Patient-Oriented Research and Mentoring in Hematopoietic Cell Transplantation

摘要 候选人 Sung Won Choi MD MS，是一位医师兼科学家，在以患者为中心的领域发展了职业生涯 研究（POR）。她是一位活跃的临床儿科肿瘤学家，专注于治疗危及生命的血液 采用新型免疫疗法治疗疾病，例如同种异体造血细胞移植（HCT）。尽管 虽然 HCT 的潜在治疗目的，但其更广泛的应用受到急性移植物抗宿主病的限制 （移植物抗宿主病）。不幸的是，急性 GVHD 会对临床和健康相关的生活质量 (HRQOL) 产生负面影响 结果并导致大量的发病率和死亡率。 Choi 博士的研究项目专注于设计 通过整合生物学、临床和心理社会相关性来减轻急性 GVHD 的创新临床试验 研究并利用现代移动医疗技术进一步加强护理服务。因此，她的研究 计划为对 POR 感兴趣的早期研究人员提供了独特的培训机会， 并辅以密歇根大学以下优秀资源： (1) NHLBI T32 培训 血液学资助（T32 HL007622）； (2) 密歇根临床与健康研究所 (MICHR) |临床 和转化科学奖（UL1TR002240）； (3) 临床试验支持单位； (4) 公立学校 健康。这种理想的培训环境将使年轻的临床研究人员能够发展并获得以下方面的新技能： 临床试验设计、定性和定量研究方法、沟通和演示技巧、 手稿和拨款写作、专业成长、个人发展计划、团队科学项目、道德 进行研究和保护人类受试者。确实，这种丰富的环境影响了崔博士的 设计严格的 POR 研究、指导下一代 POR 研究人员的总体职业目标，以及 与她的学员一起，为家庭的临床和 HRQOL 结果做出有意义的贡献 护理人员和 HCT 患者通过联合生产活动。在本申请中，Choi 博士提出了教学法 课程和小组讨论以及她目前资助的研究项目中的体验式培训： I/II 期临床试验，测试组蛋白脱乙酰酶 (HDAC) 抑制剂预防急性发作的安全性和有效性 儿童 HCT 中的 GVHD（目标 1）；在一项移动健康随机试验中，检验路线图的有效性 2.0，对 HCT 患者家庭护理人员的积极心理干预（目标 2）。 Choi博士还将开发 通过为未来的全面及时适应性干预生成初步数据来确定新的研究方向 (JITAI) 旨在促进 HCT 患者家庭护理人员的身心 HRQOL（目标 3）。这项新研究 方向是目标 2 的逻辑延伸。因此，这些项目将共同为 Choi 博士的学员提供 在以下方面的独特经验： i) 多学科团队科学； ii) 设计、实施和分析药物 和非药物（社会和行为）干预措施； iii) 早期临床试验设计和随机化 对照试验设计； iii) 产生新的研究思路以及未来资助的初步数据 应用程序。崔博士将扩大她的指导活动并建立专业发展的核心知识。

ABSTRACT The candidate, Sung Won Choi MD MS, is a physician-scientist who has developed a career in patient-oriented research (POR). She is an active clinical pediatric oncologist who focuses on treating life-threatening blood diseases with novel immune-based therapies, such as allogeneic hematopoietic cell transplant (HCT). Despite the potentially curative intent of HCT, its broader application has been limited by acute graft-versus host disease (GVHD). Unfortunately, acute GVHD can negatively impact clinical and health-related quality of life (HRQOL) outcomes and lead to substantial morbidity and mortality. Dr. Choi’s research program has focused on designing innovative clinical trials to mitigate acute GVHD by incorporating biological, clinical, and psychosocial correlative studies and leveraging modern mobile health technologies to further enhance care delivery. Thus, her research program provides a unique training opportunity for early stage investigators interested in POR, which is complemented by the following outstanding resources at the University of Michigan: (1) NHLBI T32 Training Grant in Hematology (T32 HL007622); (2) Michigan Institute for Clinical and Health Research (MICHR) | Clinical and Translational Science Award (UL1TR002240); (3) Clinical Trials Support Unit; and (4) School of Public Health. This ideal training environment will allow young, clinical investigators to develop and gain new skills in clinical trials design, qualitative and quantitative research methods, communication and presentation skills, manuscript and grant writing, professional growth, individual development plans, team science projects, ethical conduct of research, and protection of human subjects. Indeed, this rich environment has influenced Dr. Choi’s overall career goal of designing rigorous POR studies, mentoring the next generation of POR investigators, and together with her mentees, making meaningful contributions to the clinical and HRQOL outcomes of family caregivers and HCT patients through joint productive activity. In this application, Dr. Choi proposes didactic courses and group discussions coupled with experiential training in her currently funded research projects: in a phase I/II clinical trial, to test the safety and efficacy of a histone deacetylase (HDAC) inhibitor to prevent acute GVHD in pediatric HCT (Aim 1); in a mobile health randomized trial, to examine the effectiveness of Roadmap 2.0, a positive psychology intervention in family caregivers of HCT patients (Aim 2). Dr. Choi will also develop new research directions by generating preliminary data for a future, full-scale just-in-time adaptive intervention (JITAI) to promote physical and mental HRQOL in family caregivers of HCT patients (Aim 3). This new research direction is a logical extension of Aim 2. Thus, these projects will collectively provide Dr. Choi’s trainees with unique experiences in: i) multidisciplinary team science; ii) designing, conducting, and analyzing pharmaceutical and non-pharmaceutical (social and behavioral) interventions; iii) early phase clinical trial design and randomized controlled trial design; and iii) and generating new research ideas as well as preliminary data for future grant applications. Dr. Choi will expand her mentoring activities and build core knowledge in professional development.