FEIBA in Real Clinical Practice – Fresh Data from the Observational Study FEIBA GO
FEIBA GO (Global Outcome) was a prospective observational study evaluating the long-term administration of FEIBA, its safety, efficacy, and quality of life for patients with hemophilia A or B and high-titer inhibitors (high responders), who were receiving aPCC in routine clinical practice. The study, whose results were recently published, was conducted at 25 centers in 11 countries.
Introduction
For patients with hemophilia A and an inhibitor to factor VIII (FVIII), it is necessary to administer drugs with bypass activity to manage bleeding episodes or surgical procedures, as their effects are not influenced by the existing inhibitor. These drugs are also used as alternatives for bleeding prevention as part of prophylaxis. One well-established bypassing agent is the activated prothrombin complex concentrate (aPCC) known as FEIBA.
Evaluated Patient Population
The planned follow-up period for the FEIBA GO study was 4 years. All patients who received at least one dose of FEIBA were included in the safety evaluation analysis.
A total of 50 patients were included, 37 of whom received aPCC for prophylaxis and 13 for on-demand treatment. The majority (n = 49) were patients with hemophilia A with inhibitors, with a median age of 16.5 years (range 2–71). The median duration of treatment was 14.1 (0.4–188.3) months.
Findings
The average annual bleed rate (ABR) for patients receiving prophylaxis was 6.82 ± 11.52 (SD – standard deviation), and the joint ABR was 3.77 ± 5.71. For patients receiving FEIBA only on demand, the overall ABR was 10.94 ± 11.27 and the joint ABR was 6.94 ± 7.39. Regarding bleeding treatment, efficacy was deemed good in 77.8% of cases and excellent in 18.2%.
The total annual aPCC dose was 13,345.56 IU/kg for prophylaxis and 2100.69 IU/kg for on-demand treatment. The median dose per infusion was 65.76 IU/kg (range 37–100 IU/kg), with a median of 4.12 weekly infusions (0.5–14).
A total of 208 adverse events were recorded. Two serious adverse events were considered possibly related to aPCC – one case involved acute myocardial infarction associated with coronary artery embolism in a patient treated with aPCC for gastrointestinal bleeding. No cases of thrombotic microangiopathy were reported. No adverse event caused death.
Conclusion
The FEIBA GO study demonstrated the long-term efficacy and consistent safety profile of aPCC for on-demand bleeding treatment and prophylactic administration in a complex group of hemophilia patients with high-titer inhibitors.
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Source: Ettingshausen C. E., Hermans C., Holme P. A. et al. Real-world data in patients with congenital hemophilia and inhibitors: final data from the FEIBA Global Outcome (FEIBA GO) study. Ther Adv Hematol 2023 Jul 24: 14: 20406207231184323, doi: 10.1177/20406207231184323.
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