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Master Thesis

Étude rétrospective monocentrique dans un centre hospitalier universitaire : passage à un facteur VIII recombinant à demi-vie prolongée chez l’hémophile A : avantages cliniques et économiques

Abstract : Background : Recombinant factor VIII Fc fusion protein (rFVIIIFc) is the first extended half-life (EHL) recombinant clotting factor with marketing authorization, available in France since October 2016. Data and literature in clinical practice are limited. We propose one-year clinical and economic outcomes evaluation in patient with haemophilia considering adherence to treatment. Study design and method : We reviewed diaries of all patients treated with rFVIIIFc at Marseille Haemophilia Center for one year. No estimation on prescribed regimen have been realized. All data were related to patient’s infusions (i.e. annual number of infusions, weekly dose/kg, and annual consumption) and bleeding reports. Statistical significance was set at p<0.05. Only clotting factor costs are taken into account. We neglected other costs (e.g. dispositive, nurse intervention…). Results : 34 patients were concerned. The median age was 18 years (IQR = 18). Adherence occurred in 62% for FVIII and 66% for rFVIIIFc. The analysis reported a slight decrease of annual clotting factor consumption consecutive to the switch (-2%, p = 0.7339) with a total utilization of 7 001 500 IU (med = 185 000, IQR = 192 625) for FVIII and 6 892 500 IU (med = 202 000, IQR = 159 000) for rFVIIIFc. It is important to note that 23% of patients were < 12 years of age and 31% of patients were related to adjustment of doses for weight evolution, clinical need or ease of use. These data were combined with a significant reduction of annual infusions number (-22.5%, med = 138.5, IQR = 65.8 for FVIII; med = 105, IQR = 24 for rFVIIIFc, p < 0.0001) and bleedings (-50%, med = 5, IQR = 7.5 for FVIII; med = 1, IQR = 4 for rFVIIIFc, p < 0.0001). Concerning the cost, we noted a non-significant decrease (-8%, p = 0.1300) representing saving of 384 636 € for one year.
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Master Thesis
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Submitted on : Monday, September 28, 2020 - 8:49:05 AM
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  • HAL Id : dumas-02950493, version 1

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Romain Giraud. Étude rétrospective monocentrique dans un centre hospitalier universitaire : passage à un facteur VIII recombinant à demi-vie prolongée chez l’hémophile A : avantages cliniques et économiques. Sciences pharmaceutiques. 2020. ⟨dumas-02950493⟩

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