Serial controlled N-of-1 trials of topical vitamin E as prophylaxis for chemotherapy-induced oral mucositis in paediatric patients

Eur J Cancer. 2007 May;43(8):1269-75. doi: 10.1016/j.ejca.2007.02.001. Epub 2007 Mar 23.


The objectives were (1) to determine whether in children undergoing doxorubicin-containing chemotherapy, topical vitamin E decreases an objective measurement of oral mucositis compared to placebo, and (2) to assess the feasibility of an innovative trial design in paediatric cancer, combining N-of-1 trials using Bayesian meta-analysis. We conducted a series of N-of-1, double-blinded, randomised controlled trials in children > or = 6 years of age receiving repeated cycles of identical doxorubicin-containing chemotherapy. Each study cycle was followed by topical vitamin E (800 mg) or placebo. We enroled 16 children and 45 post chemotherapy cycles were randomised to vitamin E (N=22) or placebo (N=23). There was no difference in objective mucositis scores with a mean score of 0.2 with vitamin E and 0.3 with placebo. Topical vitamin E does not reduce doxorubicin-induced oral mucositis in children. The use of N-of-1 studies and Bayesian meta-analysis may facilitate the study of some therapies in paediatric oncology.

Publication types

  • Randomized Controlled Trial
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Administration, Topical
  • Adolescent
  • Anti-Inflammatory Agents / administration & dosage*
  • Antibiotics, Antineoplastic / adverse effects*
  • Child
  • Double-Blind Method
  • Doxorubicin / adverse effects*
  • Feasibility Studies
  • Female
  • Humans
  • Male
  • Stomatitis / chemically induced
  • Stomatitis / prevention & control*
  • Treatment Outcome
  • Vitamin E / administration & dosage*


  • Anti-Inflammatory Agents
  • Antibiotics, Antineoplastic
  • Vitamin E
  • Doxorubicin