Gandhi Chaitanya, Denis Marie-Claire, Holmes Daniel, Rivera Juan, van Uum Stan, Ezzat Shereen, Chik Constance
Department of Medicine, University of Alberta, Edmonton, AB, Canada.
Department of Medicine, Centre Hospitalier de l'Université Laval, Quebec City, QC, Canada.
Front Endocrinol (Lausanne). 2025 Jan 31;16:1516899. doi: 10.3389/fendo.2025.1516899. eCollection 2025.
Examine, in a real-world setting, whether strict normalization of modestly elevated insulin-like growth factor 1 (IGF1) results in clinical and health-related quality of life benefits in patients with acromegaly using an open-label, non-randomized, 6-month prospective interventional study.
In patients with acromegaly and modest IGF1 elevation, strict IGF1 control was achieved by addition or dose escalation of pegvisomant. Clinical and biochemical parameters were assessed at baseline, 1 and 3 months for pegvisomant dose titration, and at 6 months. The Patient-Assessed Acromegaly Symptom Questionnaire (PASQ), the Acromegaly Quality of Life questionnaire (AcroQoL) and the Acromegaly Disease Activity Tool (ACRODAT) were completed at baseline and at 6 months.
Ten patients (8 males) with mean age of 50.7 years participated in the study. All patients had a macroadenoma and nine had prior transsphenoidal surgeries. At time of screening, six patients were on a somatostatin analog, two on pegvisomant, and two on pegvisomant and a somatostatin analog. After six months of dose escalation or the addition of pegvisomant, IGF1 decreased from 1.22 ± 0.14 to 0.87 ± 0.20 times the upper limit of normal (p=0.001). PASQ score decreased by 3.5 (p=0.02) and the ACRODAT overall status decreased by 50.5 (p=0.001); however, there was no difference in the AcroQoL score. Hemoglobin A1c and liver enzymes did not differ and repeat MRI of the sella at 6 months showed no change.
In this pilot study, stricter control of modest IGF1 elevations led to symptomatic improvement as measured by the PASQ score. These findings prompt larger prospective trials.
在现实环境中，通过一项开放标签、非随机、为期6个月的前瞻性干预研究，检验对轻度升高的胰岛素样生长因子1（IGF1）进行严格标准化是否会给肢端肥大症患者带来临床及与健康相关的生活质量益处。
对于肢端肥大症且IGF1轻度升高的患者，通过添加培维索孟或增加其剂量来实现对IGF1的严格控制。在基线、第1个月和第3个月评估临床和生化参数以进行培维索孟剂量滴定，并在第6个月进行评估。在基线和第6个月完成患者评估的肢端肥大症症状问卷（PASQ）、肢端肥大症生活质量问卷（AcroQoL）和肢端肥大症疾病活动工具（ACRODAT）。
10名平均年龄为50.7岁的患者（8名男性）参与了该研究。所有患者均患有大腺瘤，9名患者曾接受经蝶窦手术。筛查时，6名患者使用生长抑素类似物，2名患者使用培维索孟，2名患者同时使用培维索孟和生长抑素类似物。在剂量增加或添加培维索孟6个月后，IGF1从正常上限的1.22±0.14降至0.87±0.20倍（p=0.001）。PASQ评分降低了3.5（p=0.02），ACRODAT总体状况降低了50.5（p=0.001）；然而，AcroQoL评分没有差异。糖化血红蛋白和肝酶无差异，6个月时蝶鞍的重复磁共振成像显示无变化。
在这项初步研究中，对轻度升高的IGF1进行更严格的控制导致PASQ评分所衡量的症状改善。这些发现促使开展更大规模的前瞻性试验。
