PMID- 25370326
OWN - NLM
STAT- MEDLINE
DCOM- 20160805
LR  - 20220409
IS  - 1530-891X (Print)
IS  - 1934-2403 (Electronic)
IS  - 1530-891X (Linking)
VI  - 21
IP  - 3
DP  - 2015 Mar
TI  - Long-term treatment with pegvisomant as monotherapy in patients with acromegaly: 
      experience from ACROSTUDY.
PG  - 264-74
LID - 10.4158/EP14330.OR [doi]
AB  - 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: ACROSTUDY data indicate that 
      pegvisomant used as sole medical therapy is safe and effective for patients with 
      acromegaly. The reported low incidence of pituitary tumor size increase and liver 
      enzyme elevations are reassuring and support the positive benefit-risk of 
      pegvisomant therapy.
FAU - Freda, Pamela U
AU  - Freda PU
AD  - Department of Medicine, Columbia University College of Physicians & Surgeons, New 
      York, New York.
FAU - Gordon, Murray B
AU  - Gordon MB
AD  - Department of Medicine and Neurosurgery, Allegheny Neuroendocrinology Center, 
      Allegheny General Hospital Pittsburgh, Pennsylvania.
FAU - Kelepouris, Nicky
AU  - Kelepouris N
AD  - Pfizer, Collegeville, Pennsylvania.
FAU - Jonsson, Peter
AU  - Jonsson P
AD  - Pfizer Health AB, Sollentuna, Sweden.
FAU - Koltowska-Haggstrom, Maria
AU  - Koltowska-Haggstrom M
AD  - Department of Women's and Children's Health, Uppsala University, Uppsala, Sweden.
FAU - van der Lely, A J
AU  - van der Lely AJ
AD  - Department of Medicine, Erasmus University Medical Center, Rotterdam, The 
      Netherlands.
LA  - eng
GR  - R01 DK064720/DK/NIDDK NIH HHS/United States
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
PL  - United States
TA  - Endocr Pract
JT  - Endocrine practice : official journal of the American College of Endocrinology 
      and the American Association of Clinical Endocrinologists
JID - 9607439
RN  - 0 (Receptors, Somatotropin)
RN  - 12629-01-5 (Human Growth Hormone)
RN  - 67763-96-6 (Insulin-Like Growth Factor I)
RN  - N824AOU5XV (pegvisomant)
SB  - IM
CIN - Endocr Pract. 2015 Mar;21(3):296-8. PMID: 25667381
MH  - Acromegaly/blood/*drug therapy/physiopathology
MH  - Adolescent
MH  - Adult
MH  - Aged
MH  - Aged, 80 and over
MH  - Child
MH  - Child, Preschool
MH  - Female
MH  - Human Growth Hormone/adverse effects/*analogs & derivatives/therapeutic use
MH  - Humans
MH  - Infant
MH  - Insulin-Like Growth Factor I/analysis
MH  - Liver/physiopathology
MH  - Male
MH  - Middle Aged
MH  - Receptors, Somatotropin/*antagonists & inhibitors
MH  - Treatment Outcome
PMC - PMC4618502
MID - NIHMS669192
EDAT- 2014/11/06 06:00
MHDA- 2016/08/06 06:00
PMCR- 2015/10/24
CRDT- 2014/11/06 06:00
PHST- 2014/11/06 06:00 [entrez]
PHST- 2014/11/06 06:00 [pubmed]
PHST- 2016/08/06 06:00 [medline]
PHST- 2015/10/24 00:00 [pmc-release]
AID - S1530-891X(20)43358-0 [pii]
AID - 10.4158/EP14330.OR [doi]
PST - ppublish
SO  - Endocr Pract. 2015 Mar;21(3):264-74. doi: 10.4158/EP14330.OR.