PMID- 30325178
OWN - NLM
STAT- MEDLINE
DCOM- 20181211
LR  - 20181211
IS  - 1479-683X (Electronic)
IS  - 0804-4643 (Linking)
VI  - 179
IP  - 6
DP  - 2018 Dec 1
TI  - Long-term treatment with pegvisomant: observations from 2090 acromegaly patients 
      in ACROSTUDY.
PG  - 419-427
LID - EJE-18-0616 [pii]
LID - 10.1530/EJE-18-0616 [doi]
AB  - Objectives ACROSTUDY is an international, non-interventional study of acromegaly 
      patients treated with pegvisomant (PEGV), a growth hormone receptor antagonist 
      and has been conducted since 2004 in 15 countries to study the long-term safety 
      and efficacy of PEGV. This report comprises the second interim analysis of 2090 
      patients as of May 12, 2016. Methods Descriptive analyses of safety, pituitary 
      imaging and outcomes on PEGV treatment up to 12 years were performed. Results 
      Prior to starting PEGV, 96% of patients had reported surgery, radiation, medical 
      therapy or any combinations of those. At start of PEGV, 89% of patients had IGFI 
      levels above the upper limit of normal (ULN). The percentage of patients with 
      normal IGFI levels increased from 53% at year 1 to 73% at year 10, and the 
      average daily dose of PEGV increased from 12.8 mg (year 1) to 18.9 mg (year 10). 
      A total of 4832 adverse events (AEs) were reported in 1137 patients (54.4%), of 
      which 570 were considered treatment related in 337 patients (16.1%). Serious AEs 
      were reported in 22% of patients, of which 2.3% were considered treatment 
      related. Locally reported MRIs showed most patients (72.2%) had no change in 
      tumor size relative to the prior scan; 16.8% had a decrease, 6.8% an increase and 
      4.3% both. In patients with normal liver tests at PEGV start, an ALT or AST 
      elevation of >3x ULN at any time point during their follow-up was reported in 3%. 
      Conclusions This second interim analysis confirms that long-term use of PEGV is 
      an effective and safe treatment in patients with acromegaly.
FAU - Buchfelder, Michael
AU  - Buchfelder M
AD  - Department of Neurosurgery, University of Erlangen-Nurnberg, Erlangen, Germany.
FAU - van der Lely, Aart-Jan
AU  - van der Lely AJ
AD  - Department of Medicine, Erasmus University MC, Rotterdam, the Netherlands.
FAU - Biller, Beverly M K
AU  - Biller BMK
AD  - Neuroendocrine Unit, Massachusetts General Hospital, Boston, Massachusetts, USA.
FAU - Webb, Susan M
AU  - Webb SM
AD  - Endocrinologia (Malalties de la Hipofisi), Hospital Sant Pau, Universitat 
      Autonoma de Barcelona (UAB), Barcelona, Spain.
FAU - Brue, Thierry
AU  - Brue T
AD  - Department of Endocrinology, Centre de Reference des Maladies Rares d'Origine 
      Hypophysaire, Hopital de la Conception, Marseille, France.
FAU - Strasburger, Christian J
AU  - Strasburger CJ
AD  - Department of Medicine for Endocrinology, Diabetes and Nutritional Medicine, 
      Charite Universitatsmedizin, Campus Mitte, Berlin, Germany.
FAU - Ghigo, Ezio
AU  - Ghigo E
AD  - University Hospital Citta Salute e Scienza, Turin, Italy.
FAU - Camacho-Hubner, Cecilia
AU  - Camacho-Hubner C
AD  - Endocrine Care Global Medical Affairs, Pfizer Inc., New York City, New York, USA.
FAU - Pan, Kaijie
AU  - Pan K
AD  - Endocrine Care Global Clinical Affairs, Pfizer Inc., Collegeville, Pennsylvania, 
      USA.
FAU - Lavenberg, Joanne
AU  - Lavenberg J
AD  - Endocrine Care Global Clinical Affairs, Pfizer Inc., Collegeville, Pennsylvania, 
      USA.
FAU - Jonsson, Peter
AU  - Jonsson P
AD  - Endocrine Care, Pfizer Health AB, Sollentuna, Sweden.
FAU - Hey-Hadavi, Juliana H
AU  - Hey-Hadavi JH
AD  - Endocrine Care Global Medical Affairs, Pfizer Inc., New York City, New York, USA.
LA  - eng
PT  - Journal Article
PT  - Pragmatic Clinical Trial
PL  - England
TA  - Eur J Endocrinol
JT  - European journal of endocrinology
JID - 9423848
RN  - 12629-01-5 (Human Growth Hormone)
RN  - N824AOU5XV (pegvisomant)
SB  - IM
MH  - Acromegaly/*diagnosis/*drug therapy/epidemiology
MH  - Adolescent
MH  - Adult
MH  - Aged
MH  - Aged, 80 and over
MH  - Child
MH  - Child, Preschool
MH  - Drug Administration Schedule
MH  - Female
MH  - Follow-Up Studies
MH  - Human Growth Hormone/administration & dosage/*analogs & derivatives/*metabolism
MH  - Humans
MH  - Infant
MH  - *Internationality
MH  - Male
MH  - Middle Aged
MH  - Treatment Outcome
MH  - Young Adult
EDAT- 2018/10/17 06:00
MHDA- 2018/12/12 06:00
CRDT- 2018/10/17 06:00
PHST- 2018/07/20 00:00 [received]
PHST- 2018/10/01 00:00 [accepted]
PHST- 2018/10/17 06:00 [pubmed]
PHST- 2018/12/12 06:00 [medline]
PHST- 2018/10/17 06:00 [entrez]
AID - EJE-18-0616 [pii]
AID - 10.1530/EJE-18-0616 [doi]
PST - ppublish
SO  - Eur J Endocrinol. 2018 Dec 1;179(6):419-427. doi: 10.1530/EJE-18-0616.