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Inhibitor haemophilia

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https://www.readbyqxmd.com/read/28205285/natural-history-and-clinical-characteristics-of-inhibitors-in-previously-treated-haemophilia-a-patients-a-case-series
#1
A Iorio, A M Barbara, M Makris, K Fischer, G Castaman, C Catarino, E Gilman, K Kavakli, T Lambert, R Lassila, T Lissitchkov, E Mauser-Bunschoten, M E Mingot-Castellano, N Ozdemir, I Pabinger, R Parra, J Pasi, K Peerlinck, A Rauch, V Roussel-Robert, M Serban, A Tagliaferri, J Windyga, E Zanon
BACKGROUND: Development of inhibitors is the most serious complication in haemophilia A treatment. The assessment of risk for inhibitor formation in new or modified factor concentrates is traditionally performed in previously treated patients (PTPs). However, evidence on risk factors for and natural history of inhibitors has been generated mostly in previously untreated patients (PUPs). The purpose of this study was to examine cases of de novo inhibitors in PTPs reported in the scientific literature and to the EUropean HAemophilia Safety Surveillance (EUHASS) programme, and explore determinants and course of inhibitor development...
February 15, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28198996/treatment-burden-haemostatic-strategies-and-real-world-inhibitor-screening-practice-in-non-severe-haemophilia-a
#2
Paul Batty, Steve K Austin, Kate Khair, Carolyn M Millar, Ben Palmer, Savita Rangarajan, Jan-Phillip Stümpel, Murugaiyan Thanigaikumar, Thynn T Yee, Daniel P Hart
Inhibitor formation in non-severe haemophilia A is a life-long risk and associated with morbidity and mortality. There is a paucity of data to understand real-world inhibitor screening practice. We evaluated the treatment burden, haemostatic strategies, F8 genotyping and inhibitor screening practices in non-severe haemophilia A in seven London haemophilia centres. In the 2-year study period, 44% (377/853) patients received at least one haemostatic treatment. Seventy-nine percent of those treated (296/377) received factor VIII (FVIII) concentrate...
February 15, 2017: British Journal of Haematology
https://www.readbyqxmd.com/read/28182257/inhibitor-screening-in-non-severe-haemophilia-patients-a-major-challenge
#3
EDITORIAL
Anne-Mette Hvas, Lone H Poulsen
No abstract text is available yet for this article.
February 9, 2017: British Journal of Haematology
https://www.readbyqxmd.com/read/28159192/recombinant-factor-ix-fc-fusion-protein-in-children-with-haemophilia-b-kids-b-long-results-from-a-multicentre-non-randomised-phase-3-study
#4
Kathelijn Fischer, Roshni Kulkarni, Beatrice Nolan, Johnny Mahlangu, Savita Rangarajan, Giulia Gambino, Lei Diao, Alejandra Ramirez-Santiago, Glenn F Pierce, Geoffrey Allen
BACKGROUND: Kids B-LONG was a multicentre, open-label, phase 3 study assessing the safety, efficacy, and pharmacokinetics of recombinant factor IX Fc fusion protein (rFIXFc) in previously treated paediatric patients younger than 12 years with severe haemophilia B. METHODS: The study enrolled 30 previously treated boys younger than 12 years with haemophilia B (≤2 IU/dL [≤2%] endogenous coagulation factor IX [FIX] activity). All patients were initially given rFIXFc prophylaxis (50-60 IU/kg) once per week with adjustments to dose (≤100 IU/kg per infusion) or dosing frequency (up to two times per week) as needed...
February 2017: Lancet Haematology
https://www.readbyqxmd.com/read/28124406/recombinant-porcine-factor-viii-for-high-risk-surgery-in-paediatric-congenital-haemophilia-a-with-high-titre-inhibitor
#5
S E Croteau, Y L Abajas, A S Wolberg, B I Nielsen, G R Marx, C W Baird, E J Neufeld, P E Monahan
INTRODUCTION: High-titre factor VIII (FVIII) inhibitors complicate peri-operative haemostasis. Recombinant porcine FVIII (r-pFVIII) may provide an alternative haemostatic agent for high-risk procedures and allow FVIII activity monitoring. AIM: Devise an effective haemostatic plan for repair of a progressively symptomatic aortic coarctation in a 5-year-old male with immune tolerance induction (ITI) refractory high-titre FVIII inhibitors. METHODS: Preprocedure human FVIII inhibitor titre was 58 Bethesda Units mL(-1) (BU) and cross-reacted to neutralize porcine FVIII at 30 BU...
