Regular Long-Term Continuous Replacement Therapy for Hemophilia

Masashi TAKI
2005 The Japanese Journal of Pediatric Hematology  
Hemophilia treatment in Japan is mainly on-demand therapy in which a deficient coagulation factor VIII or IX concentrate is administered to stop bleeding when it occurs. Although home infusion therapy on demand has improved QOL in hemophiliacs in Japan, progressive musculoskeletal damage such as hemophilic arthropathy still remains a problem due to frequent hemarthroses. In recent years, prophylactic therapy in which a deficient coagulation factor is regularly administered for a long period to
more » ... revent bleeding and joint damage from early infancy (primary prophylaxis) has been adopted in Europe, and prevention of the onset and progression of musculoskeletal disease has been reported. Regular long-term continuous replacement therapy (primary and secondary prophylaxis), however, has been studied by retrospective observations, and many issues, including its effectiveness and safety, still remain to be clarified. In the response to these situations, the Hemophilia Committee of the Japanese Society of Pediatric Hematology has commenced clinical research of regular long-term continuous replacement therapy for young children with severe hemophilia. In this article, background, definition, advantages and risks, current overseas situation, and the future issues of regular long-term continuous replacement therapy are discussed.
doi:10.11412/jjph1987.19.67 fatcat:b4vzwrhvfjbwzpdlvqmt7pgjvu