Dipartimento di Medicina Clinica e Chirurgia, Sezione di Endocrinologia, University Federico II, Naples, Italy.
Chair of Endocrinology, San Raffaele Vita-Salute University, Milano, Italy.
Nat Rev Dis Primers. 2019 Mar 21;5(1):20. doi: 10.1038/s41572-019-0071-6.
Acromegaly is characterized by increased release of growth hormone and, consequently, insulin-like growth factor I (IGF1), most often by a pituitary adenoma. Prolonged exposure to excess hormone leads to progressive somatic disfigurement and a wide range of systemic manifestations that are associated with increased mortality. Although considered a rare disease, recent studies have reported an increased incidence of acromegaly owing to better disease awareness, improved diagnostic tools and perhaps a real increase in prevalence. Acromegaly treatment approaches, which include surgery, radiotherapy and medical therapy, have changed considerably over time owing to improved surgical procedures, development of new radiotherapy techniques and availability of new medical therapies. The optimal use of these treatments will reduce mortality in patients with acromegaly to levels in the general population. Medical therapy is currently an important treatment option and can even be the first-line treatment in patients with acromegaly who will not benefit from or are not suitable for first-line neurosurgical treatment. Pharmacological treatments include somatostatin receptor ligands (such as octreotide, lanreotide and pasireotide), dopamine agonists and the growth hormone receptor antagonist pegvisomant. In this Primer, we review the main aspects of acromegaly, including scientific advances that underlie expanding knowledge of disease pathogenesis, improvements in disease management and new medical therapies that are available and in development to improve disease control.
肢端肥大症的特征是生长激素释放增加，进而导致胰岛素样生长因子 I（IGF1）增加，最常见的原因是垂体腺瘤。长期暴露于过量激素会导致进行性躯体畸形和广泛的全身表现，并与死亡率增加相关。尽管肢端肥大症被认为是一种罕见疾病，但由于疾病意识提高、诊断工具改进，以及患病率可能确实增加，最近的研究报告称肢端肥大症的发病率有所增加。由于手术程序的改进、新放射治疗技术的发展以及新的医疗治疗方法的出现，肢端肥大症的治疗方法（包括手术、放射治疗和药物治疗）在过去几十年中发生了很大变化。这些治疗方法的最佳应用将使肢端肥大症患者的死亡率降低到与普通人群相当的水平。药物治疗目前是一种重要的治疗选择，甚至可以成为那些不会受益于或不适合一线神经外科治疗的肢端肥大症患者的一线治疗方法。药物治疗包括生长抑素受体配体（如奥曲肽、兰瑞肽和培维索孟）、多巴胺激动剂和生长激素受体拮抗剂培维索孟。在本专题文章中，我们综述了肢端肥大症的主要方面，包括疾病发病机制研究进展、疾病管理改进以及新的药物治疗方法，这些方法可改善疾病控制并正在开发中。
