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Hur effektiv och säker är rekombinant och renad plasma faktor VIII-behandling för hemofili A patienter?
Linnaeus University, Faculty of Health and Life Sciences, Department of Chemistry and Biomedical Sciences.
2013 (Swedish)Independent thesis Basic level (degree of Bachelor), 10 credits / 15 HE creditsStudent thesis
Abstract [en]

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.

Abstract [sv]

Hemofili, blödarsjuka, är en sjukdom där blodet har svårt att koagulera efter en skada. Sjukdomen beror på en mutation i genen för koagulationsfaktor VIII (hemofili A) respektive koagulationsfaktor IX (hemofili B). I båda fallen ger mutationerna defekta koagulationsfaktorer. Vanligast idag är att dessa sjukdomar behandlas med injektioner av de humana formerna av respektive faktor. Fokus i detta examensarbete inriktas mot hemofili A och faktor VIII.

Syftet med denna litteraturstudie är att undersöka hur effektiva och säkra två olika kommersiellt tillgängliga faktor VIII preparat är med avseende på att helt förhindra blödningar. Ett av preparaten är utvunnet från human plasma, det andra är framställt med hjälp av rekombinant DNA-teknik. Båda preparaten avvänds för behandling av hemofili A-patienter.

Studien utformades som en litteraturstudie och sökningar gjordes i PubMed. Kriterierna för val av artiklar var: patienter med hemofili, behandling med renad faktor VIII från blodplasma och/eller rekombinant faktor VIII (rFVIII) samt kliniska studier. Minimala antalet ingående patienter i en klinisk prövning sattes till 20. Sökningarna ledde till granskning av sex studier, där tre studier behandlade rFVIII-behandling och tre behandlade plasmaupprenad faktor VIII-behandling.

Effekt och säkerhet bedömdes i de flesta fall med effekt på hemostas, reducerad smärta, reducerad svullnad, antal infusioner och antalet upptäckta negativa fall under behandlingen. Såväl upprenad human faktor VIII som rekombinant framställd faktor VIII gav likvärdiga  och mycket goda effekter. De största riskerna var antikropputveckling under behandlingen, vilket oftast ledde till att patienten fick avbryta behandlingen. Störst antikropputveckling påvisades vid rFVIII-behandling. En allvarlig risk, som kan förekomma vid behandling med renad human faktor VIII, är samtidig överföring av virus, framför allt hepatit B- eller C-virus.

Place, publisher, year, edition, pages
2013. , 38 p.
Keyword [sv]
blödarsjuka, hemofili
National Category
Pharmaceutical Sciences
Identifiers
URN: urn:nbn:se:lnu:diva-25679OAI: oai:DiVA.org:lnu-25679DiVA: diva2:621563
Subject / course
Pharmacy
Educational program
Bachelor of Science Programme in Pharmacy, 180 credits
Presentation
(Swedish)
Supervisors
Examiners
Available from: 2013-05-27 Created: 2013-05-15 Last updated: 2013-05-27Bibliographically approved

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