Objective

We report the final analysis of the French ACROSTUDY, using data revised and enriched since the 2013 interim analysis. Our objective was to validate the use of pegvisomant (PEGV) in the treatment of acromegaly and to determine efficacy and safety.

Patients and methods

Patients with acromegaly treated with PEGV and followed up for at least 5 years were included. Eighty-eight investigators from 62 clinical centers in France included patients from April 2007 to April 2014. PEGV dose and administration frequency were determined by the physicians, based on their clinical evaluation and local habits. No additional examinations beyond those performed in normal follow-up were required. Minimum recommended follow-up included check-ups at treatment initiation, 6 months, 12 months and then annually.

Results

In total, 312 patients were enrolled. Mean age was 46.1 ± 14.3 years at introduction of PEGV. Median PEGV treatment duration was 6.3 years and median follow-up was 5.6 years. Median dose at initiation was 10 mg/day. The percentages of patients with IGF-1 ≤ ULN (upper limit of normal) were 10% (n = 300) at baseline, 54% at 6 months (n = 278), and 61.7% (n = 253) at 2 years, then stabilizing at 64.4% (n = 180) at 5 years. Mean PEGV dose was 17.4 ± 11.7 mg in patients with controlled disease versus 21.1 ± 17.3 mg in those without control at 5 years. At 5 years, 21.8% of patients (54/248) were receiving >30 mg PEGV per day. In patients with at least one pituitary imaging procedure during the 5-year follow-up (n = 292), the most recent image showed stable tumor volume in 212 subjects (72.6%), increased volume in 13 (4.5%), and decreased volume in 30 (10.3%). No PEGV treatments were permanently discontinued due to transaminase elevation. There were no cases of liver failure.

Conclusion

The French ACROSTUDY showed normalization of IGF-1 levels in 64.4% of a real-life cohort of patients, mostly with uncontrolled disease despite multiple prior therapies. Long-term follow-up showed a sustained effectiveness and good long-term safety.

中文翻译：

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培维索孟治疗肢端肥大症的临床实践：法国 ACROSTUDY 的最终结果（312 名患者）

客观的

我们使用自 2013 年中期分析以来修订和丰富的数据报告了法国 ACROSTUDY 的最终分析。我们的目标是验证培维索孟 (PEGV) 在治疗肢端肥大症中的应用，并确定疗效和安全性。

患者和方法

包括接受 PEGV 治疗并随访至少 5 年的肢端肥大症患者。来自法国 62 个临床中心的 88 名研究人员包括 2007 年 4 月至 2014 年 4 月的患者。PEGV 剂量和给药频率由医生根据其临床评估和当地习惯确定。除了在正常随访中进行的检查外，不需要额外的检查。建议的最低随访包括治疗开始时、6 个月、12 个月和每年一次的检查。

结果

总共招募了312名患者。引入 PEGV 时的平均年龄为 46.1  ±  14.3 岁。中位 PEGV 治疗时间为 6.3 年，中位随访时间为 5.6 年。开始时的中位剂量为 10  mg/天。IGF-1 ≤ ULN（正常上限）的患者百分比在基线时为 10%（n  =  300），在 6 个月时为 54%（n  =  278），在 2 年时为 61.7%（n  =  253），然后在 5 年稳定在 64.4% ( n  =  180)。疾病控制患者的平均 PEGV 剂量为 17.4  ±  11.7 mg，而 21.1 ± 17.3    5 年时没有控制的人的毫克。5 年时，21.8% 的患者 (54/248) 每天接受 >30  mg PEGV。在 5 年随访期间（n  =  292）至少接受过一次垂体成像手术的患者中，最近的图像显示 212 名受试者（72.6%）的肿瘤体积稳定，13 名受试者（4.5%）的体积增加，并减少30 (10.3%)。没有因转氨酶升高而永久停止 PEGV 治疗。没有肝功能衰竭病例。

结论

法国 ACROSTUDY 显示，现实生活中 64.4% 的患者队列中 IGF-1 水平正常化，尽管先前接受了多种治疗，但大多数疾病未得到控制。长期随访显示持续有效和良好的长期安全性。