RUNX1 Research Program 6th Annual Scientific Conference and Patient Meeting

RUNX1 研究计划第六届年度科学会议和患者会议

• 批准号: 10539782
• 负责人: Katrin Ericson
• 金额: $ 0.8万
• 依托单位: RUNX1 FOUNDATION
• 依托单位国家: 美国
• 财政年份: 2022
• 资助国家: 美国
• 起止时间: 2022-07-15 至 2023-07-14
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/10539782
• 关键词: Automobile Driving; Basic Science; Cell Therapy; Clinical Research; Collaborations; Communities; Community Participation; Development Plans; Diagnosis; Disease; Education; Epigenetic Process; Ethnic group; Faculty; Familial Platelet Disorder; Family; Family member; Fostering; Functional disorder; Genes; Genetic; Goals; Hematologic Neoplasms; Hematology; Hematopoiesis; Hematopoietic Stem Cell Transplantation; Hemorrhage; Immune System Diseases; Immune system; Immunologist; Immunooncology; Individual; Inflammation; Inflammatory; Knowledge; Learning; Link; Malignant - descriptor; Malignant Neoplasms; Methods; Mutation; National Human Genome Research Institute; Natural History; Online Mendelian Inheritance In Man; Pathogenesis; Patients; Positioning Attribute; Postdoctoral Fellow; Predisposition; RUNX1 gene; Race; Rare Diseases; Research; Research Personnel; Role; Somatic Mutation; Students; Therapeutic Intervention; Woman; base; cancer prevention; data sharing; design; experience; graduate student; leukemia; lifetime risk; loss of function; meetings; member; patient population; premalignant; prevent; prevention clinical trial; programs; research study; symposium; translational scientist; virtual

PROJECT SUMMARY The main goal of the RUNX1 Research Program 6th Annual Scientific Conference and Patient Meeting is to foster collaboration and provide opportunities for knowledge and data sharing among researchers, clinicians, and patients and their families for a rare disease called RUNX1 familial platelet disorder with predisposition to hematologic malignancies (RUNX1-FPD or FPDMM). Individuals with the disorder have a 35-50% lifetime risk of developing a hematologic malignancy. Of those who develop a malignancy, over 50% are diagnosed with AML. Conference participation has grown each year. We had over 182 attendees for the virtual conference in 2021. In previous years, 50% of research attendees were women, 30% of attendees were post-docs or graduate students, and 15% of independent investigators were 5 years or less in their first faculty position. We plan to continue to engage a diverse audience of investigators. RRP is committed to engaging patients, clinicians and/or researchers from underrepresented racial and ethnic groups to broaden participation from these communities at our conference and beyond. The agenda includes research-specific sessions open to all stakeholders as well as a track dedicated to RUNX1-FPD patients and family members. There are some overlapping sessions designed to encourage engagement between the two communities. The objective is to promote and drive patient-engaged research forward, with direct patient input in the development and planning of the sessions via our 23-member Research Guided by Patients Committee (RGPC). The research focused portion of the meeting will bring together experts from the fields of hematology, immunology, oncology, cell therapy and basic science who are committed to uncovering the mechanisms underlying the pathogenesis of RUNX1-FPD with the goal of developing therapeutic interventions that impact both the bleeding issues and most importantly the predisposition to malignancy. The audience will include investigators devoted to studying RUNX1, leukemia progression, clonal hematopoiesis, gene editing, and hematopoietic stem cell transplants. In 2022, we will continue to expand our network by encouraging more immunologists to join and participate. It has become increasingly clear through the NHGRI-sponsored natural history study of RUNX1-FPD that immune dysfunction and inflammatory disorders are common. RUNX1-FPD includes a unique patient population where there may be a nexus between immunology and oncology based entirely on activity of a single master regulator, RUNX1.

项目摘要 RUNX1研究计划的主要目标第六年度科学会议和患者会议是 培养合作，并为研究人员，临床医生之间的知识和数据共享提供机会 患者及其家人患有一种称为Runx1家族血小板疾病的罕见疾病，易于 血液系统恶性肿瘤（RUNX1-FPD或FPDMM）。患有疾病的人终身风险为35-50％ 发展血液学恶性肿瘤。在发生恶性肿瘤的人中，超过50％被诊断出患有 AML。 会议参与每年都在增长。我们在2021年的虚拟会议上有超过182名参与者。 在过去的几年中，有50％的研究人员是女性，30％的与会者是毕业后或研究生 学生和15％的独立调查员处于第一位教职员工职位的5年或更短的时间。我们计划 继续吸引各种各样的调查员。 RRP致力于参与患者，临床医生 和/或来自代表性不足的种族和族裔的研究人员，从这些种族和族裔群体扩大参与 我们会议及以后的社区。 议程包括针对所有利益相关者开放的研究特定会议，以及专门针对的曲目 Runx1-FPD患者和家人。有一些重叠的会议旨在鼓励 两个社区之间的参与。目的是促进和推动患者参与的研究 前进，通过我们的23名成员研究，直接患者在会议的开发和计划中进行了直接的意见 在患者委员会（RGPC）的指导下。 这项研究的重点是会议的一部分将使血液学领域的专家聚集在一起， 免疫学，肿瘤学，细胞疗法和基础科学，致力于发现机制 Runx1-FPD的发病机理的根本目的是开发影响的治疗干预措施 出血问题，最重要的是恶性肿瘤的倾向。观众将包括 研究人员致力于研究Runx1，白血病进展，克隆造血，基因编辑和 造血干细胞移植。在2022年，我们将通过鼓励更多 免疫学家加入并参加。通过NHGRI赞助的自然，它变得越来越清楚 RUNX1-FPD的历史研究很常见，即免疫功能障碍和炎症性疾病很常见。 runx1-fpd 包括一个独特的患者人群，其中可能存在免疫学和基于肿瘤学之间的联系 完全基于单个主调节器Runx1的活动。