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Journal of Child Neurology
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Fidelity of Gamma-Glutamyl Transferase (GGT) in Differentiating Skeletal Muscle From Liver Damage

Xiomara Q. Rosales, MD

Departments of Pediatrics and Neurology, The Ohio State University and Center for Gene Therapy, the Research Institute at Nationwide Children's Hospital, Columbus, Ohio

Mary-Lynn Chu, MD

Neurogenetics Division, Department of Neurology, New York University School of Medicine, New York, New York

Christopher Shilling, MS

Departments of Pediatrics and Neurology, The Ohio State University and Center for Gene Therapy, the Research Institute at Nationwide Children's Hospital, Columbus, Ohio

Cheryl Wall, RN

Departments of Pediatrics and Neurology, The Ohio State University and Center for Gene Therapy, the Research Institute at Nationwide Children's Hospital, Columbus, Ohio

Gregory M. Pastores, MD

Neurogenetics Division, Department of Neurology, New York University School of Medicine, New York, New York

Jerry R. Mendell, MD

Departments of Pediatrics and Neurology, The Ohio State University and Center for Gene Therapy, the Research Institute at Nationwide Children's Hospital, Columbus, Ohio, mendellj{at}ccri.net

This study tested the hypothesis that gamma-glutamyl transferase (GGT) can be used as a reliable biomarker to distinguish skeletal muscle from liver damage. Twenty-eight Duchenne muscular dystrophy subjects with proven dystrophin gene mutations were enrolled. Included were 14 ambulatory and 14 nonambulatory patients with approximately half of each cohort taking corticosteroids. Twenty normal males served as controls. Initial blood samples for serum GGT and creatine kinase were taken between 8AM and 9AM and redrawn 8 hours later to test for variability. Between blood draws, subjects resumed normal activities in a play environment or could leave the clinic. Not a single duchenne muscular dystrophy patient showed a GGT outside the control range at any time point, while creatine kinase levels were 14 to 200 times normal. Validation of this finding is essential for management of patients with muscle disorders exposed to potentially hepatotoxic drugs for clinical management or monitoring subjects participating in clinical trials.

Key Words: myopathy • muscular dystrophy • liver disease

This version was published on July 1, 2008

Journal of Child Neurology, Vol. 23, No. 7, 748-751 (2008)
DOI: 10.1177/0883073808314365


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