title: Wegener granulomatosis: an analysis of 158 patients.
abstract: OBJECTIVE
  To prospectively study the clinical features, pathophysiology, treatment and prognosis of Wegener granulomatosis.
  
  
  METHODS
  Of the 180 patients with Wegener granulomatosis referred to the National Institute of Allergy and Infectious Diseases during the past 24 years, 158 have been followed for 6 months to 24 years (a total of 1229 patient-years).
  
  
  METHODS
  Characteristics of clinical presentation, surgical pathology, course of illness, laboratory and radiographic findings, and the results of medical and surgical treatment have been recorded in a computer-based information retrieval system.
  
  
  METHODS
  The Warren Magnuson Clinical Center of the National Institutes of Health.
  
  
  RESULTS
  Men and women were equally represented; 97% of patients were white, and 85% were more than 19 years of age. The mean period of follow-up was 8 years. One hundred and thirty-three patients (84%) received "standard" therapy with daily low-dose cyclophosphamide and glucocorticoids. Eight (5.0%) received only low-dose cyclophosphamide. Six (4.0%) never received cyclophosphamide and were treated with other cytotoxic agents and glucocorticoids. Ten patients (6.0%) were treated with only glucocorticoids. Ninety-one percent of patients experienced marked improvement, and 75% achieved complete remission. Fifty percent of remissions were associated with one or more relapses. Of 99 patients followed for greater than 5 years, 44% had remissions of greater than 5 years duration. Thirteen percent of patients died of Wegener granulomatosis, treatment-related causes, or both. Almost all patients had serious morbidity from irreversible features of their disease (86%) or side effects of treatment (42%).
  
  
  CONCLUSIONS
  The course of Wegener granulomatosis has been dramatically improved by daily treatment with cyclophosphamide and glucocorticoids. Nonetheless, disease- and treatment-related morbidity is often profound. Alternative forms of therapy have not yet achieved the high rates of remission induction and successful maintenance that have been reported with daily cyclophosphamide treatment. Despite continued therapeutic success with cyclophosphamide, our long-term follow-up of patients with Wegener granulomatosis has led to increasing concerns about toxicity resulting from prolonged cyclophosphamide therapy and has encouraged investigation of other therapeutic regimens.
authors.affiliation: National Institutes of Health, Bethesda, Maryland.
authors.forename: G S
authors.initials: GS
authors.lastname: Hoffman
authors.forename: G S
authors.initials: GS
authors.lastname: Kerr
authors.forename: R Y
authors.initials: RY
authors.lastname: Leavitt
authors.forename: C W
authors.initials: CW
authors.lastname: Hallahan
authors.forename: R S
authors.initials: RS
authors.lastname: Lebovics
authors.forename: W D
authors.initials: WD
authors.lastname: Travis
authors.forename: M
authors.initials: M
authors.lastname: Rottem
authors.forename: A S
authors.initials: AS
authors.lastname: Fauci
mesh_terms: D000293:Adolescent; D000328:Adult; D000368:Aged; D001706:Biopsy; D002648:Child; D003520:Cyclophosphamide / Q000009:adverse effects / Q000627:therapeutic use*; D004359:Drug Therapy, Combination; D005260:Female; D005500:Follow-Up Studies; D014890:Granulomatosis with Polyangiitis / Q000150:complications / Q000175:diagnosis / Q000188:drug therapy* / Q000401:mortality; D006801:Humans; D008297:Male; D008875:Middle Aged; D009017:Morbidity; D009894:Opportunistic Infections / Q000453:epidemiology; D011241:Prednisone / Q000009:adverse effects / Q000627:therapeutic use*; D012008:Recurrence; D012074:Remission Induction; D016896:Treatment Outcome
chemical_list: D003520:Cyclophosphamide; D011241:Prednisone