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


CONCLUSIONS
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.
Cabral
David A
DA
Division of Rheumatology, British Columbia Children's Hospital, Vancouver, British Columbia, Canada. dcabral@cw.bc.ca
Uribe
América G
AG
Benseler
Susanne
S
O'Neil
Kathleen M
KM
Hashkes
Philip J
PJ
Higgins
Gloria
G
Zeft
Andrew S
AS
Lovell
Daniel J
DJ
Kingsbury
Daniel J
DJ
Stevens
Anne
A
McCurdy
Deborah
D
Chira
Peter
P
Abramson
Leslie
L
Arkachaisri
Thaschawee
T
Campillo
Sarah
S
Eberhard
Anne
A
Hersh
Aimee O
AO
Huber
Adam M
AM
Kim
Susan
S
Klein-Gitelman
Marisa
M
Levy
Deborah M
DM
Li
Suzanne C
SC
Mason
Thomas
T
Dewitt
Esi Morgan
EM
Muscal
Eyal
E
Nassi
Lorien
L
Reiff
Andreas
A
Schikler
Kenneth
K
Singer
Nora G
NG
Wahezi
Dawn
D
Woodward
Amy
A
D000293:Adolescent; D000305:Adrenal Cortex Hormones / Q000627:therapeutic use; D002648:Child; D002675:Child, Preschool; D015267:Churg-Strauss Syndrome / Q000175:diagnosis; D015331:Cohort Studies; D003520:Cyclophosphamide / Q000627:therapeutic use; D003937:Diagnosis, Differential; D005060:Europe; D005260:Female; D005921:Glomerulonephritis / Q000175:diagnosis; D014890:Granulomatosis with Polyangiitis / Q000145:classification* / Q000175:diagnosis* / Q000188:drug therapy; D006801:Humans; D008297:Male; D008727:Methotrexate / Q000627:therapeutic use; D055953:Microscopic Polyangiitis / Q000175:diagnosis; D010865:Pilot Projects; D012015:Reference Standards; D012680:Sensitivity and Specificity; D012955:Societies, Medical; D014481:United States; D014657:Vasculitis / Q000175:diagnosis
D000305:Adrenal Cortex Hormones; D003520:Cyclophosphamide; D008727:Methotrexate