Inserm U1052, CNRS UMR5286, Claude Bernard Lyon 1 University, Cancer Research Center of Lyon, 69008 Lyon, France.
Endocrinology Department, "C.I. Parhon" National Institute of Endocrinology, 011863, Bucharest, Romania.
Eur J Endocrinol. 2024 Jan 3;190(1):K17-K20. doi: 10.1093/ejendo/lvad173.
Only 30% of patients with McCune-Albright syndrome (MAS)-associated acromegaly achieve biochemical control under first-generation somatostatin receptor ligands (fg-SRLs), while pegvisomant fails to normalize insulin-like growth factor 1 (IGF-I) in >20% of cases. Here, we report all the patients with MAS-associated acromegaly treated with pasireotide long-acting release (LAR) in our center. Pasireotide LAR 20 mg/month resulted in rapid and long-term IGF-I normalization in patients #1 and #3. Patient #3 was resistant to fg-SRLs, while patient #1 was also controlled on fg-SRLs. In patient #2, resistant to fg-SRLs and uncontrolled on pegvisomant 40 mg/day combined with cabergoline 0.5 mg/day, pegvisomant was replaced with pasireotide LAR 40 mg/month, resulting in the near normalization of IGF-I levels. All 3 patients developed intermittent impaired fasting glucose, without the need for glucose-lowering drugs. Thus, pasireotide LAR is clearly useful as third-line therapy, and potentially even as second-line therapy, in MAS-associated acromegaly.
仅有 30%的 McCune-Albright 综合征(MAS)相关肢端肥大症患者在第一代生长抑素受体配体(fg-SRLs)治疗下达到生化控制,而培维索孟未能使超过 20%的病例中胰岛素样生长因子 1(IGF-1)正常化。在这里,我们报告了在我们中心接受帕瑞肽长效释放剂(LAR)治疗的所有 MAS 相关肢端肥大症患者。帕瑞肽 LAR 20mg/月可使患者 #1 和 #3 的 IGF-1 迅速且长期正常化。患者 #3 对 fg-SRLs 耐药,而患者 #1 也对 fg-SRLs 控制良好。在对 fg-SRLs 耐药且不能用 40mg/天培维索孟联合 0.5mg/天卡麦角林控制的患者 #2 中,用帕瑞肽 LAR 40mg/月替代培维索孟,导致 IGF-1 水平接近正常。所有 3 名患者均出现间歇性空腹血糖受损,无需使用降糖药物。因此,帕瑞肽 LAR 显然可作为三线治疗药物,甚至可能作为二线治疗药物,用于 MAS 相关肢端肥大症。