The downsides of a major medical advance.

Abstract

When positive, the results of randomized controlled trials designed to evaluate the effectiveness of new options are released with great fanfare. This is especially true for difficult to treat conditions for which there are few treatment alternatives. This scenario recently played out at the 2009 American College of Rheumatology National meeting when the long anticipated RAVE (Rituximab versus Cyclophosphamide for Induction of Remission in ANCA-Associated Vasculitis) trial results were announced. Since then the results of the RAVE and RITUXVAS trials have demonstrated that rituximab is as effective at inducing remission in patients with ANCA positive vasculitis as cyclophosphamide. Prior to these trials, cyclophosphamide was the option of choice for this potentially life threatening disease, and physicians have long hoped for a viable alternative given the significant toxicity associated with this medication, particularly infection, infertility, and cancer. While the results of the trial were justifiably received with great enthusiasm, practical clinical experience has revealed some noteworthy downsides to this important medical advance.