Serial controlled N-of-1 trials of topical vitamin E as prophylaxis for chemotherapy-induced oral mucositis in paediatric patients.
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.
Sung
L
L
Department of Paediatrics, University of Toronto, Toronto, Canada. lillian.sung@sickkids.ca
Tomlinson
G A
GA
Greenberg
M L
ML
Koren
G
G
Judd
P
P
Ota
S
S
Feldman
B M
BM
D000287:Administration, Topical; D000293:Adolescent; D000893:Anti-Inflammatory Agents / Q000008:administration & dosage*; D000903:Antibiotics, Antineoplastic / Q000009:adverse effects*; D002648:Child; D004311:Double-Blind Method; D004317:Doxorubicin / Q000009:adverse effects*; D005240:Feasibility Studies; D005260:Female; D006801:Humans; D008297:Male; D013280:Stomatitis / Q000139:chemically induced / Q000517:prevention & control*; D016896:Treatment Outcome; D014810:Vitamin E / Q000008:administration & dosage*
D000893:Anti-Inflammatory Agents; D000903:Antibiotics, Antineoplastic; D014810:Vitamin E; D004317:Doxorubicin