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Brazilian multicenter study on pegvisomant treatment in acromegaly

Boguszewski, Cesar L; Huayllas, Martha Katherine P; Vilar, Lucio; Naves, Luciana Ansaneli; Ribeiro-Oliveira Junior, Antonio; Soares, Beatriz Santana; Czepielewski, Mauro Antonio; Abucham, Julio; Correa-Silva, Silvia Regina; Bronstein, Marcello Delano; Jallad, Raquel Soares; Duarte, Felipe Gaia; Musolino, Nina Rosa; Kasuki, Leandro; Gadelha, Monica Roberto.
Arch. endocrinol. metab. (Online) ; 63(4): 328-336, July-Aug. 2019. tab, graf
Artículo en Inglés | LILACS | ID: biblio-1019350
ABSTRACT Objective Investigate the therapeutic response of acromegaly patients to pegvisomant (PEGV) in a real-life, Brazilian multicenter study. Subjects and methods Characteristics of acromegaly patients treated with PEGV were reviewed at diagnosis, just before and during treatment. All patients with at least two IGF-I measurements on PEGV were included. Efficacy was defined as any normal IGF-I measurement during treatment. Safety data were reviewed. Predictors of response were determined by comparing controlled versus uncontrolled patients. Results 109 patients [61 women; median age at diagnosis 34 years; 95.3% macroadenomas] from 10 Brazilian centers were studied. Previous treatment included surgery (89%), radiotherapy (34%), somatostatin receptor ligands (99%), and cabergoline (67%). Before PEGV, median levels of GH, IGF-I and IGF-I % of upper limit of normal were 4.3 µg/L, 613 ng/mL, and 209%, respectively. Pre-diabetes/diabetes was present in 48.6% and tumor remnant in 71% of patients. Initial dose was 10 mg/day in all except 4 cases, maximum dose was 30 mg/day, and median exposure time was 30.5 months. PEGV was used as monotherapy in 11% of cases. Normal IGF-I levels was obtained in 74.1% of patients. Glycemic control improved in 56.6% of patients with pre-diabetes/diabetes. Exposure time, pre-treatment GH and IGF-I levels were predictors of response. Tumor enlargement occurred in 6.5% and elevation of liver enzymes in 9.2%. PEGV was discontinued in 6 patients and 3 deaths unrelated to the drug were reported. Conclusions In a real-life scenario, PEGV is a highly effective and safe treatment for acromegaly patients not controlled with other therapies.
Biblioteca responsable: BR1.1