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Inhibitor haemophilia

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https://www.readbyqxmd.com/read/27928886/f376a-m388a-solulin-a-new-promising-antifibrinolytic-for-severe-haemophilia-a
#1
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
#2
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
#3
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
#4
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
#5
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
#6
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
#7
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
https://www.readbyqxmd.com/read/27824212/regulatory-t-cells-and-their-potential-for-tolerance-induction-in-haemophilia-a-patients
#8
A Schmidt, C Königs
FVIII inhibitors still are the major concern in treatment of haemophilia A patients by FVIII replacement therapy. Immune tolerance induction to reverse inhibitor formation fails in about 30% of treated patients. These patients face increased morbidity and mortality producing a need for new therapy strategies in the treatment of FVIII inhibitor-positive patients. Regulatory T cells are important modulators of the immune response and are also involved in the immune response to FVIII in haemophilia A patients...
November 8, 2016: Hämostaseologie
https://www.readbyqxmd.com/read/27813214/complications-of-haemophilia-in-babies-first-two-years-of-life-a-report-from-the-centers-for-disease-control-and-prevention-universal-data-collection-system
#9
R Kulkarni, R J Presley, J M Lusher, A D Shapiro, J C Gill, M Manco-Johnson, M A Koerper, T C Abshire, D DiMichele, W K Hoots, P Mathew, D J Nugent, S Geraghty, B L Evatt, J M Soucie
AIM: To describe the prevalence and complications in babies ≤2 years with haemophilia. METHODS: We used a standardized collection tool to obtain consented data on eligible babies aged ≤2 years with haemophilia enrolled in the Centers for Disease Control and Prevention Universal Data Collection System surveillance project at US Hemophilia Treatment Centers (HTCs). RESULTS: Of 547 babies, 82% had haemophilia A, and 70% were diagnosed within one month of birth...
November 4, 2016: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/27770665/current-view-and-outcome-of-iti-therapy-a-change-over-time
#10
K Holstein, A Batorova, M Carvalho, K Fijnvandraat, P Holme, K Kavakli, T Lambert, A Rocino, V Jiménez-Yuste, J Astermark
INTRODUCTION: Inhibitor development in people with haemophilia is a serious complication that may require intensive and costly interventions. The goal of inhibitor management should be permanent inhibitor eradication through immune tolerance induction (ITI), but well-designed studies are lacking and the management of patients is therefore defined by the experience and views of the clinician. OBJECTIVES: To explore the current clinical practice and outcome of ITI therapy in Europe and how this may have changed over the last decade, as well as to provide consensus recommendations to guide clinicians in their clinical practice...
October 15, 2016: Thrombosis Research
https://www.readbyqxmd.com/read/27766057/different-impact-of-factor-viii-products-on-inhibitor-development
#11
H Marijke van den Berg
Inhibitors are the most serious side effect of haemophilia treatment; they occur in 25-30 % of all patients with severe haemophilia A. Over the last 2 decades, conflicting data on the impact of clotting products have been published. Due to small studies of selected cases, appreciation of the impact of any particular product has been difficult. Moreover, the emphasis on inhibitor testing has led to increased detection of low-titre inhibitors (to >10 %), while the percentage of high-titre inhibitors is still around 20 %...
2016: Thrombosis Journal
https://www.readbyqxmd.com/read/27761964/recombinant-b-domain-deleted-porcine-sequence-factor-viii-r-pfviii-for-the-treatment-of-bleeding-in-patients-with-congenital-haemophilia-a-and-inhibitors
#12
J N Mahlangu, T A Andreeva, D E Macfarlane, C Walsh, N S Key
INTRODUCTION: Development of inhibitors to human FVIII (hFVIII) significantly complicates the control of bleeding events in patients with haemophilia A. AIM: This prospective, multicentre, open-label, non-comparative, Phase II study evaluated the haemostatic activity of a recombinant B-domain-deleted porcine FVIII (r-pFVIII), in the treatment of non-life/non-limb-threatening bleeding in individuals with haemophilia A and FVIII inhibitors. METHODS: Acute bleeding episodes in patients with pFVIII inhibitor titres <0...
