Comparability of patients with ANCA-associated vasculitis enrolled in clinical trials or in observational cohorts

Research output: Contribution to journalArticlepeer-review


  • C Pagnoux
  • S Carette
  • N A Khalidi
  • M Walsh
  • T F Hiemstra
  • D Cuthbertson
  • C Langford
  • G Hoffman
  • C L Koening
  • P A Monach
  • L Moreland
  • L Mouthon
  • P Seo
  • U Specks
  • S Ytterberg
  • K Westman
  • P Hoglund
  • Lorraine Harper
  • O Flossman
  • R Luqmani
  • Caroline Savage
  • N Rasmussen
  • K de Groot
  • V Tesar
  • D Jayne
  • P A Merkel
  • L Guillevin
  • French Vasculitis Study Group (FVSG)
  • European Vasculitis Society (EUVAS)
  • Vasculitis Clinical Research Consortium (VCRC)


OBJECTIVE: 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).

CONCLUSION: 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.


Original languageEnglish
Pages (from-to)S77-S83
Number of pages7
JournalClinical and Experimental Rheumatology
Issue number2, Suppl. 89
Publication statusPublished - 29 May 2015


  • Adult, Age Distribution, Aged, Antibodies, Antineutrophil Cytoplasmic, Cohort Studies, Female, Granulomatosis with Polyangiitis (Wegener’s granulomatosis), Humans, Kidney Diseases, Male, Microscopic Polyangiitis, Middle Aged, Myeloblastin, Observational Study as Topic, Otorhinolaryngologic Diseases, Patient Selection, Peroxidase, Randomized Controlled Trials as Topic, Severity of Illness Index, ANCA, vasculitis