Comparability of patients with ANCA-associated vasculitis enrolled in clinical trials or in observational cohorts. Academic Article uri icon

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abstract

  • OBJECTIVES: To analyse the differences between patients with granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA) entered into randomised clinical trials (RCTs) and those followed in large observational cohorts. METHODS: The main characteristics and outcomes of patients with generalised and/or severe GPA or MPA with a five-factor score ≥ 1 enrolled in the French Vasculitis Study Group (FVSG) or the US-Canadian-based Vasculitis Clinical Research Consortium cohorts were compared to those enrolled in one of 2 FVSG clinical RCTs (WEG91, WEGENT) or 3 European Vasculitis Society clinical trials (CYCLOPS, CYCAZAREM, IMPROVE). RESULTS: 657 patients (65.3% with GPA) in RCTs were compared to 437 in cohorts (90.6% with GPA). RCT patients were older at diagnosis than the cohort patients (56.6 ± 13.9 vs. 46.8 ± 17.3 years), had higher Birmingham vasculitis activity score (19.5 ± 9.1 vs. 16.9 ± 7.4), and more frequent kidney disease (84.0% vs. 54.9%) but fewer ear, nose, and throat symptoms (56.8% vs. 72.2%). At 56 months post-diagnosis, mortality and relapse rates, adjusted for age and renal function, were higher for patients with GPA in RCTs vs. cohorts (10.7% vs. 2.5% [p=0.001] and 22.5% vs. 15.6% [p=0.03], respectively) but similar for patients with MPA (6.2% vs. 6.6% [p=0.92] and 16.6% vs. 10.1% [p=0.39], respectively). CONCLUSIONS: Patients with GPA or MPA in RCTs and those in observational cohorts show important differences that should be remembered when interpreting results based on these study populations.

authors

  • Pagnoux, C
  • Carette, S
  • Khalidi, Nader
  • Walsh, M
  • Hiemstra, TF
  • Cuthbertson, D
  • Langford, C
  • Hoffman, G
  • Koening, CL
  • Monach, PA
  • Moreland, L
  • Mouthon, L
  • Seo, P
  • Specks, U
  • Ytterberg, S
  • Westman, K
  • Hoglund, P
  • Harper, L
  • Flossman, O
  • Luqmani, R
  • Savage, CO
  • Rasmussen, N
  • de Groot, K
  • Tesar, V
  • Jayne, D
  • Merkel, PA
  • Guillevin, L
  • french Vasculitis Study Group (FVSG) European Vasculitis Society (EUVAS) and Vasculitis Clinical Research Consortium (VCRC)

publication date

  • March 2015

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