Chemoprevention of squamous cell carcinoma in recessive dystrophic epidermolysis bullosa: results of a phase 1 trial of systemic isotretinoin - 24/08/11
Abstract |
Background |
Patients with recessive dystrophic epidermolysis bullosa (RDEB) are at high risk of developing squamous cell carcinoma on or after midadolescence, and most patients die of metastatic squamous cell carcinoma within 5 years of diagnosis of their first squamous cell carcinoma.
Objective |
We sought to determine whether isotretinoin can be safely administered to patients with RDEB as a possible chemopreventive agent.
Methods |
A total of 20 patients with RDEB aged 15 years or older were treated daily for 8 months with isotretinoin (with a targeted dosage of 0.5 mg/kg/d).
Results |
No unusual adverse reactions were noted in this patient population. Several patients experienced reduced blistering at lower doses and increased mechanical fragility at maintenance dosage.
Conclusions |
Isotretinoin, at least up to a dosage of 0.5 mg/kg/d, may be safely used in patients with RDEB. Although increased fragility may occur, patients tolerated this drug well and were receptive to its long-term use for possible chemoprevention of cancer. Whether such an effect will occur is yet to be proven.
Le texte complet de cet article est disponible en PDF.Abbreviations : EB, FDA, NEBR, RDEB, RDEB-HS, SCC, UNC
Plan
Supported by federal grants from the National Institute of Arthritis, Musculoskeletal and Skin Diseases (NO1 AR72233; K24 AR02098), and the Food and Drug Administration (FD-R-001796-01) (Dr Fine, all; Dr Stein, FD-R-001796-01). Roche Laboratories, through its Investigator-Initiated Research Program, provided medication to each of our participants. A National Institutes of Health–supported General Clinical Research Center at the University of North Carolina at Chapel Hill provided resources. Conflicts of interest: None identified. |
Vol 50 - N° 4
P. 563-571 - avril 2004 Retour au numéroBienvenue sur EM-consulte, la référence des professionnels de santé.
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