Pudjo Hagung Widjajanto
Division of Pediatric Hematology Oncology,
Faculty of Medicine, Public Health and Nursing, Universitas Gadjah MadaDr Sardjito Hospital, Yogyakarta
Hemophilia is the most common hereditary bleeding disorder in children that generates factor deficiency. Thus, factor replacement is essential and should be administered first to stop bleeding. There are many coagulation factors ever produced, from fresh frozen plasma, cryoprecipitated, plasma-derived concentrates, recombinant, and extended half-life factors. The development of coagulation factor products play important role for the major improvement in treatment outcomes. However, the appearance of neutralizing allo-antibody or inhibitor makes haemophilia treatment more complicated and expensive. Introduction of Activated Prothrombin Complex Concentrates (APCC) and recombinant activated Factor VII (rFVIIa) has improved the treatment result of patients with inhibitor. Novel treatment modalities are under development including gene therapy for producing normal level of coagulation factor, bispecific antibody to mimic the function of factor VIII, and targeting therapies to AT3 and TFPI. These options may reduce the need of coagulation factor and the appearance of inhibitor.
The ultimate goal of haemophilia management is to maintain the patient’s quality of life and multidisciplinary comprehensive care team is mandatory. It involves medical rehabilitation and psycho-social support after bleeding already overcame. In compare to on-demand factor treatment, the prophylactic factor administration showed more economical with better outcomes in reducing debilitating and chronic joint damage.
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