Table 3

Points to evaluate in assessing a Wegener's granulomatosis therapeutic study

Type and design of study
Retrospective, prospective, open-label, randomized

Single center or multicenter
Study size
For open-label trials, how many patients were included?

For randomized trials, were the sample size per arm and power sufficient to draw conclusions?
Patient population
Did the study enroll patients with other forms of vasculitis?

How was Wegener's granulomatosis diagnosed?

Were patients enrolled at initial diagnosis or relapse?
Site of organ involvement
What was the distribution of organ involvement?

Were there any patients who had disease isolated to nonmajor organ sites such as the skin, the joint, or the sinus?

Were patients enrolled for sites known to be of limited medical responsiveness (i.e. subglottic stenosis)?

Were methods in place to rule out other processes that could have the appearance of active disease?
Definition of outcome measures
How did the study define active disease, remission, and relapse?

Were outcome measures clearly defined in the methods?
Concurrent therapies
Were immunosuppressive therapies used concurrently with the agent or regimen being studied?
Standardization
Did all enrolled patients receive a standarized dose and duration of the study agent?

Was the glucocorticoid dose and taper standardized?

At what point in time did patients enter the study (i.e. at beginning of induction or at remission)?
Study duration
What was the median follow-up time?

Were patients followed for sufficient duration to observe relapses?

Langford Arthritis Res Ther 2003 5:180   doi:10.1186/ar771