Patient-Oriented Research and Mentoring in Hematopoietic Cell Transplantation

摘要 候选人，Sung Won Choi医学博士和理学硕士，是一名医生和科学家，他在以病人为导向的医疗领域发展了自己的职业生涯。 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 新的基于免疫的疗法，如异基因造血细胞移植（HCT）的疾病。尽管 HCT的潜在治疗目的，其更广泛的应用受到急性移植物抗宿主病的限制 （GVHD）。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。崔博士的研究项目集中在设计 创新的临床试验，以减轻急性GVHD，通过整合生物学，临床和心理社会相关 研究和利用现代移动的卫生技术，以进一步加强保健服务。因此，她的研究 该计划为对POR感兴趣的早期研究人员提供了独特的培训机会， 在密歇根大学的以下优秀资源的补充：（1）NHLBI T32培训 血液学补助金（T32 HL 007622）;（2）密歇根临床和健康研究所（MICHR）|临床 和转化科学奖（UL 1 TR 002240）;（3）临床试验支持单位;以及（4）公共学院 健康这种理想的培训环境将使年轻的临床研究者能够发展和获得新的技能， 临床试验设计，定性和定量研究方法，沟通和表达技巧， 手稿和赠款写作，专业成长，个人发展计划，团队科学项目，道德 开展研究和保护人类受试者。的确，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研究者的总体职业目标，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。在这个应用程序中，崔博士提出了教学 课程和小组讨论，加上她目前资助的研究项目的经验培训：在一个 I/II期临床试验，以测试组蛋白去乙酰化酶（HDAC）抑制剂预防急性 儿科HCT中的GVHD（目标1）;在一项移动的健康随机试验中，检查Roadmap的有效性 2.0，对HCT患者家庭照顾者进行积极心理干预（目的2）。崔博士还将开发 通过为未来的全面及时适应性干预生成初步数据， （吉泰），以提高身体和精神的HCT患者的家庭照顾者的HRQOL（目的3）。这项新研究 方向是目标2的逻辑延伸。因此，这些项目将共同为崔博士的学员提供 独特的经验：i）多学科团队科学; ii）设计，进行和分析药物 和非药物（社会和行为）干预; iii）早期临床试验设计和随机 对照试验设计;及iii）产生新的研究意念及初步数据，以供日后拨款使用 应用.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.