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 (P<0.01). The response, assessed histologically, after the 8-mg lycopene treatment was significantly better than that from 4 mg lycopene (P<0.05) and than the response seen in the control group (P<0.001). Patients taking 4 mg lycopene also responded significantly better than those in the control group (P<0.05).
Conclusions: Oral lycopene appears, from this small RCT conducted over 5 months, to be effective in the treatment and management of oral leukoplakia.