塔克罗利斯或酸莫菲蒂尔治疗频繁复发或类固醇依赖性性综合征:一项随机临床试验
Jingjing Wang1, Fei Liu1, Weili Yan2
1Department of Nephrology, Children's Hospital, Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hangzhou, China.
JAMA pediatrics
|May 12, 2025
概括
与mycophenolate mofetil (MMF) 相比,Tacrolimus (TAC) 治疗显著改善了患有经常复发性瘤综合征 (FRNS) 或类固醇依赖性瘤综合征 (SDNS) 的儿童的无复发生存率. 此外,TAC还降低了累积类固醇剂量,具有类似的安全性.
科学领域:
- 儿科脏病学 儿科脏病学
- 免疫抑制疗法 免疫抑制疗法
背景情况:
- 在儿童中,经常复发的性综合征 (FRNS) 和类固醇依赖性性综合征 (SDNS) 通常需要长期免疫抑制.
- 建议使用塔克罗利木斯 (TAC) 和甲酸莫菲蒂尔 (MMF),但随机试验的直接比较数据缺乏.
研究的目的:
- 为了比较TAC与MMF在FRNS或SDNS的儿科患者中的有效性和安全性.
- 为了评估无复发存活率,复发频率和累积类固醇剂量.
主要方法:
- 一个多中心的,开放的随机临床试验,涉及270名患有FRNS或SDNS的儿童 (2-18岁).
- 患者被随机分组1:1接受TAC或MMF口服1年,同时接受类固醇逐渐减少的治疗方案.
- 主要终点是1年无复发生存期;次要终点包括复发频率和类固醇剂量.
主要成果:
- 与mycophenolate mofetil (MMF) (HR,2.86;P <.001) 相比,塔克罗利斯 (TAC) 治疗导致显著更高的1年无复发生存率.
- TAC (323.99天) 的平均时间比MMF (263.21天) 长,年复发率较低 (17.78%vs41.48%).
- 接受TAC治疗的患者需要显著降低累积类固醇剂量 (0.22毫克/千克/天与0.34毫克/千克/天),并且组间的安全性概况相似.
结论:
- 与mycophenolate mofetil (MMF) 相比,一年的tacrolimus (TAC) 治疗显著改善了FRNS或SDNS儿童的无复发生存率.
- 在这种儿科群体中,TAC在减少疾病复发和类固醇依赖方面具有潜在的优势.
- 这些发现支持TAC作为儿童FRNS/SDNS管理的首选治疗方案,因为它的疗效和可比的安全性.
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