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培维索孟治疗肢端肥大症:最新进展

Pegvisomant in acromegaly: an update.

作者信息

Giustina A, Arnaldi G, Bogazzi F, Cannavò S, Colao A, De Marinis L, De Menis E, Degli Uberti E, Giorgino F, Grottoli S, Lania A G, Maffei P, Pivonello R, Ghigo E

机构信息

Chair of Endocrinology, Vita-Salute San Raffaele University, Milano, Italy.

Clinic of Endocrinology and Metabolism Disease, Ospedali Riuniti di Ancona, Ancona, Italy.

出版信息

J Endocrinol Invest. 2017 Jun;40(6):577-589. doi: 10.1007/s40618-017-0614-1. Epub 2017 Feb 7.

Abstract

BACKGROUND

In 2007, we published an opinion document to review the role of pegvisomant (PEG) in the treatment of acromegaly. Since then, new evidence emerged on the biochemical and clinical effects of PEG and on its long-term efficacy and safety.

AIM

We here reviewed the emerging aspects of the use of PEG in clinical practice in the light of the most recent literature.

RESULTS

The clinical use of PEG is still suboptimal, considering that it remains the most powerful tool to control IGF-I in acromegaly allowing to obtain, with a pharmacological treatment, the most important clinical effects in terms of signs and symptoms, quality of life and comorbidities. The number of patients with acromegaly exposed to PEG worldwide has become quite elevated and the prolonged follow-up allows now to deal quite satisfactorily with many clinical issues including major safety issues, such as the concerns about possible tumour (re)growth under PEG. The positive or neutral impact of PEG on glucose metabolism has been highlighted, and the clinical experience, although limited, with sleep apnoea and pregnancy has been reviewed. Finally, the current concept of somatostatin receptor ligands (SRL) resistance has been addressed, in order to better define the acromegaly patients to whom the PEG option may be offered.

CONCLUSIONS

PEG increasingly appears to be an effective and safe medical option for many patients not controlled by SRL but its use still needs to be optimized.

摘要

背景

2007年,我们发表了一篇观点文章,回顾培维索孟(PEG)在肢端肥大症治疗中的作用。自那时起,关于PEG的生化和临床效应及其长期疗效与安全性出现了新的证据。

目的

我们依据最新文献,在此回顾PEG在临床实践中应用的新情况。

结果

考虑到PEG仍是控制肢端肥大症患者胰岛素样生长因子-I(IGF-I)最有效的工具,通过药物治疗能在体征和症状、生活质量及合并症方面取得最重要的临床效果,其临床应用仍未达到最佳状态。全球接受PEG治疗的肢端肥大症患者数量已相当可观,长期随访现在能够较为满意地处理许多临床问题,包括主要的安全问题,如对PEG治疗下肿瘤(再)生长的担忧。PEG对糖代谢的积极或中性影响已得到强调,并且已对睡眠呼吸暂停和妊娠方面虽有限但已有的临床经验进行了回顾。最后,探讨了生长抑素受体配体(SRL)抵抗的当前概念,以便更好地界定可选择PEG治疗的肢端肥大症患者。

结论

对于许多未被SRL控制的患者,PEG越来越显示出是一种有效且安全的药物选择,但其使用仍需优化。

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