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https://www.readbyqxmd.com/read/29139314/cost-utility-analysis-of-life-long-prophylaxis-with-recombinant-factor-viii-fc-vs-recombinant-factor-viii-for-the-management-of-severe-haemophilia-a-in-sweden
#1
Nathaniel Henry, Jelena Jovanović, Max Schlueter, Persefoni Kritikou, Koo Wilson, Karl-Johan Myrén
AIMS: Prophylaxis with recombinant factor VIII (rFVIII) is the standard of care for severe haemophilia A in Sweden. The need for frequent injections with existing rFVIII products may, however, result in poor adherence to prophylaxis leading to increased bleeding and long-term joint damage. Recombinant FVIIIFc (rFVIIIFc) is an extended half-life fusion protein which can offer prolonged protection and reduced dosing frequency. The objective of this study was to evaluate the cost-utility of prophylaxis with rFVIIIFc in severe haemophilia A from the perspective of the Swedish health system...
November 15, 2017: Journal of Medical Economics
https://www.readbyqxmd.com/read/29080391/timing-and-severity-of-inhibitor-development-in-recombinant-versus-plasma-derived-factor-viii-concentrates-a-sippet-analysis
#2
F Peyvandi, A Cannavò, I Garagiola, R Palla, P M Mannucci, F R Rosendaal
BACKGROUND: The development of neutralizing antibodies (inhibitors) against factor VIII (FVIII) is the most severe complication in the early phases of treatment of severe haemophilia A. Recently a randomized trial, the Survey of Inhibitors in Plasma-Product Exposed Toddlers (SIPPET) demonstrated a two-fold higher risk of inhibitors development in children treated with recombinant FVIII (rFVIII) products than with plasma-derived FVIII (pdFVIII) during the first 50 exposure days (EDs). OBJECTIVE/METHODS: In this post-hoc SIPPET analysis we evaluated the rate of inhibitor incidence over time by every 5 EDs (from 0 to 50 EDs) in patients treated with different classes of FVIII product, made possible by a frequent testing regime...
October 28, 2017: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/29080389/sippet-insights-into-factor-viii-immunogenicity
#3
Padraic G Fallon, Michelle Lavin, James S O'Donnell
In this issue of the Journal of Thrombosis and Haemostasis, Peyvandi et al present further insights from the Survey of Inhibitors in Plasma-Product Exposed Toddlers (SIPPET) study regarding the relative immunogenicity of recombinant- (rFVIII) versus plasma-derived FVIII (pdFVIII) in patients with haemophilia A [1]. In a post-hoc analysis of this prospective randomised controlled trial, the timing and severity of inhibitor development for previously untreated patients (PUPs) treated with rFVIII was compared with that in PUPs treated with pdFVIII...
October 28, 2017: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/29050497/simoctocog-alfa-for-the-treatment-of-hemophilia-a
#4
Massimo Morfini
Hemophilia A is the most frequent inherited bleeding disorder and most challenging coagulation disorder. To combat this, a number of new improved rFVIII/IX concentrates have recently been approved. Some of them are derived from protein fusion biotechnology or pegylation to extend their half-life (HL). However, prophylaxis has become a standard of care to prevent arthropathy in hemophiliacs though the need of frequent venipunctures is a major obstacle to primary prophylaxis. The new Extended Half-Life (EHL) rFIX concentrates allow increased intervals, while the improved HL of new rFVIII was moderate...
October 20, 2017: Expert Opinion on Biological Therapy
https://www.readbyqxmd.com/read/28921438/purification-methods-for-recombinant-factor-viii-expressed-in-human-liver-sk-hep-cells
#5
Vladimir Granovski, Mario Soares Abreu-Neto, Dimas Tadeu Covas
Coagulation factor VIII is one of the largest proteins attempted to be expressed in recombinant form. A very complex and labile protein which has a very short half-live and need a fast and efficient purification chain. Here, we describe a simple purification sequence using multimodal Capto MMC, affinity FVIII select and ion exchange SP-Fastflow chromatography steps without subjecting the target molecule to mechanical and temperature stress, separating impurities from rFVIII using net charge, hydrophobicity, and affinity of the molecules...
2018: Methods in Molecular Biology
https://www.readbyqxmd.com/read/28921428/production-of-recombinant-factor-viii-in-human-cell-lines
#6
Daianne Maciely Carvalho Fantacini, Virgínia Picanço-Castro
Human cell lines can produce recombinant proteins much more similar to their natural counterpart, compared to other mammalian cell lines, reducing potential immunogenic reactions. Recombinant proteins produced in nonhuman cells can have in its structure glycan epitopes, such as Galα1,3-Gal (alpha-Gal) and N-glycolylneuraminic acid (Neu5Gc) residues, that are antigenic to humans and can potentially affect the efficacy of the recombinant product. Therefore, the production of recombinant factor VIII (rFVIII) in human cell lines is a new approach to avoid nonhuman glycosylation...
