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Economic evaluation of rFVIIa high initial dose compared to rFVIIa standard initial dose in patients with haemophilia with inhibitors using the Czech HemoRec registry

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SALAJ Peter KUBES Radovan CETKOVSKÝ Petr CAPOVA Irena PENKA Miroslav OVESNÁ Petra MESTERTON Johan LINDGREN Peter

Rok publikování 2014
Druh Článek v odborném periodiku
Časopis / Zdroj Thrombosis Research
Fakulta / Pracoviště MU

Lékařská fakulta

Citace
Doi http://dx.doi.org/10.1016/j.thromres.2013.11.002
Obor Onkologie a hematologie
Klíčová slova Haemophilia; NovoSeven (R); rFVIIa; High dose; Costs; Time to treatment
Popis Introduction: In the treatment of bleeds in haemophilia patients with inhibitors, a high initial dose of recombinant Factor VIIa (rFVIIa) provides at least equal efficacy and a similar safety profile to a standard initial dose. However, no pharmacoeconomic comparison between these dosing regimens has previously been performed. Here, we assess the pharmacoeconomics of high (> 120 mu g/kg) versus standard (<= 120 mu g/kg) initial rFVIIa dose in inhibitor patients and the impact of time to treatment initiation on costs and outcomes. Methods: In a retrospective analysis, observational data on bleed characteristics, rFVIIa treatment, hospitalizations and outcomes were extracted from the Czech Republic HemoRec registry. Crude comparisons and generalized linear regression modelling (GLM; correcting for patient differences) were performed to compare costs and outcomes between the high and standard initial dosing groups. Results: Of 314 rFVIIa-treated bleeding episodes (12 inhibitor patients), most were spontaneous joint bleeds and 67.5% were treated with a high initial dose. In the crude comparison, high initial rFVIIa dosing was associated with a lower mean number of doses needed to achieve haemostasis compared with standard dosing (p < 0.001), but higher total dose and costs (p <= 0.008). However, regression analyses revealed that high initial dose was associated with similar costs (p = 0.891) and a shorter time to bleeding resolution (p = 0.014). Increasing time to treatment initiation increased both time to bleeding resolution and total costs. Conclusion: Compared with a standard dose, a high initial rFVIIa dose may improve treatment outcomes without increasing costs. Early treatment initiation may reduce treatment costs.

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