OBJECTIVE To report the final long-term safety and efficacy analyses of patients with acromegaly treated with pegvisomant from the ACROSTUDY. DESIGN Global (15 countries), multicentre, non-interventional study (2004-2017). METHODS The complete ACROSTUDY cohort comprised patients with acromegaly, who were being treated with pegvisomant (PEGV) prior to the study or at enrolment. The main endpoints were long-term safety (comorbidities, adverse events (AEs), pituitary tumour volumes, liver tests) and efficacy (IGF1 changes). RESULTS Patients (n = 2221) were treated with PEGV for a median of 9.3 years (range, 0-20.8 years) and followed up for a median of 7.4 years (range, 0-13.9 years). Before PEGV, 96.3% had received other acromegaly treatments (surgery/radiotherapy/medications). Before PEGV treatment, 87.2% of patients reported comorbidities. During ACROSTUDY, 5567 AEs were reported in 56.5% of patients and of these 613 were considered treatment-related (in 16.5% of patients) and led to drug withdrawal in 1.3%. Pituitary imaging showed a tumour size increase in 7.1% of patients; the majority (71.1%) reported no changes. Abnormal AST or ALT liver tests occurred in 3.2% of patients. IGF1 normalization rate improved over time, increasing from 11.4% at PEGV start to 53.7% at year 1, and reaching 75.4% at year 10 with the use of ≥30 mg PEGV/day in an increasing proportion of patients. CONCLUSION This comprehensive review of the complete cohort in ACROSTUDY confirmed the overall favourable benefit-to-risk profile and high efficacy of PEGV as mono- and combination therapy in patients with an aggressive course/uncontrolled/active acromegaly requiring long-term medical therapy for control.

中文翻译：

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培维索孟治疗肢端肥大症十多年的实际经验：ACROSTUDY。

目的 报告来自 ACROSTUDY 的用培维索孟治疗的肢端肥大症患者的最终长期安全性和有效性分析。全球设计（15 个国家），多中心、非干预性研究（2004-2017 年）。方法 完整的 ACROSTUDY 队列包括肢端肥大症患者，他们在研究前或入组时正在接受培维索孟 (PEGV) 治疗。主要终点是长期安全性（合并症、不良事件（AE）、垂体肿瘤体积、肝脏测试）和疗效（IGF1变化）。结果 患者（n = 2221）接受 PEGV 治疗的中位时间为 9.3 年（范围，0-20.8 年），中位随访时间为 7.4 年（范围，0-13.9 年）。在 PEGV 之前，96.3% 的患者接受过其他肢端肥大症治疗（手术/放疗/药物）。在 PEGV 治疗之前，87.2% 的患者报告了合并症。在 ACROSTUDY 期间，56.5% 的患者报告了 5567 例 AE，其中 613 例被认为与治疗相关（16.5% 的患者）并导致 1.3% 的患者停药。垂体成像显示 7.1% 的患者肿瘤大小增加；大多数（71.1%）报告没有变化。3.2% 的患者出现 AST 或 ALT 肝功能异常。IGF1 正常化率随着时间的推移而改善，从 PEGV 开始时的 11.4% 增加到第 1 年的 53.7%，并在越来越多的患者中使用 ≥30 mg PEGV/天，在第 10 年达到 75.4%。结.