title: 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: BACKGROUND
  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.
  
  
  CONCLUSIONS
  Compared with a standard dose, a high initial rFVIIa dose may improve treatment outcomes without increasing costs. Early treatment initiation may reduce treatment costs.
authors: Institute of Haematology and Blood Transfusion, Prague, Czech Republic. Electronic address: peter.salaj@uhkt.cz.
authors: Peter
authors: P
authors: Salaj
authors: Orthopaedic Clinic, First Faculty of Medicine, Charles University, Prague and University Hospital Bulovka, Prague, Czech Republic.
authors: Radovan
authors: R
authors: Kubes
authors: Institute of Haematology and Blood Transfusion, Prague, Czech Republic.
authors: Petr
authors: P
authors: Cetkovsky
authors: Institute of Haematology and Blood Transfusion, Prague, Czech Republic.
authors: Irena
authors: I
authors: Capova
authors: Department of Clinical Haematology at University Hospital Brno, Czech Republic.
authors: Miroslav
authors: M
authors: Penka
authors: Institute of Biostatistics and Analyses at Masaryk University, Brno, Czech Republic.
authors: Petra
authors: P
authors: Ovesná
authors: Institute of Environmental Medicine, Karolinska Institute, Stockholm, Sweden.
authors: Johan
authors: J
authors: Mesterton
authors: Institute of Environmental Medicine, Karolinska Institute, Stockholm, Sweden.
authors: Peter
authors: P
authors: Lindgren
mesh_terms: D000328:Adult; D018153:Czech Republic; D015942:Factor VIIa / Q000008:administration & dosage* / Q000191:economics*; D006467:Hemophilia A / Q000188:drug therapy* / Q000191:economics*; D002836:Hemophilia B / Q000188:drug therapy* / Q000191:economics*; D006470:Hemorrhage / Q000188:drug therapy / Q000191:economics; D006801:Humans; D016014:Linear Models; D011994:Recombinant Proteins / Q000008:administration & dosage / Q000191:economics; D012189:Retrospective Studies; D055815:Young Adult
chemical_list: D011994:Recombinant Proteins; C103587:recombinant FVIIa; D015942:Factor VIIa
keywords: BU; Bethesda Units; CZK; Costs; Czech Koruna; GLM; Haemophilia; High dose; NovoSeven®; Time to treatment; generalized linear modelling; rFVIIa; recombinant factor VIIa