帕西里欧提德:对于麦肯-阿尔布赖特相关的壮病的潜在治疗选择
Mirela-Diana Ilie1,2, Gérald Raverot1,3, Aude Brac de la Perrière3
1Inserm U1052, CNRS UMR5286, Claude Bernard Lyon 1 University, Cancer Research Center of Lyon, 69008 Lyon, France.
European journal of endocrinology
|December 21, 2023
概括
帕西雷胺长效释放 (LAR) 显示出对麦库恩-阿尔布赖特综合征 (MAS) 相关的壮症有前途. 这种治疗在对其他疗法耐药的患者中实现了IGF-I正常化,这表明它作为第二或第三线选择的实用性.
科学领域:
- 内分泌学 在内分泌学.
- 代谢障碍 代谢障碍 代谢障碍
- 药理学 药理学是指药理学的学科.
背景情况:
- 麦库恩-阿尔布赖特综合征 (MAS) 相关的壮症带来了治疗挑战,第一代体静止素受体连接体 (fg-SRLs) 和pegvisomant的疗效有限.
- 像fg-SRLs这样的标准疗法只能在30%的MAS相关壮病患者中实现生化控制,而pegvisomant在20%以上的病例中未能使IGF-I正常化.
研究的目的:
- 为了评估在患有麦库恩-阿尔布赖特综合征 (MAS) 相关的壮症的患者中,抗胺长效释放 (LAR) 的疗效和安全性.
- 评估沙里胺LAR作为这种特定患者群体的潜在第二或第三线治疗选择.
主要方法:
- 对三个患有MAS相关壮病的患者进行了回顾性分析,这些患者在一个中心接受了沙利西LAR治疗.
- 评估胰岛素样生长因子1 (IGF-I) 水平和葡萄糖代谢参数在治疗前和治疗期间.
主要成果:
- 帕西雷胺LAR (20毫克/月) 导致两名患者的IGF-I正常化迅速和持续,其中一人对fg-SRLs耐药.
- 在第三个患者中,该患者对fg-SRLs有耐药性,并且对维索曼特加卡伯戈林的控制不充分,转换为沙利西LAR (40 mg/月) 导致IGF-I近乎正常化.
- 所有患者都经历了间歇性禁食葡萄糖受损,在没有降血糖药物的情况下进行管理.
结论:
- 帕西里奥提德LAR证明了作为MAS相关壮症的第三线治疗的显著实用性.
- 帕西里奥提德LAR也可以被视为二线治疗选择,用于那些与MAS相关的壮症患者,这些患者对初始治疗没有充分的反应.
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