Classification, presentation, and initial treatment of Wegener's granulomatosis in childhood

Arthritis Rheum. 2009 Nov;60(11):3413-24. doi: 10.1002/art.24876.

Abstract

Objective: To compare the criteria for Wegener's granulomatosis (WG) of the American College of Rheumatology (ACR) with those of the European League Against Rheumatism/Pediatric Rheumatology European Society (EULAR/PRES) in a cohort of children with WG and other antineutrophil cytoplasmic antibody (ANCA)-associated vasculitides (AAVs), and to describe the interval to diagnosis, presenting features, and initial treatment for WG.

Methods: Eligible patients had been diagnosed by site rheumatologists (termed the "MD diagnosis") since 2004. This diagnosis was used as a reference standard for sensitivity and specificity testing of the 2 WG classification criteria. Descriptive analyses were confined to ACR-classified WG patients.

Results: MD diagnoses of 117 patients (82 of whom were female) were WG (n = 76), microscopic polyangiitis (n = 17), ANCA-positive pauci-immune glomerulonephritis (n = 5), Churg-Strauss syndrome (n = 2), and unclassified vasculitis (n = 17). The sensitivities of the ACR and EULAR/PRES classification criteria for WG among the spectrum of AAVs were 68.4% and 73.6%, respectively, and the specificities were 68.3% and 73.2%, respectively. Two more children were identified as having WG by the EULAR/PRES criteria than by the ACR criteria. For the 65 ACR-classified WG patients, the median age at diagnosis was 14.2 years (range 4-17 years), and the median interval from symptom onset to diagnosis was 2.7 months (range 0-49 months). The most frequent presenting features by organ system were constitutional (89.2%), pulmonary (80.0%), ear, nose, and throat (80.0%), and renal (75.4%). Fifty-four patients (83.1%) commenced treatment with the combination of corticosteroids and cyclophosphamide, with widely varying regimens; the remainder received methotrexate alone (n = 1), corticosteroids alone (n = 4), or a combination (n = 6).

Conclusion: The EULAR/PRES criteria minimally improved diagnostic sensitivity and specificity for WG among a narrow spectrum of children with AAVs. Diagnostic delays may result from poor characterization of childhood WG. Initial therapy varied considerably among participating centers.

Publication types

  • Comparative Study
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adrenal Cortex Hormones / therapeutic use
  • Child
  • Child, Preschool
  • Churg-Strauss Syndrome / diagnosis
  • Cohort Studies
  • Cyclophosphamide / therapeutic use
  • Diagnosis, Differential
  • Europe
  • Female
  • Glomerulonephritis / diagnosis
  • Granulomatosis with Polyangiitis / classification*
  • Granulomatosis with Polyangiitis / diagnosis*
  • Granulomatosis with Polyangiitis / drug therapy
  • Humans
  • Male
  • Methotrexate / therapeutic use
  • Microscopic Polyangiitis / diagnosis
  • Pilot Projects
  • Reference Standards
  • Sensitivity and Specificity
  • Societies, Medical*
  • United States
  • Vasculitis / diagnosis

Substances

  • Adrenal Cortex Hormones
  • Cyclophosphamide
  • Methotrexate