Off-label use of recombinant activated factor VII in hemophagocytic lymphohistiocytosis patients with major bleeding.

Abstract

Hemophagocytic lymphohistiocytosis (HLH) is a fatal multi-systemic disorder. Patients with HLH are more prone to bleeding, which implies a poor outcome. The objective was to investigate the efficacy of recombinant activated factor VII (rFVIIa) in major intractable bleeding of HLH. A total of 20 patients with secondary HLH received at least one dose of rFVIIa. When bleeding occurs, 88.9% cases had a nadir platelet counts below 25*10⁹/L. Median number of doses of rFVIIa was 3.5 (range, 1 to 78) with a mean dose of 76.45 µg/kg/dose. Total of 60% (12 of 20) patients obtained a response to rFVIIa therapy, however, in patients with a history of chronic active EBV infection (CAEBV), only 27.3% achieved a response. The mortality rate was 90% (18 of 20) in the cohort, none of the deaths were attributed to rFVIIa administration. Thromboembolism rate was low.