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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.

Abstract：

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 μg/kg) versus standard (≤120 μg/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.

journal_name

Thromb Res

journal_title

Thrombosis research

authors

Salaj P,Kubes R,Cetkovsky P,Capova I,Penka M,Ovesná P,Mesterton J,Lindgren P

doi

10.1016/j.thromres.2013.11.002

subject

Has Abstract

pub_date

2014-02-01 00:00:00

pages

162-7

issue

eissn

0049-3848

issn

1879-2472

pii

S0049-3848(13)00520-3

journal_volume

133

pub_type

杂志文章

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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.