title: Topical 5-fluorouracil for treatment of cervical intraepithelial neoplasia 2: a randomized controlled trial.
abstract: OBJECTIVE
  The objective of the study was to evaluate the efficacy of intravaginal application of 5% 5-fluorouracil (5-FU) for the treatment of cervical intraepithelial neoplasia (CIN) 2 in women.
  
  
  METHODS
  Women aged 18-29 years with CIN 2 were recruited for this randomized controlled trial of observation vs treatment with intravaginal 5-FU. Women in the observation group returned in 6 months for a Papanicolaou smear, colposcopy, and a human papillomavirus (HPV) deoxyribonucleic acid test. Women in the 5-FU group were treated with intravaginal 5-FU once every 2 weeks for a total of 16 weeks and were similarly evaluated at 6 months. All women who had a baseline visit were included in the intention-to-treat analysis. Values of P < .05 were considered statistically significant.
  
  
  RESULTS
  Between August 2010 and June 2013, 60 women were randomized and had a baseline visit for intervention (n = 31) vs observation (n = 29). Of women who had cervical biopsy results at 6 months, regression of disease was demonstrated in 93% of women in the 5-FU group (26 of 28) and 56% of women in the observation group (15 of 27). Under the intention-to-treat analysis, a relative risk for cervical disease regression of 1.62 (95% confidence interval [CI], 1.10-2.56) was found between the 5-FU and observation arms (P = .01). When the cervical biopsy, Papanicolaou smear, and HPV results were combined for the 6 month follow-up visit, 50% of the 5-FU group (14 of 28) had a documented normal biopsy, normal Papanicolaou smear, and negative HPV test compared with 22% in the observation group (6 of 27) (relative risk, 2.25; 95% confidence interval, 1.05-5.09; P < .05). There were no moderate or severe side effects in the intervention group.
  
  
  CONCLUSIONS
  Topical 5-FU appears to be an effective medical therapy for CIN 2 in young women. 5-FU is readily available and may be considered as an off-label treatment option for young women with CIN 2 who are interested in the treatment of disease but want to avoid excisional procedures.
authors.affiliation: Department of Obstetrics and Gynecology, University of North Carolina at Chapel Hill School of Medicine, Chapel Hill, NC. Electronic address: lisa_rahangdale@med.unc.edu.
authors.forename: Lisa
authors.initials: L
authors.lastname: Rahangdale
authors.affiliation: Department of Obstetrics and Gynecology, University of North Carolina at Chapel Hill School of Medicine, Chapel Hill, NC.
authors.forename: Quinn K
authors.initials: QK
authors.lastname: Lippmann
authors.affiliation: Department of Biostatistics, University of North Carolina at Chapel Hill School of Medicine, Chapel Hill, NC.
authors.forename: Katelyn
authors.initials: K
authors.lastname: Garcia
authors.affiliation: Department of Pathology, University of North Carolina at Chapel Hill School of Medicine, Chapel Hill, NC.
authors.forename: Debra
authors.initials: D
authors.lastname: Budwit
authors.affiliation: Department of Epidemiology, University of North Carolina Gillings School of Public Health, Chapel Hill, NC.
authors.forename: Jennifer S
authors.initials: JS
authors.lastname: Smith
authors.affiliation: Department of Obstetrics and Gynecology, University of North Carolina at Chapel Hill School of Medicine, Chapel Hill, NC.
authors.forename: Linda
authors.initials: L
authors.lastname: van Le
mesh_terms: D000282:Administration, Intravaginal; D000287:Administration, Topical; D000293:Adolescent; D000328:Adult; D000964:Antimetabolites, Antineoplastic / Q000627:therapeutic use*; D001706:Biopsy; D004279:DNA, Viral / Q000032:analysis; D005260:Female; D005472:Fluorouracil / Q000627:therapeutic use*; D006801:Humans; D056687:Off-Label Use; D065006:Papanicolaou Test; D027383:Papillomaviridae / Q000235:genetics; D011446:Prospective Studies; D002583:Uterine Cervical Neoplasms / Q000188:drug therapy*; D014626:Vaginal Smears; D055815:Young Adult; D002578:Uterine Cervical Dysplasia / Q000188:drug therapy*
chemical_list: D000964:Antimetabolites, Antineoplastic; D004279:DNA, Viral; D005472:Fluorouracil
keywords: 5-fluorouracil; cervical intraepithelial neoplasia; medical therapy