One large randomized noninferiority trial of patients with AAV has found Rituximab (RTX) to be non-inferior to cyclophosphamide as induction therapy, including among the patients with renal involvement at enrollment. A randomized controlled trial comparing the effectiveness of daily oral cyclophosphamide versus intravenous pulses suggested that the pulse regimen significantly reduced cumulative exposure and was not associated with any significant difference in time to remission, by Vasculitis Market Share

Other agents, including tacrolimus, intravenous immunoglobulin (IVIG), and anti-TNF-α compounds such as etanercept and infliximab, have previously been explored in the treatment of AAV, however, the current evidence supporting their use is limited and, in light of the demonstrable efficacy of other agents, are unlikely to be adopted as first-line induction therapy. Nevertheless, there are relatively few trials that have explored the efficacy of these agents in combination therapy.

Azathioprine (AZA) was established as the drug of choice for maintenance therapy in the trial which found introducing AZA within 3 months of inducing clinical remission did not result in more early relapses than continuing cyclophosphamide for 12 months. The role of AZA as the preferred agent for maintenance therapy was reinforced by head to head trials comparing AZA to mycophenolate mofetil (MMF) and methotrexate which did not find evidence to support the use of these alternative agents. Indeed, AZA was associated with fewer relapses compared to MMF without a significant difference in serious adverse event rates.

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