Mycophenolate mofetil: a novel immunosuppressant in the treatment of dystrophic epidermolysis bullosa, a randomized controlled trial

J Dermatolog Treat. 2013 Dec;24(6):422-6. doi: 10.3109/09546634.2013.768327. Epub 2013 May 21.


Background: No effective treatment has been found for epidermolysis bullosa dystrophica (EBD).

Objective: To evaluate the efficacy and safety mycophenolate mofetil (MMF) in treating EBD.

Methods: This randomized controlled double-blinded study included 35 patients with severe generalized EBD. Patients were randomly divided into two groups: group I (18 patients) received cyclosporine therapy (5 mg/kg/day) and group II (17 patients) received MMF therapy (500-1500 mg/day). Clinical assessment was made weekly for 3 months from the start of the treatment. Patients were assessed by measuring the extent of the disease, the % of improvement, assessing the number of new blister formation and the time of complete healing of new blisters. Side effects were recorded when detected.

Results: The % of improvement in the disease extent was statistically significantly higher (p = 0.009) in group I (mean ± SD: 59.21 ± 22.676) than in group II (mean ± SD: 44.03 ± 25.71). As regards the number of new blisters and the rate of healing of blisters, there was no statistically significant difference between both groups (p = 0.693 and 0.404, respectively). No serious side effects were reported.

Conclusion: MMF seems to be a good therapeutic option for the long-term treatment of EBD, it can be a good alternative for patients who cannot tolerate cyclosporine.

Publication types

  • Randomized Controlled Trial

MeSH terms

  • Adolescent
  • Adult
  • Child
  • Child, Preschool
  • Cyclosporine / therapeutic use
  • Double-Blind Method
  • Epidermolysis Bullosa Dystrophica / drug therapy*
  • Epidermolysis Bullosa Dystrophica / pathology
  • Female
  • Humans
  • Immunosuppressive Agents / adverse effects
  • Immunosuppressive Agents / therapeutic use*
  • Infant
  • Kidney Transplantation
  • Male
  • Mycophenolic Acid / adverse effects
  • Mycophenolic Acid / analogs & derivatives*
  • Mycophenolic Acid / therapeutic use
  • Survival Rate
  • Treatment Outcome
  • Young Adult


  • Immunosuppressive Agents
  • Cyclosporine
  • Mycophenolic Acid