治疗实践和卡波西形血管内皮瘤的反应:一个多中心队列研究
Alexandra J Borst1, Whitney Eng2, Morgan Griffin2
1Perelman School of Medicine at the University of Pennsylvania/Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, USA.
Pediatric blood & cancer
|December 10, 2023
概括
锡罗和温克里斯在治疗罕见的儿科血管瘤,如卡波西形血内皮瘤 (KHE) 和状血管瘤 (TA) 中表现出类似的有效性. 这两种疗法都有很高的响应率,指导个性化治疗决策.
科学领域:
- 儿科瘤学 儿科瘤学
- 血管生物学 血管生物学
- 罕见疾病 罕见疾病
背景情况:
- 形血管内皮瘤 (KHE) 和形血管瘤 (TA) 是罕见的儿科血管瘤.
- 从历史上看,这些疾病与显著的发病率和死亡率有关.
研究的目的:
- 在没有前性临床试验的情况下,确定KHE/TA的第一线治疗方法.
- 为了比较西罗和温克里斯作为一线治疗的疗效和耐用性.
主要方法:
- 对来自17所机构的159名KHE/TA患者 (2005-2020) 的回顾性分析.
- 3个月和6个月后对西罗和温克里斯的反应率的比较.
- 评估反应的耐久性和随后的治疗方式.
主要成果:
- 治疗总体响应率高:在3个月后>60%,在6个月后>70%.
- 3个月后,温克里斯的初始放射性反应比西洛利木斯 (72.7%对比20%,p=0.03) 高.
- 六个月后,西罗利木斯和温克里斯之间的反应率,持续时间或进展没有显著差异.
结论:
- 对KHE/TA的死亡率明显低于 (1.3%) 历史数据表明.
- 西洛利斯和温克里斯在KHE/TA的第一线治疗中表现出可比的疗效和耐久性.
- 结果支持基于临床因素和患者/医生偏好的个性化治疗计划,为未来的治疗方案提供信息.
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