title: High-dose intravenous immunoglobulin therapy for scleromyxoedema: a prospective open-label clinical trial using an objective score of clinical evaluation system.
abstract: BACKGROUND
  Scleromyxoedema is a primary fibro-mucinosis whose therapy is still challenging.
  
  
  OBJECTIVE
  To evaluate the safety and efficacy of high-dose intravenous immunoglobulin (IVIg) for the management of scleromyxoedema prospectively using an objective score.
  
  
  METHODS
  In a prospective open-label study, IVIg was administered to eight patients with scleromyxoedema in a dose of 2 g/kg per month. The patients were followed-up to a minimum of 6 months, and their disease activity and response to treatment were assessed using the Physician's Global Assessment of disease severity (PGA) and a modified objective skin scoring system for patients with scleroderma (modified Rodnan score system for scleromyxoedema or mRSSS). We used a stringent statistical nonparametric test, the Mann-Whitney U-test, to assess the changes in the mRSSS following therapy with IVIg.
  
  
  RESULTS
  Eight patients were included (five males) with a mean age of 59 years. Mean duration of scleromyxoedema was 19 months (6-37 months). The mean duration of treatment was 36.5 months (range 7-74 months).The patients were followed-up for a minimum of 15 months to a maximum of 87 months (mean 44 months). The mean baseline mRSSS of our cohort was 82.38 (37-145, SD 40.763) at the start of treatment, and this significantly decreased to 14.88 (0-37, SD 12.988) (P = 0.012) at the last clinical evaluation with a decrease in mRSSS of 81.6%. No considerable side effects were noted. Paraproteinemia remained substantially unchanged. In six cases, maintenance infusions were required to preserve disease control, while in two patients, therapy was stopped after 7 and 11 months. Relapses, however, occurred, respectively, after 6 and 25 months.
  
  
  CONCLUSIONS
  Our study is the first to demonstrate a statistically clinical objective improvement of clinical symptoms of scleromyxoedema with IVIg.
authors.affiliation: Section of Dermatology, DISSAL, University of Genoa and S. Martino University Hospital, Genoa, Italy.
authors.forename: A
authors.initials: A
authors.lastname: Guarneri
authors.affiliation: Section of Dermatology, DISSAL, University of Genoa and S. Martino University Hospital, Genoa, Italy.
authors.forename: M
authors.initials: M
authors.lastname: Cioni
authors.affiliation: Unit of Dermatology, Department of Medical Sciences and Public Health, University of Cagliari, Cagliari, Italy.
authors.forename: F
authors.initials: F
authors.lastname: Rongioletti
mesh_terms: D004305:Dose-Response Relationship, Drug; D005260:Female; D006801:Humans; D016756:Immunoglobulins, Intravenous / Q000008:administration & dosage* / Q000627:therapeutic use; D008297:Male; D008875:Middle Aged; D011446:Prospective Studies; D053718:Scleromyxedema / Q000188:drug therapy* / Q000473:pathology
chemical_list: D016756:Immunoglobulins, Intravenous