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(PRO)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幸福感的衡量，直接由患者报告。正如医学研究所指出的那样，对PRO的衡量和评估有助于以患者为中心的护理，这是提供卫生保健质量的关键因素。在介入试验中使用PROS作为次要结果可以帮助理解干预的有效性，并提供对患者健康方面的洞察。这项拟议的项目旨在确定表现为急性镰状细胞疼痛发作的儿童的健康相关生活质量(HRQL)和短期功能结果，这些儿童被随机接受镁(Mg)或安慰剂治疗，这是该拟议项目的父母试验。这个拟议的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，并且唯一能够利用这一母体临床试验来评估药物治疗对这些专业人员的有效性。该项目的总体目标是确定在急性疼痛危象期间接受静脉注射镁治疗的儿童的HRQL和短期结果。我们假设，与那些接受安慰剂治疗的儿童相比，接受治疗的儿童在完成镁治疗时会有更好的HRQL，在随访中会有更好的短期结果。此外，我们的实验室已经完成了针对镰状细胞疾病的疾病特异性HRQL测量仪器开发的第三阶段。作为一个探索性目标，我们建议确定这一测量的响应性和最小重要差异(MID)，以进一步定义其在父母临床试验环境中的心理测量学特性。我们假设PedsQL镰刀细胞疾病模块将随着时间的推移对儿童健康状况的变化做出反应，并且我们将使用基于锚定和分布的方法成功地利用父临床试验的设计来定义MID。这项拟议的项目将在出现急性镰状细胞疼痛发作的急诊室时、最后一次服用研究药物/安慰剂时、出院后一周和出院后一个月左右评估HRQL。此外，以下短期结果将在出院后一周和一个月进行评估：1)主要照顾者缺课的天数，2)儿童缺课的天数，3)标准疼痛评分(0-10)。

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.