2017 Clinical trials update: innovations in hemophilia therapy
J Hartmann, SE Croteau - American journal of hematology, 2016 - Wiley Online Library
A surge in therapeutic clinical trials over recent years is paving the way for transformative
treatment options for patients with hemophilia. The introduction of recombinant factor …
treatment options for patients with hemophilia. The introduction of recombinant factor …
The role of emicizumab, a bispecific factor IXa-and factor X-directed antibody, for the prevention of bleeding episodes in patients with hemophilia A
T Knight, MU Callaghan - Therapeutic advances in …, 2018 - journals.sagepub.com
Hemophilia A, characterized by impaired or absent expression of factor VIII, has long been
managed via direct factor replacement. Functionally, factor VIII acts as a cofactor for factor …
managed via direct factor replacement. Functionally, factor VIII acts as a cofactor for factor …
[HTML][HTML] Safety and efficacy of BAY 94‐9027, a prolonged‐half‐life factor VIII
MT Reding, HJ Ng, LH Poulsen, ME Eyster… - Journal of Thrombosis …, 2017 - Elsevier
Essentials• Recombinant factor VIII BAY 94‐9027 conjugates in a site‐specific manner with
polyethylene glycol.• BAY 94‐9027 was given to patients with severe hemophilia A as …
polyethylene glycol.• BAY 94‐9027 was given to patients with severe hemophilia A as …
[HTML][HTML] Prophylaxis vs. on‐demand treatment with BAY 81‐8973, a full‐length plasma protein‐free recombinant factor VIII product: results from a randomized trial …
K Kavakli, R Yang, L Rusen, H Beckmann… - Journal of Thrombosis …, 2015 - Elsevier
Background BAY 81‐8973 is a new full‐length human recombinant factor VIII product
manufactured with technologies to improve consistency in glycosylation and expression to …
manufactured with technologies to improve consistency in glycosylation and expression to …
Pharmacokinetics in routine haemophilia clinical practice: rationale and modalities—a practical review
C Hermans, G Dolan - Therapeutic advances in hematology, 2020 - journals.sagepub.com
Prophylactic therapy with exogenous clotting factor concentrates in haemophilia A and B
aims to achieve levels of circulating FVIII or FIX that are adequate for the prevention or …
aims to achieve levels of circulating FVIII or FIX that are adequate for the prevention or …
Inherited abnormalities of coagulation: hemophilia, von Willebrand disease, and beyond
Bleeding disorders are broadly classified into primary and secondary hemostatic defects
(Fig. 1). Primary hemostatic disorders (disorders of platelets and von Willebrand factor …
(Fig. 1). Primary hemostatic disorders (disorders of platelets and von Willebrand factor …
Practical considerations in choosing a factor VIII prophylaxis regimen: role of clinical phenotype and trough levels
R Ljung, K Fischer, M Carcao… - Thrombosis and …, 2016 - thieme-connect.com
Current therapy for haemophilia A is guided by severity of the disease, which in turn is best
reflected in patients' endogenous factor VIII activity levels. For patients with severe …
reflected in patients' endogenous factor VIII activity levels. For patients with severe …
[HTML][HTML] Pharmacokinetics of recombinant factor XIII at steady state in patients with congenital factor XIII A‐subunit deficiency
Background The use of monthly recombinant factor XIII (rFXIII) recently demonstrated
favorable safety and efficacy for congenital FXIII A‐subunit deficiency patients aged≥ 6 …
favorable safety and efficacy for congenital FXIII A‐subunit deficiency patients aged≥ 6 …
Pharmacokinetic studies of factor VIII in Chinese boys with severe hemophilia A: a single-center study
ZP Chen, PJ Li, G Li, L Tang, YZ Zhen, XY Wu… - Chinese Medical …, 2018 - mednexus.org
Background: Although much attention has been paid to the pharmacokinetics (PKs) of
different factor VIII (FVIII) concentrates in persons with hemophilia A (HA), limited information …
different factor VIII (FVIII) concentrates in persons with hemophilia A (HA), limited information …
Favorable pharmacokinetic characteristics of extended-half-life recombinant factor VIII BAY 94-9027 enable robust individual profiling using a population …
A Solms, A Iorio, MJ Ahsman, P Vis, A Shah… - Clinical …, 2020 - Springer
Background Prophylaxis with factor VIII (FVIII) should be individualized based on patient
characteristics, including FVIII pharmacokinetics. Population pharmacokinetic (popPK) …
characteristics, including FVIII pharmacokinetics. Population pharmacokinetic (popPK) …