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A Placebo-Controlled Crossover Trial

M. Vorgerd, MD; T. Grehl, MD; M Jäger, PhD; K Müller, MD; G Freitag, MSc; T Patzold, MD; N Bruns, MD; K Fabian, MD; M Tegenthoff, MD; W Mortier, MD; A Luttmann, PhD; J Zange, PhD; J. P Malin, MD

Arch Neurol. 2000;57:956-963.

Objective  To determine whether treatment with creatine can improve exercise intolerance in myophosphorylase deficiency (McArdle disease).

Design  Double-blind, placebo-controlled crossover study with oral creatine monohydrate supplementation.

Patients  Nine patients with biochemically and genetically proven McArdle disease were treated.

Intervention  Five days of daily high-dose creatine intake (150 mg/kg body weight) were followed by daily low-dose creatine intake (60 mg/kg). Each treatment phase with creatine or placebo lasted 5 weeks.

Main Outcome Measures  The effect of treatment was estimated at the end of each treatment phase by recording clinical scores, ergometer exercise test results, phosphorus 31 nuclear magnetic resonance spectroscopy, and surface electromyography.

Results  Of 9 patients, 5 reported improvement of muscle complaints with creatine. Force-time integrals (P = .03) and depletion of phosphocreatine (P = .04) increased significantly during ischemic exercise with creatine. Phosphocreatine depletion also increased significantly during aerobic exercise (P = .006). The decrease of median frequency in surface electromyograms during contraction was significantly larger (P = .03) with creatine.

Conclusion  This is the first controlled study indicating that creatine supplementation improves skeletal muscle function in McArdle disease.

From the Departments of Neurology (Drs Vorgerd, Grehl, Patzold, Tegenthoff, and Malin), Medical Biostatistics (Ms Freitag), Cardiology (Dr Bruns), and Pediatrics (Dr Mortier), Ruhr University Bochum, Bochum, Germany; the Institute of Occupational Physiology University of Dortmund, Dortmund, Germany (Drs Jäger and Luttmann); the German Aerospace Center (Deutsches Zentrum für Luft-und Raumfahrt) Cologne, Germany (Drs Müller and Zange); and the Institute of Sports Medicine, University of Dresden, Dresden, Germany (Dr Fabian).

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