Optimal treatment for children with haemophilia: a review@@@Optimalus hemofilija sergančių vaikų gydymas (apžvalga)

correspondence to: Sonata Šaulytė trakymienė, Faculty of medicine, Vilnius university, Santariškių 7, lt-08661 Vilnius, lithuania. e-mail: sonata.trakymiene@vuvl.lt Background. Haemophilia is a rare, sex-linked inherited disorder caused by deficiencies of coagulation factor VIII or IX. The hallmark of severe haemophilia is recurrent bleedings into joints, resulting in lasting and irreversible changes and leading to haemophilic arthropathy. Treatment of haemophiliacs has undergone a revolutionary development. Prevention of joint bleed and haemophiliac arthropathy is possible by early regular administration of the deficient clotting factor, defined as prophylactic treatment. The purpose of this paper is to review the current knowledge of prophylactic treatment in haemophilia and the related aspects, and to discuss haemophilia care development and capabilities in Lithuania. Materials and methods. The literature data for the period 1992–2008 concerning prophylactic treatment from historical to current knowledge and haemophilia care development in Lithuania were analysed. Results. Data from observational studies and a recent randomized trial have shown that early prophylaxis is superior to on-demand treatment to prevent joint bleeds and arthropathy development. Despite quite a long experience with prophylactic treatment in some countries and the fact that it is recommended by international authorities, opinions concerning the prophylaxis differ in the world, and there is no uniform clinical practice because of lots of barriers. On-demand treatment strategy with plasma-derived factor concentrates is the standard practice in most clinics of Lithuania. Conclusions. The importance of prophylaxis is unquestioned today. Although definitive recommendations concerning one uniform prophylactic regimen cannot be made so far, primary prophylaxis nowadays is considered to be a standard care of children with severe haemophilia. In Lithuania, haemophilia still causes a significant morbidity because of its suboptimal treatment.