2018: Methods in Molecular Biology
https://www.readbyqxmd.com/read/28836341/effect-of-late-prophylaxis-in-hemophilia-on-joint-status-a-randomized-trial
#7
M J Manco-Johnson, B Lundin, S Funk, C Peterfy, D Raunig, M Werk, C L Kempton, M T Reding, S Goranov, L Gercheva, L Rusen, V Uscatescu, M Pierdominici, S Engelen, J Pocoski, D Walker, W Hong
Essentials High-quality data are lacking on use of prophylaxis in adults with hemophilia and arthropathy. SPINART was a 3-year randomized clinical trial of late/tertiary prophylaxis vs on-demand therapy. Prophylaxis improved function, quality of life, activity and pain but not joint structure by MRI. Prophylaxis improves function but must start before joint bleeding onset to prevent arthropathy. SUMMARY: Background Limited data exist on the impact of prophylaxis on adults with severe hemophilia A and pre-existing joint disease...
August 24, 2017: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/28768627/genetic-risk-stratification-to-reduce-inhibitor-development-in-the-early-treatment-of-hemophilia-a-a-sippet-analysis
#8
RANDOMIZED CONTROLLED TRIAL
Frits R Rosendaal, Roberta Palla, Isabella Garagiola, Pier M Mannucci, Flora Peyvandi
A recent randomized trial, the Survey of Inhibitors in Plasma-Product Exposed Toddlers (SIPPET), showed a higher risk of inhibitor development with recombinant factor VIII (rFVIII) than plasma-derived concentrates (pdFVIII). We investigated whether risk stratification by F8 mutation identifies patients who do not suffer this deleterious effect of rFVIII. Among 235 randomized patients with severe hemophilia A previously untreated with FVIII concentrate, 197 with null mutations were classified as high risk and 38 with non-null mutations were classified as low risk...
October 12, 2017: Blood
https://www.readbyqxmd.com/read/28727494/efficacy-of-standard-prophylaxis-versus-on-demand-treatment-with-bayer-s-sucrose-formulated-recombinant-fviii-rfviii-fs-in-chinese-children-with-severe-hemophilia-a
#9
Yongqiang Zhao, Juan Xiao, Renchi Yang, Runhui Wu, Yu Hu, Horst Beckmann, Junde Wu, Qingsong Hou, Jing Sun
In China, care of patients with severe hemophilia primarily involves insufficient dosing of on-demand treatment and secondary low-dose prophylaxis (10 IU/kg 2× /wk). We sought to evaluate 3× /wk, standard-dose prophylaxis with sucrose-formulated recombinant factor VIII (rFVIII-FS; Bayer) compared with on-demand treatment in Chinese children with severe hemophilia A. Children and adolescents aged 2-16 years with severe hemophilia A, no inhibitors, and no prophylaxis for >6 consecutive months before study entry were eligible for this 24-week, interventional, sequential-treatment study...
April 2017: Pediatric Hematology and Oncology
https://www.readbyqxmd.com/read/28692842/pk-driven-prophylaxis-versus-standard-prophylaxis-when-a-tailored-treatment-may-be-a-real-and-achievable-cost-saving-approach-in-children-with-severe-hemophilia-a
#10
Samantha Pasca, Marta Milan, Lucia Sarolo, Ezio Zanon
BACKGROUND: Prophylaxis is the gold standard for the treatment of children with severe hemophilia. In the last years a new approach to prophylaxis based on annual bleeding rate (ABR), pharmacokinetics (PK) and lifestyle of each patient has begun to be adopted in hemophilia treatment. AIM: Aim of our observational retrospective study was to evaluate whether in a group of children with severe hemophilia A (HA) a tailored approach may be used to replace standard therapy, reducing costs...
