Early stopping of clinical trials in lupus and other uncommon rheumatologic diseases.
Use of statistical methods for early stopping of clinical trials allows more efficient and ethical utilization of subjects. In uncommon diseases, where the pool of potential subjects is limited, these methods provide a way to maximize the information gathered from trials. For trials in lupus, methods for early stopping should be more widely utilized. We discuss the ethics, practical aspects, pros and cons, and statistical foundations of some established methods for early stopping, with an emphasis on those that have available implementation in computer software. An example of the design and analysis of a treatment trial in systemic lupus erythematosus with the triangular test method of early stopping is also given.
LaValley
M P
MP
Multipurpose Arthritis and Musculoskeletal Diseases Center, Boston University, MA 02118, USA.
Felson
D T
DT
D002986:Clinical Trials as Topic / Q000379:methods*; D004992:Ethics, Medical; D006801:Humans; D008180:Lupus Erythematosus, Systemic / Q000628:therapy*; D012107:Research Design; D012216:Rheumatic Diseases / Q000628:therapy*; D017211:Treatment Failure