Azathioprine for ocular inflammatory diseases

Am J Ophthalmol. 2009 Oct;148(4):500-509.e2. doi: 10.1016/j.ajo.2009.05.008. Epub 2009 Jul 1.


Purpose: To evaluate treatment outcomes of azathioprine for noninfectious ocular inflammatory diseases.

Design: Retrospective cohort study.

Methods: Medical records of 145 patients starting azathioprine as a sole noncorticosteroid immunosuppressant at 4 tertiary uveitis services were reviewed. Main outcome measures included control of ocular inflammation, sustained control after tapering prednisone to </= 10 mg/day, and discontinuation of treatment because of side effects.

Results: Among 145 patients (255 eyes) treated with azathioprine, 63% had uveitis, 23% had mucous membrane pemphigoid, 11% had scleritis, and 3% had other inflammatory diseases. By Kaplan-Meier analysis, 62% (95% confidence interval [CI], 50% to 74%) of patients with active disease initially gained complete inactivity of inflammation sustained over at least 28 days within 1 year of therapy, and 47% (95% CI, 37% to 58%) tapered systemic corticosteroids to </= 10 mg daily while maintaining control of inflammation within 1 year of therapy. Treatment success was most common for intermediate uveitis (90% with sustained inactivity within 1 year; 95% CI, 64% to 99%). Over the median follow-up of 230 days (interquartile range, 62 to 679 days), azathioprine was discontinued at a rate of 0.45 per person-years (/PY): 0.16/PY because of side effects, 0.10/PY because of ineffectiveness, 0.09/PY because of disease remission, and 0.10/PY because of unspecified causes.

Conclusions: Azathioprine was moderately effective as a single corticosteroid-sparing immunosuppressive agent in terms of control of inflammation and corticosteroid-sparing benefits, but required several months to achieve treatment goals; it seems especially useful for patients with intermediate uveitis. Treatment-limiting side effects occurred in approximately one-fourth of patients within 1 year, but typically were reversible.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adolescent
  • Adult
  • Aged
  • Aged, 80 and over
  • Azathioprine / adverse effects
  • Azathioprine / therapeutic use*
  • Child
  • Female
  • Follow-Up Studies
  • Humans
  • Immunosuppressive Agents / adverse effects
  • Immunosuppressive Agents / therapeutic use*
  • Inflammation / drug therapy
  • Male
  • Middle Aged
  • Pemphigoid, Benign Mucous Membrane / drug therapy*
  • Prednisone / therapeutic use
  • Retrospective Studies
  • Scleritis / drug therapy*
  • Treatment Outcome
  • Uveitis / drug therapy*
  • Young Adult


  • Immunosuppressive Agents
  • Azathioprine
  • Prednisone