Patient-Oriented Research and Mentoring in Hematopoietic Cell Transplantation

摘要 候选人Sung Won Choi医学硕士是一名内科科学家，他的职业生涯是以病人为导向的 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。 新的免疫疗法，如异基因造血细胞移植(HCT)。尽管 Hct的潜在治疗意图，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，急性移植物抗宿主病会对临床和健康相关的生活质量(HRQOL)产生负面影响 这会造成严重的后果，并导致大量发病率和死亡率。崔博士的研究项目专注于设计 通过结合生物学、临床和心理社会相关性来缓解急性移植物抗宿主病的创新临床试验 研究和利用现代移动保健技术，以进一步加强保健服务。因此，她的研究 该计划为对POR感兴趣的早期调查人员提供了独特的培训机会，这是 与密歇根大学的下列突出资源相辅相成：(1)NHLBI T32培训 血液学补助金(T32 HL007622)；(2)密歇根临床与健康研究所(MICHR)|临床 和翻译科学奖(UL1TR002240)；(3)临床试验支持单位；(4)公共学院 健康。这种理想的培训环境将允许年轻的临床研究人员发展和获得新的技能 临床试验设计，定性和定量研究方法，沟通和陈述技能， 手稿和资助写作，职业成长，个人发展计划，团队科学项目，伦理 进行研究，并保护人类受试者。事实上，这种丰富的环境已经影响了崔博士的 总体职业目标是设计严格的POR研究，指导下一代POR调查人员，以及 与她的导师一起，为家庭的临床和HRQOL结果做出有意义的贡献 通过联合生产活动，帮助照顾者和高血球移植患者。在这份申请中，崔博士建议进行说教 课程和小组讨论，加上她目前资助的研究项目的体验式培训：在 I/II期临床试验，测试组蛋白脱乙酰酶(HDAC)抑制剂的安全性和有效性，以防止急性 儿童HCT中的GVHD(目标1)；在流动健康随机试验中，检查Roadmap的有效性 2.0，对HCT患者家庭照顾者的积极心理干预(目标2)。崔医生也会发展成 通过为未来的全面适时适应性干预生成初步数据来实现新的研究方向 (吉泰)促进HCT患者家庭照顾者的身心HRQOL(目标3)。这项新研究 方向是目标2的合理延伸。因此，这些项目将共同为崔博士的受训人员提供 在以下方面有独特的经验：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；iii)早期临床试验设计和随机 受控试验设计；以及iii)产生新的研究思路以及未来拨款的初步数据 申请。崔博士将扩大她的指导活动，并建立专业发展方面的核心知识。

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.