Chemoprevention of squamous cell carcinoma in recessive dystrophic epidermolysis bullosa: results of a phase 1 trial of systemic isotretinoin.
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.
Fine
Jo-David
JD
National Epidermolysis Bullosa Registry, Lexington, Kentucky, USA. ProfMoose@aol.com
Johnson
Lorraine B
LB
Weiner
Madeline
M
Stein
Amy
A
Suchindran
Chirayath
C
D000293:Adolescent; D000328:Adult; D000368:Aged; D002294:Carcinoma, Squamous Cell / Q000209:etiology / Q000517:prevention & control*; D003879:Dermatologic Agents / Q000009:adverse effects / Q000627:therapeutic use*; D016108:Epidermolysis Bullosa Dystrophica / Q000150:complications* / Q000188:drug therapy; D005260:Female; D006801:Humans; D015474:Isotretinoin / Q000009:adverse effects / Q000627:therapeutic use*; D008297:Male; D008875:Middle Aged; D012307:Risk Factors; D012878:Skin Neoplasms / Q000209:etiology / Q000517:prevention & control*
D003879:Dermatologic Agents; D015474:Isotretinoin