Publication of data collection forms from NHLBI funded sickle cell disease implementation consortium (SCDIC) registry
Publication of data collection forms from NHLBI funded sickle cell disease implementation consortium (SCDIC) registry
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DOI：
10.1186/s13023-020-01457-x
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发表时间：
2020-07-07
影响因子：
3.7
通讯作者：
Richardson, Lynne D.
中科院分区：
文献类型：
--
作者：
Glassberg, Jeffrey A.;Linton, Elizabeth A.;Richardson, Lynne D.
镰状细胞病（SCD）是一种常染色体隐性血液疾病，影响约10万美国人和全球310万人。由于罕见病相关知识和经验的缺乏，因此特别需要合作和基础设施，以克服在将基础研究进展转化为临床进展方面的挑战。尽管SCD采取了登记处的措施，但由于缺乏对选择过程的描述和最终数据收集工具的副本，加上SCD人口的不完整代表，阻碍了进一步的研究进展。本文描述了SCDIC（镰状细胞病实施联盟）注册的发展，并使SCDIC注册基线和第一次随访数据收集表可用于其他SCD研究工作。使用研究电子数据捕获（REDCap）存储和管理来自8个站点的2400名入组患者的研究数据。标准化的数据收集工具，招募和入组是通过联盟站点的共识来完善的。数据点包括从各种经过验证的来源（PHENX， PROMIS等）采取的措施。调查由研究人员直接管理，并通过DCC协调纵向随访。附加的注册表跟踪医疗记录、事件相关的病人失效、妊娠、实验室报告、心肺和肾功能。SCDIC Registry致力于为成人和青少年SCD人群提供准确、最新的特征，以及标准化、有效的数据收集工具，以指导循证研究和实践。
Background Sickle cell disease (SCD) is an autosomal recessive blood disorder affecting approximately 100,000 Americans and 3.1 million people globally. The scarcity of relevant knowledge and experience with rare diseases creates a unique need for cooperation and infrastructure to overcome challenges in translating basic research advances into clinical advances. Despite registry initiatives in SCD, the unavailability of descriptions of the selection process and copies of final data collection tools, coupled with incomplete representation of the SCD population hampers further research progress. This manuscript describes the SCDIC (Sickle Cell Disease Implementation Consortium) Registry development and makes the SCDIC Registry baseline and first follow-up data collection forms available for other SCD research efforts. Results Study data on 2400 enrolled patients across eight sites was stored and managed using Research Electronic Data Capture (REDCap). Standardized data collection instruments, recruitment and enrollment were refined through consensus of consortium sites. Data points included measures taken from a variety of validated sources (PHENX, PROMIS and others). Surveys were directly administered by research staff and longitudinal follow-up was coordinated through the DCC. Appended registry forms track medical records, event-related patient invalidation, pregnancy, lab reporting, cardiopulmonary and renal functions. Conclusions The SCDIC Registry strives to provide an accurate, updated characterization of the adult and adolescent SCD population as well as standardized, validated data collecting tools to guide evidence-based research and practice.