title: A randomized, double-blind, placebo-controlled trial of photopheresis in systemic sclerosis.
abstract: BACKGROUND
  Systemic sclerosis is a multisystemic connective tissue disease with marked involvement of the skin and joints for which few effective evidence based therapies are available. To further investigate the efficacy of extracorporeal photochemotherapy on early aggressive cutaneous disease, a randomized, double-blind, placebo-controlled trial was performed.
  
  
  OBJECTIVE
  Our aim was to evaluate the efficacy of photopheresis in the treatment of patients with systemic sclerosis (scleroderma).
  
  
  METHODS
  This randomized, double-blind, placebo-controlled clinical trial was conducted at 16 investigational sites in the United States, Canada, and Europe. Sixty-four patients with typical clinical and histologic findings of scleroderma, of less than 2 years' duration, were studied. Patients did not receive any other concomitant treatment for scleroderma. Patients were randomized to receive either active or sham photopheresis treatment on two consecutive days monthly for 12 months. Severity of skin (skin scores assessed in 22 body regions) and joint involvement (60 joints examined for contractures) were assessed on a monthly basis.
  
  
  RESULTS
  A statistically significant improvement in skin scores as compared with baseline was observed at 6 months (P = .0024) and 12 months (P = .008) among those who received active photopheresis, but not among those who received sham photopheresis. Comparison of skin scores between the two study arms did not achieve statistical significance because of the small sample size of the study arms. Joint involvement was also significantly improved after 6 months (P = .002) and 12 months (P = .001) of active photopheresis when compared with baseline.
  
  
  CONCLUSIONS
  The study lacks sufficient statistical power to reveal a significant difference in skin and joint manifestations between the active and sham photopheresis arms.
  
  
  CONCLUSIONS
  Photopheresis induced significant improvement of skin and joint involvement in patients with scleroderma of recent onset; however, any effect when compared with sham treatment and a possible placebo effect may be modest.
authors.affiliation: Department of Dermatology, Medical University of Vienna, Vienna, Austria. robert.knobler@meduniwien.ac.at
authors.forename: Robert M
authors.initials: RM
authors.lastname: Knobler
authors.forename: Lars E
authors.initials: LE
authors.lastname: French
authors.forename: Youn
authors.initials: Y
authors.lastname: Kim
authors.forename: Emil
authors.initials: E
authors.lastname: Bisaccia
authors.forename: Winfried
authors.initials: W
authors.lastname: Graninger
authors.forename: Hesam
authors.initials: H
authors.lastname: Nahavandi
authors.forename: Frank J
authors.initials: FJ
authors.lastname: Strobl
authors.forename: Edward
authors.initials: E
authors.lastname: Keystone
authors.forename: Marilyn
authors.initials: M
authors.lastname: Mehlmauer
authors.forename: Alain H
authors.initials: AH
authors.lastname: Rook
authors.forename: Irwin
authors.initials: I
authors.lastname: Braverman
mesh_terms: D000328:Adult; D004311:Double-Blind Method; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D017893:Photopheresis; D010919:Placebos; D012595:Scleroderma, Systemic / Q000188:drug therapy*; D016896:Treatment Outcome
chemical_list: D010919:Placebos