Core B: MPN-RC Tissue Bank

核心B摘要 核心负责人：布里奇特·马塞利诺，医学博士 组织库提供运输、接收、处理、存储和分发组织的基础设施 取自骨髓增生性肿瘤(MPN)患者的纵向组织标本 银行和登记参加临床试验的患者(项目4)。这些标本被提供给MPN-RC 为开发创新疗法而进行转译研究的研究人员(项目1-3)。 它们还被提供给生物标记和生物信息学核心D以执行生物标记分析以评估 耗尽MPN造血的特定疗法的疗效或耐药原因正在评估 干细胞和祖细胞。组织也可供科学界广大成员使用 翻译研究。翻译研究依赖于充足的组织的现成可用性。 准确诊断的患者数量，以生成具有统计意义的数据。因为MPN是 相对少见，它们的准确诊断可能很困难，一直以来都存在组织稀少，与 临床数据，可供单个机构的个人研究人员使用。核心B填补了这一空白。库存有 超过1700名MPN患者的样本，Core B保持着世界上最大的之一 可供研究的MPN组织清单。此外，Core B还收集患者的样本 参加MPN-RC赞助的临床试验(项目4)，目的是进行生物标记物研究 证明特定疗法对MPN造血干祖细胞耗竭的有效性 细胞。组织库核心B的具体目标如下：(1)接收、处理、冷冻保存和存储 来自参加纵向组织库(MPN-RC 106)的MPN患者的组织以及登记的患者 关于MPN-RC临床站点的临床试验(项目4)。(2)为符合以下条件的MPN组织提供中央存储库 与存储在由维护的基于网络的数据库中的临床和突变注释数据相关联 生物统计和数据管理核心C。这种组织的注释、选择和分布是为 每个翻译研究项目。(3)a.将组织分发给MPN-RC调查人员和更大的科学界 翻译研究社区致力于阐明驱动发病机制和 MPN的进展和开发针对骨髓间充质干细胞的新治疗策略。B.分发临床资料 试验样本以分析新制剂的靶向效应并创建生物标记物，以确定 对治疗的治疗和药效学反应(核心D)。核心B是必不可少的共享资源 使转化性研究最终导致针对MPN的疗法的开发 干细胞。

Core B Abstract Core Lead: Bridget Marcellino, MD, PhD Core B, Tissue Bank provides the infrastructure to transport, receive, process, store, and distribute tissue specimens obtained from patients with myeloproliferative neoplasms (MPN) enrolled on the longitudinal tissue bank as well as patients enrolled on clinical trials (Project 4). These specimens are provided to MPN-RC investigators performing translational research for the purpose of developing innovative therapies (Projects 1-3). They are also provided to the Biomarker and Bioinformatics Core D to perform biomarker analyses to evaluate the effectiveness or causes of resistance of specific therapies being evaluated to deplete MPN hematopoietic stem and progenitor cells. Tissues are also available to members of the scientific community at large for translational research. Translational research is dependent on the ready availability of tissues from a sufficient number of accurately diagnosed patients to generate statistically significant data. Because the MPNs are relatively uncommon and their accurate diagnosis can be difficult, there has been a paucity of tissue, linked to clinical data, available to individual investigators at single institutions. Core B fills this void. With an inventory of specimens from more than 1700 individual patients with MPNs, Core B maintains one of the world’s largest inventories of MPN tissues available for research. In addition, Core B collects specimens from patients participating in MPN-RC sponsored clinical trials (Project 4) for the purpose of performing biomarker studies to demonstrate the effectiveness of specific therapies on the depletion of MPN hematopoietic stem and progenitor cells. The specific aims of Tissue Bank Core B are the following: (1) Receive, process, cryopreserve, and store tissues from MPN patients participating in a longitudinal tissue bank (MPN-RC 106) as well as patients enrolled on clinical trials (Project 4) at MPN-RC clinical sites. (2) Provide a central repository for MPN tissues that are associated with clinical and mutational annotation data stored in the web-based data bank maintained by Biostatistics and Data Management Core C. This annotation, selection and distribution of tissues is tailored for each translational research project. (3) a. Distribute tissues to MPN-RC investigators and the larger scientific community for translational research dedicated to elucidating the mechanisms driving the pathogenesis and progression of MPNs and developing novel therapeutic strategies to target MF stem cells. b. Distribute clinical trial specimens to analyze on-target effects of novel agents and create biomarkers which will determine therapeutic and pharmacodynamic responses to therapy (Core D). Core B is an essential shared resource that enables translational research that will ultimately lead to the development of therapies, which target the MPN stem cell.