Safety and tolerability of growth hormone therapy in multiple system atrophy: A double-blind, placebo-controlled study

• Published in: Movement disorders 2007-06, Vol.22 (8), p.1138-1144
• Main Authors: Holmberg, Björn, Johansson, Jan-Ove, Poewe, Werner, Wenning, Gregor, Quinn, Niall P., Mathias, Chris, Tolosa, Eduardo, Cardozo, Adriana, Dizdar, Nil, Rascol, Olivier, Slaoui, Tarik
• Format: Article
• Language: English
• Subjects: autonomic testing; Biological and medical sciences; Body Mass Index; cerebrospinal-fluid; Constipation - chemically induced; Constipation - epidemiology; diagnosis; Double-Blind Method; Drug Tolerance; Female; group emsa-sg; growth hormone treatment; Heart Rate - drug effects; heart-rate-variability; Human Growth Hormone - adverse effects; Human Growth Hormone - therapeutic use; Humans; idiopathic parkinsons-disease; Immunomodulators; Male; Medical sciences; MEDICIN; MEDICINE; Middle Aged; mortality; Multiple sclerosis and variants. Guillain barré syndrome and other inflammatory polyneuropathies. Leukoencephalitis; multiple system atrophy; Multiple System Atrophy - drug therapy; Multiple System Atrophy - pathology; neurogenesis; Neurologi; Neurology; Pharmacology. Drug treatments; progression; rat hippocampus; stimulation; UMSARS; UPDRS
• ISSN: 0885-3185; 1531-8257; 1531-8257
• DOI: 10.1002/mds.21501

The objective of this study was to investigate tolerability and possible neurotrophic effects of growth hormone (GH) in treatment of multiple system atrophy (MSA). In this double‐blind pilot study, MSA patients were randomized to recombinant human growth hormone (r‐hGH, n = 22), 1 mg every second day (6 months) followed by alternating daily injections of 1 mg and 0.5 mg (6 months), or matched placebo (n = 21). Safety analysis demonstrated no obvious between‐group differences. In both groups, there was progressive worsening of Unified Parkinson's Disease Rating Scale total score, which tended to be less in r‐hGH‐treated patients (12.9% at 6 months, 25.3% at 12 months) than in placebo (17.0% and 35.7%). Similarly, there was a trend to less worsening in Unified MSA Rating Scale total score with r‐hGH (13.2% and 21.2%) than with placebo (21.1% and 36.5%). Cardiovascular reflex autonomic testing also tended to show less deterioration with r‐hGH than with placebo at 12 months. However, 95% CI did not indicate treatment differences for any efficacy measures. In conclusion, r‐hGH administration in MSA patients for up to 1 year appears safe and might influence disease symptoms, signs and, possibly, progression. The results support further studies utilizing higher doses in more patients. © 2007 Movement Disorder Society