Approach

For any white patch involving the oral mucosa, where no other definable cause can be identified and a 'true' diagnosis of oral leukoplakia can be made, elimination of contributory factors is recommended (smoking cessation, elimination of areca nut (betel quid) use, and reduction of/abstinence from alcohol use). However, except for such measures, there is no reason to separate forms of leukoplakia (e.g., tobacco-associated forms of leukoplakia from idiopathic forms) in terms of overall management.[138]

For definitive treatment of leukoplakia, options are influenced by the perceived malignant potential of the lesion based on clinical characteristics (e.g., site and size of lesion) and the degree of any associated epithelial dysplasia present. Biopsy may miss dysplastic lesions or even early carcinomas. To date there is no reliably effective management protocol in preventing malignant transformation of leukoplakia.[139] In general, careful observation is least recommended for all cases of leukoplakia. For many, such as those with an assured benign histopathology, this is adequate. However, for those cases of oral leukoplakia that have malignant potential or have been shown to have moderate to severe epithelial dysplasia, excision by scalpel or laser should be the preferred management modality.

Lesions with malignant potential

Surgical treatment remains the first option; however, follow-up studies of rates of malignant transformation in patients who have and have not undergone surgical removal of leukoplakias are not comparable because of differences in study design and techniques used (scalpel or laser excision, cryotherapy). The study results that are available are highly variable and are sometimes conflicting in their conclusions.[140][141]

Although surgical removal is the most common treatment for dysplastic lesions, either by cold steel or laser, there are no randomised controlled trials comparing surgery to surveillance alone, and there is some evidence that surgery reduces the risk of malignant transformation, but does not completely eliminate the risk.[142]​ Furthermore, recurrence rates after surgery range from 15% to 30%.[143][144]​​ 

Nevertheless, because studies have shown previously undetected carcinomas in leukoplakias, it seems prudent to remove leukoplakias by one recognised modality or another.[80][81]

Treatments may be effective in the resolution of a lesion; however, relapses and adverse effects are common.[145]

Homogeneous leukoplakia

This variant of oral leukoplakia carries the lowest risk of malignant progression, with one study indicating the frequency of malignant development at 3% (compared with 20% of cases of non-homogeneous leukoplakias developing carcinomas).[146]

Where a representative biopsy shows benign disease and absence of epithelial dysplasia, frequent routine observation is an acceptable strategy. Repeat incisional biopsy of any clinical change is necessary, with excision of any confirmed dysplasia. Follow-up is mandatory.

Sublingual leukoplakia (sublingual keratosis)

The anatomical distribution of leukoplakia over the floor of the mouth, sometimes extending to the ventral tongue surface, is often characterised by wide areas of smooth to heterogeneous surface alterations with an increased degree of dysplastic to malignant change, with some stating that nearly 1 in 2 cases may become malignant, although lower rates have also been reported.[128][129]​ Where possible, surgical excision with or without autologous graft coverage is preferred. Alternatives include laser excision.

Speckled leukoplakia

The high risk of significant dysplasia and malignant transformation of these forms of leukoplakia require total excision, with or without autologous graft coverage.[147] Alternative treatment strategies to scalpel excisional surgery involve the use of laser excision.

Proliferative verrucous leukoplakia

Due to extensive areas involved in proliferative verrucous leukoplakia (PVL) and its multifocal nature, it is difficult to eliminate by surgery without compromising function. PVL lesions often recur following surgery. PVL requires careful follow-up and surgery if malignancy is suspected.[148]

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