UMBILICAL CORD BLOOD BANK FOR HEMOGLOBINOPATHY PATIENTS

在过去的十年中，已经取得了一些进展， 治疗或病人镰状细胞贫血和地中海贫血。其中 是异基因骨髓移植，目前唯一的治疗方法 提供治愈的可能性 在镰状细胞性贫血中， 移植已经在患有晚期癌症的患者中进行， 器官损伤，而在地中海贫血中， 在出现铁相关组织损伤之前 由于担心 移植和移植物抗宿主病（GVHD）， 血红蛋白病患者仅限于以下情况， 存在HLA相容的捐赠者。 不幸的是，一个HLA匹配的相关 捐助者往往无法获得。 脐带血，最近 公认的造血干细胞来源，已被用于 成功移植了500多名患者。 的潜在优势 脐带血干细胞的来源与造血干细胞的来源有关。 即使没有完全的HLA兼容性， 高度GVHD是最小的。 这项建议的目的是 增加提供移植作为治疗选择的机会， 血红蛋白病患者通过建立一个遗传相关的 脐带血是什么？ 我们的具体目标是：（1）建立一个 一个拥有2500个脐带血单位（CBU）的脐带血库，将为 作为血红蛋白病中心的资源，（2）实施，验证 并监测这些中心的CBU收集程序，（3）确定 如果可以在多个地点收集高质量的CBU并将其运送到 儿童医院奥克兰研究所（CHORI）进行处理 和冷冻保存，以及（4）建立一个研究资源， 评价CBU移植在脑梗死患者中的作用 血红蛋白病 脐带血采集、处理和 数据收集改编自NIH无关脐带血 移植研究（COBLT）。 统计评价和计算机化 数据收集将由The EMMES，Corp.，一组提供 类似于COBLT研究。 分子HLA分型和 血红蛋白病诊断服务将提供给 合作方案。 本文中描述的相关CBU资源 应用程序将为研究人员提供一个研究 CBU在血红蛋白病患者移植中的作用， 在分子水平上评估HLA差异之间的关系， 使用CBU进行移植和GVHD，并确定CBU收集是否 应该提供给所有的家庭谁要么有一个孩子与 血红蛋白病或有风险有一个孩子与 血红蛋白病 相关脐带血库提供的资源 可能为一些患者提供治愈的可能性， 患有镰状细胞性贫血和地中海贫血

During the past decade, a number of advances have been made in the treatment or patients sickle cell anemia and thalassemia. Among these is allogeneic bone marrow transplantation, the only current treatment that offers the potential for cure. In sickle cell anemia, transplantation has been performed in patients who have had advanced organ damage while in thalassemia, transplantation has been performed before evidence of iron related tissue damage. Due to concerns over engraftment and graft versus host disease (GVHD), transplants for patients with hemoglobinopathies have been limited to situations where an HLA compatible donor exists. Unfortunately, an HLA matched related donor is often not available. Umbilical cord blood, a recently recognized source of hematopoetic stem cells, has been used to successfully transplant over 500 patients. The potential advantages of cord blood over other donor sources of stem cells is related to the observation that even without complete HLA compatibility, the risk of high-grade GVHD is minimal. The objective of this proposal is to increase the chance to offer transplantation as a therapeutic option for patients with hemoglobinopathies by establishing a genetically related umbilical cord blood bank. Our specific aims are (1) to establish a cord blood bank containing 2500 cord blood units (CBU) which will serve as a resource to hemoglobinopathy centers, (2) to implement, validate and monitor CBU collection procedures in these centers, (3) to determine if quality CBUs can be collected at multiple sites and shipped to Children's Hospital Oakland Research Institute (CHORI) for processing and cryopreservation, and (4) to establish a research resource for evaluation of the role of CBU transplantation in patients with hemoglobinopathies. Protocols for cord blood collection, processing and data collection have been adapted from the NIH unrelated Cord Blood Transplantation study (COBLT). Statistical evaluation and computerized data collection will be performed by The EMMES, Corp., a group providing similar assistance to the COBLT study. Molecular HLA typing and hemoglobinopathy diagnostic services will be made available to collaborating programs. The related CBU resource described in this application will provide investigators with an opportunity to study the role of CBU for transplantation in patients with hemoglobinopathies, to evaluate at the molecular level relationships between HLA disparity, engraftment and GVHD using CBUs, and to determine if CBUs collection should be offered to all families who either have a child with a hemoglobinopathy or are at risk of having a child with a hemoglobinopathy. The resource provided by this related cord blood bank may offer the possibility of cure to a number of patients who are affected by sickle cell anemia and thalassemia.