Patient Oriented Research and Mentoring in Interstitial Lung Disease

 描述（由申请人提供）：我的职业目标是成为以患者为导向的研究领域的国际领导者，直接改善间质性肺疾病患者的生活。我目前是加州大学旧金山分校 (UCSF) 的副教授，机构对我作为一名临床研究人员的成功做出了坚定的承诺；我已经发展了独立的研究生涯，并得到了 NHLBI 的大力资助；我建立了一个富有成效且有影响力的研究小组，拥有大量原创研究出版物和良好的合作记录；我已经成为间质性肺疾病领域广受欢迎的导师，有源源不断的早期研究人员（来自加州大学旧金山分校和世界各地）想要与我一起工作。我相信我正处于职业生涯获得 K24 奖项的理想阶段。在本次K24的支持下，我计划投入大量精力进行导师制和与导师制相关的研究活动。我的指导和学员发展方法基于三个核心组成部分：为学员建立切合实际的目标；确定实现学员目标所需的机会、知识和技能；提供充分的互动、指导和资源，以确保受训者利用这些机会、知识和技能来实现他或她的目标。重要的是，我根据每个学员的目标和经验水平定制我的学员发展计划。加州大学旧金山分校是美国领先的学术医疗中心之一，拥有广泛的基础设施来支持以患者为导向的研究。 UCSF 机构支持的基础包括长期存在的 K30 临床研究课程奖（1999 年至今）和临床与转化科学奖（CTSA，2006 年至今）。 K30 和 CTSA 奖项共同提供必要的基础设施、服务和培训，以支持以患者为导向的研究和指导。加州大学旧金山分校肺科、重症监护、睡眠和过敏科以及加州大学旧金山分校医学系全力支持我的研究职业发展，并支持对以患者为中心的研究人员的指导。该奖项将支持两个新的以患者为导向的研究项目，这两个项目都利用了我现有的 NHLBI 资助的研究项目。第一个涉及特发性肺纤维化（IPF）临床试验中结果测量的新方法——使用家庭监测。第二个扩展了我们对 IPF 风险预测的研究，以研究基于血液的生物标记物对经过验证的临床风险预测模型的附加价值。项目 1 采用一种新颖的方法（家庭评估）来测量 IPF 患者的两个关键结果指标（用力肺活量和呼吸困难），这将加深我们对 IPF 自然史的理解。我预计，基于家庭的用力肺活量和呼吸困难严重程度的测量将具有良好的耐受性、可重复性，并且与基于办公室的测量具有良好的相关性。此外，我预计家庭评​​估能够比办公室评估更快地识别疾病潜在的急性恶化，并证明许多 IPF 患者有急性恶化的亚临床发作。项目 2 以我们小组在 IPF 死亡风险预测领域的开创性工作为基础，研究疾病活动和进展的重要死亡前指标。血液生物标志物的基线值将被添加到基线临床风险预测模型（GAP模型）中并确定附加预测值。我预计基于血液的生物标记物的添加将大大提高 GAP 模型的预测性能。这将为希望了解个体患者在接下来的 12 个月内可能经历什么的临床研究人员（以及潜在的临床医生）提供一个重要的工具。

 DESCRIPTION (provided by applicant): My career goal is to be an international leader in patient oriented research that directly improves the lives of people with interstitial lung disease I am currently an Associate Professor at the University of California San Francisco (UCSF) with a strong institutional commitment to my success as a clinical researcher; I have developed an independent research career that is well funded by the NHLBI; I have established a productive and impactful research group with a large number of original research publications and a strong record of collaboration; and I have become a sought-after mentor in the field of interstitial lung disease with a steady stream of early investigators (from UCSF and around the world) who want to come work with me. I believe I am at an ideal stage in my career for a K24 award. With the support of this K24, I plan to devote substantial effort to mentorship and mentorship-related research activities. My approach to mentorship and mentee development is based around three core components: Establishing realistic goals for the mentee; Identifying the opportunities, knowledge and skills necessary to achieve the mentee's goals; and Providing adequate interaction, direction, and resources to insure that mentee's take advantage of these opportunities, knowledge and skills to progress toward his or her goals. Importantly, I tailor my mentee development program to the goals and experience level of each mentee. UCSF is one of the nation's leading academic medical centers, and it has an extensive infrastructure to support patient oriented research. The foundation for UCSF's institutional support includes a longstanding K30 Clinical Research Curriculum award (1999 to present) and a Clinical and Translational Science Award (CTSA, 2006 to present). Together, the K30 and CTSA awards provide essential infrastructure, services, and training to support patient oriented research and mentorship. The UCSF Division of Pulmonary, Critical Care, Sleep and Allergy, and the UCSF Department of Medicine are fully committed to my research career development and to supporting mentorship of patient oriented researchers. This award will support two new patient oriented research projects, both leveraging my existing NHLBI-funded research projects. The first involves a novel approach to outcome measurement in clinical trials of idiopathic pulmonary fibrosis (IPF) - the use of home monitoring. The second extends our research on risk prediction in IPF to investigate the additive value of blood-based biological markers to a validated clinical risk prediction model. Project 1 utilizes a novel approach (home-based assessment) to measure two key outcome measures in patients with IPF (forced vital capacity and dyspnea) that will refine our understanding of the natural history of IPF. I anticipate that home-based measurement of forced vital capacity and dyspnea severity will be well- tolerated, reproducible, and well-correlated with office-based measurements. Further, I expect home-based assessments to identify potential acute worsenings of disease more quickly than office-based assessments, and demonstrate that many patients with IPF have sub-clinical episodes of acute worsening. Project 2 builds upon seminal work from our group in the field of mortality risk prediction in IPF to look at important pre-mortality measures of disease activity and progression. Baseline values of blood-based biological markers will be added to the baseline clinical risk prediction model (GAP model) and the additive predictive value determined. I anticipate that the addition of blood-based biological markers will improve predictive performance of the GAP model substantially. This will provide an important tool to clinical researchers (and potentially, clinicians) looking to understand what individual patients are likely to experience over the subsequent 12 months.