title: Subconjunctival sirolimus for the treatment of chronic active anterior uveitis: results of a pilot trial.
abstract: OBJECTIVE
  To evaluate the safety and possible efficacy of subconjunctival sirolimus for the treatment of chronic active anterior uveitis.
  
  
  METHODS
  Prospective, nonrandomized, open-label clinical trial.
  
  
  METHODS
  This single-center pilot trial enrolled 5 patients with chronic active anterior uveitis. The study drug was administered as a single subconjunctival injection of 30 μL (1320 μg) sirolimus in the study eye at the baseline visit. Study visits were performed at baseline, at 2 weeks, at 4 weeks, and monthly until 4 months, and included a complete ophthalmic examination, review of systems, adverse event assessment at each visit, physical examination, and ancillary ophthalmic testing at some visits. The primary outcome measure was a 2-step reduction in the anterior chamber inflammation within 4 weeks of injection of the study drug.
  
  
  RESULTS
  There were 3 female and 2 male patients; 4 patients had idiopathic anterior uveitis and 1 had psoriatic arthritis-associated anterior uveitis. Three of the 5 patients met the primary outcome criteria by showing at least a 2-step decrease in inflammation within 4 weeks; 2 patients showed a 1-step decrease in inflammation within the same time frame. No recurrence was encountered during a 4-month follow-up. There were no serious adverse events.
  
  
  CONCLUSIONS
  Subconjunctival sirolimus appears to be well tolerated in this pilot trial and shows promise as a treatment for active inflammation in patients with chronic anterior uveitis. Larger studies are needed to assess its usefulness in uveitis.
authors.affiliation: National Eye Institute, National Institutes of Health, Bethesda, MD 20892, USA. senh@nei.nih.gov
authors.forename: H Nida
authors.initials: HN
authors.lastname: Sen
authors.forename: Theresa A
authors.initials: TA
authors.lastname: Larson
authors.forename: Annal D
authors.initials: AD
authors.lastname: Meleth
authors.forename: Wendy M
authors.initials: WM
authors.lastname: Smith
authors.forename: Robert B
authors.initials: RB
authors.lastname: Nussenblatt
mesh_terms: D000328:Adult; D002908:Chronic Disease; D003228:Conjunctiva / Q000187:drug effects; D005260:Female; D006801:Humans; D007166:Immunosuppressive Agents / Q000008:administration & dosage* / Q000009:adverse effects; D056965:Injections, Intraocular; D008297:Male; D008875:Middle Aged; D010865:Pilot Projects; D011446:Prospective Studies; D020123:Sirolimus / Q000008:administration & dosage* / Q000009:adverse effects; D016896:Treatment Outcome; D014606:Uveitis, Anterior / Q000188:drug therapy* / Q000503:physiopathology; D014792:Visual Acuity / Q000502:physiology
chemical_list: D007166:Immunosuppressive Agents; D020123:Sirolimus