Therapeutic efficacy of phenytoin in recessive dystrophic epidermolysis. A comparison of short- and long-term treatment.

To assess the potential short- and long-term efficacy of phenytoin in treating recessive dystrophic epidermolysis bullosa (RDEB), we treated 22 patients with therapeutic doses of oral phenytoin (blood level, 8 to 10 micrograms/mL) for periods ranging from eight to 99 weeks. Fourteen (64%) of the 22 patients had greater than a 40% mean percentage decrease in blistering of the skin. Nine (41%) of the 22 patients were treated for longer than 75 weeks, and seven (78%) of these nine patients had a mean decrease in blistering of at least 40% during phenytoin therapy. These data, along with previous reports, suggest that phenytoin has therapeutic efficacy in RDEB.