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.

中文翻译：

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肢端肥大症的药物治疗。

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）水平持续升高的结果，对这些因子的严格控制可改善健康状况，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。蝶骨手术是大多数患者的一线治疗。但是，由于大多数肿瘤是大腺瘤，因此治愈率很低。放射疗法的作用正在发展，尽管在控制肿瘤生长方面极为有效，但控制GH和IGF-I水平可能需要长达15年的时间。在此期间，必须进行药物治疗。多巴胺激动剂是廉价的口服剂，但是，尽管大多数患者都受益匪浅，GH和IGF-I水平仅在约35-40％的患者中恢复正常，且副作用很常见。生长抑素类似物是药物治疗的金标准。它们可以在一部分患者中引起肿瘤缩小，并且可以使大约65％的患者的GH / IGF-I轴正常化（最多）。但是，这使得大量队列无法控制。GH受体拮抗剂pegvisomant的出现为几乎每位患者提供了使IGF-I正常化的潜力，但是这种不作用于垂体的新型疗法也提出了许多问题。它们可以在一定比例的患者中引起肿瘤缩小，并且可以使大约65％的患者的GH / IGF-I轴正常化（至多）。但是，这使得大量队列无法控制。GH受体拮抗剂pegvisomant的出现为几乎每位患者提供了使IGF-I正常化的潜力，但是这种不作用于垂体的新颖疗法也引起了许多问题。它们可以在一部分患者中引起肿瘤缩小，并且可以使大约65％的患者的GH / IGF-I轴正常化（最多）。但是，这使得大量队列无法控制。GH受体拮抗剂pegvisomant的出现为几乎每位患者提供了使IGF-I正常化的潜力，但是这种不作用于垂体的新型疗法也提出了许多问题。