Roberto Salvatori1, Pietro Maffei2, Susan M Webb3, Thierry Brue4, Jane Loftus5, Srinivas Rao Valluri6, Roy Gomez7, Michael P Wajnrajch6,8, Maria Fleseriu9. 1. Division of Endocrinology and Metabolism and Pituitary Center, School of Medicine, Johns Hopkins University, Baltimore, MD, USA. salvator@jhmi.edu. 2. Department of Medicine (DIMED), University of Padua, Padua, Italy. 3. Department of Endocrinology/Medicine, Centro de Investigación Biomédica en Red de Enfermedades Raras, Hospital Sant Pau, Universitat Autónoma de Barcelona, Carrer de Sant Quintí 89, 08041, Barcelona, Spain. 4. Hopital de la Conception, and Aix-Marseille Université, Marseille Medical Genetics, Assistance Publique-Hôpitaux de Marseille, Marseille, France. 5. Pfizer Ltd, Tadworth, UK. 6. , Pfizer, New York, NY, USA. 7. Pfizer, Brussels, Belgium. 8. Division of Pediatric Endocrinology, Grossman School of Medicine, New York University, New York, NY, USA. 9. Pituitary Center, Departments of Medicine, Division of Endocrinology, Diabetes, & Clinical Nutrition) and Neurological Surgery, Oregon Health & Science University, Portland, OR, USA.
Abstract
PURPOSE: To report the effects of pegvisomant (PEGV) treatment on patient-reported outcomes in acromegaly patients. METHODS: We conducted an extension study of an open-label, multinational, non-interventional study (ACROSTUDY) evaluating the long-term safety and efficacy of PEGV for acromegaly in routine clinical practice. Enrolled patients were rollover patients from ACROSTUDY, or treatment naïve/semi-naïve (NSN; no PEGV within 6 months of enrollment). Exploratory efficacy endpoints were changes in symptoms with the Patient-Assessed Acromegaly Symptom Questionnaire (PASQ) and quality of life with the Acromegaly Quality of Life questionnaire (AcroQoL) analyzed by controlled or uncontrolled IGF-I levels. Results were analyzed in all patients, in NSN patient subgroup, and by diabetes status. RESULTS: A total of 544 patients with acromegaly were enrolled, including 434 rollover subjects from ACROSTUDY and 110 NSN patients. Mean PEGV treatment duration was 7.8 years (range, 0-19.6 years). Overall, the majority of PASQ scores improved over time, but there was no significant difference between IGF-I controlled or uncontrolled groups. In the NSN subgroup, most PASQ and AcroQoL scores remained similar to baseline up to 1 year, regardless of IGF-I control. Patients with diabetes reported better PASQ scores over time with PEGV treatment, regardless of IGF-I control. IGF-I normalization increased from 10% of patients at baseline to more than 78% at year 10, with a mean daily PEGV dose of 18.7 mg. CONCLUSIONS: Overall, patients treated with PEGV had small improvements in PASQ. While IGF-I normalization increased with PEGV treatment, IGF-I control had no effects on PASQ and AcroQoL scores.
PURPOSE: To report the effects of pegvisomant (PEGV) treatment on patient-reported outcomes in acromegaly patients. METHODS: We conducted an extension study of an open-label, multinational, non-interventional study (ACROSTUDY) evaluating the long-term safety and efficacy of PEGV for acromegaly in routine clinical practice. Enrolled patients were rollover patients from ACROSTUDY, or treatment naïve/semi-naïve (NSN; no PEGV within 6 months of enrollment). Exploratory efficacy endpoints were changes in symptoms with the Patient-Assessed Acromegaly Symptom Questionnaire (PASQ) and quality of life with the Acromegaly Quality of Life questionnaire (AcroQoL) analyzed by controlled or uncontrolled IGF-I levels. Results were analyzed in all patients, in NSN patient subgroup, and by diabetes status. RESULTS: A total of 544 patients with acromegaly were enrolled, including 434 rollover subjects from ACROSTUDY and 110 NSN patients. Mean PEGV treatment duration was 7.8 years (range, 0-19.6 years). Overall, the majority of PASQ scores improved over time, but there was no significant difference between IGF-I controlled or uncontrolled groups. In the NSN subgroup, most PASQ and AcroQoL scores remained similar to baseline up to 1 year, regardless of IGF-I control. Patients with diabetes reported better PASQ scores over time with PEGV treatment, regardless of IGF-I control. IGF-I normalization increased from 10% of patients at baseline to more than 78% at year 10, with a mean daily PEGV dose of 18.7 mg. CONCLUSIONS: Overall, patients treated with PEGV had small improvements in PASQ. While IGF-I normalization increased with PEGV treatment, IGF-I control had no effects on PASQ and AcroQoL scores.
Authors: Michael Buchfelder; Aart-Jan van der Lely; Beverly M K Biller; Susan M Webb; Thierry Brue; Christian J Strasburger; Ezio Ghigo; Cecilia Camacho-Hubner; Kaijie Pan; Joanne Lavenberg; Peter Jönsson; Juliana H Hey-Hadavi Journal: Eur J Endocrinol Date: 2018-12-01 Impact factor: 6.664
Authors: Maria Fleseriu; Leon Fogelfeld; Murray B Gordon; Jill Sisco; Ross D Crosby; William H Ludlam; Asi Haviv; Susan D Mathias Journal: Pituitary Date: 2020-08 Impact factor: 4.107
Authors: Maria Fleseriu; Beverly M K Biller; Pamela U Freda; Monica R Gadelha; Andrea Giustina; Laurence Katznelson; Mark E Molitch; Susan L Samson; Christian J Strasburger; A J van der Lely; Shlomo Melmed Journal: Pituitary Date: 2020-10-20 Impact factor: 4.107
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