A multicenter, randomized, double-blinded trial of pyridostigmine in postpolio syndrome

D. A. Trojan, J. P. Collet, S. Shapiro, B. Jubelt, R. G. Miller, J. C. Agre, T. L. Munsat, D. Hollander, R. Tandan, C. Granger, A. Robinson, L. Finch, T. Ducruet, N. R. Cashman

Research output: Contribution to journalArticlepeer-review

68 Scopus citations

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. Objectives: 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.

Original languageEnglish (US)
Pages (from-to)1225-1233
Number of pages9
JournalNeurology
Volume53
Issue number6
DOIs
StatePublished - Oct 12 1999

Keywords

  • Insulin-like growth factor-I
  • Poliomyelitis
  • Pyridostigmine
  • Quality of life
  • Strength

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