Is “compassionate use” compassionate?

The possibility that recombinant activated factor VII (rFVIIa) improves outcome following acute intracerebral hemorrhage (ICH)¹ has clinicians racing their engines in anticipation of at last having an effective treatment for ICH. At this exciting time, two articles^2,3 published in this issue of Neurology wave, if not the red “stop” flag, at least a yellow flag for “hazard on the track.”

In non-randomized, open-label series of patients with ICH treated with rFVIIa, Subramaniam et al.² report an unexpectedly high incidence of post-ICH hydrocephalus and Sugg et al.³ an increased incidence of post-ICH troponin-T elevation and myocardial infarction (MI). Five of nine ICH patients (56%) receiving 40 μg/kg rFVIIa in the study by Subramaniam et al. developed hydrocephalus. Although all five had intraventricular blood, their baseline CT scans suggested relatively low risk of subsequent hydrocephalus according to validated radiographic criteria⁴; the study did not include a concurrent control group. Sugg et al. evaluated 20 patients with ICH treated with 80 μg/kg rFVIIa and found troponin elevations in 4 (20%) and symptomatic MI in 2 (10%), …