Treatment of Disorders of Keratinization with an Oral Stereoisomer of Retinoic Acid

Abstract

Fifty-four patients with treatment-resistant disorders of keratinization were treated with oral 13-cis retinoic acid, a synthetic stereoisomer of retinoic acid, for one to 29 weeks. The dosage varied from 0.5 to 6.0 mg/kg/day. The median patient dosage was 2 mg/kg daily for 8 weeks. Excellent responses have been observed in patients with congenital ichthyosiform erythroderma, lamellar ichthyosis, Darier’s disease, and pityriasis rubra pilaris. However, patients with epidermolytic hyperkeratosis have responded only partially, and patients with psoriasis, nevus comedonicus, X-linked ichthyosis, and Netherton’s syndrome have either not responded or have worsened. Commonly observed side effects included cheilitis, facial dermatitis, xerosis, skin fragility, and conjunctivitis. The erythrocyte sedimentation rate was elevated in 7 patients; no other abnormal laboratory tests, including liver function tests, were noted in this group of patients. The mechanism by which this synthetic retinoid alters these disease states is not clear but may be related to the observed ability of vitamin A to affect glycoprotein synthesis and epithelial differentiation. Our results indicate that synthetic retinoids, such as 13-cis retinoic acid, may represent a potent new class of drugs in the treatment of cutaneous disease.