A randomized, placebo-controlled trial of creatine in children with spinal muscular atrophy

Brenda L. Wong, Linda S. Hynan, Susan T. Iannaccone, S. T. Iannaccone, K. Rabb, D. Carman, K. Harris, A. Morton, L. S. Hynan, J. S. Reisch, J. Smith, J. C. Webster, C. Goldsmith, A. R. Gorman, P. N. Schochet, P. M. Luckett, P. Walters, B. L. Wong, F. J. Samaha, A. FritchP. J. Morehart, B. S. Russman, K. Zilke, S. Sienko-Thomas, R. T. Leshner, J. Mayhew, B. Grillo, S. A. Smith, J. Stout, A. Nugent, J. T. Parke, A. Clark, C. P. Earnest, R. J. Kryscio, L. Timberman, C. S. Moy, C. Newton

Research output: Contribution to journalArticle

13 Citations (Scopus)

Abstract

OBJECTIVE: To determine whether oral creatine produces positive changes in any of 4 outcomes in children with spinal muscular atrophy (SMA). METHODS: The authors conducted a randomized, double-blind, placebo-controlled trial on 55 patients aged 2-18 years with SMA. Patients aged younger than 5 years received 2 g/day of creatine/placebo for 6 months. Patients aged 5 years and older received 5 g/day. The primary outcome measure was the Gross Motor Function Measure (GMFM). Secondary outcome measures were Quantitative Muscle Testing (QMT), Parent Questionnaire for the PedsQL™ Neuromuscular Module (QOL), and Pulmonary Function Tests (PFT). RESULTS: Forty of the 55 patients completed the protocol. There was no significant difference in the 4 outcome measures between creatine and placebo groups. CONCLUSIONS: Under the experimental conditions of our study, creatine supplementation for 6 months did not improve motor function, muscle strength, pulmonary function, or quality of life in children with SMA.

Original languageEnglish (US)
Pages (from-to)101-110
Number of pages10
JournalJournal of Clinical Neuromuscular Disease
Volume8
Issue number3
DOIs
StatePublished - Mar 2007

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Spinal Muscular Atrophy
Creatine
Randomized Controlled Trials
Placebos
Outcome Assessment (Health Care)
Respiratory Function Tests
Muscle Strength
Quality of Life
Muscles
Lung

Keywords

  • Creatine
  • Spinalmuscular atrophy (SMA)

ASJC Scopus subject areas

  • Clinical Neurology

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A randomized, placebo-controlled trial of creatine in children with spinal muscular atrophy. / Wong, Brenda L.; Hynan, Linda S.; Iannaccone, Susan T.; Iannaccone, S. T.; Rabb, K.; Carman, D.; Harris, K.; Morton, A.; Hynan, L. S.; Reisch, J. S.; Smith, J.; Webster, J. C.; Goldsmith, C.; Gorman, A. R.; Schochet, P. N.; Luckett, P. M.; Walters, P.; Wong, B. L.; Samaha, F. J.; Fritch, A.; Morehart, P. J.; Russman, B. S.; Zilke, K.; Sienko-Thomas, S.; Leshner, R. T.; Mayhew, J.; Grillo, B.; Smith, S. A.; Stout, J.; Nugent, A.; Parke, J. T.; Clark, A.; Earnest, C. P.; Kryscio, R. J.; Timberman, L.; Moy, C. S.; Newton, C.

In: Journal of Clinical Neuromuscular Disease, Vol. 8, No. 3, 03.2007, p. 101-110.

