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Real-World Use of Albutrepenonacog Alfa, A Recombinant Coagulation Factor IX Albumin Fusion Protein, for Personalized Prophylaxis in Japanese Individuals With Hemophilia B: A Case Series
Currently, the mainstay of disease management for hemophilia B, a hemorrhagic disease caused by a congenital deficiency or molecular abnormalities of blood coagulation factor IX (FIX), is prophylaxis using FIX concentrate. On-demand injections of FIX concentrate may also be required, even during pro...
Autores principales: | , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Cureus
2023
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9909124/ https://www.ncbi.nlm.nih.gov/pubmed/36779089 http://dx.doi.org/10.7759/cureus.33573 |
Sumario: | Currently, the mainstay of disease management for hemophilia B, a hemorrhagic disease caused by a congenital deficiency or molecular abnormalities of blood coagulation factor IX (FIX), is prophylaxis using FIX concentrate. On-demand injections of FIX concentrate may also be required, even during prophylaxis, when a patient with hemophilia B is bleeding. Albutrepenonacog alfa (rFIX-FP) is a human albumin fusion gene recombinant FIX, which is administered once every seven, 14, or 21 days, depending on patient preferences and symptoms. Studies have demonstrated its efficacy and safety in a range of patients; however, to date, reports of real-world experiences of the use of rFIX-FP in Japan are limited. We present a case series of three Japanese individuals with moderately severe (FIX activity 1 to <2%) or severe (FIX activity <1%) hemophilia B who benefited from prophylaxis using rFIX-FP in our clinical practice setting. We highlighted the good effectiveness of rFIX-FP in a patient with moderately severe hemophilia B who required prophylaxis due to joint bleeding, which was causing deterioration of his left ankle joint, as well as in a patient with severe hemophilia B and atherothrombotic cerebral infarction, whose trough level had to be ≥5% for concomitant use of an antiplatelet drug, and in a patient with severe hemophilia B who was working in nursing care, which involved heavy labor and night shifts, and who had previously been treated with on-demand FIX concentrate. In all three cases, rFIX-FP improved disease symptoms, and the patients were able to maintain steady states of therapy due to the treatment characteristics of rFIX-FP, which stabilizes FIX at high trough levels. |
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