Abstract | OBJECTIVE: To evaluate use of pegvisomant, a growth hormone (GH) receptor antagonist, as monotherapy in ACROSTUDY, a global safety surveillance study set in 14 countries (373 sites). METHODS: A descriptive analysis of safety, magnetic resonance imaging (MRI) reading, and treatment outcomes in 710 subjects who received at least 1 pegvisomant dose as monotherapy during and up to 5 years follow-up in ACROSTUDY. RESULTS: Subjects received a mean of 5.4 years of pegvisomant and were followed in ACROSTUDY for a mean of 3.8 years. A total of 1,255 adverse events (AEs) were reported in 345 subjects (48.6%). Serious AEs (SAEs) were reported in 133 (18.7%) subjects, including 22 deaths, none of which were attributed to pegvisomant use. Of 670 (94%) subjects with at least 1 liver function test (LFT) reported in ACROSTUDY, 8 (1.2%) had reported increases in transaminases >3 times the upper limit of normal (ULN). No liver failure was reported. Based on central MRI reading, 12 of 542 subjects (2.2%) had a confirmed increase or increase/decrease in tumor size. Injection-site reactions were reported in 2.3%. At 5 years of therapy, insulin-like growth factor 1 (IGF-1) level was reported normal in 67.5% (mean dose 17.2 mg/day) and elevated in 29.9% (mean dose 19.8 mg/day). Subjects on 20 mg per day or more rose from 36% at 3 years to 41% at 5 years of therapy. CONCLUSIONS:
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Authors | Pamela U Freda, Murray B Gordon, Nicky Kelepouris, Peter Jonsson, Maria Koltowska-Haggstrom, A J van der Lely |
Journal | Endocrine practice : official journal of the American College of Endocrinology and the American Association of Clinical Endocrinologists
(Endocr Pract)
Vol. 21
Issue 3
Pg. 264-74
(Mar 2015)
ISSN: 1530-891X [Print] United States |
PMID | 25370326
(Publication Type: Journal Article, Research Support, Non-U.S. Gov't)
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Chemical References |
- Receptors, Somatotropin
- Human Growth Hormone
- Insulin-Like Growth Factor I
- pegvisomant
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Topics |
- Acromegaly
(blood, drug therapy, physiopathology)
- Adolescent
- Adult
- Aged
- Aged, 80 and over
- Child
- Child, Preschool
- Female
- Human Growth Hormone
(adverse effects, analogs & derivatives, therapeutic use)
- Humans
- Infant
- Insulin-Like Growth Factor I
(analysis)
- Liver
(physiopathology)
- Male
- Middle Aged
- Receptors, Somatotropin
(antagonists & inhibitors)
- Treatment Outcome
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