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

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.

• Authors: David A Cabral, América G Uribe, Susanne Benseler, Kathleen M O'Neil, Philip J Hashkes, Gloria Higgins, Andrew S Zeft, Daniel J Lovell, Daniel J Kingsbury, Anne Stevens, Deborah McCurdy, Peter Chira, Leslie Abramson, Thaschawee Arkachaisri, Sarah Campillo, Anne Eberhard, Aimee O Hersh, Adam M Huber, Susan Kim, Marisa Klein-Gitelman, Deborah M Levy, Suzanne C Li, Thomas Mason, Esi Morgan Dewitt, Eyal Muscal, Lorien Nassi, Andreas Reiff, Kenneth Schikler, Nora G Singer, Dawn Wahezi, Amy Woodward, ARChiVe (A Registry for Childhood Vasculitis: e-entry) Investigators Network
• Journal: Arthritis and rheumatism (Arthritis Rheum) Vol. 60 Issue 11 Pg. 3413-24 (Nov 2009) ISSN: 0004-3591 [Print] United States
• PMID: 19877069 (Publication Type: Comparative Study, Journal Article, Research Support, Non-U.S. Gov't)

Chemical References

• Adrenal Cortex Hormones
• Cyclophosphamide
• Methotrexate

Topics

• 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, diagnosis, drug therapy)
• Humans
• Male
• Methotrexate (therapeutic use)
• Microscopic Polyangiitis (diagnosis)
• Pilot Projects
• Reference Standards
• Sensitivity and Specificity
• Societies, Medical
• United States
• Vasculitis (diagnosis)