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针对毛细血管静脉形的向治疗

Lola Zerbib^1,2, Sophia Ladraa^1,2, Antoine Fraissenon^2,3,4,5

¹Université Paris Cité, Paris, France.

Signal transduction and targeted therapy

|June 16, 2024

概括

阿尔佩利西布通过抑制PI3Kα.有效地治疗与PIK3CA相关的毛细血管静脉形. 在小鼠模型中,这种治疗改善了病变和延长了生存时间,并且在患有PIK3CA或TEK突变的患者中显示出显著的体积减少.

科学领域:

遗传学遗传学是一个
血管生物学血管生物学
药理学药理学是指药理学的学科.

背景情况:

零星静脉形是由PIK3CA或TEK突变引起的,在没有经批准的治疗方法的情况下引起了严重的健康问题.
这些形导致疼痛,出血,血栓形成和可能危及生命的并发症.
目前的手术和硬化疗法等治疗方法往往不足.

研究的目的:

开发和验证与PIK3CA相关的毛细血管静脉形小鼠模型.
评估PI3Kα抑制剂,特别是alpelisib在治疗这些发育不良的疗效.
评估alpelisib在人类耐火性静脉形患者中的安全性和有效性.

主要方法:

创建了一个基因小鼠模型,模仿人类PIK3CA相关的毛细血管静脉形.
在小鼠模型中比较了alpelisib (PI3Kα抑制剂) 与其他药物,如拉巴素和米兰塞蒂布.
进行了一项临床试验,使用alpelisib治疗25名患有PIK3CA或TEK突变的患者 (包括儿童),并通过MRI评估结果.

主要成果:

在小鼠模型中,阿尔佩利西布在预防,改善和延长生存方面表现出有效性.
所有25名用alpelisib治疗的患者在6个月内都出现了改善,血管形体积减少 (33.4%用于PIK3CA,27.8%用于TEK).
临床症状减弱,以前难以治疗的形对阿尔佩利西布治疗有了反应.

结论:

用alpelisib抑制PI3Kα是PIK3CA或TEK相关的毛细血管静脉形的一个有前途的治疗策略.
阿尔佩利西布为患有严重或耐火性静脉形的患者 (包括儿童) 提供了可行的治疗选择.
这项研究验证了针对血管形中的PI3Kα途径的治疗潜力.

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