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Randomized Controlled Trial
. 2025 Mar;31(3):807-814.
doi: 10.1111/odi.15058. Epub 2024 Jun 27.

Surgical treatment compared with "wait and see" in patients affected by oral leukoplakia to prevent oral cancer: Preliminary data from a multicenter randomized controlled trial

Niccolò Lombardi  1   2 , Paolo G Arduino  3 , Marta Lampiano  3 , Alessio Gambino  3 , Roberto Broccoletti  3 , Elena M Varoni  1   2 , Giovanni Lodi  1   2
Affiliations
Randomized Controlled Trial

Surgical treatment compared with "wait and see" in patients affected by oral leukoplakia to prevent oral cancer: Preliminary data from a multicenter randomized controlled trial

Niccolò Lombardi et al. Oral Dis. 2025 Mar.

Abstract

Objective: Oral leukoplakia (OL) is one of the most common and investigated oral potentially malignant disorders (OPMD). Preventing OSCC occurrence should be the primary outcome in the clinical management of OL. Surgical removal of OL is performed by most clinicians, although its effectiveness in reducing OSCC onset has still not been established by randomized controlled trials (RCT). Wait and see approach is characterized by frequent clinical examinations and periodical biopsies of OL, avoiding unnecessary surgical procedures. This is the first multicenter RCT in literature aiming at comparing the effectiveness of surgical removal and the "wait and see" approach in preventing OSCC onset in patients affected by dysplastic and non-dysplastic OL.

Methods: Two Italian referral care centres for oral diseases were involved in this multicenter two-arm RCT comparing the surgical removal of OL (group A) and the "wait and see" approach (group B), with the aim of reducing oral cancer onset.

Results: This report shows preliminary data on the first 161 patients, with a mean follow-up of 19.14 ± 11.25 months. Eight cases of OSCC occurred (6 out 8 involving the tongue): one case in group A and seven cases in group B. Moreover, OL recurred in 13 (20%) cases after surgical excision.

Conclusions: Within the limitations of this preliminary report, these initial data underline the increased risk of OSCC onset in the case of OL of the tongue in the presence of epithelial dysplasia in group B ("wait and see") compared to group A (surgery). This RCT is currently ongoing at the same clinical departments, with the aim of enrolling 310 patients and collecting data at 5-year follow-up, in order to achieve conclusive results, in an evidence-based medicine approach.

Keywords: leukoplakia; oral carcinoma; oral potentially malignant disorders; oral surgery; wait and see approach.

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Conflict of interest statement

The authors declare no conflict of interest.

Figures

FIGURE 1
FIGURE 1
Descriptive flowchart of the protocol.
FIGURE 2
FIGURE 2
Oral leukoplakia on the left border of the tongue at the time of enrolment (a); OSCC onset 20 months later (b).
See this image and copyright information in PMC

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