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标题：Brazilian multicenter study on pegvisomant treatment in acromegaly
其他标题：Brazilian multicenter study on pegvisomant treatment in acromegaly
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作者：Boguszewski, Cesar L. ; Huayllas, Martha Katherine P ; Vilar, Lucio 等
期刊名称：Archives of Endocrinology and Metabolism
印刷版ISSN：2359-3997
出版年度：2019
卷号：63
期号：4
页码：328-336
DOI：10.20945/2359-3997000000159
出版社：Archives of Endocrinology and Metabolism
摘要：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.
关键词：Acromegaly; human growth hormone derivatives; pegvisomant; multicenter study.
其他关键词：Acromegaly;human growth hormone derivatives;pegvisomant;multicenter study