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Sökning: WFRF:(van der Bom JG)

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  • Eckhardt, CL, et al. (författare)
  • Factor VIII gene (F8) mutation and risk of inhibitor development in nonsevere hemophilia A
  • 2013
  • Ingår i: Blood. - : American Society of Hematology. - 1528-0020 .- 0006-4971. ; 122:11, s. 1954-1962
  • Tidskriftsartikel (refereegranskat)abstract
    • The inhibitor incidence in nonsevere hemophilia A patients with certain F8 mutations approaches the inhibitor incidence in severe patients. These findings are highly relevant for clinical practice, as they facilitate identification of high-risk patients based on F8 genotype.
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  • Fischer, K, et al. (författare)
  • Prophylactic treatment for severe haemophilia: comparison of an intermediate-dose to a high-dose regimen
  • 2002
  • Ingår i: Haemophilia. - : Wiley. - 1351-8216. ; 8:6, s. 753-760
  • Tidskriftsartikel (refereegranskat)abstract
    • A multicentre study was performed in Sweden and the Netherlands, comparing effects of two prophylactic regimens in 128 patients with severe haemophilia, born 1970-90. 42 Swedish patients (high-dose prophylaxis), were compared with 86 Dutch patients (intermediate-dose prophylaxis). Patients were evaluated at the date of their last radiological score according to Pettersson. Annual clotting factor consumption and bleeding frequency were registered for a period of three years before evaluation. Patients in the high-dose group were younger at evaluation (median 15.2 vs. 17.9 years), started prophylaxis earlier (median 2 vs. 5 years), and used 2.19 times more clotting factor kg(-1) year(-1). Patients treated with high-dose prophylaxis had fewer joint bleeds (median 0.3 year(-1) vs. 3.3 year(-1)) and the proportion of patients without arthropathy as measured by the Pettersson score was higher (69% vs. 32%), however, the age-adjusted difference in scores (median 0 points vs. 4 points) was small and at present not statistically significant. Clinical scores and quality of life were similar. These findings suggest that, compared with intermediate-dose prophylaxis, high-dose prophylaxis significantly increases treatment costs and reduces joint bleeds over a period of 3 years, but only slightly reduces arthropathy after 17 years of follow-up.
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