Real-World Early Treatment with Room Temperature-Stable Recombinant Factor VIIa in Hemophilia A/B and Inhibitors: SMART-7™ Post Hoc Analyses.

Treating hemophilia A or B patients with inhibitors is particularly challenging, as they do not respond to replacement therapy with factor VIII or factor IX concentrates. A room temperature-stable formulation of recombinant activated factor VII (rFVIIa; NovoSeven ® ), which provides improved convenience and treatment access to patients compared with the earlier formulation of rFVIIa, was shown to be safe and effective in a post-authorization, multinational, observational study (Study Monitoring Antibodies against Room Temperature-stable factor 7 [SMART-7™]). In post hoc, subgroup analyses of SMART-7™ data, the hemostatic response following rFVIIa monotherapy in patients with hemophilia A or B with inhibitors by time to first treatment and in different age cohorts was assessed. A total of 482/618 bleeding episodes treated with rFVIIa monotherapy and with (1) valid efficacy assessment, (2) no missing time for bleed start, (3) no missing time for any dose administration, and (4) valid time to first treatment were included in the analyses. Data on the type and location of bleeding episodes treated with rFVIIa monotherapy were also collected. The majority of bleeding episodes treated with rFVIIa monotherapy were treated within 1 hour after bleeding onset (318/482 [66%]) and, among them, 96.5% (307/318) were effectively treated (i.e., bleeding stopped). Hemostatic efficacy remained high for bleeding episodes treated >1 to ≤4 hours after the onset, with 94/101 (93.1%) treated effectively. Cause and location of bleeding varied across the different age groups assessed. Real-world evidence from post hoc, subgroup analyses of SMART-7™ data confirmed that patients were able to treat themselves quickly and that early treatment with rFVIIa was associated with high efficacy.

The use of recombinant activated factor VII in patients with acquired haemophilia.

Acquired haemophilia (AH) is a rare, often severe bleeding disorder characterised by autoantibodies to coagulation factor VIII (FVIII). Observational studies offer crucial insight into the disease and its treatment. Recombinant activated factor VII (rFVIIa, eptacog alfa activated) was available on an emergency and compassionate use basis from 1988 to 1999 at sites in Europe and North America. In 1996, rFVIIa was approved in Europe for the treatment of AH; it was licensed for this indication in the United States in 2006. Recombinant activated FVII is approved for first-line treatment of bleeding episodes and prevention of bleeding in surgical/invasive procedures in patients with AH. This review provides an up-to-date summary of the haemostatic efficacy of rFVIIa in patients with AH, from the first emergency and compassionate use programmes, to patient registries and a post-marketing surveillance study. In acute bleeding episodes, rFVIIa provided high and consistent rates of control, and available data showed that acute bleed control rates were higher for first-line rFVIIa versus salvage rFVIIa. In surgical procedures, rFVIIa also provided high rates of control. In patients with AH, rFVIIa has a high rate of haemostatic efficacy in acute and surgical bleeding episodes.