Safety and tolerability of growth hormone therapy in multiple system atrophy: a double-blind, placebo-controlled study.
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.
Holmberg
Björn
B
Movement Disorders Unit, Sahlgrenska University Hospital, Göteborg University, Göteborg, Sweden. bjorn.holmberg@neuro.gu.se
Johansson
Jan-Ove
JO
Poewe
Werner
W
Wenning
Gregor
G
Quinn
Niall P
NP
Mathias
Chris
C
Tolosa
Eduardo
E
Cardozo
Adriana
A
Dizdar
Nil
N
Rascol
Olivier
O
Slaoui
Tarik
T
D015992:Body Mass Index; D003248:Constipation / Q000139:chemically induced / Q000453:epidemiology; D004311:Double-Blind Method; D004361:Drug Tolerance; D005260:Female; D006339:Heart Rate / Q000187:drug effects; D019382:Human Growth Hormone / Q000009:adverse effects / Q000627:therapeutic use*; D006801:Humans; D008297:Male; D008875:Middle Aged; D019578:Multiple System Atrophy / Q000188:drug therapy* / Q000473:pathology*
D019382:Human Growth Hormone