Cost-effectiveness of erythropoietin in traumatic brain injury: a multinational trial-based economic analysis

Rachel J. Knott, Anthony Harris, Alisa Higgins, Alistair Nichol, Craig French, Lorraine Little, Samir Haddad, Jeffrey Presneill, Yaseen Arabi, Michael Bailey, D. James Cooper, Jacques Duranteau, Olivier Huet, Anne Mak, Colin McArthur, Ville Pettila, Markus Skrifvars, Shirley Vallance, Dinesh Varma, Judy WillsRinaldo Bellomo, for the EPO-TBI investigators and the ANZICS Clinical Trials Group

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The EPO-TBI multi-national randomized controlled trial found that erythropoietin (EPO), when compared to placebo, did not affect 6-month neurological outcome, but reduced illness severity-adjusted mortality in patients with traumatic brain injury (TBI), making the cost-effectiveness of EPO in TBI uncertain. The current study uses patient-level data from the EPO-TBI trial to evaluate the cost-effectiveness of EPO in patients with moderate or severe TBI from the healthcare payers' perspective. We addressed the issue of transferability in multi-national trials by estimating costs and effects for specific geographical regions of the study (Australia/New Zealand, Europe, and Saudi Arabia). Unadjusted mean quality-adjusted life-years (QALYs; 95% confidence interval [CI]) at 6 months were 0.027 (0.020-0.034; p < 0.001) higher in the EPO group, with an adjusted QALY increment of 0.014 (0.000-0.028; p = 0.04). Mean unadjusted costs (95% CI) were $US5668 (-9191 to-2144; p = 0.002) lower in the treatment group; controlling for baseline IMPACT-TBI score and regional heterogeneity reduced this difference to $2377 (-12,446 to 7693; p = 0.64). For a willingness-to-pay threshold of $US50,000 per QALY, 71.8% of replications were considered cost-effective. Therefore, we did not find evidence that EPO was significantly cost-effective in the treatment of moderate or severe TBI at 6-month follow-up.

Original languageEnglish
Pages (from-to)2541-2548
Number of pages8
JournalJournal of Neurotrauma
Issue number17
Publication statusPublished - 1 Sep 2019


  • cost-effectiveness
  • erythropoietin
  • multi-national trial
  • QALYs
  • traumatic brain injury

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