title: Topical 5-Fluorouracil 1% as Primary Treatment for Ocular Surface Squamous Neoplasia.
abstract: To determine the efficacy of topical 5-fluorouracil 1% (5-FU) as a primary treatment of ocular surface squamous neoplasia (OSSN).
  
  
  
  Retrospective study.
  
  
  
  Topical 5-FU was used as primary therapy in 44 patients with OSSN.
  
  
  
  5-Fluorouracil 1% administered topically 4 times daily for 1 week followed by a drug holiday of 3 weeks. Patients were identified through a pharmacy database. Patients were excluded if 5-FU was used as adjuvant therapy, if they did not complete therapy, or if they were still actively receiving treatment for OSSN at the time of last follow-up.
  
  
  
  The primary outcome measures were the frequency of complete resolution with topical 5-FU treatment and the rate of OSSN recurrence.
  
  
  
  Of the 44 patients identified, 32 were men and 12 were women. The mean age was 68 years. Complete resolution of OSSN was noted in 82% of patients (36/44); 18% (8/44) were considered treatment nonresponders. Patients were treated with a median of 4 cycles (range, 2-9 cycles). Nasal location was the only risk factor identified for nonresponse to therapy (P = 0.04). The median follow-up after resolution was 10 months (range, 2-77 months). In the 36 patients who showed complete resolution, 4 experienced tumor recurrence. Recurrence rates at 1 and 2 years were 6% and 15%, respectively, using Kaplan-Meier survival analysis. At least 1 side effect from the medication was reported by 61% of patients (21/44), but only 1 patient discontinued the medication because of intolerance. The most common side effect was pain (n = 17; 39%), followed by tearing (n = 10; 23%), photophobia (n = 6; 14%), itching (n = 4; 9%), swelling (n = 2; 5%), and infection (n = 1; 2%). No long-term complications were reported.
  
  
  
  5-Fluorouracil is effective and well tolerated as a primary treatment for OSSN, with 82% of tumors responding completely to therapy.
authors.affiliation: Bascom Palmer Eye Institute, University of Miami, Miami, Florida.
authors.forename: Madhura G
authors.initials: MG
authors.lastname: Joag
authors.affiliation: Bascom Palmer Eye Institute, University of Miami, Miami, Florida.
authors.forename: Adam
authors.initials: A
authors.lastname: Sise
authors.affiliation: Bascom Palmer Eye Institute, University of Miami, Miami, Florida.
authors.forename: Juan Carlos
authors.initials: JC
authors.lastname: Murillo
authors.affiliation: Bascom Palmer Eye Institute, University of Miami, Miami, Florida.
authors.forename: Ibrahim Osama
authors.initials: IO
authors.lastname: Sayed-Ahmed
authors.affiliation: Bascom Palmer Eye Institute, University of Miami, Miami, Florida.
authors.forename: James R
authors.initials: JR
authors.lastname: Wong
authors.affiliation: Bascom Palmer Eye Institute, University of Miami, Miami, Florida.
authors.forename: Carolina
authors.initials: C
authors.lastname: Mercado
authors.affiliation: Bascom Palmer Eye Institute, University of Miami, Miami, Florida; Veterans Affairs Medical Center, Miami, Florida.
authors.forename: Anat
authors.initials: A
authors.lastname: Galor
authors.affiliation: Bascom Palmer Eye Institute, University of Miami, Miami, Florida. Electronic address: ckarp@med.miami.edu.
authors.forename: Carol L
authors.initials: CL
authors.lastname: Karp
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000964:Antimetabolites, Antineoplastic / Q000008:administration & dosage*; D002294:Carcinoma, Squamous Cell / Q000188:drug therapy*; D003230:Conjunctival Neoplasms / Q000188:drug therapy*; D003316:Corneal Diseases / Q000188:drug therapy; D005260:Female; D005472:Fluorouracil / Q000008:administration & dosage*; D006801:Humans; D053208:Kaplan-Meier Estimate; D008297:Male; D008875:Middle Aged; D009364:Neoplasm Recurrence, Local / Q000453:epidemiology; D009883:Ophthalmic Solutions / Q000627:therapeutic use*; D012189:Retrospective Studies; D012307:Risk Factors
chemical_list: D000964:Antimetabolites, Antineoplastic; D009883:Ophthalmic Solutions; D005472:Fluorouracil