Design This was a randomised controlled trial (RCT) of the treatment of oral leukoplakia with the carotenoid lycopene. Intervention A total of 58 patients received either 8 mg oral lycopene in two doses daily (n = 20), 4 mg oral lycopene in two doses daily (n = 18) or placebo capsules (n = 18), for a 3-month period. Progress of patients was followed for a further 2 months. Outcome measures An objective clinical response, evaluated by bidimensional measurement of the lesion and colour photography, was classified as complete, partial, stable or progression. Histological status was categorised and ranked as normal (0), atypical hyperplasia (1), mild dysplasia (2), moderate dysplasia (3) or severe dysplasia (4). Histological response was then described by the change in rank, for example, from moderate dysplasia (3) to atypical hyperplasia (1) would indicate an improvement of 2 units. Results There was no significant difference in the clinical response of people who took 8 mg lycopene compared with those taking 4 mg lycopene. The clinical responses measured in both these groups were significantly greater, however, than those in the control group (Po0.01). The response, assessed histologically, after the 8-mg lycopene treatment was significantly better than that from 4 mg lycopene (Po0.05) and than the response seen in the control group (Po0.001). Patients taking 4 mg lycopene also responded significantly better than those in the control group (Po0.05).Conclusions Oral lycopene appears, from this small RCT conducted over 5 months, to be effective in the treatment and management of oral leukoplakia.
CommentaryOral leukoplakia is a diagnosis given to a white patch that cannot be categorised. Once a histological diagnosis is made, it is useful to refer to it either by causal factor, for example, candidal leukoplakia, or by degree of dysplasia. 1 An international meeting clarifying these definitions reported its findings and also suggested a method of staging these lesions. 1 This has been further commented upon by Van der Waal and Axell 2 and Schepman and van der Waal 3 and it is a pity that this study did not adopt this methodology. This staging not only includes the different forms of dysplasia but also takes into account the size of the lesion.A recent Cochrane systematic review on treatment of leukoplakia also provides some guidelines for future RCT in this field. 4 In their paper, Lodi et al 5 point out that no RCT conducted to date exceeds 15 months and yet there is evidence to show that malignant transformation increases with duration of followup. 6 They also point out that many researchers use outcomes other than malignant transformation, for example, histological diagnosis or resolution of lesion. There are problems with using these outcomes because there is little evidence for their predictive value and it has been shown that outcomes such as dysplasia are subject to high observer variation. 7,8 Many journals have now adopted the CONSORT guidelines for reporting of RCT: this study would have been easier to foll...