Quality of Life After Treatment with Magnesium for Acute Sickle Cell Pain Episode

描述（由申请人提供）：越来越多的人认识到，患者报告的结果（PROs）是衡量拟议治疗有效性的重要结果。PRO是从患者的角度对患者的健康和福祉进行衡量，并由患者直接报告。正如医学研究所指出的那样，对赞成意见的测量和评价有助于以病人为中心的护理，这是提供保健质量的关键因素。将pro作为介入试验的次要结果有助于理解干预的有效性，并提供对患者健康方面的见解。本项目旨在确定急性镰状细胞性疼痛发作儿童的健康相关生活质量（HRQL）和短期功能结局，这些儿童随机接受镁（Mg）或安慰剂治疗，这是本项目的母试验。该项目建立在我们之前在HRQL和短期功能结果方面的工作基础上，并且能够独特地利用该母临床试验来评估药物治疗对这些pro的有效性。该项目的总体目标是确定急性疼痛危机期间静脉注射镁治疗的儿童的HRQL和短期结果。我们假设治疗组的儿童在完成Mg治疗时的HRQL和随访时的短期结果比接受安慰剂的儿童好。此外，我们的实验室已经完成了镰状细胞病特定疾病HRQL测量仪器开发的第三阶段。我们提出作为一个探索性的目的，以确定该措施的反应性和最小重要差异（MID），以进一步确定其心理测量属性在母体临床试验的设置。我们假设，随着时间的推移，PedsQL镰状细胞疾病模块将对儿童健康状况的变化做出反应，并且我们将利用父临床试验的设计成功地用锚定和基于分布的方法定义MID。这个拟议的项目将评估急性镰状细胞性疼痛出现在急诊室时的HRQL，大约在最后一剂研究药物/安慰剂的时间，出院后一周和出院后一个月。此外，在出院后一周和一个月评估以下短期结果：1)主要看护人旷工/旷课天数，2)儿童旷课天数，3)标准疼痛评分（0-10）。公共卫生相关性：该项目具有独特的潜力，可以证明急性镰状细胞病疼痛治疗后儿童的健康状况得到改善。这将从患者的角度为卫生保健提供者提供有关治疗有效性的信息，并将有助于今后对这些儿童的直接护理。这是支持以患者为中心的护理的关键步骤，这是提供高质量卫生保健的关键因素。（摘要结束）

DESCRIPTION (provided by applicant): It is increasingly recognized that patient reported outcomes (PROs) are important outcomes to measure effectiveness of proposed therapies. A PRO is a measurement of the patient's health and well being from their perspective that is reported directly from the patient. The measurement and evaluation of PROs contribute to patient-centered care which is a key ingredient in provision of health care quality as noted by the Institute of Medicine. Utilizing PROs as a secondary outcome in an interventional trial can aid in understanding the effectiveness of the intervention and provide insight into aspects of a patient's well being. This proposed project seeks to determine the health-related quality of life (HRQL) and short term functional outcomes of children presenting with an acute sickle cell pain episode who are randomized to receive treatment with magnesium (Mg) or placebo, the parent trial to this proposed project. This proposed project builds on our previous work in HRQL and short term functional outcomes and is uniquely able to utilize this parent clinical trial to evaluate the effectiveness of drug therapy on these PROs. The overall goal of this project is to determine the HRQL and short term outcomes of children treated with intravenous magnesium during an acute painful crisis. We hypothesize that children in the treatment arm will have better HRQL at completion of Mg therapy and better short term outcomes at follow-up than those receiving placebo. In addition, our laboratory has completed phase 3 in the instrument development of a disease specific HRQL measure for sickle cell disease. We propose as an exploratory aim to determine the responsiveness and minimally important difference (MID) of this measure to further define its psychometric properties in the setting of the parent clinical trial. We hypothesize that the PedsQL Sickle Cell Disease module will be responsive to change in health status of the child over time and that we will define the MID with an anchor and distribution- based method successfully utilizing the design of the parent clinical trial. This proposed project will assess HRQL at time of presentation to the emergency room with an acute sickle cell pain episode, around the time of the last dose of study medication/placebo, one week post-discharge from the hospital, and one month post-discharge from the hospital. In addition, the following short term outcomes will be assessed one week and one month post-discharge from the hospital: 1) Days of work/school missed by primary caretaker, 2) Days of school missed by child and 3) Pain score (0-10) on standard pain scale. PUBLIC HEALTH RELEVANCE: This project has the unique potential to demonstrate improvement in a child's well being after treatment for acute sickle cell disease pain. This will give health care providers information on the effectiveness of therapy from the patient's perspective and will help direct care of these children in the future. This is a critical step to support patient-centered care, a key ingredient in provision of quality health care. (End of Abstract)