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 journalArticle

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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
StatePublished - Oct 12 1999

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Pyridostigmine Bromide
Postpoliomyelitis Syndrome
Insulin-Like Growth Factor I
Quality of Life
Fatigue
Placebos
Serum
Muscle Fatigue
Neuromuscular Junction
Cholinesterase Inhibitors
Presynaptic Terminals
Muscle Strength
Growth Hormone
Safety
Muscles
Control Groups

Keywords

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

Cite this

Trojan, D. A., Collet, J. P., Shapiro, S., Jubelt, B., Miller, R. G., Agre, J. C., ... Cashman, N. R. (1999). A multicenter, randomized, double-blinded trial of pyridostigmine in postpolio syndrome. Neurology, 53(6), 1225-1233.

A multicenter, randomized, double-blinded trial of pyridostigmine in postpolio syndrome. / Trojan, D. A.; Collet, J. P.; Shapiro, S.; Jubelt, B.; Miller, R. G.; Agre, J. C.; Munsat, T. L.; Hollander, D.; Tandan, R.; Granger, C.; Robinson, A.; Finch, L.; Ducruet, T.; Cashman, N. R.

In: Neurology, Vol. 53, No. 6, 12.10.1999, p. 1225-1233.

Research output: Contribution to journalArticle

Trojan, DA, Collet, JP, Shapiro, S, Jubelt, B, Miller, RG, Agre, JC, Munsat, TL, Hollander, D, Tandan, R, Granger, C, Robinson, A, Finch, L, Ducruet, T & Cashman, NR 1999, 'A multicenter, randomized, double-blinded trial of pyridostigmine in postpolio syndrome', Neurology, vol. 53, no. 6, pp. 1225-1233.
Trojan DA, Collet JP, Shapiro S, Jubelt B, Miller RG, Agre JC et al. A multicenter, randomized, double-blinded trial of pyridostigmine in postpolio syndrome. Neurology. 1999 Oct 12;53(6):1225-1233.
Trojan, D. A. ; Collet, J. P. ; Shapiro, S. ; Jubelt, B. ; Miller, R. G. ; Agre, J. C. ; Munsat, T. L. ; Hollander, D. ; Tandan, R. ; Granger, C. ; Robinson, A. ; Finch, L. ; Ducruet, T. ; Cashman, N. R. / A multicenter, randomized, double-blinded trial of pyridostigmine in postpolio syndrome. In: Neurology. 1999 ; Vol. 53, No. 6. pp. 1225-1233.
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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.",
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T1 - A multicenter, randomized, double-blinded trial of pyridostigmine in postpolio syndrome

AU - Trojan, D. A.

AU - Collet, J. P.

AU - Shapiro, S.

AU - Jubelt, B.

AU - Miller, R. G.

AU - Agre, J. C.

AU - Munsat, T. L.

AU - Hollander, D.

AU - Tandan, R.

AU - Granger, C.

AU - Robinson, A.

AU - Finch, L.

AU - Ducruet, T.

AU - Cashman, N. R.

PY - 1999/10/12

Y1 - 1999/10/12

N2 - 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.

AB - 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.

KW - Insulin-like growth factor-I

KW - Poliomyelitis

KW - Pyridostigmine

KW - Quality of life

KW - Strength

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