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标题：Pegvisomant in acromegaly: a multicenter real-life study in Argentina
其他标题：Pegvisomant in acromegaly: a multicenter real-life study in Argentina
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作者：Basavilbaso, Natalia Ximena Garcia ; Ballarino, Maria Carolina ; Bruera, Darío 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：320-327
DOI：10.20945/2359-3997000000160
出版社：Archives of Endocrinology and Metabolism
摘要：ABSTRACT Objective To describe the long term safety and efficacy of pegvisomant (PEGV), and the predictors of treatment response in patients with acromegaly in the real life setting. Subjects and methods We retrospectively reviewed the clinical, hormonal and radiological data of acromegalic patients treated with PEGV in 17 Argentine centers. Results Seventy-five patients (age range 22-77, 51 females) with acromegaly have been treated with PEGV for up to 118 months (median 27 months). Before PEGV, 97.3% of patients had been treated with medical therapy, surgery and/or radiotherapy, two patients had no previous treatment. At that time, all patients had an IGF-1 above the upper normal limit (ULN) (mean 2.4 x ULN ± 0.98, range 1.25-7). At diagnosis of acromegaly 84% presented macroadenomas, prior to PEGV only 23,5% of patients remained with tumor remnant > 1 cm, the remaining showed normal or less than 1 cm images. Disease control (IGF-1 ≤ 1.2 x ULN) was achieved in 62.9% of patients with a mean dose of 11.8 mg/day. Thirty-four patients (45%) received PEGV monotherapy, while 41 (55%) received combined therapy with either somatostatin analogues and/or cabergoline. Adverse events related to PEGV were: local injection site reaction in 5.3%, elevated liver enzymes in 9.3%, and tumor size growth in 9.8%. Pre-PEGV IGF-I level was the only predictor of treatment response: 2.1 x ULN vs 2.8 x ULN in controlled and uncontrolled patients respectively (p < 0.001). Conclusion this long term experience indicates PEGV treatment was highly effective and safe in our series of Argentine patients with acromegaly refractory to standard therapies.
关键词：Pegvisomant; acromegaly; GH receptor antagonist; clinical trial.
其他关键词：Pegvisomant;acromegaly;GH receptor antagonist;clinical trial