January 25, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28111886/low-dose-factor-viii-infusion-in-chinese-adult-haemophilia-a-patients-pharmacokinetics-evidence-that-daily-infusion-results-in-higher-trough-level-than-with-every-other-day-infusion-with-similar-factor-viii-consumption
#6
B Hua, A Lee, L Fan, K Li, Y Zhang, M-C Poon, Y Zhao
INTRODUCTION: Pharmacokinetics (PK) modelling suggests improvement of trough levels are achieved by using more frequent infusion strategy. However, no clinical study data exists to confirm or quantify improvement in trough level, particularly for low-dose prophylaxis in patients with haemophilia A. AIM: To provide evidence that low dose daily (ED) prophylaxis can increase trough levels without increasing FVIII consumption compared to every-other-day (EOD) infusion...
January 22, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28088606/choice-of-factor-viii-ix-regimen-in-adolescents-and-young-adults-with-severe-or-moderately-severe-haemophilia-a-french-national-observational-study-orthem-15-25
#7
Sandrine Meunier, Roseline d'oiron, Hervé Chambost, Edita Dolimier, Benoît Guillet
INTRODUCTION: The value and challenges of long-term prophylaxis (LTP) in adolescents and young adults need further characterisation. AIM: To determine the proportions of adolescents and young adults with severe or moderately severe haemophilia in France under LTP and treatment on demand (OD). METHODS: Patients 15 to 25years old with haemophilia A or B, factor VIII/IX ≤2% and no current inhibitor could be included if they had been under factor VIII/IX treatment at least 12months and kept a treatment and bleeding diary...
December 28, 2016: Thrombosis Research
https://www.readbyqxmd.com/read/28026073/use-of-the-ukhcdo-database-for-a-postmarketing-surveillance-study-of-different-doses-of-recombinant-factor-viia-in-haemophilia
#8
C R M Hay, T Sharpe, G Dolan
INTRODUCTION: Recombinant factor VIIa (rFVIIa) is recommended in Europe at standard (3 × 90 μg kg(-1) ) or high (1 × 270 μg kg(-1) ) doses. When granting the license for the high dose, the European Medicines Agency (EMA) requested postmarketing surveillance for thrombosis. This was conducted by the United Kingdom National Haemophilia Database (NHD) on behalf of Novo Nordisk and the EMA. AIM: To assess the use and safety of rFVIIa utilizing prospective data collected by the NHD (1 January 2008 to 30 June 2011)...
December 27, 2016: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28004057/long-term-safety-and-efficacy-of-extended-interval-prophylaxis-with-recombinant-factor-ix-fc-fusion-protein-rfixfc-in-subjects-with-haemophilia-b
#9
K John Pasi, Kathelijn Fischer, Margaret Ragni, Beatrice Nolan, David J Perry, Roshni Kulkarni, Margareth Ozelo, Johnny Mahlangu, Amy D Shapiro, Ross I Baker, Carolyn M Bennett, Christopher Barnes, Johannes Oldenburg, Tadashi Matsushita, Huixing Yuan, Alejandra Ramirez-Santiago, Glenn F Pierce, Geoffrey Allen, Baisong Mei
The safety, efficacy, and prolonged half-life of recombinant factor IX Fc fusion protein (rFIXFc) were demonstrated in the Phase 3 B-LONG (adults/adolescents ≥12 years) and Kids B-LONG (children <12 years) studies of subjects with haemophilia B (≤2 IU/dl). Here, we report interim, long-term safety and efficacy data from B-YOND, the rFIXFc extension study. Eligible subjects who completed B-LONG or Kids B-LONG could enrol in B-YOND. There were four treatment groups: weekly prophylaxis (20-100 IU/kg every 7 days), individualised prophylaxis (100 IU/kg every 8-16 days), modified prophylaxis (further dosing personalisation to optimise prophylaxis), and episodic (on-demand) treatment...
