在儿童白血病患者中使用人口药理动力学选择伊萨图西马布剂量方案
Claire Brillac1, Dorothée Sémiond2, Corina Oprea1
1Sanofi Research and Development, Vitry-sur-Seine, 94403, France.
Cancer chemotherapy and pharmacology
|December 2, 2025
概括
种群药动力学建模证实,20mg/kg的伊萨图西马布剂量适用于患有复发性或耐药性急性白血病的儿科患者,包括婴儿.
科学领域:
- 药理学 药理学是指药理学的学科.
- 儿科瘤学 儿科瘤学
- 免疫治疗是一种免疫疗法.
背景情况:
- 伊萨图西马布是一种针对CD38的免疫球蛋白G单克隆抗体,已被批准用于复发性或耐火性多发性髓瘤.
- 临床前研究表明,伊萨图西马布具有抗白血病活性.
- 在ISAKIDS研究中,对儿科患者进行了评估,这些患者患有复发性或耐药性急性淋巴细胞白血病和急性髓性白血病.
研究的目的:
- 在ISAKIDS研究中使用人口药理动力学 (PopPK) 确定1至24个月龄的婴儿适合的伊萨图西马布剂量.
- 确认老年儿科患者的20mg/kg伊萨图西马布剂量,并探索年幼婴儿的剂量.
主要方法:
- 使用了人口药理动力学 (PopPK) 建模和模拟.
- 在ISAKIDS研究中的儿科患者的数据与ISLAY研究中的成人数据相结合.
- 一个初步的策略证实了20毫克/公斤剂量用于24个月及以上的患者.
主要成果:
- 与成年人相比,PopPK模型在10-24个月 (9-12公斤) 的儿科患者中显示了轻微的药物暴露不足 (大约30%的减少).
- 这种微小的差异不需要调整剂量.
- 在研究的临床环境中,没有观察到伊萨图西马布暴露与疗效之间的关系.
结论:
- 建模和模拟支持所有儿科年龄组的20mg/kg伊萨图西马布剂量.
- 这种方法降低了剂量选择的风险,并促进了进一步的儿科研究.
- 这些发现支持在最年轻的急性白血病患者中继续研究伊萨图西马布.
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