PMID- 34342594
OWN - NLM
STAT- MEDLINE
DCOM- 20210908
LR  - 20220426
IS  - 1479-683X (Electronic)
IS  - 0804-4643 (Print)
IS  - 0804-4643 (Linking)
VI  - 185
IP  - 4
DP  - 2021 Aug 27
TI  - More than a decade of real-world experience of pegvisomant for acromegaly: 
      ACROSTUDY.
PG  - 525-538
LID - EJE-21-0239 [pii]
LID - 10.1530/EJE-21-0239 [doi]
AB  - OBJECTIVE: To report the final long-term safety and efficacy analyses of patients 
      with acromegaly treated with pegvisomant from the ACROSTUDY. DESIGN: Global (15 
      countries), multicentre, non-interventional study (2004-2017). METHODS: The 
      complete ACROSTUDY cohort comprised patients with acromegaly, who were being 
      treated with pegvisomant (PEGV) prior to the study or at enrolment. The main 
      endpoints were long-term safety (comorbidities, adverse events (AEs), pituitary 
      tumour volumes, liver tests) and efficacy (IGF1 changes). RESULTS: Patients (n = 
      2221) were treated with PEGV for a median of 9.3 years (range, 0-20.8 years) and 
      followed up for a median of 7.4 years (range, 0-13.9 years). Before PEGV, 96.3% 
      had received other acromegaly treatments (surgery/radiotherapy/medications). 
      Before PEGV treatment, 87.2% of patients reported comorbidities. During 
      ACROSTUDY, 5567 AEs were reported in 56.5% of patients and of these 613 were 
      considered treatment-related (in 16.5% of patients) and led to drug withdrawal in 
      1.3%. Pituitary imaging showed a tumour size increase in 7.1% of patients; the 
      majority (71.1%) reported no changes. Abnormal AST or ALT liver tests occurred in 
      3.2% of patients. IGF1 normalization rate improved over time, increasing from 
      11.4% at PEGV start to 53.7% at year 1, and reaching 75.4% at year 10 with the 
      use of >/=30 mg PEGV/day in an increasing proportion of patients. CONCLUSION: This 
      comprehensive review of the complete cohort in ACROSTUDY confirmed the overall 
      favourable benefit-to-risk profile and high efficacy of PEGV as mono- and 
      combination therapy in patients with an aggressive course/uncontrolled/active 
      acromegaly requiring long-term medical therapy for control.
FAU - Fleseriu, Maria
AU  - Fleseriu M
AUID- ORCID: 0000-0001-9284-6289
AD  - Pituitary Center Division of Endocrinology, Diabetes, & Clinical Nutrition, 
      Department of Medicine.
AD  - Pituitary Center, Department of Neurological Surgery, Oregon Health & Science 
      University, Portland, Oregon, USA.
FAU - Fuhrer-Sakel, Dagmar
AU  - Fuhrer-Sakel D
AD  - Department of Endocrinology Diabetology and Metabolism, Endocrine Tumour Center 
      at West German Cancer Center, University Hospital Essen, University of 
      Duisburg-Essen, Essen, Germany.
FAU - van der Lely, Aart J
AU  - van der Lely AJ
AD  - Division of Endocrinology, Department of Internal Medicine, Erasmus Medical 
      Centre, Rotterdam, Netherlands.
FAU - De Marinis, Laura
AU  - De Marinis L
AD  - Pituitary Unit, Department of Endocrinology, Fondazione A Gemelli, IRCCS, 
      Universita Cattolica del Sacro Cuore, Rome, Italy.
FAU - Brue, Thierry
AU  - Brue T
AUID- ORCID: 0000-0001-8482-6691
AD  - Assistance Publique-Hopitaux de Marseille, Hopital de la Conception, and 
      Aix-Marseille Universite, Marseille Medical Genetics, Marseille, France.
FAU - van der Lans-Bussemaker, Joli
AU  - van der Lans-Bussemaker J
AD  - Pfizer, Capelle aan den IJssel, Netherlands.
FAU - Hey-Hadavi, Judith
AU  - Hey-Hadavi J
AD  - Pfizer, New York, New York, USA.
FAU - Camacho-Hubner, Cecilia
AU  - Camacho-Hubner C
AD  - Pfizer, New York, New York, USA.
FAU - Wajnrajch, Michael P
AU  - Wajnrajch MP
AD  - Pfizer, New York, New York, USA.
AD  - Division of Pediatric Endocrinology, Department of Pediatrics, New York 
      University Langone Medical Center, New York, New York, USA.
FAU - Valluri, Srinivas Rao
AU  - Valluri SR
AD  - Pfizer, New York, New York, USA.
FAU - Palladino, Andrew Anthony
AU  - Palladino AA
AD  - Pfizer, Collegeville, Pennsylvania, USA.
FAU - Gomez, Roy
AU  - Gomez R
AD  - Pfizer, Brussels, Belgium.
FAU - Salvatori, Roberto
AU  - Salvatori R
AD  - Division of Endocrinology and Pituitary Center, Johns Hopkins University School 
      of Medicine, Baltimore, Maryland, USA.
LA  - eng
PT  - Historical Article
PT  - Journal Article
PT  - Multicenter Study
DEP - 20210827
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/*drug therapy/epidemiology
MH  - Adenoma/drug therapy/epidemiology
MH  - Adolescent
MH  - Adult
MH  - Aged
MH  - Aged, 80 and over
MH  - Child
MH  - Child, Preschool
MH  - Cohort Studies
MH  - Europe/epidemiology
MH  - Female
MH  - Follow-Up Studies
MH  - Growth Hormone-Secreting Pituitary Adenoma/drug therapy/epidemiology
MH  - History, 21st Century
MH  - Human Growth Hormone/*analogs & derivatives/therapeutic use
MH  - Humans
MH  - Infant
MH  - Male
MH  - Middle Aged
MH  - Retrospective Studies
MH  - Treatment Outcome
MH  - United States/epidemiology
MH  - Young Adult
PMC - PMC8428076
EDAT- 2021/08/04 06:00
MHDA- 2021/09/09 06:00
PMCR- 2021/09/09
CRDT- 2021/08/03 12:20
PHST- 2021/03/10 00:00 [received]
PHST- 2021/08/03 00:00 [accepted]
PHST- 2021/08/04 06:00 [pubmed]
PHST- 2021/09/09 06:00 [medline]
PHST- 2021/08/03 12:20 [entrez]
PHST- 2021/09/09 00:00 [pmc-release]
AID - EJE-21-0239 [pii]
AID - 10.1530/EJE-21-0239 [doi]
PST - epublish
SO  - Eur J Endocrinol. 2021 Aug 27;185(4):525-538. doi: 10.1530/EJE-21-0239.