Hur effektiv och säker är rekombinant och renad plasma faktor VIII-behandling för hemofili A patienter?
Haemophilia is an inherited disease which causes increased bleeding due to defect clotting factors VIII and/or IX. There are two forms of haemophilia, A and B, which both are X-linked and due to mutations of the genes of factor VIII and factor IX respectively. This study focuses on Haemophilia A and thereby factor VIII.The drugs that are available on the Swedish market today for patients with haemophilia A include Factor VIII(FVIII), which has been purified from human plasma and recombinant factor VIII(rFVIII). The main goal of this study is to gain a better understanding of the effects of treatment of haemophilia A with either purified plasma factor VIII or recombinant factor VIII on reducing or preventing bleeding and on side effects. The study was designed as a literature review and searches were carried out in PubMed at the Linnaeus University library. The criteria for selection of articles were: patients with haemophilia, treatment with plasma derived and/or recombinant factor VIII as well as clinical studies. The minimum number of patients was set at 20. The searches led to the review of six studies, where three used recombinant factor treatment and the other three referred to plasma-derived treatment.Efficacy and safety were evaluated in most cases by the hemostasis effect, reduced pain, reduced swelling, number of infusions, and number of reported adverse events. The incidence of inhibitor formation during treatment often resulted in the patient discontinuing the treatment. An increased development of neutralizing antibodies in the patient was evident in recombinant factor treatment, while there was an increased risk for transmission of hepatitis B or C in treatment with plasma derived from humans.In Sweden, one can choose to be treated on-demand, which reduces the risks tremendously for the haemophilia patient. However, this can increase the risk of inhibitor formation that can lead to ineffective treatment. The risk of bleeding in haemophilia patients can vary and is connected to the parent?s genes. Caution and self-awareness about the limitations decreases the risks of severe cases of bleeding.