A novel natural history study of medullary thyroid carcinoma: Incorporating the patient perspective to inform advanced disease management

甲状腺髓样癌的新型自然史研究：结合患者的观点为先进的疾病管理提供信息

• 批准号: 10237146
• 负责人: Elizabeth Gardner Grubbs
• 金额: $ 39.86万
• 依托单位: UNIVERSITY OF TX MD ANDERSON CAN CTR
• 依托单位国家: 美国
• 财政年份: 2019
• 资助国家: 美国
• 起止时间: 2019-09-01 至 2023-07-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10237146
• 关键词: novel; natural; history; study; medullary

Abstract Medullary thyroid carcinoma (MTC) is a rare tumor with a current prevalence in the United States of 13,500 Americans. Advanced disease is mortal; patients with extra-cervical metastases have 5- and 10-year survival rates of 26% and 14%, respectively. After decades of limited effective available therapies, the Food and Drug Administration recently approved two new drugs, vandetanib (2011) and cabozantinib (2012), for the treatment of progressive, metastatic MTC; unfortunately, these drugs are not curative, response to therapy is not durable, and individual institutional and investigator experience remains limited due to the rarity of the disease. As a result, expert clinicians are left with unanswered questions regarding optimal drug use, and there continues to be a need for innovative drug research. Quality of life was not definitively studied as a goal of therapy in the trials for either drug, or little is known about how patients with advanced MTC make decisions regarding taking these newly-approved drugs and/or participating in clinical trials. The goal of this research is to describe the natural history of MTC via the utilization of a multi-institutional registry in order to understand the pathway to progressive disease requiring drug therapy, and integrating for the first time the patient perspective in order to identify patient reported outcome measures necessary to consider in current treatment and to include in clinical trial design. This data will inform emerging drug therapies for advanced MTC. This study will gather a comprehensive natural history to fully characterize the disease longitudinally from both the clinical and patient perspectives to identify demographic, clinical, pathologic, and genotypic predictors of progression to advanced MTC requiring approved drugs or enrollment in clinical trial, and patients’ response to such therapy. Additionally, the study will characterize patients’ experience with the progressive stages of MTC and treatment initiation through a systematic, longitudinal evaluation of patient-reported outcomes (PROs) that are relevant to the disease, including quality of life, symptoms and other outcomes.

摘要 甲状腺髓样癌（MTC）是一种罕见的肿瘤，目前在美国的患病率为13，500 美国人晚期疾病是致命的;颈外转移的患者有5年和10年的生存期 分别为26%和14%。经过几十年的有限有效的可用疗法，食品和药物管理局 FDA最近批准了两种新药，vandetanib（2011）和cabozantinib（2012），用于治疗 进行性、转移性MTC;不幸的是，这些药物不能治愈，对治疗的反应不持久， 并且由于该疾病的罕见性，个体机构和研究者的经验仍然有限。作为 结果，专家临床医生留下了关于最佳药物使用的未回答的问题， 需要创新药物研究。生活质量并没有作为治疗的目标进行明确的研究， 对于任何一种药物的临床试验，或者对于晚期MTC患者如何决定服用 这些新批准的药物和/或参与临床试验。这项研究的目的是描述 通过利用多机构登记研究了解MTC的自然史，以了解 需要药物治疗的进展性疾病，并首次整合患者的观点， 确定当前治疗中需要考虑的患者报告结局指标，并纳入临床 试验设计这些数据将为晚期MTC的新兴药物治疗提供信息。 这项研究将收集一个全面的自然史，从两个方面全面描述疾病的纵向特征。 临床和患者的观点，以确定人口统计学，临床，病理和基因型预测因子， 进展为需要批准药物或入组临床试验的晚期MTC，以及患者对 这样的治疗。此外，该研究将描述患者在MTC进展阶段的经历， 通过对患者报告结局（PRO）进行系统性纵向评价， 与疾病有关，包括生活质量、症状和其他结果。