Major surgery in severe haemophilia A with inhibitors using a recombinant factor VIIa and activated prothrombin complex concentrate hybrid regimen

Haemophilia ◽  
2014 ◽  
Vol 20 (4) ◽  
pp. 587-592 ◽  
Author(s):  
J. J. van Veen ◽  
R. M. Maclean ◽  
K. K. Hampton ◽  
A. Hamer ◽  
M. Makris
Keyword(s):  
Prothrombin Complex Concentrate ◽  
Recombinant Factor Viia ◽  
Factor Viia ◽  
Major Surgery ◽  
Haemophilia A ◽  
Severe Haemophilia ◽  
Activated Prothrombin Complex Concentrate

scholarly journals Determining the value contribution of emicizumab (Hemlibra®) for the prophylaxis of haemophilia A with inhibitors in Spain by multi-criteria decision analysis

2019 ◽  
Vol 2019 ◽  
pp. 228424031988053
Author(s):  
Maria Teresa Álvarez-Román ◽  
Ignacio Cuervo-Arango ◽  
Rafael Pérez-Santamarina ◽  
José Luís Poveda ◽  
José Antonio Romero ◽  
...  
Keyword(s):  
Decision Analysis ◽  
Prothrombin Complex Concentrate ◽  
Recombinant Factor Viia ◽  
Factor Viia ◽  
Therapeutic Benefit ◽  
Tolerability Profile ◽  
Haemophilia A ◽  
Multi Criteria Decision Analysis ◽  
Value Contribution ◽  
Activated Prothrombin Complex Concentrate

Patients with moderate to severe haemophilia A are at a higher risk of developing FVIII inhibitors that require the use of more costly and less effective treatments. The objective of this study was to determine the value of emicizumab for the prophylaxis of haemophilia A with inhibitors compared to the current therapeutic alternatives, activated prothrombin complex concentrate and recombinant factor VIIa through reflective Multi-Criteria Decision Analysis. The EVIDEM framework adapted to orphan drugs and weighted by a sample of 98 national and regional Spanish evaluators was used. Two structured evidence matrices were developed: emicizumab against activated prothrombin complex concentrate and emicizumab against recombinant factor VIIa. A multidisciplinary team of haemophilia experts rated each of the criteria. Mean and standard deviation were calculated by each criterion and discussed among all participants. Haemophilia A with inhibitors was perceived as a severe disease with high unmet needs. Emicizumab was rated with higher efficacy, therapeutic benefit and quality of life than comparators. When administered alone for the prevention of bleeding events, emicizumab had slightly better safety and tolerability profile than activated prothrombin complex concentrate and similar with recombinant factor VIIa. The inclusion of emicizumab in clinical practice guidelines was valued positively by the members of the panel. Overall, value of emicizumab was higher than activated prothrombin complex concentrate and recombinant factor VIIa, mostly because of efficacy and therapeutic benefit in reducing treated haemorrhages. Reflective Multi-Criteria Decision Analysis has proven to be a feasible method to determine the value contribution of comparative therapies in haemophilia.

Sign in / Sign up

Export Citation Format

Share Document