Department of Internal Medicine, Hacettepe University Hospital, 06230, Ankara, Turkey.
Koru Hospital, 06510, Ankara, Turkey.
Pituitary. 2024 Oct;27(5):468-479. doi: 10.1007/s11102-024-01461-5. Epub 2024 Oct 1.
Acromegaly is a rare chronic endocrine disorder associated with significant comorbidities. Many patients fail to achieve biochemical control with current medical therapies, including surgery and first-generation somatostatin ligands (fg-SRLs). We aimed to perform a systematic review and single-arm meta-analysis to evaluate the efficacy of the multi-receptor somatostatin ligand pasireotide in patients with active or uncontrolled acromegaly.
We systematically searched PubMed, Embase, and Cochrane databases for studies that assessed the efficacy of pasireotide in patients with acromegaly and reported the outcomes of (1) biochemical control and its composite indicators; (2) normalized IGF-1 level and (3) low GH level. For the statistical analysis, we used R software.
We included nine studies with a total of 590 patients: four clinical trials and five observational cohorts. 82.2% of the overall population consisted of inadequately controlled acromegaly patients. After a follow-up of 12 months, the pooled biochemical control rate was 26.50% (95% CI 14.87-42.66). The prevalence of normalized IGF-1 and low GH levels was 36.27% (95% CI 29.15-43.39) and 34.76% (95% CI 24.58-44.95), respectively. Additionally, biochemical response rates were sustained throughout the extension phase of these studies. In a pooled analysis including four studies with extension phase results, the prevalence of biochemical control rate was 29.03% (95% CI 11.49-46.58) with 76 events out of 281 patients. The most commonly reported adverse events were gastrointestinal disturbances in 31.26% (95% CI 7.44-72.01) and hyperglycemia in 29.55% (95% CI 21.80-37.29) of patients. The incidence of new-onset diabetes mellitus significantly increased after pasireotide treatment, with a rate of 23.36% (95% CI 19.58-27.13).
Pasireotide demonstrates biochemical control in patients with active or uncontrolled acromegaly. Although a high rate of hyperglycemic adverse events and diabetes mellitus related to the treatment were observed, most of them were manageable.
肢端肥大症是一种罕见的慢性内分泌疾病，与多种严重合并症相关。许多患者即使接受当前的医学治疗（包括手术和第一代生长抑素类似物（fg-SRLs））也无法达到生化控制。我们旨在进行系统评价和单臂荟萃分析，以评估多受体生长抑素类似物培高利特在活动性或未控制的肢端肥大症患者中的疗效。
我们系统地检索了 PubMed、Embase 和 Cochrane 数据库，以评估培高利特治疗肢端肥大症患者的疗效，并报告了以下结果：（1）生化控制及其综合指标；（2）正常 IGF-1 水平；（3）低 GH 水平。统计分析使用 R 软件进行。
我们纳入了 9 项研究，共 590 例患者：4 项临床试验和 5 项观察性队列研究。总体人群中有 82.2%为未得到充分控制的肢端肥大症患者。随访 12 个月后，总体生化控制率为 26.50%（95% CI 14.87-42.66）。正常 IGF-1 和低 GH 水平的发生率分别为 36.27%（95% CI 29.15-43.39）和 34.76%（95% CI 24.58-44.95）。此外，在这些研究的扩展阶段，生化反应率持续存在。在包括四项扩展阶段研究结果的汇总分析中，生化控制率的发生率为 29.03%（95% CI 11.49-46.58），281 例患者中有 76 例发生。最常见的不良反应报告为胃肠道紊乱，发生率为 31.26%（95% CI 7.44-72.01）和高血糖，发生率为 29.55%（95% CI 21.80-37.29）。使用培高利特治疗后，新发糖尿病的发生率显著增加，为 23.36%（95% CI 19.58-27.13）。
培高利特在活动性或未控制的肢端肥大症患者中具有生化控制作用。尽管观察到与治疗相关的高血糖不良事件和糖尿病发病率较高，但大多数是可以管理的。
