作者: J. Oldenburg , T. Albert
DOI: 10.1111/HAE.12428
关键词: Haemophilia 、 Bispecific antibody 、 Bioinformatics 、 Medicine 、 Haemophilia A 、 Subcutaneous route 、 Clotting factor 、 Recombinant factor viii 、 Von Willebrand factor 、 Factor IX 、 Pharmacology
摘要: Summary Currently, new clotting factor concentrates are becoming available or in advanced clinical studies that will significantly improve the treatment of patients with Haemophilia A B. Various technologies applied to extend half-life and/or allow for alternative routes administration, e.g. subcutaneous route. Today, advances recombinant IX extensions up 100 h, allowing substitution intervals 1–2 weeks. For VIII (FVIII) products effect so far is only moderate, as extension limited about 15–18 h by clearance FVIII through its binding von Willebrand factor. However, novel applying extended already at horizont, a bispecific antibody mimics FVIII. The pharmacokinetic improvements lead revision our current regimens, regard intended trough levels, number tolerated bleeds and likely drive greater individualization regimens. Clearly, potential anti drug response these modified proteins must not be higher than products. Another challenge increasingly diverse biochemical characteristics products, have considered when determining potencies also monitoring various assays. Despite challenges, quality life haemophilia.