title: Randomized controlled open-label trial of four treatment regimens for pemphigus vulgaris.
abstract: BACKGROUND
  Pemphigus is a severe autoimmune blistering disease affecting the skin and mucosa. Mortality is high in the absence of treatment. Nowadays, treatment is based mainly on corticosteroids and cytotoxic drugs; however, because of the rarity of the disease worldwide, there is not yet a standard treatment based on randomized controlled trials, and the treatment used is based mainly on the experience of experts.
  
  
  OBJECTIVE
  The aim of this study was to compare the efficacy and safety of 4 treatment regimens for pemphigus vulgaris: prednisolone alone, prednisolone plus azathioprine, prednisolone plus mycophenolate mofetil, and prednisolone plus intravenous cyclophosphamide pulse therapy.
  
  
  METHODS
  One hundred twenty new cases of pemphigus vulgaris were enrolled. These patients were randomly allocated into 1 of 4 treatment groups (each comprising 30 patients) and received prednisolone (P), prednisolone and azathioprine (P/A), prednisolone and mycophenolate mofetil (P/MM), and prednisolone and intravenous cyclophosphamide pulse therapy (P/PC). They were followed up for 1 year at the Pemphigus Research Unit.
  
  
  RESULTS
  In groups P, P/A, P/MM, and P/PC, 23 (76.5%), 24 (80%), 21 (70%), and 22 (73.3%) of the patients, respectively, followed the regimen for the full 1-year period. The mean total dose of prednisolone administered in groups P, P/A, P/MM, and P/PC was 11631 mg (standard deviation [SD] = 7742), 7712 mg (SD = 955), 9798 mg (SD = 3995), and 8276 mg (SD = 810), respectively. The mean total dose of prednisolone in group P (prednisolone alone) was 11,631 mg, The mean total dose of prednisolone in the 3 cytotoxic groups was 8652 mg. By using analysis of variance, the difference was statistically significant (P = .047). In the cytotoxic groups, there was a significant difference between the P/A and P/MM groups (P = .007), but not between P/A and P/PC (P = .971), and P/MM and P/PC (P = .670). Side effects were not significantly different among the 4 groups.
  
  
  CONCLUSIONS
  Larger sample sizes and blind design are suggested for future studies.
  
  
  CONCLUSIONS
  The efficacy of prednisolone is enhanced when it is combined with a cytotoxic drug. The most efficacious cytotoxic drug to reduce steroid was found to be azathioprine, followed by cyclophosphamide (pulse therapy), and mycophenolate mofetil.
authors.affiliation: Pemphigus Research Unit, Department of Dermatology, Tehran University for Medical Sciences, Razi Hospital, Tehran, Iran. cheyda@davatchi.net
authors.forename: Cheyda
authors.initials: C
authors.lastname: Chams-Davatchi
authors.forename: Nafiseh
authors.initials: N
authors.lastname: Esmaili
authors.forename: Maryam
authors.initials: M
authors.lastname: Daneshpazhooh
authors.forename: Mahin
authors.initials: M
authors.lastname: Valikhani
authors.forename: Kamran
authors.initials: K
authors.lastname: Balighi
authors.forename: Zahra
authors.initials: Z
authors.lastname: Hallaji
authors.forename: Masoumeh
authors.initials: M
authors.lastname: Barzegari
authors.forename: Maryam
authors.initials: M
authors.lastname: Akhyani
authors.forename: S Zahra
authors.initials: SZ
authors.lastname: Ghodsi
authors.forename: Hassan
authors.initials: H
authors.lastname: Seirafi
authors.forename: Mohammad-Javad Tabrizi
authors.initials: MJ
authors.lastname: Nazemi
authors.forename: Hossein
authors.initials: H
authors.lastname: Mortazavi
authors.forename: Mostafa
authors.initials: M
authors.lastname: Mirshams-Shahshahani
mesh_terms: D000328:Adult; D001379:Azathioprine / Q000627:therapeutic use*; D003520:Cyclophosphamide / Q000627:therapeutic use*; D005260:Female; D006801:Humans; D007166:Immunosuppressive Agents / Q000627:therapeutic use*; D008297:Male; D008875:Middle Aged; D009173:Mycophenolic Acid / Q000031:analogs & derivatives* / Q000627:therapeutic use; D010392:Pemphigus / Q000188:drug therapy*; D011239:Prednisolone / Q000627:therapeutic use*; D016896:Treatment Outcome
chemical_list: D007166:Immunosuppressive Agents; D003520:Cyclophosphamide; D011239:Prednisolone; D009173:Mycophenolic Acid; D001379:Azathioprine