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  • Title: Chemoprevention of squamous cell carcinoma in recessive dystrophic epidermolysis bullosa: results of a phase 1 trial of systemic isotretinoin.
    Author: Fine JD, Johnson LB, Weiner M, Stein A, Suchindran C.
    Journal: J Am Acad Dermatol; 2004 Apr; 50(4):563-71. PubMed ID: 15034505.
    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.
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