Patient-reported outcomes in patients with acromegaly treated with pegvisomant in the ACROSTUDY extension: A real-world experience

Roberto Salvatori (1) , Pietro Maffei (2) , Susan M. Webb (3) , Thierry Brue (4, 5) , Jane Loftus (6) , Srinivas Rao Valluri (6) , Roy Gomez (6) , Michael P. Wajnrajch (6, 7) , Maria Fleseriu (8)

1 JHU - Johns Hopkins University
2 Unipd - Università degli Studi di Padova = University of Padua
3 UAB - Universitat Autònoma de Barcelona
4 MMG - Marseille medical genetics - Centre de génétique médicale de Marseille
5 Service d'endocrinologie, diabète, maladies métaboliques [Hôpital de la Conception - APHM]
6 Pfizer
7 NYU Grossman School of Medicine - New York University School of Medicine
8 OHSU - Oregon Health and Science University [Portland]

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.

Keywords: ACROSTUDY; Acromegaly; Patient-reported outcomes; Pegvisomant

Domains: Life Sciences [q-bio]; Genetics

Roberto Salvatori, Pietro Maffei, Susan M. Webb, Thierry Brue, Jane Loftus, et al.. Patient-reported outcomes in patients with acromegaly treated with pegvisomant in the ACROSTUDY extension: A real-world experience. Pituitary, 2022, 25 (3), pp.420-432. ⟨10.1007/s11102-022-01206-2⟩. ⟨hal-03780362⟩