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依帕伐单抗(gamifant)治疗原发性噬血细胞性淋巴组织细胞增多症如何?

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印度代购提示,本品2020057日  瑞典制药公司Swedish Orphan Biovitrum ABSOBI)宣布,评估依帕伐单抗(emapalumab)治疗原发性噬血细胞性淋巴组织细胞增多症(HLH)疗效的关键II/III期临床研究(NCT01818492)的结果已于202057日发表于国际顶级医学期刊《新英格兰医学杂志》(NEJM)。

 

依帕伐单抗

 

  印度代购提示,本品NEJM上发表的这项研究,是一项全球性、多中心、开放标签、单臂、关键性研究,共入组了34例年龄≤18岁的原发性HLH患者,评估了依帕伐单抗与地塞米松联合用药,其中地塞米松剂量在研究期间可以逐渐降低。

 

  印度代购提示,本品34例患者中,7例在入组研究前未接受过治疗(初治组),27例接受过常规治疗(经治组)。

 

  印度代购提示,本品所有患者在入组研究时均有活动性疾病,经治组27例患者接受常规治疗期间或之后病情恶化或复发、或接受常规疗法未获得满意应答、或因副作用无法继续接受常规治疗。

 

  印度代购提示,本品截至2017720日,接受依帕伐单抗治疗的34例患者中,有26例完成了研究。数据显示:在完成8周治疗后,经治组(n=27)和整个研究组(n=34)分别有63%65%的患者病情缓解,这些百分比显著高于预先指定的40%的无效假设阈值(分别为p=0.02p=0.005)。经治组(n=27)和整个研究组(n=34)分别有70%65%的患者能够开展造血干细胞移植并且接受了移植。在最后一次观察中,经治组(n=27)、整个研究组(n=34)分别有74%71%的患者仍然存活。患者移植后表现出非常高的存活率,经治组和整个研究组接受了移植的患者中,12个月存活率分别为89.5%90.2%

As of July 20, 2017, 26 of 34 patients treated with epvacizumab have completed the study. Data showed that after 8 weeks of treatment, 63% of patients in the treated group (n=27) and 65% of patients in the study group (n=34) were in remission. These percentages were significantly higher than the prespecified null hypothesis threshold of 40% (P =0.02 and P =0.005, respectively). 70% of patients in the treated group (n=27) and 65% of patients in the study group (n=34) were able to undergo hematopoietic stem cell transplantation and received transplantation, respectively. At the last observation, 74% of patients in the treated group (n=27) and 71% of patients in the overall study group (n=34) were still alive. Patients showed a very high survival rate after transplantation, with a 12-month survival rate of 89.5% in the treated group and 90.2% in the entire study group.


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