From: Hallmark trials in ANCA-associated vasculitis (AAV) for the pediatric rheumatologist
Reference, Country | Study Design | Patient Selection | Induction therapy | Maintenance therapy | Therapy-related outcomes | Adverse Events |
---|---|---|---|---|---|---|
Akikusa et al., 2007, Canada | Retrospective 1984–2005 | n = 25 GPA 100% | GC 100% CYC 76% AZA 40% MTX 32% | n = 7* AZA (n = 3) MTX (n = 4) | 100% achieved remission (median 5 months, range 3–6 months) 75% relapse rate (median 10 months) | 12 hospitalizations in 5 patients for infections 0 malignancies 0 deaths |
Iudici et al., 2015, France | Retrospective | n = 35 GPA 71.4% EGPA 17.1% MPA 11.4% | GC 91.4% GC + IS 77.1% CYC IV 54.3% CYC PO 5.7% AZA 8.6% MTX 5.7% SZ 2.9% PLEX 2.9% | – | Inactive disease at last follow-up (n = 33, median 96 months): GPA 68.2% on treatment EGPA 100% on treatment (50% on GC + IS) MPA 100% on treatment (66.7% on GC + IS) Relapse rates: 76% overall (GPA 83%, EGPA 50%, MPA 33%) | 9 infections 1 pancreatitis (GC) 2 cataracts (GC) 1 transaminitis (AZA) 1 hematologic (CYC) 1 hypertension (GC) 4 deaths |
Sacri et al., 2015, France | Retrospective 1986–2011 | n = 66 GPA 42% MPA 58% | GC 100% IV 86%, PO 14% CYC IV 47% CYC PO 20% RTX 13.6% MMF 4.5% PLEX 16.7% | GC alone 28.8% GC + AZA/ MTX/ MMF 63.6% AZA alone 1.5% None 6% | 92.4% achieved remission Post-induction: 70% achieved remission 24.2% (n = 16) had refractory disease; 15/16 achieved secondary remission with addition of CYC (n = 8), IVIG (n = 2), RTX (n = 3) PLEX (n = 3) 40.9% relapse rate (median 29 mos) | 4 deaths |
ARChiVe, Morishita et al., 2017, International | 2004–2007 retrospective, 2007–2008 prospective | n = 105 GPA 81% MPA 13% EGPA 6% | GC 100% (IV pulse 70%) CYC 70.5% MTX 16.2% RTX 13.3% AZA 1.9% MMF 1.0% PLEX 23.8% | AZA 42.3% MTX 22.8% MMF 13.3% CYC 9.5% RTX 9.5% None 4.7% | 44/105 (42%) achieved remission by 12 months 21/44 (48%) discontinued GC 41/44 (93%) remained on maintenance 24% relapse rate | 80 hospitalizations in 43 patients: 46% flares 16% infection 5% treatment-related 15% other disease-related 18% unrelated to vasculitis 0 deaths |