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Comparative Effectiveness of In-Hospital Use of Recombinant Factor VIIa for Off-Label Indications vs. Usual Care

Slide: 33 of 36

Summary of Outcomes for Most Common Off-Label, In-Hospital Uses of rFVIIa

Overall study quality is fair to poor and the strength of evidence is low, with the exception of meta-analyses of intracranial hemorrhage that had moderate strength of evidence for all outcomes and of a meta-analysis of adult cardiac surgery studies that had moderate strength of evidence for the thromboembolic event outcome. Clinical efficacy is often defined via indirect/surrogate outcomes, such as transfusion requirements, change in hematoma volume, or ICU length of stay. Safety is defined via thromboembolic events and mortality, but individual studies often lack the statistical power to assess these outcomes. Evidence of rFVIIa benefit is suggested for several indications, but largely via the surrogate outcomes used in the included studies and with an uncertain relationship to improved patient survival or functional status. In addition, for some uses, rFVIIa produces an increased risk of thromboembolism. Current evidence of low strength suggests the potential for benefits to exceed harms for bleeding from body trauma. There are no indications where potential risks are likely to greatly exceed the benefits.

Intracranial hemorrhage: There are four RCTs and one observational study involving 968 rFVIIa-treated patients. Treatment with rFVIIa reduced expansion of intracranial hematoma volume relative to usual care, but increased the risk of arterial thromboembolic events and did not reduce the rates of mortality or poor functional outcome. Current evidence of moderate strength suggests that neither benefits nor harms substantially exceed each other.

Adult cardiac surgery: There are two RCTs and four included comparative observational studies with 251 patients receiving prophylactic or therapeutic rFVIIa. These studies showed that rFVIIa likely increased the risk of thrombembolic events, but failed to show an effect of rFVIIa on other outcomes, including mortality. rFVIIa use for this indication is increasing in the U.S.

Body trauma: There are two RCTs and two comparative observational studies examining rFVIIa treatment in 257 patients experiencing massive blood loss from trauma. These suggested a possible reduced rate of ARDS, most likely to be present in cases of blunt trauma, but these findings are complicated by the exclusion of patients with early mortality from both of the RCTs and one of the cohort studies. There is no evidence of effect on mortality or of increased thromboembolic events with treatment. Current evidence of low strength suggests the potential for benefit and little evidence of increased harm.