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Endocrine Abstracts (2020) 70 AEP662 | DOI: 10.1530/endoabs.70.AEP662

1Fondazione Policlinico Gemelli, IRCCS, Università Cattolica del Sacro Cuore, Rome, Pituitary unit, Department of Endocrinology, Italy; 2University of Pittsburgh and Oregon Health and Science University, United States; 3Fondazione Policlinico Gemelli, IRCCS, Università Cattolica del Sacro Cuore, Rome, Department of Neurosurgery, Italy; 4Fondazione Policlinico Gemelli, IRCCS, Università Cattolica del Sacro Cuore, Rome, Department of Pathology, Rome, Italy; 5Vita Salute San Raffaele, Endocrinology, Milan, Italy


Background: Pegvisomant(PegV) and Pasireotide LAR(Pasi) are commonly used in acromegaly patients resistant to first-generation SRLs. Predictors of response to therapy with PegV and Pasi in this subset of patients are still unclear. These findings may be useful in choosing the most appropriate therapeutic option for the personalized treatment of patients affected by more aggressive disease. We aimed to identify individual predictors of responses.

Methods: We conducted a longitudinal retrospective cross-sectional study in a multicenter study on 64 acromegaly patients resistant to first-generation SRLs. We enrolled in the study patients (1) who underwent surgical resection as first-line therapeutic option, (2) defined resistant to adjuvant treatment first-generation SRL at high dose, after at least 6 consecutive months of therapy, (3) treated with Pasi or PegV for at least 6 months. Patients treated with radiotherapy in the previous 10 years were excluded. Biochemical control was defined as normal IGF-I.

Results: 51 patients were treated with PegV and 31 with Pasi. 57 patients were female. Mean age was 37.5 months (SDS: 13.4). Mean IGF-I × ULN at study entry was 3.4 (SDS: 1,1). The clinical, biochemical and morphological features was similar between the two groups. In the Pasi-treatment group, 18 patients were switched from PegV (15 as not-controlled and 3 for intolerance).Of the 51 patients treated with PegV, biochemical control was obtained in 36 (70.6%) and among the 31 patients treated with Pasi, biochemical control was obtained in 20 (64.5%). In the PegV-treatment group, patients resistant totherapy showed significantly higher Ki67 compared to the responding patients (P = 0.04), a more frequent invasion of the ventricular system (P = 0.006), and higher values of IGF-I × ULN before treatment (P = 0.01). In the Pasi-treatment group, patients resistant to therapy showed significantly higher Ki67 compared to responders (P = 0.001), higher IGF-I × ULN before treatment (P = 0.04), a higher prevalence of d3-isoform of the GH receptor (P = 0.04) and a low/absent expression of subtype 5 of somatostatin receptors (P = 0.04). The SSTR2A expression was similar between the two groups of treatment (P = 0.06).

Conclusions: Our data confirm in real life scenario the efficacy of Pegvisomant and Pasireotide LAR in obtainingbiochemical control of acromegaly in patients affected by more aggressive disease and suggest that molecular markers may be useful for optimizing an individualized treatment outcome.

Volume 70

22nd European Congress of Endocrinology

Online
05 Sep 2020 - 09 Sep 2020

European Society of Endocrinology 

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