在第一线设置中青少年使用卡普拉西祖马布的现实世界经验:病例报告
Thuy B Tran1, Jennifer Boyce, Heather Mitchell
1Department of Pharmacy, Specialty Pharmacy Services, Atrium Health, Charlotte, NC.
Journal of pediatric hematology/oncology
|August 1, 2023
概括
卡普拉西祖马布在青少年中有效治疗了获得的血栓性血栓性缺血性紫外线 (aTTP),使得早期停止治疗成为可能. 这为在aTTP的年轻患者中中止治疗提供了潜在的新标准.
科学领域:
- 血液学 血液学 血液学
- 免疫学 免疫学 免疫学
- 药理学 药理学是指药理学的学科.
背景情况:
- 获得的血栓性血栓细胞性紫外线 (aTTP) 是一种危及生命的血栓性微血管病变.
- 卡普拉西祖马布是成年人TTP的批准治疗方法,在血小板正常化和减少疾病复发方面表现出有效性.
- 关于青少年群体使用卡普拉西祖马布的数据有限.
研究的目的:
- 报告卡普拉西祖马布在两个青少年aTTP病例中的非标签使用情况.
- 在这个儿科队列中评估卡普拉齐祖马布的疗效和安全性.
- 探索在青少年中提前停止卡普拉西祖马布治疗的潜在标准.
主要方法:
- 两名患有aTTP的青少年患者接受了非标签的卡普拉西祖马布治疗.
- 在血小板正常化和ADAMTS13活性恢复 (>20-30%) 后考虑停止治疗.
- 治疗的持续时间和血交换被监测.
主要成果:
- 这两名青少年患者都实现了血小板正常化和ADAMTS13恢复,允许在血交换后30天之前停止治疗.
- 在这些病例中观察到的疗效与其他接受caplacizumab治疗的儿科患者报告的结果一致.
- 在这些情况下,没有注意到与卡普拉西祖马布相关的不良事件.
结论:
- 卡普拉西祖马布在患有aTTP的青少年患者中显示出有效性,与成人和其他儿科报告相似.
- 在青少年中,一旦达到临床和实验室缓解标志物,早期停止卡普拉西祖马布治疗似乎是可行的.
- 这些发现可能有助于制定儿童aTTP治疗中卡普拉齐祖马布早期戒断指南.
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