title: Mycophenolate mofetil: a novel immunosuppressant in the treatment of dystrophic epidermolysis bullosa, a randomized controlled trial.
abstract: 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.
  
  
  CONCLUSIONS
  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.
authors: Dermatology Department, Faculty of Medicine, Cairo University , Cairo , Egypt.
authors: Mohammad A
authors: MA
authors: El-Darouti
authors: Marwa M
authors: MM
authors: Fawzy
authors: Iman M
authors: IM
authors: Amin
authors: Rania M
authors: RM
authors: Abdel Hay
authors: Rehab A
authors: RA
authors: Hegazy
authors: Dalia M
authors: DM
authors: Abdel Halim
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D002675:Child, Preschool; D016572:Cyclosporine / Q000627:therapeutic use; D004311:Double-Blind Method; D016108:Epidermolysis Bullosa Dystrophica / Q000188:drug therapy* / Q000473:pathology; D005260:Female; D006801:Humans; D007166:Immunosuppressive Agents / Q000009:adverse effects / Q000627:therapeutic use*; D007223:Infant; D016030:Kidney Transplantation; D008297:Male; D009173:Mycophenolic Acid / Q000009:adverse effects / Q000031:analogs & derivatives* / Q000627:therapeutic use; D015996:Survival Rate; D016896:Treatment Outcome; D055815:Young Adult
chemical_list: D007166:Immunosuppressive Agents; D016572:Cyclosporine; D009173:Mycophenolic Acid