Implicazioni economiche del trattamento dei pazienti emofilici con inibitori: Una revisione sistematica della letteratura

Translated title of the contribution: Economic implications of treating patients with hemophilia and inhibitors: A systematic literature review

Cristina Negrini, F. Daniele, M. E. Mancuso, G. F. Rivolta, A. Tagliaferri

Research output: Contribution to journalArticlepeer-review

Abstract

Objectives: To evaluate resource utilization and related costs in the management of hemophilic patients with Factor VIII inhibitors. Methods: A systematic literature review was performed to identify studies that evaluated costs of the management of severe hemophilic patients with inhibitors from January 1998 to June 2005. Results: 26 papers meeting search criteria were found. Selected papers were examined and articles having no relevance to economic aspects were excluded as well as review articles; of the ten remaining papers, three were full economic evaluations and seven were cost-analyses. Most studies evaluated the economic burden of treating patients with inhibitors with an on-demand regimen. Two modelling studies were aimed at assessing lifetime costs and outcomes of the immune tolerance (ITI) approach to Factor VIII inhibitors eradication. Conclusions: Observational, non-controlled studies reported high treatment costs for inhibitors patients, while case-control experiences highlight that excess costs may be due to a minority of patients. This conflicting evidence makes the assessment of the optimal inhibitor treatment a difficult one. ITI treatment resulted in better outcomes, in terms of survival and quality-adjusted survival, at lower costs than on-demand treatment, when projected lifetime; ITI requires an initial prominent investment but, due to the high success rate, it allows a subsequent cost reduction in most subjects. Given the limited healthcare resources, a priority setting policy for ITI should consider the careful identification of clinical and non-clinical predictors of future medical needs and costs of hemophilic patients with inhibitors.

Translated title of the contributionEconomic implications of treating patients with hemophilia and inhibitors: A systematic literature review
Original languageItalian
Pages (from-to)33-44
Number of pages12
JournalPharmacoEconomics - Italian Research Articles
Volume8
Issue number1
Publication statusPublished - 2006

ASJC Scopus subject areas

  • Pharmacology
  • Health Policy

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