Treatment of oral leukoplakia with a low-dose of beta-carotene and vitamin C supplements

A randomized controlled trial

Toru Nagao, Saman Warnakulasuriya, Tomoyasu Nakamura, Shinichiro Kato, Keiichi Yamamoto, Hideo Fukano, Koji Suzuki, Kazuo Shimozato, Shuji Hashimoto

Research output: Contribution to journalArticle

19 Citations (Scopus)

Abstract

Management of oral leukoplakia - a potentially malignant disorder - is currently not evidence-based. Of the few randomized trials that have been reported, most have negative data. Therefore, a multi-centre, randomized, double-blind controlled trial (RCT) was undertaken to evaluate the use of low-dose beta-carotene combined with vitamin C supplements for the treatment and to prevent malignant transformation of oral leukoplakia. 46 Japanese participants with oral leukoplakia were allocated randomly either to an experimental arm (10 mg day-1 of beta-carotene and 500 mg day-1 of vitamin C) or placebo arm (50 mg day-1 of vitamin C). Current or ex-smokers within 3 months of cessation were excluded. The supplements were continued over a period of 1 year. The primary endpoint was clinical remission at 1-year and the likelihood of malignant transformation during a 5-year follow-up period as a secondary endpoint. The overall clinical response rate in the experimental arm was 17.4% (4/23) and 4.3% (1/23) in the placebo arm (p = 0.346). During the median 60-month follow-up period, two subjects in the experimental arm and three in the control arm developed oral cancer. Under the intention-to-treat principle, relative risk by supplementing with beta-carotene and vitamin C was 0.77 (95%CI: 0.28-1.89) (p = 0.580) by the Cox proportional hazards model. No unfavorable side-effects were noted. Beta-carotene (10 mg day-1) and vitamin C were neither effective for clinical remission, nor for protection against the development of cancer. Data from this RCT does not support the hypothesis that chemoprevention with this treatment is effective for oral leukoplakia.

Original languageEnglish
Pages (from-to)1708-1717
Number of pages10
JournalInternational Journal of Cancer
Volume136
Issue number7
DOIs
Publication statusPublished - 01-04-2015

Fingerprint

Oral Leukoplakia
beta Carotene
Ascorbic Acid
Randomized Controlled Trials
Placebos
Therapeutics
Mouth Neoplasms
Chemoprevention
Proportional Hazards Models
Neoplasms

All Science Journal Classification (ASJC) codes

  • Oncology
  • Cancer Research

Cite this

Nagao, Toru ; Warnakulasuriya, Saman ; Nakamura, Tomoyasu ; Kato, Shinichiro ; Yamamoto, Keiichi ; Fukano, Hideo ; Suzuki, Koji ; Shimozato, Kazuo ; Hashimoto, Shuji. / Treatment of oral leukoplakia with a low-dose of beta-carotene and vitamin C supplements : A randomized controlled trial. In: International Journal of Cancer. 2015 ; Vol. 136, No. 7. pp. 1708-1717.
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Treatment of oral leukoplakia with a low-dose of beta-carotene and vitamin C supplements : A randomized controlled trial. / Nagao, Toru; Warnakulasuriya, Saman; Nakamura, Tomoyasu; Kato, Shinichiro; Yamamoto, Keiichi; Fukano, Hideo; Suzuki, Koji; Shimozato, Kazuo; Hashimoto, Shuji.

In: International Journal of Cancer, Vol. 136, No. 7, 01.04.2015, p. 1708-1717.

Research output: Contribution to journalArticle

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T1 - Treatment of oral leukoplakia with a low-dose of beta-carotene and vitamin C supplements

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AU - Nagao, Toru

AU - Warnakulasuriya, Saman

AU - Nakamura, Tomoyasu

AU - Kato, Shinichiro

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