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的国际领导者，直接改善间质性肺病患者的生活。我目前是加州大学旧金山分校(UCSF)的副教授，对我作为一名临床研究人员的成功有着强有力的机构承诺；我发展了一项由NHLBI资助的独立研究生涯；我建立了一个多产和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病领域的一名受欢迎的导师，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)希望与我合作。我相信我正处于我职业生涯中获得K24奖项的理想阶段。在K24的支持下，我计划将大量的精力投入到导师和导师相关的研究活动中。我对导师和学员发展的方法基于三个核心组成部分：为学员确立现实的目标；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，以确保学员利用这些机会、知识和技能向目标迈进。重要的是，我根据每个学员的目标和经验水平定制了学员发展计划。加州大学旧金山分校是美国领先的学术医疗中心之一，它拥有广泛的基础设施来支持以患者为导向的研究。加州大学旧金山分校的机构支持基础包括长期的K30临床研究课程奖(1999年至今)和临床与翻译科学奖(CTSA，2006年至今)。K30和CTSA奖项共同提供基本的基础设施、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、重症监护、睡眠和过敏科以及加州大学旧金山分校医学部完全致力于我的研究事业发展，并支持以患者为导向的研究人员的指导。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。第一个涉及一种在特发性肺纤维化(IPF)临床试验中测量结果的新方法--家庭监护的使用。第二部分扩展了我们对特发性肺间质纤维化风险预测的研究，探讨了血液生物标志物对临床风险预测模型的附加价值。项目1利用一种新的方法(基于家庭的评估)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自然历史的理解。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很好的耐受性、重复性，并与基于办公室的测量具有很好的相关性。此外，我预计基于家庭的评估将比基于办公室的评估更快地确定疾病的潜在急性恶化，并表明许多IPF患者有亚临床发作的急性恶化。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，着眼于疾病活动和进展的重要死亡前衡量标准。将以血液为基础的生物标志物的基线值添加到基线临床风险预测模型(GAP模型)中，并确定相加预测值。我预计基于血液的生物标志物的加入将显著改善GAP模型的预测性能。这将为临床研究人员(以及潜在的临床医生)提供一个重要的工具，帮助他们了解个别患者在接下来的12个月里可能经历的情况。

 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.