Prospective Study of Clinically Non-functioning Pituitary Adenomas

项目描述（由申请人提供）：本项目将开展临床无功能垂体腺瘤（CNFA）的首个综合性前瞻性研究。目前对这些垂体腺瘤患者的评估、治疗和随访的临床实践是基于回顾性收集的数据，这些数据有许多不足之处。因此，我们在如何优化这些实践方面的知识存在重大差距。改善我们治疗这些患者的方法的一个主要障碍是缺乏前瞻性收集的证据来支持或改变它。我们将克服这一障碍，回答有关CNFA护理的重要未决问题。该项目的目的1是前瞻性研究无症状垂体病变，不需要手术干预，以确定适当的初步评估和随访，以及保守，非手术治疗的安全性。该项目的目的2是前瞻性评估有症状的CNFAs的手术治疗结果，手术是首选的初始治疗方法。手术后复发是常见的，但不能可靠地预测，因此我们将研究肿瘤再生长的潜在危险因素，特别是“沉默”的皮质性肿瘤类型。我们还将评估术后小肿瘤残留患者保守随访的安全性，并通过检查残留/复发肿瘤术后RT的风险与收益来确定哪些患者需要RT。该项目的目的3是首次前瞻性地研究该疾病和我们的治疗对CNFAs患者的生活质量和神经认知功能的影响。该项目的目标4将是建立一个新的垂体肿瘤标本库，这将与我们前瞻性研究中收集的广泛临床数据相关联。该信息库将为未来肿瘤标志物及其临床意义的合作研究提供宝贵的资源。我们的调查团队拥有内分泌学、神经外科、认知神经科学和病理学方面的专业知识，他们有能力承担这项研究。大量的病人转介给我们的团队，使我们有条件进行这项研究。该项目建立了一个庞大而独特的前瞻性随访CNFA患者队列，并创建了一个与临床数据相关的垂体肿瘤库，从而进一步加强了该项目。本项目将为临床无功能垂体腺瘤患者的评估、治疗和管理提供新的数据。

DESCRIPTION (provided by applicant): This project will conduct the first comprehensive prospective study of clinically non-functioning pituitary adenomas (CNFA). Current clinical practices for the evaluation, treatment and follow up of patients with these pituitary adenomas are based on retrospectively collected data, which have many shortcomings. As a result, significant gaps exist in our knowledge about how to optimize these practices. A major barrier to improving our approach to these patients has been the lack of prospectively collected evidence to support or change it. We will overcome this barrier and answer the important outstanding questions about CNFA care. Aim 1 of this project is to prospectively study asymptomatic pituitary lesions that do not require surgical intervention in order to determine the appropriate initial evaluation and follow up as well as the safety of their conservative, non-surgical management. Aim 2 of this project is to prospectively assess the outcome of symptomatic CNFAs treated with surgery, the initial treatment of choice. Recurrences after surgery are common, but cannot be reliably predicted so we will study potential risk factors for tumor re-growth in particular the "silent" corticotroph tumor type. We will also assess the safety of conservative follow up for patients with small tumor remnants after surgery and determine in which of these patients RT is needed by examining the risks vs. benefits of post-operatively RT for residual/recurrent tumors. Aim 3 of the project is to examine for the first time, prospectively, the impact of the disease and our therapies on quality of life and neurocognitive function in patients with CNFAs. Aim 4 of the project will be to establish a novel bank of pituitary tumor specimens from our cohort that will be linked to the extensive clinical data collected in our prospective study. This bank will serve as a valuable resource for future collaborative studies of tumor markers and their clinical significance. Our investigative team with expertise in Endocrinology, Neurosurgery, Cognitive Neuroscience and Pathology is well qualified and equipped to undertake this study. The large number of patients referred to the team make us well situated to conduct this study. The project is further strengthened by its establishment of a large and unique prospectively followed cohort of CNFA patients and by its creation of a bank of pituitary tumors that is linked to its clinical data. This project will provide novel data on the evaluation, treatment and management of patients with clinically non-functioning pituitary adenomas. PUBLIC HEALTH RELEVANCE: In this project we will conduct a unique prospective study of a large cohort of patients with clinically non-functioning pituitary tumors. We will investigate important unanswered questions about the evaluation, management and treatment of patients with these tumors. We will investigate the safety of conservative nonsurgical management of patients with asymptomatic pituitary lesions. We will also investigate the most appropriate follow up of patients who have undergone surgery as well as identify patients who are at greatest risk for tumor regrowth after surgery. We will also establish a bank of pituitary tumors from this cohort that can serve as a novel resource for future investigations into the molecular mechanisms of pituitary tumor growth. Our study will lead to important new knowledge about the care of patients with clinically nonfunctioning pituitary tumors.