July 5, 2017: Thrombosis Research
https://www.readbyqxmd.com/read/28692108/safety-and-efficacy-of-a-glycopegylated-rfviii-turoctocog-alpha-pegol-n8-gp-in-paediatric-patients-with-severe-haemophilia-a
#11
Sandrine Meunier, Jayanthi Alamelu, Silke Ehrenforth, Hideji Hanabusa, Faraizah Abdul Karim, Kaan Kavakli, Melanie Khodaie, Janice Staber, Oleksandra Stasyshyn, Donald L Yee, Lina Rageliene
Turoctocog alfa pegol (N8-GP, Novo Nordisk, Bagsværd, Denmark), an extended half-life glycoPEGylated recombinant factor VIII (rFVIII), is being developed for prophylaxis and treatment of bleeds in haemophilia A patients. pathfinder™5 is a multinational, open-label, single-arm trial to assess safety, efficacy and pharmacokinetics of N8-GP in paediatric (<12 years), previously treated patients. Boys with severe haemophilia A (<1 % FVIII), no history of inhibitors and previously treated with FVIII products (>50 exposure days [ED] for patients aged 0-5 years [younger cohort]; >150 ED for patients aged 6-11 years [older cohort]) were included...
August 30, 2017: Thrombosis and Haemostasis
https://www.readbyqxmd.com/read/28678027/meta-analysis-on-incidence-of-inhibitors-in-patients-with-haemophilia-a-treated-with-recombinant-factor-viii-products
#12
Matteo Rota, Paolo A Cortesi, Katharina N Steinitz-Trost, Armin J Reininger, Alessandro Gringeri, Lorenzo G Mantovani
: Recent cohort studies showed differences in inhibitor incidence in previously untreated patients (PUPs) with haemophilia A treated with recombinant factor VIII (rFVIII) concentrates. We carried out a systematic literature search and meta-analysis for all randomized clinical trials and observational studies published from 1 January 1988 to 31 August 2015, to assess the incidence of inhibitor development and the relationship with rFVIII product used in PUPs and minimally treated patients (MTPs, ≤5 previous exposure days), with severe haemophilia...
July 3, 2017: Blood Coagulation & Fibrinolysis: An International Journal in Haemostasis and Thrombosis
https://www.readbyqxmd.com/read/28670147/many-factor-viii-products-available-in-the-treatment-of-hemophilia-a-an-embarrassment-of-riches
#13
REVIEW
Kenneth Lieuw
Hemophilia A (HA) is a common bleeding disorder caused by the deficiency of factor VIII (FVIII) with an incidence of ~1 in 5000 male births. Replacement of FVIII is necessary to prevent and treat bleeding episodes. However, with multiple new drugs in addition to old standards, choosing among the different FVIII treatment options is harder than ever. There are FVIII products that are plasma derived or recombinant, FVIII products designed to extend the half-life of FVIII, and the first single-chain FVIII product, recombinant factor VIII single chain (rFVIII-SC)...
2017: Journal of Blood Medicine
https://www.readbyqxmd.com/read/28544263/impact-of-a-product-specific-reference-standard-for-the-measurement-of-a-pegylated-rfviii-activity-the-swiss-multicentre-field-study
#14
O Bulla, A Poncet, L Alberio, L M Asmis, A Gähler, L Graf, M Nagler, J-D Studt, D A Tsakiris, P Fontana
INTRODUCTION: Measuring factor VIII (FVIII) activity can be challenging when it has been modified, such as when FVIII is pegylated to increase its circulating half-life. Use of a product-specific reference standard may help avoid this issue. AIM: Evaluate the impact of using a product-specific reference standard for measuring the FVIII activity of BAX 855 - a pegylated FVIII - in eight of Switzerland's main laboratories. METHODS: Factor VIII-deficient plasma, spiked with five different concentrations of BAX 855, plus a control FVIII sample, was sent to the participating laboratories...
May 24, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28532239/status-and-trend-analysis-of-prophylactic-usage-of-recombinant-factor-viii-in-chinese-pediatric-patients-with-hemophilia-a-recare-a-retrospective-phase-iv-non-interventional-study
#15
Changgang Li, Xinsheng Zhang, Yongqiang Zhao, Runhui Wu, Qun Hu, Vicky Xu, Jing Sun, Renchi Yang, Xiaojing Li, Rongfu Zhou, Shinmei Lian, Jian Gu, Junde Wu, Qingsong Hou
BACKGROUND: No study has reported the status and chronological trend of prophylactic recombinant factor VIII (rFVIII) use in Chinese pediatric patients with hemophilia A (HA). OBJECTIVE: We aimed to analyze the status and trend of rFVIII-containing prophylaxis in Chinese pediatric patients with HA. METHODS: ReCARE (Retrospective study in Chinese pediatric hemophilia A patients with rFVIII contained REgular prophylaxis) was a retrospective study conducted in 12 hemophilia treatment centers across China...
