The changing treatment landscape in haemophilia: from standard half-life clotting factor concentrates to gene editing
ME Mancuso, JN Mahlangu, SW Pipe - The Lancet, 2021 - thelancet.com
Congenital haemophilia A (factor VIII deficiency) and B (factor IX deficiency) are X-linked
bleeding disorders. Replacement therapy has been the cornerstone of the management of …
bleeding disorders. Replacement therapy has been the cornerstone of the management of …
Advances and innovations in haemophilia treatment
R Peters, T Harris - Nature Reviews Drug Discovery, 2018 - nature.com
Haemophilia is a rare disease for which the approved therapeutic options have remained
virtually unchanged for 50 years. In the past decade, however, there has been an explosion …
virtually unchanged for 50 years. In the past decade, however, there has been an explosion …
Efficacy, safety, and pharmacokinetics of emicizumab prophylaxis given every 4 weeks in people with haemophilia A (HAVEN 4): a multicentre, open-label, non …
Background Emicizumab, a subcutaneously administered, humanised, bispecific,
monoclonal antibody, is approved to treat people with haemophilia A of all ages with and …
monoclonal antibody, is approved to treat people with haemophilia A of all ages with and …
Current and emerging factor VIII replacement products for hemophilia A
LA Cafuir, CL Kempton - Therapeutic advances in …, 2017 - journals.sagepub.com
Hemophilia A is a congenital X-linked bleeding disorder caused by coagulation factor VIII
(FVIII) deficiency. Routine infusion of factor replacement products is the current standard of …
(FVIII) deficiency. Routine infusion of factor replacement products is the current standard of …
[HTML][HTML] Status of recombinant factor VIII concentrate treatment for hemophilia a in Italy: characteristics and clinical benefits
M Schiavoni, M Napolitano, G Giuffrida… - Frontiers in …, 2019 - frontiersin.org
The current interest in recombinant factor VIII (rFVIII) products stems from the fact that they
offer a technological solution to prolonging the half-life of and reducing the risk of formation …
offer a technological solution to prolonging the half-life of and reducing the risk of formation …
[HTML][HTML] Efficient and safe correction of hemophilia A by lentiviral vector-transduced BOECs in an implantable device
C Olgasi, C Borsotti, S Merlin, T Bergmann… - … Therapy Methods & …, 2021 - cell.com
Hemophilia A (HA) is a rare bleeding disorder caused by deficiency/dysfunction of the FVIII
protein. As current therapies based on frequent FVIII infusions are not a definitive cure, long …
protein. As current therapies based on frequent FVIII infusions are not a definitive cure, long …
Bleeding and safety outcomes in persons with haemophilia A without inhibitors: results from a prospective non‐interventional study in a real‐world setting
R Kruse‐Jarres, J Oldenburg, E Santagostino… - …, 2019 - Wiley Online Library
Introduction Prospectively collected real‐world data on bleeds, haemophilia treatment and
safety in persons with haemophilia A (PwHA) without factor VIII (FVIII) inhibitors are limited …
safety in persons with haemophilia A (PwHA) without factor VIII (FVIII) inhibitors are limited …
Population pharmacokinetic characterization of BAY 81‐8973, a full‐length recombinant factor VIII: lessons learned‒importance of including samples with factor VIII …
D Garmann, S McLeay, A Shah, P Vis… - …, 2017 - Wiley Online Library
Introduction The pharmacokinetics (PK), safety and efficacy of BAY 81‐8973, a full‐length,
unmodified, recombinant human factor VIII (FVIII), were evaluated in the LEOPOLD trials …
unmodified, recombinant human factor VIII (FVIII), were evaluated in the LEOPOLD trials …
[HTML][HTML] Factor VIII products and inhibitor development in previously treated patients with severe or moderately severe hemophilia A: a systematic review
Essentials• Data on product‐related immunogenicity in previously treated haemophilia A
patients is scarce.• A systematic review and meta‐analysis of all currently available …
patients is scarce.• A systematic review and meta‐analysis of all currently available …
[HTML][HTML] Improved pharmacokinetics with BAY 81-8973 versus antihemophilic factor (recombinant) plasma/albumin-free method: a randomized pharmacokinetic study …
A Shah, A Solms, D Garmann, Y Katterle… - Clinical …, 2017 - Springer
Background BAY 81-8973 is a full-length, unmodified, recombinant human factor VIII (FVIII)
for the treatment of hemophilia A. Objective The aim of this study was to compare the …
for the treatment of hemophilia A. Objective The aim of this study was to compare the …