2014年12月9日讯 /生物谷BIOON/ --2014年第56届美国血液学会年会（ASH）于12月6日-9日在美国旧金山举行。近日，诺和诺德（Novo Nordisk）在会上公布了A型血液病药物NovoEight®（turoctocog alpha，重组凝血因子VIII）III期Guardian项目新的分析数据。Guardian-TM1研究显示，NovoEight初始治疗时年平均出血率（ABR）最高的A型血液病患者群体，在治疗持续期间出血次数表现出最大幅度的降低。Guardian-TM2研究的事后分析（post-doc analysis）进一步发现，与Guardian-TM1研究中的ABR相比，当成人和青少年患者群体继续接受NovoEight®治疗时ABR分别降低69%和63%。Guardian-TM1和Guardian-TM3研究显示，NovoEight®能够有效预防和减少出血，在既往接受NovoEight®治疗的213例患者体内未产生针对NovoEight®的中和性抗体（抑制剂）。
III期Guardian项目是迄今为止在A型血液病患者中开展的最大的临床预注册试验项目，涉及超过200例A型血有病患者，这些III期临床试验包括既往接受过治疗的重度A型血有病患者和儿科患者，研究结果证明了NovoEight®能够有效预防和治疗出血事件，同时患者体内未产生抑制剂。低ABR基线患者群体和高基线ABR患者群体都能够从NovoEight®预防性治疗中受益。
关于NovoEight®
NovoEight®是诺德诺德第三代重组凝血因子VIII，利用了最新的基因重组及蛋白纯化技术，开发用于A型血友病患者出血事件的预防和治疗。目前，NovoEight®已获美国、欧盟、日本、澳大利亚、瑞士批准，用于A型血有病患者预防和治疗出血事件。A型血液病是一种遗传性疾病，因缺乏凝血因子八（VIII）所致，是临床上最常见的血液病类型，约占血液病的80%-85%。
英文原文：NovoEight®, phase 3 data show reduction in annualised bleeding rate over time in people with haemophilia A
San Francisco, US, 7 December 2014 - Today, Novo Nordisk announced a new analysis of phase 3 data demonstrating people with haemophilia A who had the highest annualised bleeding rate (ABR) during initial treatment with NovoEight® (turoctocog alfa) showed the largest reduction in bleeding over the duration of treatment. Data for the recombinant factor VIII (rFVIII) product NovoEight® were presented at the 56th Annual Meeting of the American Society of Hematology (ASH).1
The post hoc analysis from the guardian(TM)2 trial further found that, when continued on treatment with NovoEight®, adults and adolescents showed reductions of 69% and 63%, respectively, in ABR compared with their ABRs from guardian(TM)1. The guardian(TM)1 trial (along with guardian(TM)3) showed that NovoEight® was efficacious in preventing and reducing bleeds with no confirmed inhibitor development in 213 previously treated patients.2,3
"This post hoc analysis gives an indication that patients with high baseline bleed rate can benefit from prophylaxis with NovoEight®, as well as patients who maintain low ABR," said Margareth Ozelo, M.D., Ph.D., Hemocentre, IHTC, University of Campinas, Sao Paulo, Brazil, and lead investigator.
The most common adverse reactions (=>0.5%) seen in the study were injection site reactions, increased hepatic enzymes, and fever.4
About NovoEight®
NovoEight® is approved by the US Food and Drug Administration (October 2013) and the European Medicines Agency (November 2013) for the treatment and prophylaxis of bleeding in patients with haemophilia A. NovoEight® is also approved in Japan (January 2014), Australia (January 2014), and Switzerland (February 2014), and applications for regulatory approvals have been submitted in a range of other countries.
About guardian(TM)
The pivotal guardian(TM) clinical programme is one of the largest and most comprehensive preregistration clinical trial programmes in haemophilia, with more than 210 patients with severe haemophilia A treated. The guardian(TM)2 trial is an extension of the clinical programme. The post hoc analysis of the guardian(TM)2 trial involved 166 patients who had participated in guardian(TM)2 and either guardian(TM)1 or 3, had <=1 week of surgery treatment in guardian(TM)1 or 3 (initial period), and had =>3 months of exposure to turoctocog alfa prophylaxis during a selected time period (1 January 2012-30 June 2013) in guardian(TM)2.
About Haemophilia A
Haemophilia is a chronic, inherited bleeding disorder that primarily affects males. People with haemophilia A are either missing or have a malfunctioning factor VIII protein, which is essential for proper blood clotting. People with haemophilia A have a tendency to bleed longer than most or to bleed internally into muscles, joints or organs because they are missing this clotting factor. To manage the disease and stop bleeding, people with haemophilia A must replace the missing factor VIII protein, which is accomplished by intravenous injection of the clotting factor.5
Globally, it is estimated that more than 400,000 people have haemophilia.6 The disease is severely underdiagnosed in developing countries.