A Trend of Primary Regular Replacement Therapy for Hemophilia

概要

論文の詳細を見る
A common strategy for replacement therapy in patients with hemophilia is replacement of the deficient co-agulation factor (factor VIII or IX) in the event of bleeding, for a certain period until the bleeding stops, which is referred to as on-demand therapy or episodic therapy. The Scandinavian countries have recently begun toemploy regular replacement therapy (referred to as prophylaxis in the US and Europe), with regular replacement of the coagulation factor over the long term, even in the absence of hemorrhage, in order to prevent bleeding as well as prevent the development and progression of arthropathy, and the usefulness of this strategy in the prevention of arthropathy has been reported. However, as the outcomes of regular replacement therapy are mainly derived from retrospective observational studies, lack of sufficiently strong evidence for the effectiveness and safety of the therapy has been pointed out. Therefore, the Hemophilia Committee of the Japanese Society of Pediatric Hematology has launched a prospective clinical study. With accumulation of further evidence in regard to the effectiveness of regular replacement therapy from progress reports of the ongoing prospective randomized controlled trials in the US and Europe, replacement therapy for hemophilia is now expected to enter a new era.
特定非営利活動法人　日本小児血液・がん学会の論文