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

Cabral, D. A.; Uribe, A. G.; Benseler, S.; O'Neil, K. M.; Hashkes, P. J.; Higgins, G.; Zeft, A. S.; Lovell, D. J.; Kingsbury, D. J.; Stevens, A.; McCurdy, D.; Chira, P.; Abramson, L.; Arkachaisri, T.; Campillo, S.; Eberhard, A.; Hersh, A. O.; Huber, A. M.; Kim, S.; Klein-Gitelman, M.; Levy, D. M.; Li, S. C.; Mason, T.; Dewitt, E. M.; Muscal, E.; Nassi, L.; Reiff, A.; Schikler, K.; Singer, N. G.; Wahezi, D.; Woodward, A.

Arthritis Rheum. 2009 Oct 31; 60(11):3413-24


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.

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