Bone marrow transplant clinical network

描述(由申请人提供)： 第一次成功的造血干细胞移植(HCT)是在1968年为三名患有先天性免疫缺陷疾病的儿童进行的。在每一种情况下，干细胞都是从与受者基因相同或密切匹配的同胞捐赠者的骨髓中收集的。从那时起，数以千计的HCT被用于治疗恶性和非恶性疾病。目前，全世界每年的HCT数量估计约为45,000例，美国每年约为15-20,000例。增加使用的原因包括已证实的和潜在的对许多疾病的疗效，更好地了解适当的移植和患者选择时间，更多的捐赠者和更好的确定人类白细胞抗原配型的技术，更容易的干细胞采集，以及更好的支持性护理，从而降低与移植相关的死亡率。尽管有这些改进， HCT的应用仍然受到发病率和死亡率相对较高的风险的限制，就异基因HCT而言，还受到可获得合适捐赠者的限制。HCT的结果受许多患者和疾病相关因素的影响，如年龄、疾病阶段和先前的治疗，以及与移植相关的因素，如干细胞来源和移植方案。理想情况下，大多数移植策略的有效性将在大型随机临床试验中进行评估。然而，各种因素阻碍了在这种情况下进行随机试验的能力。许多用红细胞压积治疗的疾病是不常见的。此外，对于异基因移植，并不是所有的患者都有合适的 捐赠者身份已确定。因此，即使考虑到最常见的HCT适应症，个别中心符合条件的患者数量也很少。以明确的方式测试新的移植策略的能力需要存在一个致力于开发和实施大型多中心研究的网络。根据RFA HL-01-004，国家心肺血液研究所(NHLBI)和国家癌症研究所(NCI)共同建立了血液和骨髓移植临床试验网络(BMT CTN)。该网络的目标是在多中心环境下有效地评估来自单中心研究的新的HCT方法和管理策略。与癌症临床试验组不同，这个小组的重点是改善移植程序本身，而不是总体上的癌症治疗。 (摘要结束。)

DESCRIPTION (provided by applicant): The first successful hematopoietic stem cell transplantations (HCT) were done in 1968 in three children with congenital immune deficiency diseases. In each instance, stem cells were collected from the bone marrow of sibling donors who were genotypically identical or closely matched to the recipient for human leukocyte antigens (HLA). Since then, thousands of HCTs have been performed to treat malignant and non-malignant diseases. Current estimates of annual numbers of HCTs are about 45,000 worldwide, with 15-20,000 annually in the United State. Reasons for increasing use include proven and potential efficacy in many diseases, better understanding of the appropriate timing of transplantation and patient selection, greater availability of donors and better techniques for determining HLA match, greater ease of stem cell collection, and improved supportive care resulting in less transplant-related mortality. Despite these improvements, application of HCT is still limited by relatively high risks of morbidity and mortality and, in the case of allogeneic HCT, availability of suitable donors. HCT outcomes are influenced by many patient- and disease-related factors such as age, disease stage and prior treatment, as well as transplant-related factors such as stem cell source and transplant regimen. Ideally, the effectiveness of most transplant strategies would be evaluated in large randomized clinical trials. However, various factors hamper the ability to conduct randomized trials in this setting. Many diseases treated with HCT are uncommon. Additionally, for allogeneic transplants, not all patients have a suitable donor identified. Consequently, even when one considers the most common indications for HCT, numbers of eligible patients in individual centers are small. The ability to test new transplant strategies in a definitive way requires the existence of a network committed to development and implementation of large, multicenter studies. Under RFA HL-01-004, the National Heart, Lung, and Blood Institute (NHLBI) and the National Cancer Institute (NCI) jointly established a Blood and Marrow Transplant Clinical Trials Network (BMT CTN). The goal of the Network is to efficiently assess novel HCT methods and management strategies derived from single center studies in a multi-center setting. The focus of this group, in contrast to cancer clinical trials groups, is on improving the transplant procedure per se rather than cancer treatment in general. (End of abstract.)