• 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 Pettilä, Markus B Skrifvars, Shirley Vallance, Dinesh Varma, Judy Wills, and Rinaldo Bellomo.
• Centre for Health Economics, Monash Business School, Monash University, Melbourne, Victoria, Australia.
• J. Neurotrauma. 2019 Sep 1; 36 (17): 2541-2548.

AbstractThe 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.

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