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延长半衰期凝血因子浓缩剂:已发表临床试验的结果

Extended half-life clotting factor concentrates: results from published clinical trials.

作者信息

Young G, Mahlangu J N

机构信息

Children's Hospital Los Angeles, University of Southern California Keck School of Medicine, Los Angeles, CA, USA.

Haemophilia Comprehensive Care Centre, Charlotte Maxeke Johannesburg Academic Hospital, Faculty of Health Sciences, University of the Witwatersrand and National Health Laboratory Service, Johannesburg, South Africa.

出版信息

Haemophilia. 2016 Jul;22 Suppl 5:25-30. doi: 10.1111/hae.13028.

Abstract

Extended half-life clotting factor concentrates have been recently introduced into the armamentarium of treatments for patients with haemophilia A and B. In general, the data from published studies have demonstrated these products to be safe with no inhibitors reported in previously treated patients and efficacious with the advantage of a longer half-life allowing for less frequent intravenous infusions of factor. This enhanced convenience has led to some patients not previously on prophylaxis to begin prophylaxis while for others, especially children, has led to the ability to provide prophylaxis with reduced use of central venous catheters. The extended half-life factor IX products are now allowing patients to dose every 1-2 weeks while maintaining higher trough levels while the extended half-life factor VIII products have reduced the frequency of administration for patients on prophylaxis to as infrequent as once per week for some patients and to twice per week for all patients including younger children. It is important to note that data from previously untreated patients have not been published yet and the incidence for inhibitors in this patient population is as of yet unknown. The era of extended half-life clotting factor products has begun and the challenge for the haemophilia community will be how to best integrate these products into haemophilia clinical practice.

摘要

延长半衰期的凝血因子浓缩物最近已被引入到治疗甲型和乙型血友病患者的手段中。总体而言,已发表研究的数据表明,这些产品是安全的,在先前接受治疗的患者中未报告有抑制剂出现,并且是有效的,其优势在于半衰期更长,从而减少了凝血因子静脉输注的频率。这种便利性的提高使得一些以前未接受预防治疗的患者开始进行预防治疗,而对于其他患者,尤其是儿童,使得能够在减少使用中心静脉导管的情况下进行预防治疗。延长半衰期的凝血因子IX产品现在允许患者每1至2周给药一次,同时保持较高的谷浓度,而延长半衰期的凝血因子VIII产品已将接受预防治疗患者的给药频率降低至一些患者每周一次,所有患者(包括年幼儿童)每周两次。需要注意的是,以前未治疗患者的数据尚未发表,该患者群体中抑制剂的发生率目前尚不清楚。延长半衰期凝血因子产品的时代已经开始,血友病领域面临的挑战将是如何最好地将这些产品整合到血友病临床实践中。

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