October 20, 2016: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/27761962/european-retrospective-study-of-real-life-haemophilia-treatment
#13
E Berntorp, G Dolan, C Hay, S Linari, E Santagostino, A Tosetto, G Castaman, M T Álvarez-Román, R Parra Lopez, J Oldenburg, T Albert, U Scholz, M Holmström, J-F Schved, M Trossaërt, C Hermans, A Boban, C Ludlam, S Lethagen
INTRODUCTION: Haemophilia treatment varies significantly between individuals, countries and regions and details of bleed rates, factor consumption and injection frequency are often not available. AIM: To provide an overview of the FVIII/FIX treatment practice and outcome for patients with haemophilia A (HA) or haemophilia B (HB) across Europe. METHODS: Non-interventional, 12-month retrospective study where anonymized data were retrieved from haemophilia centres/registers in Belgium, France, Germany, Italy, Spain, Sweden and the United Kingdom...
October 20, 2016: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/27734074/application-of-a-molecular-diagnostic-algorithm-for-haemophilia-a-and-b-using-next-generation-sequencing-of-entire-f8-f9-and-vwf-genes
#14
Jose Maria Bastida Bermeja, Jose Ramon González-Porras, Cristina Jiménez, Rocio Benito, Gonzalo R Ordoñez, Maria Teresa Álvarez-Román, M Elena Fontecha, Kamila Janusz, David Castillo, Rosa María Fisac, Luis Javier García-Frade, Carlos Aguilar, María Paz Martínez, Nuria Bermejo, Sonia Herrero, Ana Balanzategui, Jose Manuel Martin-Antorán, Rafael Ramos, Maria Jose Cebeiro, Emilia Pardal, Carmen Aguilera, Belen Pérez-Gutierrez, Manuel Prieto, Susana Riesco, Maria Carmen Mendoza, Ana Benito, Ana Hortal Benito-Sendin, Víctor Jiménez-Yuste, Jesus Maria Hernández-Rivas, Ramon García-Sanz, Marcos González-Díaz, Maria Eugenia Sarasquete
Currently, molecular diagnosis of haemophilia A and B (HA and HB) highlights the excess risk-inhibitor development associated with specific mutations, and enables carrier testing of female relatives and prenatal or preimplantation genetic diagnosis. Molecular testing for HA also helps distinguish it from von Willebrand disease (VWD). Next-generation sequencing (NGS) allows simultaneous investigation of several complete genes, even though they may span very extensive regions. This study aimed to evaluate the usefulness of a molecular algorithm employing an NGS approach for sequencing the complete F8, F9 and VWF genes...
October 13, 2016: Thrombosis and Haemostasis
https://www.readbyqxmd.com/read/27698522/systemic-therapy-in-acquired-haemophilia-a-single-institute-experience
#15
Lawless Sarah, Das Prantik, Benson Gary
A cornerstone of the management of Acquired Haemophilia A (AHA) involves inhibitor eradication. First line immunosuppressive agents are usually steroids, either alone or in combination with cyclophosphamide. We present the use of Rituximab, cyclophosphamide, vincristine and prednisolone (RCVP) combination as immunosuppressant in AHA in a small cohort of patients in order to control their symptoms and eradicate inhibitors. This was a retrospective analysis of all AHA patients treated at the Northern Ireland Haemophilia centre over a six year period...
September 2016: Ulster Medical Journal
https://www.readbyqxmd.com/read/27683758/combination-therapy-for-inhibitor-reversal-in-haemophilia-a-using-monoclonal-anti-cd20-and-rapamycin
#16
Moanaro Biswas, Geoffrey L Rogers, Alexandra Sherman, Barry J Byrne, David M Markusic, Haiyan Jiang, Roland W Herzog
Development of antibodies (inhibitors) against coagulation factor VIII (FVIII) is a major complication of intravenous replacement therapy in haemophilia A (HA). Current immune tolerance induction (ITI) regimens are not universally effective. Rituximab, a B cell-depleting antibody against CD20, has shown mixed results for inhibitor reversal in patients. This study aims to develop a combinatorial therapy for inhibitor reversal in HA, using anti-murine CD20 (anti-mCD20) antibody and rapamycin, which targets both B and T cell responses...
