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

• Published in: The Journal of dermatological treatment Vol. 24; no. 6; pp. 422 - 426
• Main Authors: El-Darouti, Mohammad A., Fawzy, Marwa M., Amin, Iman M., Abdel Hay, Rania M., Hegazy, Rehab A., Abdel Halim, Dalia M.
• Format: Journal Article
• Language: English
• Published: Oslo Informa Healthcare USA on behalf of Informa UK Ltd 01-12-2013; Taylor & Francis
• Subjects: Adolescent; Adult; Biological and medical sciences; Bullous diseases of the skin; Child; Child, Preschool; Cyclosporine; Cyclosporine - therapeutic use; Dermatology; Double-Blind Method; epidermolysis bullosa dystrophica; Epidermolysis Bullosa Dystrophica - drug therapy; Epidermolysis Bullosa Dystrophica - pathology; Female; Humans; Immunosuppressive Agents - adverse effects; Immunosuppressive Agents - therapeutic use; Infant; Kidney Transplantation; Male; Medical sciences; mycophenolate mofetil; Mycophenolic Acid - adverse effects; Mycophenolic Acid - analogs & derivatives; Mycophenolic Acid - therapeutic use; Survival Rate; Treatment Outcome; Young Adult; Bullous dermatosis; Immunopathology; Skin disease; Calcineurin inhibitor; Epidermolysis bullosa; epidermolysis bullosa dystrophica; Enzyme; IMP dehydrogenase; Enzyme inhibitor; Autoimmune disease; Cyclosporine; Genetic disease; Immunomodulator; Randomization; Treatment; Clinical trial; Ciclosporin; Oxidoreductases; Immunosuppressive agent; Mycophenolate mofetil; Dystrophic epidermolysis bullosa
• ISSN: 0954-6634; 1471-1753
• DOI: 10.3109/09546634.2013.768327

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.