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Wanru Yao, Juan Xiao, Xiaoling Cheng, Guoshuang Feng, Changgang Li, Xinsheng Zhang, Qun Hu, Weiqun Xu, Jing Sun, Renchi Yang, Xiaojing Li, Rongfu Zhou, Shimei Lian, Jian Gu, Junde Wu, Yongqiang Zhao, Runhui Wu
OBJECTIVE: This study explores the efficacy of recombinant factor VIII (rFVIII) low-dose prophylaxis in Chinese pediatric patients with severe hemophilia A from the Retrospective Study in Chinese Pediatric Hemophilia A Patients With rFVIII Contained Regular Prophylaxis (ReCARE) population. METHODS: This is additional analysis of the multicenter, retrospective ReCARE study, in which the annual bleeding rate (ABR), annual joint bleeding rate (AJBR), and safety of >12-week, low dose (10-30 IU/kg/wk) rFVIII prophylaxis divided into primary, secondary, and tertiary groups based on the joint status and joint bleeding history were analyzed...
December 5, 2016: Clinical and Applied Thrombosis/hemostasis
Héctor Eduardo Castro Jaramillo, Mabel Moreno Viscaya, Aurelio E Mejia
OBJECTIVES: This article presents a cost-utility analysis from the Colombian health system perspective comparing primary prophylaxis to on-demand treatment using exogenous clotting factor VIII (FVIII) for patients with severe hemophilia type A. METHODS: We developed a Markov model to estimate expected costs and outcomes (measured as quality-adjusted life-years, QALYs) for each strategy. Transition probabilities were estimated using published studies; utility weights were obtained from a sample of Colombian patients with hemophilia and costs were gathered using local data...
December 6, 2016: International Journal of Technology Assessment in Health Care
Maissaa Janbain, Steven Pipe
A 10-year-old boy presents with a history of severe hemophilia A and high-titer inhibitor that had failed high-dose immune tolerance induction (ITI) with a recombinant factor VIII (rFVIII) product and a plasma-derived FVIII product. You are asked by his mother whether he should be tried on ITI with an extended half-life product, in particular, consideration of a rFVIIIFc concentrate.
December 2, 2016: Hematology—the Education Program of the American Society of Hematology
Alfred Weber, Andrea Engelmaier, Gabriele Mohr, Sonja Haindl, Hans Peter Schwarz, Peter L Turecek
BAX 855 (ADYNOVATE) is a PEGylated recombinant factor VIII (rFVIII) that showed prolonged circulatory half-life compared to unmodified rFVIII in hemophilic patients. Here, the development and validation of a novel assay is described that selectively measures the activity of BAX 855 as cofactor for the serine protease factor IX, which actives factor X. This method type, termed modification-dependent activity assay, is based on PEG-specific capture of BAX 855 by an anti-PEG IgG preparation, followed by a chromogenic FVIII activity assay...
September 29, 2016: Journal of Pharmaceutical and Biomedical Analysis
N Suzuki, A Hirakawa, M Kishimoto, T Kanematsu, M Ogawa, H Kiyoi, T Matsushita
BACKGROUND: Continuous infusion (CI) of recombinant FVIII (rFVIII) concentrates has been reported as an effective and safe method to achieve haemostasis during major surgeries or severe bleeding events. For more effective and safer CI, better understanding of in vivo recovery (IVR) and clearance (CL) issues is imperative. OBJECTIVE: We investigated the following factors affecting IVR and CL using univariate and multivariate regression analyses during 47 CIs in 34 patients: rFVIII concentrate type, haemophilia severity, blood type, the presence of hepatitis C virus (HCV) or human immunodeficiency virus (HIV), age and body mass index (BMI)...
October 5, 2016: Haemophilia: the Official Journal of the World Federation of Hemophilia
Steven W Pipe, Robert R Montgomery, Kathleen P Pratt, Peter J Lenting, David Lillicrap
A normal hemostatic response to vascular injury requires both factor VIII (FVIII) and von Willebrand factor (VWF). In plasma, VWF and FVIII normally circulate as a non-covalent complex, and each has a critical function in the maintenance of hemostasis. Furthermore, the interaction between VWF and FVIII plays a crucial role in FVIII function, immunogenicity, and clearance, with VWF essentially serving as a chaperone for FVIII. Several novel recombinant FVIII (rFVIII) therapies for hemophilia A are in clinical development, which aim to increase the half-life of FVIII (~12 hours) and reduce dosing frequency by utilizing bioengineering techniques including PEGylation, Fc fusion, and single-chain design...
September 1, 2016: Blood
Massimo Morfini, Ezio Zanon
INTRODUCTION: Replacement therapy with clotting factor concentrates is the most appropriate and effective way to treat bleedings of Hemophilia A&B to prevent chronic arthropathy. Unfortunately, the short half-life (HL) of FVIII/IX concentrates obliges the patients to receive frequent infusions, a big concern for children. The development of inhibitors in about 30-45% of hemophilia A and in 3-5% of hemophilia B patient is the major adverse event of replacement therapy. AREAS COVERED: In the last few years, new rFIX have been developed with HL...
