Barrow Pituitary Center, Barrow Neurological Institute, Departments of Neuroendocrinology and Neurosurgery, University of Arizona College of Medicine and Creighton School of Medicine, Phoenix, Arizona.
Departments of Medicine and Neurologic Surgery, Mayo Clinic, Jacksonville, Florida.
Endocr Pract. 2022 Jun;28(6):637-645. doi: 10.1016/j.eprac.2022.04.009. Epub 2022 Apr 19.
Acromegaly is characterized by chronic growth hormone (GH) and insulin-like growth factor 1 (IGF-1) hypersecretion, often caused by a GH-secreting pituitary adenoma. Even though surgery remains the first line of treatment, medical therapy is essential if surgery is contraindicated, does not achieve remission, or does not prevent recurrence despite apparent surgical remission. Oral octreotide capsules (OOCs) that combine octreotide with a transient permeability enhancer technology are the first oral somatostatin receptor ligands (SRLs) approved in the United States for acromegaly.
We review the literature and clinical trial data on OOC therapy in patients with acromegaly and discuss the clinical assessment of OOC use, potential drug-drug interactions, drug initiation, dose titration, and monitoring of drug efficacy and tolerability.
In 4 pivotal clinical trials involving 238 patients with acromegaly treated with OOC, effective suppression of serum GH and IGF-1 levels, maintenance of disease control, decreased breakthrough symptoms and symptomatic improvement with non-inferiority of OOCs to injectable SRLs in maintaining biochemical response was seen. Additionally, the safety profile of OOC therapy is comparable to that of injectable SRLs. Most patients who completed the clinical trials of OOCs have also expressed preference for oral compared with injectable SRL administration.
OOCs are an effective treatment option for patients with acromegaly who previously responded to injectable SRLs, with the benefits of avoiding injection-related side effects. This article provides a review of the pharmacology, safety, and efficacy and offers practical recommendations on the use of OOCs to treat injectable SRL-responsive patients with acromegaly.
肢端肥大症的特征是慢性生长激素（GH）和胰岛素样生长因子 1（IGF-1）分泌过多，通常由 GH 分泌垂体腺瘤引起。即使手术仍然是一线治疗方法，如果手术禁忌、未达到缓解、或尽管手术明显缓解但仍未预防复发，则医学治疗也是必不可少的。奥曲肽口服胶囊（OOC）将奥曲肽与瞬时渗透增强技术结合在一起，是美国批准的第一种用于肢端肥大症的口服生长抑素受体配体（SRL）。
我们回顾了肢端肥大症患者 OOC 治疗的文献和临床试验数据，并讨论了 OOC 使用的临床评估、潜在的药物-药物相互作用、药物起始、剂量滴定以及药物疗效和耐受性监测。
在涉及 238 例接受 OOC 治疗的肢端肥大症患者的 4 项关键临床试验中，OOC 可有效抑制血清 GH 和 IGF-1 水平，维持疾病控制，减少突破性症状，并在不劣于注射用 SRL 的情况下改善症状，与注射用 SRL 相比，OOC 维持生化缓解。此外，OOC 治疗的安全性与注射用 SRL 相当。大多数完成 OOC 临床试验的患者也表示更喜欢口服给药而不是注射用 SRL 给药。
OOC 是先前对注射用 SRL 有反应的肢端肥大症患者的有效治疗选择，具有避免注射相关副作用的优势。本文综述了 OOC 的药理学、安全性和疗效，并就 OOC 治疗对注射用 SRL 有反应的肢端肥大症患者的使用提供了实用建议。
