Bush Zachary M, Vance Mary Lee
Department of Medicine, Division of Endocrinology and Metabolism, University of Virginia, Charlottesville, VA, USA.
Rev Endocr Metab Disord. 2008 Mar;9(1):83-94. doi: 10.1007/s11154-007-9061-1.
Acromegaly is a chronic, debilitating disease caused by chronic growth hormone (GH) hypersecretion which results in chronic medical comorbidities, poor quality of life and high mortality rates. Successful treatment can improve clinical signs and symptoms and normalize mortality rates. Over 95% of acromegaly is caused by a somatotroph adenoma of the pituitary, and the first-line treatment is generally transsphenoidal surgery, which can be curative in 50-60% of patients. Nonetheless, high rates of persistent acromegaly following surgery and the limited efficacy of radiation therapy necessitate chronic medical treatment for many patients. Somatostatin analogues have become the preferred first-line medical therapy for many practitioners, as they achieve better biochemical and direct tumor control than the dopamine agonists, and long-acting preparations make once monthly administration possible. Cabergoline, a dopamine agonist, offers a lower-cost option and may be effective in patients with a pituitary tumor that co-secretes GH and prolactin. Pegvisomant is a GH receptor antagonist that produces exceptional biochemical response rates but lacks any direct effects on the tumor, which may limit its effectiveness as life-long monotherapy. Combinations of these three drug classes have not been rigorously studied, and preliminary trials do not suggest improved clinical outcomes. While medical treatment options for acromegaly have significantly improved over the last 30 years, limitations remain, and a multi-specialty team approach is necessary for the effective long-term management of patients with acromegaly.
肢端肥大症是一种由慢性生长激素(GH)分泌过多引起的慢性、使人衰弱的疾病,会导致慢性合并症、生活质量差和高死亡率。成功的治疗可以改善临床体征和症状,并使死亡率正常化。超过95%的肢端肥大症由垂体生长激素细胞腺瘤引起,一线治疗通常是经蝶窦手术,50-60%的患者可通过该手术治愈。尽管如此,手术后肢端肥大症持续存在的比例较高,且放射治疗效果有限,许多患者仍需要长期药物治疗。生长抑素类似物已成为许多医生首选的一线药物治疗,因为它们比多巴胺激动剂能更好地实现生化和直接肿瘤控制,长效制剂使每月给药一次成为可能。卡麦角林是一种多巴胺激动剂,成本较低,对同时分泌GH和催乳素的垂体瘤患者可能有效。培维索孟是一种GH受体拮抗剂,能产生优异的生化反应率,但对肿瘤没有直接作用,这可能限制其作为终身单一疗法的有效性。这三类药物的联合应用尚未得到严格研究,初步试验也未显示临床结果有所改善。虽然过去30年中肢端肥大症的药物治疗选择有了显著改善,但仍存在局限性,对于肢端肥大症患者的有效长期管理,多学科团队方法是必要的。