遺伝子組み換え型Fc融合第VIII因子製剤による免疫寛容導入療法を行ったインヒビター保有先天性血友病Aの３小児例

Abstract

Previous basic studies in vitro and in vivo have shown that recombinant Fe-fusion factor (F) VIII (rFVIIIFc) could induce and promote immunotolerance due to increasing the number of regulatory T cells, thereby reducing antigenicity to FVIII. Therefore, immune tolerance induction therapy (ITI) is expected to be effective in patients with hemophilia A and FVIII inhibitors (PwHAI) , aiming at eliminating the inhibitor. We experienced 3 pediatric cases of PwHAI including high responder on ITI using rFVIIIFc. Two of them had successful ITI and one had unsuccessful ITI, which appeared to be similar to the success rates reported in domestic and international studies. We suggest that ITI using rFVIIIFc would be useful.