December 22, 2016: Thrombosis and Haemostasis
https://www.readbyqxmd.com/read/27996087/gene-therapy-for-haemophilia
#10
REVIEW
Akshay Sharma, Manu Easow Mathew, Vasumathi Sriganesh, Ulrike M Reiss
BACKGROUND: Haemophilia is a genetic disorder characterized by spontaneous or provoked, often uncontrolled, bleeding into joints, muscles and other soft tissues. Current methods of treatment are expensive, challenging and involve regular administration of clotting factors. Gene therapy has recently been prompted as a curative treatment modality. This is an update of a published Cochrane Review. OBJECTIVES: To evaluate the safety and efficacy of gene therapy for treating people with haemophilia A or B...
20, 2016: Cochrane Database of Systematic Reviews
https://www.readbyqxmd.com/read/27990785/inhibitor-development-in-haemophilia
#11
F Peyvandi, M Makris
No abstract text is available yet for this article.
January 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/27990784/new-findings-on-inhibitor-development-from-registries-to-clinical-studies
#12
REVIEW
F Peyvandi, C E Ettingshausen, J Goudemand, V Jiménez-Yuste, E Santagostino, M Makris
The high incidence of inhibitors against factor VIII (FVIII) concentrates in patients with haemophilia A has encouraged debate as to whether product-type plays a role. There is debate in the literature as to whether rFVIII concentrates are associated with a higher incidence of inhibitors compared to pdFVIII products. The management of haemophilia in patients with inhibitors includes on-demand/prophylaxis treatment with bypassing agents, and/or immune tolerance induction (ITI). However, these options create an economic and emotional burden on patients, their families and healthcare practitioners...
January 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/27988873/coagulation-factor-ix-recombinant-albumin-fusion-protein-albutrepenonacog-alfa-idelvion-%C3%A2-a-review-of-its-use-in-haemophilia-b
#13
Katherine A Lyseng-Williamson
Albutrepenonacog alfa (Idelvion(®)), a fusion protein that genetically fuses recombinant factor IX (rFIX) with recombinant human albumin (rAlbumin), is indicated in the treatment of haemophilia B. This narrative review discusses the pharmacological properties and clinical data related to the use of this novel fusion protein, hereafter referred to as rIX-FP. The fusion of rFIX to rAlbumin prolongs the elimination half-life of rIX-FP in the circulation, allowing routine prophylaxis to be administered once every 7-14 days...
December 17, 2016: Drugs
https://www.readbyqxmd.com/read/27928886/f376a-m388a-solulin-a-new-promising-antifibrinolytic-for-severe-haemophilia-a
#14
J Parcq, K U Petersen, A Borel-Derlon, P Gautier, M Ebel, D Vivien, Y Repessé
INTRODUCTION: Haemophilia is a major bleeding disorder due to a deficiency of procoagulant factor VIII (type A) or IX (type B). The treatment is substitutive and based on infusion of factor concentrates. Main limitations of this therapy are cost, short factor half-life and the development of inhibitors (up to 30% of severe HA patients). An important aggravating factor of haemophilia is due to a premature fibrinolysis, directing attention to the therapeutic potential of suitable antifibrinolytics...
December 8, 2016: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/27893354/summary-report-of-the-first-international-conference-on-inhibitors-in-haemophilia-a
#15
Sebastien Lacroix-Desmazes, David W Scott, Jenny Goudemand, Marijke Van Den Berg, Michael Makris, Alice S Van Velzen, Elena Santagostino, David Lillicrap, Frits R Rosendaal, Anneliese Hilger, Zuben E Sauna, Johannes Oldenburg, Lorenzo Mantovani, M Elisa Mancuso, Craig Kessler, Charles R M Hay, Paul Knoebl, Giovanni Di Minno, Keith Hoots, Amanda Bok, Mark Brooker, Erica Buoso, Pier Mannuccio Mannucci, Flora Peyvandi
No abstract text is available yet for this article.
