title: A multicenter, randomized, double-blinded trial of pyridostigmine in postpolio syndrome.
abstract: BACKGROUND
  Postpoliomyelitis syndrome (PPS) is likely due to degeneration and dysfunction of terminal axons of enlarged postpolio motor units. Age-related decline in growth hormone and insulin-like growth factor (IGF-I) may be a contributing factor. Neuromuscular junction abnormalities and decreased IGF-I levels may respond to the anticholinesterase pyridostigmine, with consequent improvement in strength, fatigue, and quality of life.
  
  
  OBJECTIVE
  To determine the effect of pyridostigmine in PPS on health-related quality of life, isometric muscle strength, fatigue, and serum IGF-I levels; and to assess the safety of pyridostigmine in PPS.
  
  
  METHODS
  The study was a multicenter, randomized, double-blinded, placebo-controlled trial of a 6-month course of pyridostigmine 60 mg three times per day in 126 PPS patients. The primary data analysis compared mean changes of outcomes between treatment and control groups at 6 months using an intention to treat approach. Secondary analyses included a comparison of outcomes at 6 and 10 weeks, and in compliant patients.
  
  
  RESULTS
  The study showed no significant differences in pyridostigmine and placebo-treated patients with regard to changes in quality of life, isometric strength, fatigue, and IGF-I serum levels at 6 months in the primary analysis and in compliant patients. There were no differences in outcomes at 6 and 10 weeks between groups. However, very weak muscles (1 to 25% predicted normal at baseline) were somewhat stronger (p = 0.10, 95% CI of difference -9.5 to 73.3%), and in compliant patients IGF-I was somewhat increased (p = 0.15, 95% CI of difference -6.4 to 44.8 ng/mL) at 6 months with the medication. Pyridostigmine was generally well tolerated.
  
  
  CONCLUSIONS
  This study showed no significant differences between pyridostigmine and placebo-treated PPS patients on measures of quality of life, isometric strength, fatigue, and serum IGF-I.
authors.affiliation: Department of Neurology, Montreal Neurological Institute and Hospital, Quebec, Canada.
authors.forename: D A
authors.initials: DA
authors.lastname: Trojan
authors.forename: J P
authors.initials: JP
authors.lastname: Collet
authors.forename: S
authors.initials: S
authors.lastname: Shapiro
authors.forename: B
authors.initials: B
authors.lastname: Jubelt
authors.forename: R G
authors.initials: RG
authors.lastname: Miller
authors.forename: J C
authors.initials: JC
authors.lastname: Agre
authors.forename: T L
authors.initials: TL
authors.lastname: Munsat
authors.forename: D
authors.initials: D
authors.lastname: Hollander
authors.forename: R
authors.initials: R
authors.lastname: Tandan
authors.forename: C
authors.initials: C
authors.lastname: Granger
authors.forename: A
authors.initials: A
authors.lastname: Robinson
authors.forename: L
authors.initials: L
authors.lastname: Finch
authors.forename: T
authors.initials: T
authors.lastname: Ducruet
authors.forename: N R
authors.initials: NR
authors.lastname: Cashman
mesh_terms: D000368:Aged; D004311:Double-Blind Method; D005260:Female; D006801:Humans; D008297:Male; D008875:Middle Aged; D016262:Postpoliomyelitis Syndrome / Q000188:drug therapy*; D011729:Pyridostigmine Bromide / Q000627:therapeutic use*
chemical_list: D011729:Pyridostigmine Bromide