title: A cost evaluation of treatment alternatives for mild-to-moderate bleeding episodes in patients with haemophilia and inhibitors in Brazil.
abstract: The first-line treatment for mild-to-moderate bleeding episodes in patients with haemophilia and inhibitors in Brazil is currently activated prothrombin complex concentrate (aPCC), with recombinant activated factor VII (rFVIIa) used as second-line therapy or as a last resort. The aim of this study was to determine the cost and effectiveness of these treatments from the perspective of the Brazilian National Health Service. A decision analysis model was constructed to assess total direct medical costs (including drug costs, costs of outpatient or inpatient care, ambulance transportation and cost of concomitant medications) of first-line treatment with aPCC or rFVIIa. Clinical outcome and resource utilization data were obtained both retrospectively and prospectively and validated by the consensus of an expert panel of Brazilian haematologists. A total of 103 bleeds in 25 patients were included in the analysis. rFVIIa resolved bleeds more quickly (4.4 h) than aPCC (62.6 h) and was more effective (100% vs. 56.7% respectively). Mean total direct medical costs (from initiation to cessation of bleed) were estimated to be US$13 500 (aPCC) and US$7590 (rFVIIa). Extensive sensitivity analyses confirmed the cost-effectiveness of rFVIIa. Compared with aPCC, rFVIIa was more effective and less expensive when used as first-line treatment for mild-to-moderate bleeding episodes in patients with haemophilia and inhibitors in Brazil. rFVIIa should be considered a first-line treatment for the management of these patients.
authors: Hemocentro da UNICAMP, Rua Carlos Chagas, CEP 13084-878 Campinas, São Paulo, Brazil. margaret@unicamp.br
authors: M C
authors: MC
authors: Ozelo
authors: P R
authors: PR
authors: Villaça
authors: J O S C
authors: JO
authors: De Almeida
authors: T M F
authors: TM
authors: Bueno
authors: P A P
authors: PA
authors: De Miranda
authors: W M
authors: WM
authors: Hart
authors: M
authors: M
authors: Karamalis
mesh_terms: D000293:Adolescent; D000328:Adult; D001938:Brazil; D002648:Child; D015331:Cohort Studies; D017281:Cost of Illness; D005167:Factor VII / Q000191:economics / Q000627:therapeutic use*; D015942:Factor VIIa; D005260:Female; D006467:Hemophilia A / Q000188:drug therapy* / Q000191:economics; D006470:Hemorrhage / Q000191:economics / Q000517:prevention & control*; D006801:Humans; D008297:Male; D011446:Prospective Studies; D011994:Recombinant Proteins / Q000191:economics / Q000627:therapeutic use; D012189:Retrospective Studies; D016896:Treatment Outcome
chemical_list: D011994:Recombinant Proteins; D005167:Factor VII; C103587:recombinant FVIIa; D015942:Factor VIIa