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

Sep 21, 2026

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Objective: Oral leukoplakia (OL) is a common oral potentially malignant disorder where preventing oral squamous cell carcinoma (OSCC) is a primary management goal. Surgical removal is widely used, but randomized controlled trials proving OSCC risk reduction are limited. Methods: A multicenter two-arm randomized controlled trial compared surgical excision (group A) versus a “wait and see” strategy with frequent examinations and periodical biopsies (group B). Results: Preliminary data from 161 patients with 19.14 ± 11.25 months follow-up showed eight OSCC cases, mostly involving the tongue, with one in group A and seven in group B. Conclusions: In this preliminary report, tongue OL with epithelial dysplasia had higher OSCC onset risk in group B than in group A; enrollment is ongoing for 310 patients with 5-year follow-up.

LanguageEnglish

CategoryResearch & Report

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Pages8

Reading Time20 min

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Abstract

Objective

Methods

Results

Conclusions

Introduction

Oral potentially malignant disorders and cancer risk

Definition and clinical features of oral leukoplakia

DOI: 10. 1111/odi.15058 OR I G I NAL ART I C L E 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 Lombardi1,2 | Paolo G. Arduino3 | Marta Lampiano3 | Alessio Gambino3 | Roberto Broccoletti3 | Elena M. Varoni1,2 | Giovanni Lodi1,2 1Dipartimento di Scienze Biomediche Chirurgiche e Odontoiatriche, Università Degli Studi di Milano, Milan, Italy 2ASST Santi Paolo e Carlo, SCOdontostomatologia II, Ospedale San Paolo, Milan, Italy 3 Dipartimento di Scienze Chirurgiche, CIR Dental School, Università Degli Studi di Torino, Turin, Italy Correspondence Niccolò Lombardi, Dipartimento di Scienze Biomediche Chirurgiche e Odontoiatriche, Università Degli Studi di Milano, Milan, Italy. Email: [niccolo.lombardi@unimi.it](niccolo.lombardi@unimi.it) 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. K E Y WO R D S leukoplakia, oral carcinoma, oral potentially malignant disorders, oral surgery, wait and see approach All authors gave the final approval of the version to be published and agreed to be accountable for all aspects of the work in ensuring that questions related to the accuracy or integrity of any part of the work are appropriately investigated and resolved. This is an open access article under the terms of the Creative Commons Attribution-NonCommercial-NoDerivs License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non-commercial and no modifications or adaptations are made. © 2024 The Author(s) . Oral Diseases published by Wiley Periodicals LLC. 1 | INTRODUCTION The oral potentially malignant disorders (OPMDs) are a group of mucosal disorders characterized by a variable increased risk of developing oral squamous cell carcinoma (OSCC) (Warnakulasuriya et al., 2007) . According to the WHO Collaborating Centre for Oral Cancer's workshop, held in 2020, patients affected by any OPMDs show higher susceptibility to develop cancer anywhere in their mouth during their lifetime (Warnakulasuriya et al., 2021) . Since OSCC could not necessarily occur in the site of the clinically altered mucosa, a “clinically normal muc

FAQs

What is being compared in this randomized controlled trial?

The trial compares surgical removal of oral leukoplakia (group A) with a “wait and see” approach involving frequent clinical examinations and periodical biopsies (group B) to prevent OSCC onset.

How many OSCC cases occurred in the preliminary results?

Eight OSCC cases occurred in the first 161 patients, with six cases involving the tongue: one in group A and seven in group B.

What does the study conclude about tongue OL with epithelial dysplasia?

Within the preliminary limits, the data suggest a higher risk of OSCC onset for tongue OL with epithelial dysplasia in the “wait and see” group (group B) compared with surgery (group A).

Sep 21, 2026

Uploaded by Ophelia