A staged approach to the treatment of Wegener's granulomatosis: induction of remission with glucocorticoids and daily cyclophosphamide switching to methotrexate for remission maintenance.
OBJECTIVE
To determine the efficacy of a daily cyclophosphamide (CYC) and glucocorticoid induction and methotrexate (MTX) remission-maintenance regimen for the treatment of Wegener's granulomatosis (WG).


METHODS
An open-label, prospective, standardized trial for the treatment of WG was performed using CYC and glucocorticoids for remission induction and MTX for remission maintenance. Thirty-one patients were enrolled in this study. Outcome was assessed using predetermined definitions based on clinical characteristics and pathologic, laboratory, and radiographic findings.


RESULTS
The use of CYC and glucocorticoids for remission induction and MTX for remission maintenance resulted in disease remission for all 31 patients. The median time to remission was 3 months and the median time to discontinuation of glucocorticoids was 8 months. No patients have died, and 5 patients (16%) have had disease relapses at a median of 13 months after achieving remission. Only 2 patients (6%) have had to withdraw from the trial as a result of medication toxicity.


CONCLUSIONS
The use of CYC and glucocorticoids for remission induction and MTX for remission maintenance was shown by this study to be an acceptable alternative therapy for patients with active WG, including those with severe disease at onset.
Langford
C A
CA
Laboratory of Immunoregulation, National Institute of Allergy and Infectious Diseases, National Institutes of Health, Bethesda, Maryland 20892, USA.
Talar-Williams
C
C
Barron
K S
KS
Sneller
M C
MC
D000293:Adolescent; D000328:Adult; D000368:Aged; D002648:Child; D003520:Cyclophosphamide / Q000008:administration & dosage; D005260:Female; D005938:Glucocorticoids / Q000494:pharmacology; D014890:Granulomatosis with Polyangiitis / Q000628:therapy*; D006801:Humans; D008297:Male; D008727:Methotrexate / Q000494:pharmacology; D008875:Middle Aged; D012074:Remission Induction; D055502:Secondary Prevention
D005938:Glucocorticoids; D003520:Cyclophosphamide; D008727:Methotrexate