ISSN 1392-0138
ISSN 2029-4174 (online)
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2009 m. Nr. 1-2
Optimal treatment for children with haemophilia: a review
Sonata ŠAULYTĖ TRAKYMIENĖ, Lina RAGELIENĖ
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.
Keywords: haemophilia, prophylaxis, bleeding, joints
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