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Topical 1% 5-fluoruracil as a sole treatment of corneoconjunctival ocular surface squamous neoplasia: long-term study
  1. Raffaele Parrozzani1,
  2. Luisa Frizziero2,
  3. Sara Trainiti2,
  4. Ilaria Testi2,
  5. Giacomo Miglionico2,
  6. Elisabetta Pilotto2,
  7. Stella Blandamura3,
  8. Ambrogio Fassina3,
  9. Edoardo Midena1,2
  1. 1G.B. Bietti Foundation, IRCCS, Ocular Oncology and Toxicology Research Unit, Roma, Italy
  2. 2Department of Ophthalmology, University of Padova, Padova, Italy
  3. 3Department of Medicine, Surgical Pathology and Cytopathology Unit, University of Padova, Padova, Italy
  1. Correspondence to Professor Edoardo Midena, Clinica Oculitica di Padova—Policlinico Universitario, Via Giustiniani 2, 35128 Padova, Italy; edoardo.midena{at}


Aims To report long-term clinical outcome of topical 1% 5-fluoruracil (5-FU) as a sole treatment of ocular surface squamous neoplasia (OSSN).

Methods 41 patients affected by OSSN were included. Each patient underwent full ophthalmological examination at baseline, with cytological or histological confirmation. Patients were treated by topical chemotherapy with 1% 5-FU four times a day for 4 weeks. One course was defined as 4 weeks of topical chemotherapy. Adjunctive courses were administered after 1 month of chemotherapy-free interval.

Results Mean follow-up was 105±32 months (range 60–171 months). Complete tumour regression was achieved in 34 cases (83%) after a mean of 1.5 courses (range, 1–3 courses). Univariate analysis revealed that complete response was significantly related to tumour thickness <1.5 mm (p=0.005), lack of fornix or tarsal involvement (p=0.015 and p=0.009, respectively) and the absence of multifocality (p=0.002). Histopathological diagnosis (intraepithelial neoplasia vs squamous cell carcinoma, p=0.019) and American Joint Committee on Cancer (AJCC) classification (T1 vs T2 or T3) (p=0.028) were also related to incomplete tumour response. In a multivariate analysis, just tumour thickness >1.5 mm (p=0.045) and multifocality (p=0.023) were correlated with incomplete tumour response. Transient and reversible low-to-mild local side effects were documented in 19 (48%) eyes.

Conclusion Topical 5-FU, as a sole therapy, is a long-term safe and effective treatment for patients affected by preinvasive OSSN and for a limited proportion (50%) of invasive OSSN.

  • Neoplasia
  • Conjunctiva
  • Cornea
  • Ocular surface
  • Treatment Medical
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  • Contributors RP and EM: study conception, design, interpretation of data, drafting and revising, final approval. LF, SR, IT, GM, EP, SB and AF: data acquisition/analysis/interpretation, drafting and revising of work, final approval.

  • Funding The research for this paper was financially supported by Ministry of Health and Fondazione Roma.

  • Competing interests None.

  • Patient consent Obtained.

  • Ethics approval Institutional Review Board of the University of Padova and G.B. Bietti Foundation.

  • Provenance and peer review Not commissioned; externally peer reviewed.

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