CRESTIC EA 3804, université de Reims Champagne Ardenne, UFR Sciences Exactes et Naturelles, Moulin de la Housse, BP 1039, 51687 Reims cedex 2, France; Service d'Endocrinologie-Diabète-Nutrition, CHU de Reims, hôpital Robert Debré, avenue du Général Koenig, 51092 Reims cedex, France.
Service d'endocrinologie et maladies métaboliques, pôle cardio-vasculaire et métabolique, hôpital Larrey, CHU de Toulouse, 24, chemin de Pouvourville, TSA 30030, 31059 Toulouse cedex 9, France.
Ann Endocrinol (Paris). 2021 Dec;82(6):582-589. doi: 10.1016/j.ando.2021.05.004. Epub 2021 Jul 10.
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 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.
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 10mg/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±11.7mg in patients with controlled disease versus 21.1±17.3mg in those without control at 5 years. At 5 years, 21.8% of patients (54/248) were receiving >30mg 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.
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.
我们报告了法国 ACROSTUDY 的最终分析结果，该分析使用了自 2013 年中期分析以来修订和丰富的数据。我们的目的是验证培维索孟（PEGV）在治疗肢端肥大症中的应用，并确定其疗效和安全性。
纳入接受 PEGV 治疗并至少随访 5 年的肢端肥大症患者。来自法国 62 个临床中心的 88 名研究者于 2007 年 4 月至 2014 年 4 月期间纳入患者。根据医生的临床评估和当地习惯，确定 PEGV 的剂量和给药频率。除正常随访中进行的检查外，无需进行其他额外检查。最低推荐随访包括治疗开始时、6 个月、12 个月和每年进行检查。
共纳入 312 例患者。PEGV 起始时患者的平均年龄为 46.1±14.3 岁。中位 PEGV 治疗持续时间为 6.3 年，中位随访时间为 5.6 年。起始时的中位剂量为 10mg/天。基线时 IGF-1≤ULN（正常值上限）的患者百分比为 10%（n=300），6 个月时为 54%（n=278），2 年时为 61.7%（n=253），然后在 5 年时稳定在 64.4%（n=180）。疾病控制患者的平均 PEGV 剂量为 17.4±11.7mg，而 5 年时未控制疾病患者的剂量为 21.1±17.3mg。在 5 年随访期间至少进行了一次垂体影像学检查的患者（n=292）中，最近的影像学检查显示 212 例（72.6%）肿瘤体积稳定，13 例（4.5%）肿瘤体积增大，30 例（10.3%）肿瘤体积缩小。由于转氨酶升高，没有患者永久停止 PEGV 治疗。没有发生肝衰竭的病例。
法国 ACROSTUDY 显示，在现实生活中，大多数患者（尽管之前接受过多次治疗）疾病仍未得到控制，但仍有 64.4%的患者 IGF-1 水平正常。长期随访显示其持续有效且长期安全性良好。