June 21, 2017: Current Medical Research and Opinion
https://www.readbyqxmd.com/read/28458688/long-term-safety-of-pegylated-coagulation-factor-viii-in-the-immune-deficient-rowett-nude-rat
#16
Caroline E Rasmussen, Jette Nowak, Julie M Larsen, Emma Moore, David Bell, Kai Chiu Liu, Nanna Skall Sorensen, Wendela A Kappers, Thomas Krogh-Meibom, Hanne Offenberg
Turoctocog alfa pegol (N8-GP) is a glycoPEGylated human recombinant factor VIII for the treatment of hemophilia A. The safety profile of rFVIII, and polyethylene glycols (PEG) technology, is well-established. Conducting long-term toxicity studies in animals using human proteins can be complicated by anti-drug antibody (ADA) development. To evaluate long-term safety of N8-GP, 26- and 52-week toxicity studies were conducted in immune-deficient rats dosed intravenously every fourth day with 0, 50, 150, 500, or 1200 IU/kg N8-GP...
2017: Journal of Toxicology
https://www.readbyqxmd.com/read/28326849/long-term-efficacy-and-safety-of-prophylaxis-with-recombinant-factor-viii-in-chinese-pediatric-patients-with-hemophilia-a-a-multi-center-retrospective-non-interventional-phase-iv-recare-study
#17
Changgang Li, Xinsheng Zhang, Yongqiang Zhao, Runhui Wu, Qun Hu, Weiqun Xu, Jing Sun, Renchi Yang, Xiaojing Li, Rongfu Zhou, Shinmei Lian, Jian Gu, Junde Wu, Qingsong Hou
BACKGROUND: The first recombinant factor VIII (rFVIII) product was launched in China in 2007. However, until now, no study has been conducted to describe the efficacy and safety of prophylaxis with rFVIII in Chinese pediatric patients with hemophilia A (HA). OBJECTIVE: To summarize the efficacy and safety data on prophylaxis with rFVIII in Chinese pediatric patients with HA. METHODS: ReCARE (Retrospective study in Chinese pediatric hemophilia A patients with rFVIII contained regular prophylaxis) was a retrospective study conducted in 12 hemophilia treatment centers (HTCs) across China...
April 25, 2017: Current Medical Research and Opinion
https://www.readbyqxmd.com/read/28306186/sippet-methodology-analysis-and-generalizability
#18
REVIEW
F Peyvandi, P M Mannucci, R Palla, F R Rosendaal
The development of anti-FVIII neutralizing alloantibodies (inhibitors), occurring in about one-third of previously untreated patients (PUPs) with severe haemophilia A, depends on various genetic and environmental risk factors. Several previous studies have reported on the immunogenicity of FVIII concentrates, and due to differences in study design, study period, inhibitor testing frequency and follow-up duration the results were inconclusive. The first randomized trial on this unresolved question (SIPPET) included 251 previously untreated or minimally treated patients with severe haemophilia A treated with either a single plasma-derived FVIII (pdFVIII) containing VWF or a recombinant FVIII (rFVIII)...
May 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28198071/measuring-fviii-activity-of-glycopegylated-recombinant-factor-viii-n8-gp-with-commercially-available-one-stage-clotting-and-chromogenic-assay-kits-a-two-centre-study
#19
A Hillarp, A Bowyer, M Ezban, P Persson, S Kitchen
INTRODUCTION: Factor VIII activity (FVIII:C) assays of samples containing glycoPEGylated recombinant FVIII such as turoctocog alfa pegol (N8-GP) can be associated with differences in FVIII recovery in vitro between various one-stage activated partial thromboplastin time (APTT)-based clotting assays and some chromogenic assays. Careful validation and qualification of specific assays and conditions is therefore necessary for the assessment of FVIII:C in samples containing modified FVIII molecules...
February 14, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28166608/safety-efficacy-and-pharmacokinetics-of-rviii-singlechain-in-children-with-severe-hemophilia-a-results-of-a-multicenter-clinical-trial
#20
O Stasyshyn, C Djambas Khayat, G Iosava, J Ong, F Abdul Karim, K Fischer, A Veldman, N Blackman, K St Ledger, I Pabinger
Essentials rVIII-SingleChain is a novel recombinant factor VIII with covalently bonded heavy and light chains. Efficacy, safety and pharmacokinetics were studied in pediatric patients with severe hemophilia A. Across all prophylaxis regimens, the median annualized spontaneous bleeding rate was 0.00. rVIII-SingleChain showed excellent hemostatic efficacy and a favorable safety profile. SUMMARY: Background rVIII-SingleChain is a novel B-domain truncated recombinant factor VIII (rFVIII) comprised of covalently bonded FVIII heavy and light chains, demonstrating a high binding affinity to von Willebrand factor...
April 2017: Journal of Thrombosis and Haemostasis: JTH
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