Research output: Contribution to journalArticle

Wong, BL, Hynan, LS, Iannaccone, ST, Iannaccone, ST, Rabb, K, Carman, D, Harris, K, Morton, A, Hynan, LS, Reisch, JS, Smith, J, Webster, JC, Goldsmith, C, Gorman, AR, Schochet, PN, Luckett, PM, Walters, P, Wong, BL, Samaha, FJ, Fritch, A, Morehart, PJ, Russman, BS, Zilke, K, Sienko-Thomas, S, Leshner, RT, Mayhew, J, Grillo, B, Smith, SA, Stout, J, Nugent, A, Parke, JT, Clark, A, Earnest, CP, Kryscio, RJ, Timberman, L, Moy, CS & Newton, C 2007, 'A randomized, placebo-controlled trial of creatine in children with spinal muscular atrophy', Journal of Clinical Neuromuscular Disease, vol. 8, no. 3, pp. 101-110. https://doi.org/10.1097/CND.0b013e3180315c99
Wong, Brenda L. ; Hynan, Linda S. ; Iannaccone, Susan T. ; Iannaccone, S. T. ; Rabb, K. ; Carman, D. ; Harris, K. ; Morton, A. ; Hynan, L. S. ; Reisch, J. S. ; Smith, J. ; Webster, J. C. ; Goldsmith, C. ; Gorman, A. R. ; Schochet, P. N. ; Luckett, P. M. ; Walters, P. ; Wong, B. L. ; Samaha, F. J. ; Fritch, A. ; Morehart, P. J. ; Russman, B. S. ; Zilke, K. ; Sienko-Thomas, S. ; Leshner, R. T. ; Mayhew, J. ; Grillo, B. ; Smith, S. A. ; Stout, J. ; Nugent, A. ; Parke, J. T. ; Clark, A. ; Earnest, C. P. ; Kryscio, R. J. ; Timberman, L. ; Moy, C. S. ; Newton, C. / A randomized, placebo-controlled trial of creatine in children with spinal muscular atrophy. In: Journal of Clinical Neuromuscular Disease. 2007 ; Vol. 8, No. 3. pp. 101-110.
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abstract = "OBJECTIVE: To determine whether oral creatine produces positive changes in any of 4 outcomes in children with spinal muscular atrophy (SMA). METHODS: The authors conducted a randomized, double-blind, placebo-controlled trial on 55 patients aged 2-18 years with SMA. Patients aged younger than 5 years received 2 g/day of creatine/placebo for 6 months. Patients aged 5 years and older received 5 g/day. The primary outcome measure was the Gross Motor Function Measure (GMFM). Secondary outcome measures were Quantitative Muscle Testing (QMT), Parent Questionnaire for the PedsQL™ Neuromuscular Module (QOL), and Pulmonary Function Tests (PFT). RESULTS: Forty of the 55 patients completed the protocol. There was no significant difference in the 4 outcome measures between creatine and placebo groups. CONCLUSIONS: Under the experimental conditions of our study, creatine supplementation for 6 months did not improve motor function, muscle strength, pulmonary function, or quality of life in children with SMA.",
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T1 - A randomized, placebo-controlled trial of creatine in children with spinal muscular atrophy

AU - Wong, Brenda L.

AU - Hynan, Linda S.

AU - Iannaccone, Susan T.

AU - Iannaccone, S. T.

AU - Rabb, K.

AU - Carman, D.

AU - Harris, K.

AU - Morton, A.

AU - Hynan, L. S.

AU - Reisch, J. S.

AU - Smith, J.

AU - Webster, J. C.

AU - Goldsmith, C.

AU - Gorman, A. R.

AU - Schochet, P. N.

AU - Luckett, P. M.

AU - Walters, P.

AU - Wong, B. L.

AU - Samaha, F. J.

AU - Fritch, A.

AU - Morehart, P. J.

AU - Russman, B. S.

AU - Zilke, K.

AU - Sienko-Thomas, S.

AU - Leshner, R. T.

AU - Mayhew, J.

AU - Grillo, B.

AU - Smith, S. A.

AU - Stout, J.

AU - Nugent, A.

AU - Parke, J. T.

AU - Clark, A.

AU - Earnest, C. P.

AU - Kryscio, R. J.

AU - Timberman, L.

AU - Moy, C. S.

AU - Newton, C.

PY - 2007/3

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N2 - OBJECTIVE: To determine whether oral creatine produces positive changes in any of 4 outcomes in children with spinal muscular atrophy (SMA). METHODS: The authors conducted a randomized, double-blind, placebo-controlled trial on 55 patients aged 2-18 years with SMA. Patients aged younger than 5 years received 2 g/day of creatine/placebo for 6 months. Patients aged 5 years and older received 5 g/day. The primary outcome measure was the Gross Motor Function Measure (GMFM). Secondary outcome measures were Quantitative Muscle Testing (QMT), Parent Questionnaire for the PedsQL™ Neuromuscular Module (QOL), and Pulmonary Function Tests (PFT). RESULTS: Forty of the 55 patients completed the protocol. There was no significant difference in the 4 outcome measures between creatine and placebo groups. CONCLUSIONS: Under the experimental conditions of our study, creatine supplementation for 6 months did not improve motor function, muscle strength, pulmonary function, or quality of life in children with SMA.

AB - OBJECTIVE: To determine whether oral creatine produces positive changes in any of 4 outcomes in children with spinal muscular atrophy (SMA). METHODS: The authors conducted a randomized, double-blind, placebo-controlled trial on 55 patients aged 2-18 years with SMA. Patients aged younger than 5 years received 2 g/day of creatine/placebo for 6 months. Patients aged 5 years and older received 5 g/day. The primary outcome measure was the Gross Motor Function Measure (GMFM). Secondary outcome measures were Quantitative Muscle Testing (QMT), Parent Questionnaire for the PedsQL™ Neuromuscular Module (QOL), and Pulmonary Function Tests (PFT). RESULTS: Forty of the 55 patients completed the protocol. There was no significant difference in the 4 outcome measures between creatine and placebo groups. CONCLUSIONS: Under the experimental conditions of our study, creatine supplementation for 6 months did not improve motor function, muscle strength, pulmonary function, or quality of life in children with SMA.

KW - Creatine

KW - Spinalmuscular atrophy (SMA)

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