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

项目总结 慢性粒细胞白血病(CML)生物学认识的演变，最终 转化为高效的分子靶向治疗，在癌症医学中是无与伦比的。这 知识导致了许多口服活性分子酪氨酸激酶抑制剂(TKI)的开发 极大地改善了临床结果。在不到二十年的时间里，十年的生存概率 从之前的干扰素疗法的20%增加到53%，到TKI时代达到90%左右。预期寿命 这些患者现在接近普通人群的水平。虽然口腔TKI现在是护理的标准 对于慢性粒细胞白血病，应该考虑到治疗是终身的，要求患者服用药物。 每天一次。重要的是，有令人信服的证据表明，完全坚持治疗是治疗的关键因素 获得并保持对治疗的最佳反应。然而，不遵守是慢性粒细胞白血病的一大挑战， 由于这些药物引起的副作用严重影响了患者的生活质量 破坏了对治疗计划的完全遵守，从而经常导致次优的临床反应 为毒品干杯。从之前的初步数据中，我们推断出以下证据：系统性监测 患者报告的结果(PRO)在常规护理中有几个优点，如改善症状 控制，加强医患沟通，以及患者满意度和幸福感。 此外，研究还发现，不良事件(AEs)是导致不遵守 而且，即使是有经验的医生也倾向于低估患者的TKI负担。 这种不匹配可能会对疾病管理产生重大的临床影响，而医生可能不会。 能够及早识别那些可能存在不良依从性行为的高风险患者。vt.给出 这些发现，我们假设对慢性粒细胞白血病患者报告的AEs进行系统的电子监测 常规治疗可提高对治疗的依从性、生活质量和对治疗的临床反应。这 假设将在以下具体目标的实验中得到解决：1)开发一个在线 用于系统监控患者报告的AE评估的平台，该平台是根据独特需求量身定做的 慢性粒细胞白血病的TKI疗法。2)评估患者和医生对使用本产品的可接受性和满意度 CML常规实践平台，并评估其在改善症状管理、生活质量、 坚持治疗以及初步疗效。我们预计这项研究将提供前所未有的 系统收集患者报告的不良事件信息在慢性粒细胞白血病常规护理中的价值的信息。 如果阳性，我们的结果将为大型国际随机对照试验(RCT)的发展提供参考。 目的：研究这种实验方法是否可以改善TKI的临床反应深度和比率 慢性粒细胞白血病患者的治疗。

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.