Prospective Study of Clinically Non-functioning Pituitary Adenomas

描述（由申请人提供）：该项目将对临床无功能垂体腺瘤（CNFA）进行首次全面的前瞻性研究。目前对垂体腺瘤患者的评估、治疗和随访的临床实践都是基于回顾性收集的数据，存在许多缺陷。因此，我们在如何优化这些实践方面的知识存在重大差距。改进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并回答有关 CNFA 护理的重要悬而未决的问题。该项目的目标 1 是前瞻性研究不需要手术干预的无症状垂体病变，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。该项目的目标 2 是前瞻性评估通过手术（首选治疗）治疗症状性 CNFA 的结果。手术后复发很常见，但无法可靠预测，因此我们将研究肿瘤重新生长的潜在危险因素，特别是“沉默”促肾上腺皮质激素肿瘤类型。我们还将评估手术后有小肿瘤残留的患者保守随访的安全性，并通过检查残留/复发肿瘤术后放疗的风险与益处来确定哪些患者需要放疗。该项目的目标 3 是首次前瞻性地研究该疾病和我们的疗法对 CNFA 患者生活质量和神经认知功能的影响。该项目的目标 4 是从我们的队列中建立一个新的垂体瘤标本库，并将其与我们前瞻性研究中收集的广泛临床数据联系起来。该库将成为未来肿瘤标志物及其临床意义合作研究的宝贵资源。 我们的研究团队拥有内分泌学、神经外科、认知神经科学和病理学方面的专业知识，完全有资格并有能力开展这项研究。转介到该团队的大量患者使我们有条件进行这项研究。该项目通过建立一个大型且独特的 CNFA 患者前瞻性随访队列以及创建与其临床数据相关的垂体肿瘤库而得到进一步加强。该项目将为临床无功能垂体腺瘤患者的评估、治疗和管理提供新数据。 公共健康相关性：在这个项目中，我们将对一大群患有临床无功能垂体瘤的患者进行一项独特的前瞻性研究。我们将调查有关这些肿瘤患者的评估、管理和治疗的重要未解答问题。我们将研究对无症状垂体病变患者进行保守非手术治疗的安全性。我们还将研究对接受手术的患者进行最适当的随访，并确定手术后肿瘤再生风险最大的患者。我们还将建立一个来自该队列的垂体肿瘤库，可以作为未来研究垂体肿瘤生长分子机制的新资源。我们的研究将带来有关临床无功能垂体瘤患者护理的重要新知识。

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.