Design and Analysis of Palliative Care Trials Evaluating Early Interventions

评估早期干预的姑息治疗试验的设计和分析

• 批准号: 8858688
• 负责人: Zhigang Li
• 金额: $ 7.86万
• 依托单位: DARTMOUTH COLLEGE
• 依托单位国家: 美国
• 财政年份: 2014
• 资助国家: 美国
• 起止时间: 2014-06-03 至 2016-05-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/8858688
• 关键词: Accounting; Address; Adoption; Advanced Malignant Neoplasm; Affect; Caring; Cessation of life; Clinical Trials; Complex; Data; Data Set; Dependence; Early Intervention; Epidemiology; Evaluation; Future; Goals; Grant; Health; Hospitals; Institution; Intervention; Intervention Studies; Joints; Life; Measurement; Methodology; Methods; Modeling; Outcome; Pain; Pain Disorder; Pain Research; Pain quality; Palliative Care; Patients; Phase; Procedures; Process; Quality of life; Randomized; Reporting; Request for Proposals; Research Design; Research Personnel; Sample Size; Standardization; Statistical Methods; Supportive care; Symptoms; Techniques; Terminal Disease; Time; Waiting Lists; arm; base; depressive symptoms; design; end of life; improved; innovation; insight; joint function; novel; prevent; primary outcome; programs; research study; standard care; time interval; treatment effect; trial design

DESCRIPTION (provided by applicant): Palliative care focuses on helping relieve pain and prevent the suffering of patients, especially those patients facing advanced cancer and other life-threatening illnesses. More than 80% of large U.S. hospitals have or currently are developing palliative care programs. Although the main goal of palliative care is to improve patient outcomes such as pain and quality of life (QoL), studies have demonstrated that palliative care may also affect survival. Evaluation and improvement of palliative care programs is a primary goal for these research studies. To accomplish this goal, new statistical methods are needed, ranging from improved definition of processes and interventions to standardization of outcomes and improved statistical analysis. In this project, we propose to develop novel methods for the design and analysis of palliative care research studies that have the potential to increase scientific rigor and to fully utilize the valuable data that these trials generate. One of the major statistical issues raised in palliative care studies regards the interplay and tradeoffs between the quality and duration of life, commonly used as primary outcomes. It has been widely noted that patient outcomes worsen at the end of life for patients with advanced cancer. This suggests dependence between survival and longitudinal measurements of patient outcomes. Palliative care intervention studies have reinforced this hypothesis by showing gains in survival and QoL and symptoms including pain. However, this dependence has not been taken into account in analyzing or designing palliative care clinical trials. We have recently developed a method for jointly modeling longitudinal outcomes and survival in palliative care studies using a novel terminal decline model (TDM) where outcomes are modeled in terms of time from death on a retrospective time scale. Randomized "early intervention" or "fast-track" trials (also sometimes called delayed intervention, delayed-start, or wait list designs) have been proposed as an alternative to traditional RCTs comparing palliative care interventions to non-palliative or other supportive care groups. However, many issues remain with respect to implementing such designs. We propose to study this trial design and more general "stepped wedge" designs in the context of TDMs. Specifically, we will address methods for estimating treatment effects and for choosing optimal design features such as overall sample size and the optimal timing of interventions..

描述（由申请人提供）：姑息治疗的重点是帮助减轻疼痛，防止患者的痛苦，特别是那些面临晚期癌症和其他危及生命的疾病的患者。超过80%的美国大型医院已经或正在开发姑息治疗项目。虽然姑息治疗的主要目标是改善患者的结局，如疼痛和生活质量（QoL），但研究表明姑息治疗也可能影响生存率。评估和改善姑息治疗计划是这些研究的主要目标。为了实现这一目标，需要新的统计方法，从改进定义， （c）将各种程序和干预措施纳入成果标准化和改进统计分析。在这个项目中，我们建议开发新的方法来设计和分析姑息治疗研究，这些研究有可能增加科学的严谨性，并充分利用这些试验所提供的有价值的数据。 生成.姑息治疗研究中提出的主要统计问题之一是生命质量和持续时间之间的相互作用和权衡，通常用作主要结局。人们广泛注意到，晚期癌症患者在生命结束时的患者结局会恶化。这表明生存率和患者结局的纵向测量之间存在依赖性。姑息治疗干预研究通过显示生存率和生活质量以及包括疼痛在内的症状的改善，强化了这一假设。然而，在分析或设计姑息治疗临床试验时没有考虑到这种依赖性。我们最近开发了一种方法，用于联合建模的纵向结果和生存的姑息治疗研究中使用一种新的终端下降模型（TDM），结果是从死亡的时间在回顾性时间尺度上建模。 随机"早期干预"或"快速通道"试验（有时也称为延迟干预、延迟开始或 等待名单设计）已被提议作为传统RCT的替代方案，将姑息治疗干预与非姑息治疗或其他支持性治疗组进行比较。然而，关于实现这样的设计仍然存在许多问题。我们建议在TDM的背景下研究这种试验设计和更一般的"阶梯楔"设计。具体来说，我们将讨论估计治疗效果和选择最佳设计特征，如总样本量和最佳干预时机的方法。

项目成果

Assessing Preparatory Grief in Advanced Cancer Patients as an Independent Predictor of Distress in an American Population.

评估晚期癌症患者的预备性悲伤作为美国人群痛苦的独立预测因子。

• DOI: 10.1089/jpm.2016.0136
• 发表时间: 2017
• 期刊: Journal of palliative medicine
• 影响因子: 2.8
• 作者: Vergo,MaxwellT;Whyman,Jeremy;Li,Zhigang;Kestel,Jeanne;James,SpencerL;Rector,Christopher;Salsman,JohnM
• 通讯作者: Salsman,JohnM