5 5 9 0 6

论文已发表


注册即可获取德孚的最新动态



IF 收录期刊



更多详情 >>



作者优惠计划

Favored Author Program


很高兴为我们作者提供一种切实的方法以支持开放获取,并鼓励教师和研究人员通过开放获取模式,尽可能广泛地传播他们的作品。


作者优惠计划的成员将获得:


文章发表费(APC) 10% 的折扣


这 10% 的折扣从您加入 “作者优惠计划” 之时开始,并将适用于之后提交的所有论文



更多详情 >>



 

已发表论文

Safety and Efficacy of Turoctocog Alfa in the Prevention and Treatment of Bleeding Episodes in Previously Treated Patients from China with Severe Hemophilia A: Results from the Guardian 7 Trial

 

Authors Wu R, Sun J, Xu W, Hu Q, Li W, Xiao J, Yang F, Zeng X, Zeng Y, Zhou J, Matytsina I, Zhang S, Pluta M, Yang R

Received 24 December 2019

Accepted for publication 30 April 2020

Published 23 June 2020 Volume 2020:16 Pages 567—578

DOI https://doi.org/10.2147/TCRM.S243146

Checked for plagiarism Yes

Review by Single-blind

Peer reviewer comments 2

Editor who approved publication: Professor Deyun Wang

Purpose: Hemophilia care in China is characterized by widespread use of on-demand regimens and low-dose prophylaxis. With a limited number of approved recombinant factor VIII (FVIII) products, the incidence of arthropathy and disability in hemophilia patients remains high in China. The purpose of this trial was to evaluate the safety and efficacy of turoctocog alfa for prophylaxis and treatment of bleeding episodes in patients from China with severe hemophilia A across all age groups.
Patients and Methods: In this Phase 3, open-label trial, previously treated males of all ages with severe hemophilia A from China received turoctocog alfa for prophylaxis or on-demand treatment of bleeds. The primary endpoint was hemostatic effect for the treatment of bleeds during the main phase of the trial. Secondary endpoints included annualized bleeding rate during prophylaxis and the frequency of FVIII inhibitor development.
Results: Overall, 42 pediatric patients (age < 12 years) and 26 adolescent/adult patients (≥ 12 years) were dosed with turoctocog alfa; 51 patients initiated treatment with prophylaxis, while 17 patients initiated on-demand treatment. During the main phase of the trial (6 months), hemostatic success was 95.1%. During the full trial (up to 24 months), hemostatic success was 95.4%; the overall median ABR was 1.18 bleeds/patient/year for prophylaxis patients; and 25 (51.0%) of 49 patients with target joints at baseline had all target joints resolved. No FVIII inhibitors (≥ 0.6 BU) were reported.
Conclusion: Turoctocog alfa was safe and effective for prophylaxis and treatment of bleeding episodes and for surgery in patients from China with severe hemophilia A across all ages.
Keywords: hemophilia A, prophylaxis, recombinant factor VIII, turoctocog alfa, China




Figure 2 Mean FVIII activity profile following administration of...