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 …

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 …

[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 …

[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 …

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 …

Inherited abnormalities of coagulation: hemophilia, von Willebrand disease, and beyond

R Kumar, M Carcao - Pediatric Clinics, 2013 - pediatric.theclinics.com
Bleeding disorders are broadly classified into primary and secondary hemostatic defects
(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 …

[HTML][HTML] Pharmacokinetics of recombinant factor XIII at steady state in patients with congenital factor XIII A‐subunit deficiency

B Kerlin, B Brand, A Inbal, S Halimeh, D Nugent… - Journal of Thrombosis …, 2014 - Elsevier
Background The use of monthly recombinant factor XIII (rFXIII) recently demonstrated
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 …

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) …