September 2016: Expert Opinion on Emerging Drugs
A H Miners, S Krishnan, K J Pasi
Essentials No randomized trials have compared long-acting factor VIII (FVIII) with currently used products. A comparison was undertaken using a decision model to predict FVIII use and number of bleeds. In the base case, longer acting FVIII reduced factor use by 17% while resulting in similar bleeds. The value of longer acting FVIII will be largely determined by existing regimens and unit price. Click to hear Prof. Makris's presentation on new treatments in hemophilia SUMMARY: Background Recently, factor VIII (FVIII) products with longer half-lives, such as recombinant FVIII Fc fusion protein (rFVIIIFc), have become available...
November 2016: Journal of Thrombosis and Haemostasis: JTH
T Burnouf, P F W Strengers
No abstract text is available yet for this article.
October 5, 2016: Journal of Thrombosis and Haemostasis: JTH
L Aledort, M Recht, R Kulkarni, N Kobrinsky
No abstract text is available yet for this article.
August 6, 2016: Journal of Thrombosis and Haemostasis: JTH
James E Frampton
Efmoroctocog alfa (Elocta(®), Eloctate(®), Eloctate™), a first-in-class recombinant factor VIII-Fc fusion protein (rFVIIIFc), has an extended half-life compared with conventional factor VIII (FVIII) preparations, including recombinant FVIII (rFVIII) products. It is approved for the treatment and prophylaxis of bleeding in patients with haemophilia A in multiple countries worldwide. Data accumulated from pivotal phase III studies (A-LONG in adults and adolescents aged ≥12 years; Kids A-LONG in children aged <12 years) and their ongoing extension study (ASPIRE) have demonstrated the long-term effectiveness of efmoroctocog alfa for the treatment of acute bleeding episodes, perioperative management and routine prophylaxis in previously treated males with severe haemophilia A...
September 2016: Drugs
Jennifer Pocoski, Nanxin Li, Rajeev Ayyagari, Nikki Church, Monika Maas Enriquez, Quer Xiang, Sneha Kelkar, Ella X Du, Eric Q Wu, Jipan Xie
BACKGROUND: No head-to-head trials comparing recombinant factor VIII (rFVIII) products currently exist. This was a matching-adjusted indirect comparison (MAIC) study of efficacy of BAY 81-8973 with antihemophilic factor (recombinant) plasma/albumin-free method (rAHF-PFM) and turoctocog alfa for the prophylaxis of severe hemophilia A. METHODS: A systematic literature review was conducted to identify trials of rAHF-PFM and turoctocog alfa. Comparisons were conducted using BAY 81-8973 individual patient data (IPD) from LEOPOLD trials and published data from rAHF-PFM and turoctocog alfa trials...
2016: Journal of Blood Medicine
Monika Maas Enriquez, John Thrift, Stephen Garger, Yvonne Katterle
BAY 81-8973 is a full-length, unmodified recombinant human factor VIII (FVIII) approved for the treatment of hemophilia A. BAY 81-8973 has the same amino acid sequence as the currently marketed sucrose-formulated recombinant FVIII (rFVIII-FS) product and is produced using additional advanced manufacturing technologies. One of the key manufacturing advances for BAY 81-8973 is introduction of the gene for human heat shock protein 70 (HSP70) into the rFVIII-FS cell line. HSP70 facilitates proper folding of proteins, enhances cell survival by inhibiting apoptosis, and potentially impacts rFVIII glycosylation...
November 2016: Protein Expression and Purification
R Klamroth, M Simpson, M von Depka-Prondzinski, J C Gill, M Morfini, J S Powell, E Santagostino, J Davis, A Huth-Kühne, C Leissinger, P Neumeister, D Bensen-Kennedy, A Feussner, T Limsakun, M Zhou, A Veldman, K St Ledger, N Blackman, I Pabinger
BACKGROUND: rVIII-SingleChain, a novel recombinant factor VIII (rFVIII), has been designed as a B-domain truncated construct with covalently bonded heavy and light chains, aiming to increase binding affinity to von Willebrand factor (VWF). Preclinical studies confirmed greater affinity for VWF, giving improved pharmacokinetic and pharmacodynamic properties compared with full-length rFVIII. AIM: To investigate the pharmacokinetics of rVIII-SingleChain and compare them against those of full-length rFVIII...
