Recent advances in anti-neutrophil cytoplasmic antibody-associated vasculitis.

One large randomized noninferiority trial of 197 patients with AAV has found Rituximab ® (RTX) to be noninferior to cyclophosphamide as induction therapy,[ 38 ] including among the 102 patients with renal involvement at enrollment.[ 39 ] This finding has also been demonstrated in a separate randomized noninferiority trial of 44 patients with AAV and renal involvement.[ 40 ] Adverse events at 6 months and 1 year were not reduced in the RTX group, which had a higher number of cancers at 1 year, although this difference did not reach statistical significance.[ 38 ] Both trials were conducted in the context of case series and retrospective observational studies suggesting that RTX is efficacious as second-line therapy in patients not adequately responding to cyclophosphamide.[ 41 42 ] A prospective cohort of 10 patients with refractory PR3 positive GPA tolerated and responded to RTX, although one patient relapsed after B-cell reconstitution.[ 43 ] Most centers currently use cyclophosphamide as first-line therapy, and reserve RTX for refractory cases or for those who wish to preserve their fertility[ 44 ] [ Supplementary Table 1 ].

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