title: Prediction of therapy response in acromegalic patients under pegvisomant therapy within the German ACROSTUDY cohort.
abstract: OBJECTIVE
  This study aimed at investigating predicting factors for therapy response under growth hormone receptor antagonist therapy with a focus on subjective and patient-oriented measures.
  
  
  METHODS
  Observational, multicenter nested-cohort study including 271 selected patients with the diagnosis of acromegaly and a minimum of one-year follow-up period within the German ACROSTUDY cohort (total cohort: n = 514). Outcome measures were the change of the biomarker IGF-1 (IGF-1 change and IGF-1 normalisation) between baseline and after 1 year of pegvisomant therapy (12 ± 6 months). Main predictors were patient-assessed subjective measures according to the Patient-Assessed Acromegaly Symptom Questionnaire (PASQ) in conjugation with age, gender, BMI, max. dosage of pegvisomant at follow-up and IGF-1 before the start of pegvisomant therapy.
  
  
  RESULTS
  The mean age of the study population was 51.2 (13.9) years and the mean BMI was 29.5 (5.1) kg/m(2). In adjusted analyses, none of the individual perceived health (PASQ) scores, but age, BMI and IGF-1 at baseline were predictive for an IGF-1 decrease after 1 year of pegvisomant therapy and BMI and IGF-1, but equally none of the PASQ items, were predicting IGF-1 normalisation.
  
  
  CONCLUSIONS
  Age, BMI and baseline IGF-1 but not subjective perceived health measures predict therapy response under second line medical therapy with pegvisomant.
authors.affiliation: Clinical Neuroendocrinology, Clinical Research Department, Max Planck Institute of Psychiatry, Kraepelinstr. 2-10, 80804, Munich, Germany. csievers@mpipsykl.mpg.de.
authors.forename: Caroline
authors.initials: C
authors.lastname: Sievers
authors.affiliation: II Medizinische Klinik und Poliklinik, Klinikum rechts der Isar, Ismaningerstrasse 22, 81675, Munich, Germany.
authors.forename: Dorothee M
authors.initials: DM
authors.lastname: Baur
authors.affiliation: Endocrine Care, Pfizer Pharma GmbH, Berlin, Germany.
authors.forename: Anja
authors.initials: A
authors.lastname: Schwanke
authors.affiliation: Department of Neurosurgery, University of Erlangen-Nuremberg, Erlangen, Germany.
authors.forename: Michael
authors.initials: M
authors.lastname: Buchfelder
authors.affiliation: Endokrinologische Praxis, Oldenburg, Germany.
authors.forename: Michael
authors.initials: M
authors.lastname: Droste
authors.affiliation: Department of Endocrinology and Metabolism, University of Duisburg-Essen and Endokrinologiezentrum, Alter Hof, Munich, Germany.
authors.forename: Klaus
authors.initials: K
authors.lastname: Mann
authors.affiliation: Clinical Neuroendocrinology, Clinical Research Department, Max Planck Institute of Psychiatry, Kraepelinstr. 2-10, 80804, Munich, Germany.
authors.forename: Günter K
authors.initials: GK
authors.lastname: Stalla
mesh_terms: D000172:Acromegaly / Q000097:blood / Q000188:drug therapy*; D000328:Adult; D001786:Blood Glucose / Q000378:metabolism; D015331:Cohort Studies; D005260:Female; D019382:Human Growth Hormone / Q000031:analogs & derivatives* / Q000627:therapeutic use; D006801:Humans; D007334:Insulin-Like Growth Factor I / Q000378:metabolism; D008297:Male; D008875:Middle Aged; D011788:Quality of Life
chemical_list: D001786:Blood Glucose; D019382:Human Growth Hormone; D007334:Insulin-Like Growth Factor I; C406545:pegvisomant
keywords: Acromegaly; Growth hormone; Health-related quality of life; IGF-1; Pegvisomant