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新一代rhFVIII治疗患者抑制物形成发生率低

Low inhibitor incidence seen with new generation rhFVIII
来源:Frontline Medical News 2017-03-07 10:17点击次数:3030发表评论


正在进行中的NuProtect研究已获中期结果:新一代人类细胞系源性重组第8因子(human-cl rhFVIII)治疗甲型血友病重症患者的抑制物形成发生率较低。


Human-cl rhFVIII, a new generation recombinant factor VIII of human origin, appears to be associated with a low inhibitor incidence in patients with severe hemophilia A, according to interim findings from the ongoing NuProtect study.


Ellis J. Neufeld博士在欧洲血友病及相关疾病协会(EAHAD)年会报告里声称,66例初治患者在接受human-cl rhFVIII治疗至少20天后,高滴度和低滴度抑制物累计发生率分别为12.8%和8.4%。


In 66 evaluable previously untreated patients with at least 20 days of exposure to human-cl rhFVIII, the cumulative incidence of high-titer and low-titer inhibitor development was 12.8% and 8.4%, respectively, Ellis J. Neufeld, MD, reported at the annual meeting of the European Association for Haemophilia and Allied Disorders.


哈佛大学医学院的Neufeld博士称,患者初始治疗的中位年龄为13个月(3~135个月)。在接受F8基因分析的59例患者中,44例为高风险突变,47例为无效突变,1例未见突变。药物中位暴露时间11.5天后,66例患者中有8例出现高滴度抑制物,5例出现低滴度抑制物,其中4例为一过性。


The median age at first treatment was 13 months (range of 3-135 months). Of 59 patients with available F8 gene analysis, 44 had high-risk mutations and 47 had null mutations; 1 had no mutation identified. After a median of 11.5 treatment exposure days, 8 of the 66 patients developed high-titer inhibitors and 5 developed low-titer inhibitors; 4 of those were transient, said Dr. Neufeld of Harvard Medical School, Boston.


暴露20天后,仅2例患者出现抑制物,包括高滴度和低滴度抑制物各1例。在13例出现抑制物的患者中,12例进行了F8突变检测,全部为无效突变,11例为高风险突变。


Only two of the patients developed an inhibitor, including one high- and one low-titer inhibitor, after 20 exposure days. Among the 13 inhibitor patients, 12 had an F8 mutation identified; all were null, and 11 were high risk.


Neufeld博士指出,在此前开展的针对201例甲型血友病重症经治患者的human-cl rhFVIII(由人类细胞产生,未经化学修饰或蛋白融合)研究中,未见抑制物出现。NuProtect研究旨在评估human-cl rhFVIII用于初治患者的免疫原性、有效性和安全性,最终结果预期在2018年公布。


In a prior study of 201 patients with previously treated severe hemophilia A, no inhibitors were reported with human-cl rhFVIII – which is produced in human cells without chemical modification or protein fusion. The NuProtect study is looking at immunogenicity, efficacy, and safety in previously untreated patients. Final data are expected in 2018, Dr. Neufeld noted.


Neufeld博士披露与NuProtect研究申办方Octapharma公司及其他多家制药公司存在经济利益。


Dr. Neufeld disclosed financial ties to Octapharma, the sponsor of NuProtect, and numerous other pharmaceutical companies.


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学科代码: 血液病学     关键词:rhFVIII 血友病 抑制物形成 ,新闻 爱思唯尔医学网, Elseviermed
来源: Frontline Medical News
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