November 25, 2016: Blood Transfusion, Trasfusione del Sangue
https://www.readbyqxmd.com/read/27891721/extended-half-life-pegylated-full-length-recombinant-factor-viii-for-prophylaxis-in-children-with-severe-haemophilia-a
#16
E S Mullins, O Stasyshyn, M T Alvarez-Román, D Osman, R Liesner, W Engl, M Sharkhawy, B E Abbuehl
INTRODUCTION: Primary factor VIII (FVIII) prophylaxis is the optimal treatment in children with severe haemophilia A. They are expected to benefit from extended half-life (T1/2 ) FVIII coverage by reduced infusion frequency while maintaining haemostatic efficacy. AIMS: To determine immunogenicity, pharmacokinetics (PK), efficacy, safety and quality of life of prophylaxis with a polyethylene glycol (peg)-ylated FVIII (BAX 855) based on full-length recombinant FVIII (ADVATE) in paediatric previously treated patients (PTPs) with severe haemophilia A...
November 27, 2016: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/27885373/a-new-era-of-treatment-for-patients-with-haemophilia-a
#17
Robert Klamroth
Treatment and prevention of bleeding episodes in patients with severe haemophilia A require frequent intravenous injection of factor VIII. Inhibitory antibodies against factor VIII occur in approximately 30 % of these patients during the first exposure days and immune tolerance induction to eradicate the inhibitor is challenging. Prevention of bleeds in patients with haemophilia A and inhibitors is less effective and there is ongoing research for alternative treatment options. A promising approach in 2016 is the development of emicizumab (ACE910), a bi-specific IgG antibody to factor IXa and factor X, that mimics the cofactor function of factor VIII...
November 25, 2016: Hämostaseologie
https://www.readbyqxmd.com/read/27878207/plasma-derived-versus-recombinant-factor-concentrates-in-pups-a-never-ending-debate
#18
Erik Berntorp
Inhibitor development in haemophilia is a serious complication to treatment with factor concentrates. Since the advent of more pure products, especially developed using recombinant DNA technology, some studies have shown an increased incidence of inhibitors in previously untreated patients (PUPs) receiving recombinant products whereas plasma-derived concentrates sometimes have been claimed to have a protective role, probably due to the content of von Willebrand factor (VWF). In fact, experiments indicate that the VWF may block uptake of factor VIII into macrophages for further processing to the immune system...
November 23, 2016: Hämostaseologie
https://www.readbyqxmd.com/read/27862687/anti-factor-viii-antibodies-in-brothers-with-haemophilia-a-share-similar-characteristics
#19
J Kahle, A Orlowski, D Stichel, J F Healey, E T Parker, S M Donfield, J Astermark, E Berntorp, P Lollar, D Schwabe, C Königs
INTRODUCTION: The development of neutralizing antibodies (inhibitors) against coagulation factor VIII (FVIII) is currently the most serious complication for patients with haemophilia A undergoing FVIII replacement therapy. Several genetic factors have been acknowledged as risk factors for inhibitor development. AIM: To analyze the influence of genetic factors on the nature of the humoral immune response to FVIII in eight brother pairs with inhibitors. METHODS: The domain specificity of FVIII-specific IgG was analysed by antibody binding to FVIII fragments and homologue-scanning mutagenesis (HSM)...
November 8, 2016: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/27852679/acquired-haemophilia-a-an-unusual-postoperative-complication
#20
Sumant Arora, Gaurav Goyal, Rehan Sarmad, Kenneth J Wool
An African-American man aged 65 years with multiple malignancies in remission was admitted for small bowel obstruction. He was treated with laparotomy following failure of conservative management. Postoperatively, he developed intra-abdominal bleed, which persisted, despite surgical haematoma evacuation. Further haematological workup revealed isolated prolongation of activated partial thromboplastin time (aPTT) with reduced factor VIII (FVIII) activity and raised FVIII inhibitor titre. Assuming acquired haemophilia A (AHA), FVIII inhibitor bypassing activity and corticosteroids were started with subsequent resolution of the bleeding from the surgical site...
November 16, 2016: BMJ Case Reports
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