September 29, 2016: Thrombosis and Haemostasis
https://www.readbyqxmd.com/read/27609590/aspirin-unmasking-acquired-haemophilia-a-in-a-patient-with-prostate-cancer
#17
Julie Omolola Okiro, Amjad Zaman Khan, Fergus Keane, Faiza Murad
A 72-year-old man, on treatment for prostate cancer, attended the emergency department with his 2nd episode of spontaneous extensive bruising and haematomas. His first presentation was 2 months prior but this was thought to be because of his aspirin and he improved when aspirin was discontinued. On this occasion aspirin had been restarted 7 days before he developed his symptoms. His blood investigation was significant for a much raised activated partial thromboplastin time (aPTT). On his 3rd day of admission he deteriorated clinically with a drastic drop in his haemoglobin and worsening tense haematomas...
2016: BMJ Case Reports
https://www.readbyqxmd.com/read/27583313/long-acting-recombinant-fusion-protein-linking-coagulation-factor-ix-with-albumin-rix-fp-in-children-results-of-a-phase-3-trial
#18
Gili Kenet, Hervé Chambost, Christoph Male, Thierry Lambert, Susan Halimeh, Tatiana Chernova, Maria Elisa Mancuso, Julie Curtin, Christine Voigt, Yanyan Li, Iris Jacobs, Elena Santagostino
A global phase 3 study evaluated the pharmacokinetics, efficacy and safety of a recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in 27 previously treated male children (1-11 years) with severe and moderately severe haemophilia B (factor IX [FIX] activity ≤2 IU/dl). All patients received routine prophylaxis once every seven days for up to 77 weeks, and treated any bleeding episodes on-demand. The mean terminal half-life of rIX-FP was 91.4 hours (h), 4.3-fold longer than previous FIX treatment and clearance was 1...
September 27, 2016: Thrombosis and Haemostasis
https://www.readbyqxmd.com/read/27577234/bay-81-8973-octocog-alfa-kovaltry-%C3%A2-a-review-in-haemophilia-a
#19
Gillian M Keating
BAY 81-8973 (octocog alfa; Kovaltry(®)) is an unmodified, full-length, recombinant factor VIII (FVIII) concentrate with the same amino acid sequence as Kogenate(®) FS, but produced with innovative manufacturing technologies. This narrative review discusses the clinical efficacy and tolerability of BAY 81-8973 in haemophilia A, as well as summarizing its pharmacological properties. Results of the LEOPOLD I, LEOPOLD II and LEOPOLD Kids trials demonstrated that routine prophylaxis with intravenous BAY 81-8973 was associated with a low annualized bleeding rate (ABR) in previously treated adult and paediatric patients with severe haemophilia A...
October 2016: BioDrugs: Clinical Immunotherapeutics, Biopharmaceuticals and Gene Therapy
https://www.readbyqxmd.com/read/27562315/non-genetic-risk-factors-in-haemophilia-a-inhibitor-management-the-danger-theory-and-the-use-of-animal-models
#20
K M Lövgren, H Søndergaard, S Skov, B Wiinberg
In haemophilia A (HA) management, antidrug antibodies, or inhibitors, are a serious complication that renders factor VIII (FVIII) replacement therapy ineffective, increases morbidity and reduces quality of life for affected patients. Inhibitor development aetiology is multifactorial and covers both genetic and therapy related risk factors. Many therapy-related risk factors have proven difficult to confirm due to several confounding factors and the small study populations available. However, clinical studies indicate that e...
September 2016: Haemophilia: the Official Journal of the World Federation of Hemophilia
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