Variability in experts' opinions regarding risks and treatment choices in patients with antineutrophil cytoplasmic antibody-associated vasculitis.

OBJECTIVE

There are insufficient toxicity data available to guide treatment decisions in patients with ANCA (antineutrophil cytoplasmic antibody)-associated vasculitis. The objective of this study was to obtain expert input related to available treatment options.

METHODS

We performed a Web-based survey of experts (defined as physicians whose practices focus on vasculitis and physicians engaged in research in vasculitis) regarding adverse events (AEs) associated with cyclophosphamide and/or rituximab, the 2 treatment options for remission induction of severe ANCA-associated vasculitis (AAV). Using scaled measures, experts rated (i) the probability of 30 AEs associated with the treatments and (ii) the importance of disclosing each AE and reported (iii) their treatment preferences using standardized scenarios.

RESULTS

Ratings of the probabilities of specific AEs associated with cyclophosphamide and rituximab varied significantly among the experts. The majority agreed that AEs related to fertility, infections, and serious infusion reactions were "extremely" or "very important" to disclose. Less than half of the experts surveyed endorsed disclosing the risks of progressive multifocal leukoencephalopathy, hepatitis reactivation, or zoster. For patients with newly diagnosed AAV, the majority of experts preferred intravenous cyclophosphamide for older adults and rituximab for younger women with newly diagnosed AAV. For patients with recurrent disease who had been previously treated with cyclophosphamide, the majority of experts preferred rituximab, regardless of age or sex.

CONCLUSIONS

The variability noted in this study suggests that the information and treatment patients receive may differ depending on where they receive their care. This type of unwarranted variability could be reduced if data from long-term extension and observational studies generate more precise outcome estimates for treatment-related AEs in AAV.