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: 8389606
• 负责人: Julie A Panepinto
• 金额: $ 28.96万
• 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 2015-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8389606
• 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.

描述（由申请人提供）：越来越多地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。专业人士是直接从患者报告的角度来衡量患者的健康和福祉。对以患者为中心的护理的测量和评估，这是医学研究所指出的提供医疗保健质量的关键要素。 在介入试验中利用专业人士作为次要结果可以帮助理解干预的有效性，并洞悉患者健康状况的各个方面。该拟议的项目旨在确定与健康相关的生活质量（HRQL）以及出现急性镰状细胞疼痛发作的儿童的短期功能结果，他们被随机接受镁（MG）或安慰剂，这是该拟议项目的父母试验。这项拟议的项目基于我们先前在HRQL和短期功能结果中的工作，并且具有独特的能力利用该父临床试验来评估药物治疗对这些职业的有效性。该项目的总体目标是确定在急性痛苦危机期间用静脉注射镁治疗的儿童的HRQL和短期结局。我们假设治疗臂中的儿童在完成MG疗法时将具有更好的HRQL，而随访时的短期结局比接受安慰剂的儿童会更好。 此外，我们的实验室已经完成了第3阶段，以开发特定于疾病的HRQL措施针对镰状细胞疾病。我们建议作为探索性目的，以确定该措施的响应能力和最小重要差异（MID），以进一步定义其在父临床试验中的心理测量特性。我们假设PEDSQL镰状细胞疾病模块会随着时间的流逝而响应儿童健康状况的变化，我们将通过锚定和分布的方法成功地使用父母临床试验的设计来定义MID。 该拟议的项目将在急诊室的急性细胞疼痛发作，最后一次剂量的研究药物/安慰剂/安慰剂，医院的分期收取后一周以及医院分期收取后的一个月收费时，将评估HRQL。此外，下一个短期的结果将在医院内一周和一个月的收费后一个月进行评估：1）小学看护人错过的工作/学校，2）儿童错过的学校和3）疼痛得分（0-10）在标准疼痛量表上得分（0-10）。

项目成果

Determining the longitudinal validity and meaningful differences in HRQL of the PedsQL™ Sickle Cell Disease Module.

• DOI: 10.1186/s12955-017-0700-2
• 发表时间: 2017-06-12
• 期刊: Health and quality of life outcomes
• 影响因子: 3.6
• 作者: Panepinto JA;Paul Scott J;Badaki-Makun O;Darbari DS;Chumpitazi CE;Airewele GE;Ellison AM;Smith-Whitley K;Mahajan P;Sarnaik SA;Charles Casper T;Cook LJ;Leonard J;Hulbert ML;Powell EC;Liem RI;Hickey R;Krishnamurti L;Hillery CA;Brousseau DC;Pediatric Emergency Care Applied Research Network (PECARN)
• 通讯作者: Pediatric Emergency Care Applied Research Network (PECARN)