Evaluating Patient-Reported Outcomes Monitoring in Routine Care of Patients with Chronic Myeloid Leukemia for Increasing Adherence and Clinical Response to THerapY: TheEMPATHY Pilot Study

评估慢性粒细胞白血病患者日常护理中患者报告的结果监测，以提高治疗的依从性和临床反应：EMPATHY 试点研究

• 批准号: 9982264
• 负责人: DAVID CELLA
• 金额: $ 16.14万
• 依托单位: NORTHWESTERN UNIVERSITY AT CHICAGO
• 依托单位国家: 美国
• 财政年份: 2019
• 资助国家: 美国
• 起止时间: 2019-08-01 至 2022-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/9982264
• 关键词: Address; Adherence; Adult; Adverse effects; Adverse event; Antineoplastic Agents; Biology; Caring; Chronic Myeloid Leukemia; Client satisfaction; Clinic; Clinical; Clinical Management; Clinical Trials; Collection; Common Terminology Criteria for Adverse Events; Communication; Coupled; Data; Development; Disease Management; Dose; European Organization for Research and Treatment of Cancer; Evolution; Exposure to; General Population; Goals; Hematological Disease; Interferons; International; Intervention; Italy; Knowledge; Lead; Libraries; Life Expectancy; Malignant Neoplasms; Medicine; Modernization; Monitor; Oral; Outcome; Patient Care; Patient Monitoring; Patient Outcomes Assessments; Patient Self-Report; Patient-Focused Outcomes; Patients; Personal Satisfaction; Pharmaceutical Preparations; Physicians; Pilot Projects; Probability; Provider; Quality of life; Randomized Controlled Trials; Reporting; Risk; Symptoms; Tablets; Testing; Time; Translating; Treatment Effectiveness; Treatment Protocols; Treatment outcome; Tyrosine Kinase Inhibitor; Universities; Work; base; behavioral adherence; chemotherapy; cost; experience; experimental study; health care delivery; health care service utilization; health related quality of life; improved; molecular targeted therapies; negative affect; novel; patient-clinician communication; pill; programs; response; routine care; routine practice; satisfaction; secondary outcome; side effect; standard of care; symptom management; symptomatic improvement; therapy development; treatment organization; treatment site

PROJECT SUMMARY The evolution in the understanding of the biology of Chronic Myeloid Leukemia (CML), that eventually translated into highly effective molecular targeted therapies, is unparalleled in cancer medicine. This knowledge led the development of a number of orally active molecule tyrosine kinase inhibitors (TKIs) that have dramatically improved clinical outcomes. In less than two decades, the 10-year survival probability increased from 20 to 53% with previous (IFN)-therapies to about 90% in the TKI era. Life expectancy of these patients now approaches that of the general population. While oral TKIs are now the standard of care for CML, it should be considered that therapy is lifelong and patients are requested to take medication on a daily basis. Importantly, there is convincing evidence that full adherence to therapy is a critical factor to obtain and maintain an optimal response to therapy. However, non-adherence is a major challenge in CML, since side effects induced by these drugs negatively impact on patient's quality of life (QoL), seriously undermine full adherence with treatment schedule and thereby often lead to sub-optimal clinical responses to drugs. From previous Preliminary Data we extrapolated the following evidences: Systematic monitoring of Patient-Reported Outcomes (PRO) in routine care has several advantages, such as improved symptom control, enhanced patient-physician communication, as well as patient satisfaction and wellbeing. Furthermore, it was found that Adverse Events (AEs) are the most frequent cause for non-adherence to CML therapy and that even experienced physicians tend to underestimate burden of TKIs of their patients. This mismatch might have major clinical implications in disease management, as physicians might not be able to early identify those patients who might be at heightened risk of poor adherence behavior. Given these findings, we hypothesized that systematic electronic monitoring of Patient-Reported AEs in CML routine practice may improve adherence to therapy, quality of life, and clinical response to therapy. This hypothesis will be addressed in the experiments of the following Specific Aims: 1) To develop an online platform for systematic monitoring of patient-reported AE assessment that is tailored to the unique demands of TKI therapy for CML. 2) To assess patient and physician acceptability and satisfaction with use of this platform in CML routine practice and evaluate its value in improving symptom management, quality of life, adherence to therapy as well as preliminary efficacy. We anticipate this study will provide unprecedented information on the value of systematically collecting patient-reported AEs information in CML routine care. If positive, our results will inform the development of large international randomized controlled trial (RCT) to investigate whether this experimental approach can improve depth and rates of clinical responses to TKI therapies in CML patients.

项目摘要 理解慢性髓样白血病（CML）生物学的发展，最终 转化为高效的分子靶向疗法，在癌症医学中是无与伦比的。这 知识领导了许多口服活性分子酪氨酸激酶抑制剂（TKI）的发展 已经大大改善了临床结果。在不到二十年的时间里，10年的生存概率 在以前的（IFN）涉及的情况下，将20％增加到53％，达到TKI时代的约90％。预期寿命 这些患者现在接近普通人群。而口服TKI现在是护理标准 对于CML，应认为治疗是终生的，并要求患者服用药物 每天。重要的是，有令人信服的证据表明，完全遵守治疗是 获得并保持对治疗的最佳反应。但是，不遵守是CML的主要挑战， 由于这些药物引起的副作用对患者的生活质量产生负面影响（QOL），因此认真 破坏了完全遵守治疗时间表，从而经常导致次级最佳临床反应 吸毒。从以前的初步数据中，我们推断了以下证据：系统监控 常规护理中患者报告的结果（PRO）具有多个优点，例如改善症状 控制，增强的患者 - 医学沟通以及患者的满意度和福祉。 此外，发现不良事件（AES）是不遵守的最常见原因 CML疗法，即使经验丰富的医生也倾向于低估其患者的TKI负担。 这种不匹配可能对疾病管理具有重大临床影响，因为医生可能不是 能够尽早确定那些可能处于依从性行为差的风险增加的患者。给出 这些发现，我们假设对CML中患者报告的AES的系统电子监测 常规实践可以提高对治疗，生活质量和对治疗的临床反应的依从性。这 假设将在以下具体目的的实验中解决：1）在线开发 针对独特需求量身定制的患者报告的AE评估的系统监控平台 TKI治疗CML。 2）评估患者和医师的可接受性和满意度 CML常规实践中的平台并评估其在改善症状管理，生活质量的价值， 遵守治疗以及初步疗效。我们预计这项研究将提供前所未有的 有关系统收集患者报告的AES信息在CML常规护理中的信息的信息。 如果积极，我们的结果将为大型国际随机对照试验（RCT）的发展提供信息 研究这种实验方法是否可以改善对TKI的深度和临床反应率 CML患者的疗法。