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

镁治疗急性镰状细胞疼痛发作后的生活质量

• 批准号: 8078458
• 负责人: Julie A Panepinto
• 金额: $ 34.51万
• 依托单位: MEDICAL COLLEGE OF WISCONSIN
• 依托单位国家: 美国
• 财政年份: 2011
• 资助国家: 美国
• 起止时间: 2011-01-18 至 2013-11-30
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8078458
• 关键词: Absenteeism at work; Accident and Emergency department; Acute; Adult; Aftercare; Caregivers; Caring; Child; Child Care; Child health care; Clinical; Clinical Trials; Complement; Complication; Data; Development; Device or Instrument Development; Disease; Dose; Effectiveness; Effectiveness of Interventions; Evaluation; Event; Future; Goals; Health; Health Personnel; Health Status; Hospitals; Institute of Medicine (U.S.); Intravenous; Knowledge; Laboratories; Length of Stay; Magnesium; Magnesium Sulfate; Measurement; Measures; Medical; Methodology; Methods; Morbidity - disease rate; Nature; Outcome; Pain; Parents; Patient Outcomes Assessments; Patient-Centered Care; Patients; Pharmaceutical Preparations; Pharmacotherapy; Phase; Physicians; Placebos; Property; Protocols documentation; Psychometrics; Quality of life; Randomized; Randomized Clinical Trials; Randomized Controlled Trials; Reporting; Resources; Risk; Schools; Sickle Cell; Sickle Cell Anemia; Therapeutic Intervention; Time; Treatment Efficacy; Work; abstracting; arm; base; child well being; design; effectiveness measure; experience; follow-up; functional outcomes; health care quality; health related quality of life; improved; innovation; insight; mortality; response; secondary outcome; sound; tumor

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)

描述（由申请人提供）：人们越来越认识到患者报告的结果（PRO）是衡量拟议疗法有效性的重要结果。 PRO 是从患者的角度对患者健康状况和幸福感的衡量，由患者直接报告。 PRO 的测量和评估有助于以患者为中心的护理，正如医学研究所指出的那样，这是提供医疗保健质量的关键要素。 在干预试验中利用 PRO 作为次要结果可以帮助了解干预的有效性并深入了解患者健康状况的各个方面。该拟议项目旨在确定患有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，这是该项目的母试验拟议的项目。该拟议项目建立在我们之前在 HRQL 和短期功能结果方面的工作的基础上，并且能够独特地利用该母体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在急性疼痛危机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，与接受安慰剂的儿童相比，治疗组中的儿童在完成镁治疗后将具有更好的 HRQL，并且在随访中具有更好的短期结果。 此外，我们的实验室还完成了针对镰状细胞病的疾病特异性 HRQL 测量仪器开发的第 3 阶段。我们建议作为一个探索性目标来确定该措施的反应性和最小重要差异（MID），以进一步定义其在母体临床试验中的心理测量特性。我们假设 PedsQL 镰状细胞病模块将对儿童健康状况随时间的变化做出响应，并且我们将成功地利用母体临床试验的设计，使用基于锚点和基于分布的方法来定义 MID。 该拟议项目将评估因急性镰状细胞性疼痛发作而被送往急诊室时、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的 HRQL从医院。此外，将在出院后一周和一个月评估以下短期结果：1) 主要看护人缺勤/上学的天数，2) 儿童缺课的天数和 3) 疼痛评分（0 -10) 标准疼痛量表。公共健康相关性：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健康状况有所改善。这将从患者的角度向医疗保健提供者提供有关治疗有效性的信息，并将有助于将来直接护理这些儿童。这是支持以患者为中心的护理的关键一步，而以患者为中心的护理是提供优质医疗保健的关键要素。 （摘要完）