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肢端肥大症的医学治疗。

Medical treatment in acromegaly.

作者信息

Paisley Angela N, Trainer Peter J

机构信息

Department of Endocrinology, Christie Hospital, Wilmslow Road, Manchester M20 4BX, UK.

出版信息

Curr Opin Pharmacol. 2003 Dec;3(6):672-7. doi: 10.1016/j.coph.2003.07.007.

DOI:10.1016/j.coph.2003.07.007
PMID:14644022
Abstract

Acromegaly is a rare disabling disorder that results in premature death. The excess mortality and morbidity are the result of prolonged elevation of growth hormone (GH) and insulin-like growth factor-I (IGF-I) levels, and vigorous control of these improves well-being and restores life expectancy to normal. Recognition of the benefits of treatment has emphasised the need for optimal control of the GH/IGF-I axis. Transsphenoidal surgery is first-line therapy in the majority of patients; however, as most tumours are macroadenomas, cure rates are low. The role of radiotherapy is evolving and, although extremely effective at controlling tumour growth, it can take up to 15 years to control GH & IGF-I levels. In the interim, medical therapy is necessary. Dopamine agonists are inexpensive oral agents but, although most patients experience some benefit, GH and IGF-I levels are only normalised in around 35-40% of patients, and side effects are common. Somatostatin analogues are the gold standard of medical treatment. They can induce tumour shrinkage in a proportion of patients and can normalise the GH/IGF-I axis (at best) in approximately 65% of individuals; however, this leaves a significant cohort uncontrolled. The advent of the GH receptor antagonist pegvisomant provides the potential for IGF-I to be normalised in virtually every patient, but this novel form of therapy, which does not act on the pituitary, also raises many questions.

摘要

肢端肥大症是一种罕见的致残性疾病,可导致过早死亡。过高的死亡率和发病率是生长激素(GH)和胰岛素样生长因子-I(IGF-I)水平长期升高的结果,积极控制这些指标可改善健康状况并使预期寿命恢复正常。对治疗益处的认识强调了对GH/IGF-I轴进行最佳控制的必要性。经蝶窦手术是大多数患者的一线治疗方法;然而,由于大多数肿瘤是大腺瘤,治愈率较低。放射治疗的作用正在不断演变,虽然它在控制肿瘤生长方面极其有效,但控制GH和IGF-I水平可能需要长达15年的时间。在此期间,药物治疗是必要的。多巴胺激动剂是便宜的口服药物,虽然大多数患者会有一些益处,但只有约35-40%的患者的GH和IGF-I水平可恢复正常,且副作用常见。生长抑素类似物是药物治疗的金标准。它们可使一部分患者的肿瘤缩小,并使约65%的个体的GH/IGF-I轴(最佳情况下)恢复正常;然而,仍有相当一部分患者无法得到控制。GH受体拮抗剂培维索孟的出现为几乎每位患者的IGF-I恢复正常提供了可能,但这种不作用于垂体的新型治疗方法也引发了许多问题。

相似文献

1
Medical treatment in acromegaly.肢端肥大症的医学治疗。
Curr Opin Pharmacol. 2003 Dec;3(6):672-7. doi: 10.1016/j.coph.2003.07.007.
2
Pegvisomant: an advance in clinical efficacy in acromegaly.培维索孟:肢端肥大症临床疗效的一项进展。
Eur J Endocrinol. 2003 Apr;148 Suppl 2:S27-32. doi: 10.1530/eje.0.148s027.
3
Experience with pegvisomant in the treatment of acromegaly.培维索孟治疗肢端肥大症的经验。
Growth Horm IGF Res. 2001 Jun;11 Suppl A:S111-4. doi: 10.1016/s1096-6374(01)80018-6.
4
How effective are current therapies for acromegaly?目前治疗肢端肥大症的疗法效果如何?
Growth Horm IGF Res. 2003 Aug;13 Suppl A:S144-51. doi: 10.1016/s1096-6374(03)00072-8.
5
Additional metabolic effects of adding GH receptor antagonist to long-acting somatostatin analog in patients with active acromegaly.在活动性肢端肥大症患者中,将生长激素受体拮抗剂添加到长效生长抑素类似物中的额外代谢效应。
Neuro Endocrinol Lett. 2008 Aug;29(4):571-6.
6
Pharmacological management of acromegaly: a current perspective.肢端肥大症的药物治疗管理:当前视角。
Neurosurg Focus. 2010 Oct;29(4):E14. doi: 10.3171/2010.7.FOCUS10168.
7
[Possibilities of medical treatment in acromegaly].[肢端肥大症的医学治疗可能性]
Cas Lek Cesk. 2005;144 Suppl 3:33-4, 36-7.
8
Pharmacological approach to the treatment of acromegaly.肢端肥大症的药物治疗方法
Neurosurg Focus. 2004 Apr 15;16(4):E3. doi: 10.3171/foc.2004.16.4.4.
9
The place of pegvisomant in the management of acromegaly.培维索孟在肢端肥大症治疗中的地位。
Expert Opin Investig Drugs. 2001 Sep;10(9):1725-35. doi: 10.1517/13543784.10.9.1725.
10
A Canadian multi-centre, open-label long-term study of Pegvisomant treatment in refractory acromegaly.一项关于培维索孟治疗难治性肢端肥大症的加拿大多中心、开放标签长期研究。
Clin Invest Med. 2009 Dec 1;32(6):E265. doi: 10.25011/cim.v32i6.10662.

引用本文的文献

1
Pituitary tumor disappearance in a patient with newly diagnosed acromegaly primarily treated with octreotide LAR.一名初诊肢端肥大症患者主要接受长效奥曲肽治疗后垂体肿瘤消失
J Endocrinol Invest. 2005 Feb;28(2):166-9. doi: 10.1007/BF03345361.