Guerrettaz Ryan D, Spellman Stephen R, Page Kristin M
Department of Pediatrics, Medical College of Wisconsin (MCW), Milwaukee, WI, USA.
Center for International Blood and Marrow Transplantation Research (CIBMTR), NMDP, Minneapolis, MN, USA.
Bone Marrow Transplant. 2025 Feb;60(2):161-164. doi: 10.1038/s41409-024-02446-5. Epub 2024 Nov 8.
Observational studies using registry-based data have become indispensable in our management of patients receiving hematopoietic cell transplantation (HCT) or cellular therapies (CT) for hematologic malignancies. These registries support studies that describe the natural history of disease, evaluate clinical effectiveness, or measure safety and quality of patient care. To complement registries, biobanks exist as an essential partner to provide insight on genetic, molecular, and cellular processes. In this review, we discuss and provide examples of several biobank and registry collaborations that have advanced our knowledge in the field of HCT and CT, using the Center for International Blood and Marrow Transplantation Research (CIBMTR) as a highlighted example. The CIBMTR has collected data on over 650,000 patients and has over 60,000 paired donor recipient banked samples available for research. Biospecimens and clinical data from the CIBMTR have led to advancements in our knowledge and care of conditions such as acute myeloid leukemia and myelodysplastic syndrome (MDS), as well as our understanding of the roles of HLA typing and social determinants of health. Despite the utility in advancing research, registries and biobanks do not exist without challenges which are briefly reviewed. Nevertheless, the contributions of these resources in supporting HCT/CT research are noteworthy.
利用基于登记处的数据进行的观察性研究,在我们管理接受造血细胞移植(HCT)或细胞疗法(CT)治疗血液系统恶性肿瘤的患者过程中已变得不可或缺。这些登记处支持开展描述疾病自然史、评估临床疗效或衡量患者护理安全性和质量的研究。作为重要合作伙伴,生物样本库的存在有助于深入了解基因、分子和细胞过程。在本综述中,我们将讨论并举例说明一些生物样本库与登记处的合作,这些合作推动了我们在HCT和CT领域的认知,其中特别以国际血液和骨髓移植研究中心(CIBMTR)为例。CIBMTR已收集了超过65万名患者的数据,并有超过6万对供体-受体配对的样本可供研究使用。CIBMTR的生物样本和临床数据推动了我们对急性髓系白血病和骨髓增生异常综合征(MDS)等病症的认知和治疗,以及我们对HLA分型和健康的社会决定因素作用的理解。尽管在推动研究方面具有实用性,但登记处和生物样本库并非没有挑战,本文将简要回顾这些挑战。然而,这些资源在支持HCT/CT研究方面的贡献值得关注。