作者
Aya A Mitani, Sebastien Haneuse
发表日期
2020/3/2
期刊
JAMA network open
卷号
3
期号
3
页码范围
e201965-e201965
出版商
American Medical Association
简介
The age of big data is in full swing, with researchers in both clinical medicine and public health seeking to take advantage of the increasing availability of massive amounts of electronic and administrative health data. In turn, this has led to substantial resources and efforts being poured into the development and teaching of methods for data collection and storage as well as machine learning analytic methods. 1
However, big data are not always available, especially in the study of rare diseases. Indeed, in the study of rare diseases, small sample sizes are inevitable, especially when the primary end point is also uncommon. As an example, Avadhanula et al2 used data from a cohort of 125 patients with alkaptonuria, a rare autosomal recessive disorder. Patients were recruited between 2000 and 2018 as part of a prospective longitudinal study conducted at the National Human Genome Research Institute to investigate …
引用总数
20202021202220232024414292632
学术搜索中的文章