Ineffective off-label use of recombinant activated factor VII in a case of bone-marrow transplantation-related gastrointestinal bleeding

We present a 45-year old, Caucasian male with persistent intestinal bleeding due to enterocolitis associated with cytomegalovirus infection and acute graft-versus-host-disease. He had received allogeneic peripheral blood stem cell transplantation from an unrelated HLA-identical donor because of chronic myelogenous leukaemia diagnosed two years earlier. Bleeding started at day 18 after transplantation with bloody diarrhea, which was treated with multiple transfusions of fresh frozen plasma, platelet, and red blood cell concentrates, and continued relentlessly, despite all efforts, including continued transfusions, high-dose prednisolone, broad antibiotic and antiviral coverage, and tranexamic acid. Recombinant FVIIa was started at boluses of 90–120 μg/kg every 4–8 hours. Despite more than 10 doses, recurrent severe bleeding progressed to refractory shock, multiorgan failure and death.Little can be concluded from single case reports of clinical improvement, because publication bias in favour of positive effects is likely. Our case suggests that rFVIIa is not a panacea, in particular for severe bleeding after bone-marrow transplantation. As long as rigorous, controlled studies or comprehensive registries are lacking, conventional interventions remain the standard of care in non-haemophilic patients with severe bleeding.Recombinant activated factor VII (rFVIIa) is an effective haemostatic agent in approximately 90% of patients with hemophilia and inhibiting antibodies, and in other types of complex coagulation disorders [1,2]. It has also been used in patients with a normal coagulation system, who experience serious bleeding. For these other patients, sound evidence from controlled clinical trials is only scarcely available so far. According to a recent systematic review, rFVIIa appears to be relatively safe with a 1–2% incidence of thrombotic complications based on published trials [3]. The main safety outcome in a recent trial on the use of rFVIIa in cerebral haemorrh