慢性 疼痛，药物和住院的影响。理想情况下，既是通用的，也是针对疾病的 应用于测量HRQL。PedsQL？是在以下方面使用最广泛的HRQL通用测量 具有与疾病相关的模块的儿童。本提案旨在开发和验证 用于镰状细胞疾病儿童的特定疾病HRQL模块。这背后的假设是 工作是，PedsQL镰刀细胞疾病模块将成为衡量镰刀影响的有效工具 从孩子和家长的角度看细胞疾病和治疗。在这里提出的项目将在 检验这一假说的基础。这项建议的具体目标包括以下四个阶段 指标的发展情况如下：确定代表疾病的特定HRQL项目 镰状细胞病儿童的身体、社会、情绪和认知健康状况 PedsQL镰状细胞疾病模块，包含与儿童相关的HRQL区域 并确定飞行员标准分数和PedsQL的心理测量特性 镰状细胞疾病HRQL模块。该项目将产生一个可靠和有效的测量HRQL的 患有镰状细胞病的儿童，可用于促进结果研究，并提供 在这些儿童的临床试验中衡量治疗效果的替代方法。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。 这份针对疾病的调查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.