September 2016: Haemophilia: the Official Journal of the World Federation of Hemophilia
C I Øie, K Roepstorff, C Behrens, J Bøggild Kristensen, D M Karpf, G Bolt, C N Gudme, M Kjalke, B Smedsrød, R S Appa
UNLABELLED: Essentials Von Willebrand factor (VWF) stabilizes factor VIII (FVIII) and prevents its premature clearance. Rat anatomical and hepatocellular distribution studies assessed the VWF effect on FVIII clearance. Hepatocytes and liver sinusoidal endothelial cells play a key role in FVIII clearance. Anatomical and hepatocellular distribution of FVIII is independent of high-affinity VWF binding. ABSTRACT: Background Von Willebrand factor (VWF) stabilizes factor VIII in the circulation and prevents its premature clearance...
September 2016: Journal of Thrombosis and Haemostasis: JTH
P L Turecek, S Romeder-Finger, C Apostol, A Bauer, A Crocker-Buqué, D A Burger, R Schall, H Gritsch
INTRODUCTION: Discrepancies have been previously reported for one-stage clotting and chromogenic assays for FVIII activity analysis. Inter-laboratory variations in instruments, method of clot detection, assay set-up, reference standard calibration, reagent source and reagent composition all contribute to assay variability. AIM: To characterise multilaboratory assay variability in measuring ADYNOVATE, OBIZUR and ADVATE FVIII activity in human plasma and survey multinational FVIII activity assay preferences...
November 2016: Haemophilia: the Official Journal of the World Federation of Hemophilia
K Vepsäläinen, R Lassila, M Arola, P Huttunen, S Koskinen, R Ljung, P Lähteenmäki, M Möttönen, P Riikonen
INTRODUCTION: Currently the most serious treatment complication of haemophilia is the inhibitor development (ID), i.e. neutralizing antibody development. AIM: This nationwide multicentre study in Finland evaluated the incidence and risk factors of ID in previously untreated patients (PUPs) with severe haemophilia A (FVIII:C < 0.01 IU mL(-1) ). METHODS: We enrolled all PUPs (N = 62) born between June 1994 and May 2013 with at least 75 exposure days (EDs) to screen ID during follow-up extending to September 2013...
September 2016: Haemophilia: the Official Journal of the World Federation of Hemophilia
Johnny Mahlangu, Kazimierz Kuliczkowski, Faraizah Abdul Karim, Oleksandra Stasyshyn, Marina V Kosinova, Lynda Mae Lepatan, Aleksander Skotnicki, Lisa N Boggio, Robert Klamroth, Johannes Oldenburg, Andrzej Hellmann, Elena Santagostino, Ross I Baker, Kathelijn Fischer, Joan C Gill, Stephanie P'Ng, Pratima Chowdary, Miguel A Escobar, Claudia Djambas Khayat, Luminita Rusen, Debra Bensen-Kennedy, Nicole Blackman, Tharin Limsakun, Alex Veldman, Katie St Ledger, Ingrid Pabinger
Recombinant VIII (rVIII)-SingleChain is a novel B-domain-truncated recombinant factor VIII (rFVIII), comprised of covalently bonded factor VIII (FVIII) heavy and light chains. It was designed to have a higher binding affinity for von Willebrand factor (VWF). This phase 1/3 study investigated the efficacy and safety of rVIII-SingleChain in the treatment of bleeding episodes, routine prophylaxis, and surgical prophylaxis. Participants were ≥12 years of age, with severe hemophilia A (endogenous FVIII <1%)...
August 4, 2016: Blood
B Brand, R Gruppo, T T Wynn, L Griskevicius, M F Lopez Fernandez, M Chapman, T Dvorak, B G Pavlova, B E Abbuehl
INTRODUCTION: BAX 855 is a pegylated full-length recombinant factor VIII (rFVIII) with an extended half-life, built on a licensed rFVIII (ADVATE(®) ). BAX 855 demonstrated efficacy and safety in prophylaxis and the treatment of bleeding episodes in previously treated patients (PTPs) with severe haemophilia A. AIM: This phase 3 surgery study evaluates the haemostatic efficacy and safety of BAX 855 for perioperative haemostasis in PTPs with severe haemophilia A undergoing surgery...
July 2016: Haemophilia: the Official Journal of the World Federation of Hemophilia
Angelika Batorova, Denisa Jankovicova, Anna Morongova, Eva Bubanska, Tatiana Prigancova, Julia Horakova, Marianna Machyniakova, Jan Cervenka, Jan Chandoga, Daniel Böhmer, Martin Mistrik
UNLABELLED: We present 25-year experience with inhibitors in previously untreated patients (PUPs) with severe hemophilia A in Slovakia, where safe factor VIII (FVIII) concentrates have been used since 1990. A prospective study focused on inhibitor incidence in PUPs was established in 1997. Out of a total 61 PUPs born between January 1997 and October 2015, 59 were eligible for evaluation; 50 and 9 were treated with > 20 exposure days (ED) of plasma-derived FVIII (pdFVIII) and recombinant FVIII (rFVIII) products, respectively...
July 2016: Seminars in Thrombosis and Hemostasis
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