Patient-Oriented Research in Skin Diseases

描述（由申请人提供）：本申请为加州大学旧金山分校（UCSF）皮肤科教授，医学博士玛丽-玛格丽特·陈（Mary-Margaret Chren）提出更新面向患者研究的职业中期研究者奖。她的长期职业目标是进行严格的研究，以改善皮肤病患者的健康，并利用她的研究培养一流的皮肤病患者导向研究人员。这一新的奖项将建立在她之前的工作基础上，并完成她近期的职业目标：首先，扩大并完成非黑色素瘤皮肤癌（NMSC）队列研究的下一阶段，这是一项针对NMSC（最常见的恶性肿瘤）治疗的10年比较有效性研究；第二，在这些发现的基础上，开发和实施针对NMSC治疗的靶向多中心临床试验；第三，以这些研究数据为基础，指导临床研究人员开展面向患者的研究；第四，通过在加州大学旧金山分校临床与转化科学研究所（CTSI）的师友发展项目中发挥更大的领导作用，扩大她的指导活动范围。拟议的研究将填补目前关于两种最常见的治疗NMSC的比较疗效的知识空白，手术切除和组织学引导连续切除，或Mohs手术。该研究将采取渐进的发展步骤，设计和实施多中心随机对照试验，切除和莫氏手术治疗原发性面部基底细胞癌（BCC）。这些步骤包括建立研究、临床和利益相关者咨询小组、研究工具的组成、研究程序的起草和程序的试点测试。基础设施建设完成后，1200例原发性面部基底细胞癌患者将随机接受手术切除或莫氏手术。主要预后是5年肿瘤复发；次要结果是与皮肤相关的生活质量。拟议的指导计划包括三个部分：教学课程、“动手”研究和职业发展；随着新奖项的获得，教学选项将与CTSI的高定向课程的综合计划完全整合。Chren博士作为CTSI导师发展计划导师评估计划的负责人，将促进这种增强的整合。研究计划和指导计划共同提供了一个新的五年战略，与NIH和NIAMS追求和应用知识以减轻疾病负担以及培训科学家进行此类研究的使命直接相关。

DESCRIPTION (provided by applicant): This application proposes a renewal of a Midcareer Investigator Award in Patient-Oriented Research for Mary-Margaret Chren, MD, Professor of Dermatology at the University of California, San Francisco (UCSF). Dr. Chren's long-term career objectives are to perform rigorous research to improve the health of patients with skin diseases, and to use her research to develop first-rate investigators in patient-oriented research in skin diseases. This renewed award will build on her previous work and accomplish her immediate career objectives: first, to expand and complete the next phase of the Nonmelanoma Skin Cancer (NMSC) Cohort Study, a 10-year comparative effectiveness study of treatments for NMSC (the most common malignancy); second, to build on these findings to develop and perform a targeted multicenter clinical trial of treatments for NMSC; third, to use these research data as a substrate for the mentoring of clinician investigators in patient-oriented research; and fourth, to increase the scope of her mentoring activity by serving an expanded leadership role with the Mentorship Development Program of the UCSF Clinical & Translational Science Institute (CTSI). The proposed research will fill a gap in current knowledge about the comparative efficacy of the two most common treatments for NMSC, surgical excision and histologically-guided serial excision, or Mohs surgery. The research will undertake the incremental developmental steps to design and conduct a multicenter randomized controlled trial of excision and Mohs surgery for primary facial basal cell carcinomas (BCC). These steps include establishment of research, clinical, and stakeholder advisory groups, composition of study tools, drafting of study procedures, and pilot-testing of procedures. After infrastructure development, 1200 patients with primary facial BCC will be randomized to surgical excision or Mohs surgery. The primary outcome is tumor recurrence at five years; secondary outcome is skin-related quality of life. The proposed mentoring plan has three parts: didactic curricula, "hands-on" research, and career development; with the renewed award, didactic options will be fully integrated with CTSI's comprehensive program of highly-directed courses. This enhanced integration will be fostered by Dr. Chren's role as leader of the Mentor Evaluation Plan for the CTSI's Mentorship Development Program. Together, the Research Plan and Mentoring Plan provide a new five-year strategy that is directly relevant to the NIH and NIAMS missions of pursuing and applying knowledge to reduce disease burden, and training scientists to carry out such research.