Basic reserch on hemostasis to establish a novel hemostatic therapy for hemophilia A patients with inhibitors

2014 Fiscal Year Final Research Report

• Project/Area Number: 25860878
• Research Category: Grant-in-Aid for Young Scientists (B)
• Allocation Type: Multi-year Fund
• Research Field: Pediatrics
• Research Institution: Nara Medical University
• Principal Investigator: YADA Koji 奈良県立医科大学, 医学部, 特任助教 (30635785)
• Project Period (FY): 2013-04-01 – 2015-03-31
• Keywords: 血液凝固 / 新規治療 / インヒビター / 第VIII因子 / 出血性疾患

Outline of Final Research Achievements

Hemophilia A (HA) is a severe bleeding disease caused by quantitative and/or qualitative defect of factor (F) VIII. The development of anti-FVIII alloantibodies (inhibitors) during the hemostatic treatment with FVIII concentrates, making the hemostatic effect unstable, is one of the most serious events for HA patients. In this study, we prepared the treatment model for HA with inhibitors in vitro and investigated the inhibitory mechanism(s) of the inhibitors as well as the hemostatic effect of the therapy. As results of the several experiments with the various assays, it was found that the differences in epitope(s) and inhibitory kinetics of inhibitors affected the neutralizing effect with high dose of FVIII and that the hemostatic effect of the bypassing agent was significantly enhanced by the presence of FVIII even with the inhibitor. These findings will make a basis for establishment of a novel hemostatic therapy for HA with inhibitors.

Free Research Field

医歯薬，内科系臨床医学，小児科学，血液血栓止血学