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Inhibitor haemophilia

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https://www.readbyqxmd.com/read/28332238/outcome-measures-for-adult-and-pediatric-hemophilia-patients-with-inhibitors
#1
REVIEW
Cedric Hermans, Günter Auerswald, Gary Benson, Gerry Dolan, Anne Duffy, Victor Jiménez-Yuste, Rolf Ljung, Massimo Morfini, Thierry Lambert, Mehdi Osooli, Silva Zupančić Šalek
Recent advancements in almost all aspects of hemophilia treatment have vastly improved patient care and management, and new and emerging treatments hold the promise of further progress. However, there remains a scarcity of data on long-term outcomes in hemophilia, particularly among those patients with inhibitors, for whom no validated outcome assessment tools are currently available. At the 15(th) Zürich Haemophilia Forum, an expert panel reviewed the most important outcome measures in inhibitor patients and considered the challenges associated with assessing outcomes in this population...
March 22, 2017: European Journal of Haematology
https://www.readbyqxmd.com/read/28331929/confirmation-of-longer-fix-activity-half-life-with-prolonged-sample-collection-after-single-doses-of-nonacog-alfa-in-patients-with-haemophilia-b
#2
Baolai Hua, Runhui Wu, FeiFei Sun, Binyu Luo, Christine Alvey, Robert Labadie, Peng Roger Qu, Joan M Korth-Bradley, Pablo Rendo
A multicentre, single-dose study enrolled 12 previously treated patients with moderately severe to severe (factor IX [FIX] levels ≤2 IU/dl) haemophilia B to assess FIX pharmacokinetics after nonacog alfa administration and to evaluate the impact of length of sampling time on half-life (t½). After refraining from FIX replacement for four days, patients received 50 IU/kg as an intravenous (IV) infusion over 10 minutes. Blood samples were collected predose and 0.25, 0.5, 1, 3, 6, 9, 24, 50, 72, and 96 h post dose...
March 23, 2017: Thrombosis and Haemostasis
https://www.readbyqxmd.com/read/28306186/sippet-methodology-analysis-and-generalizability
#3
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)...
March 17, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28220685/incidence-of-low-titre-factor-viii-inhibitors-in-patients-with-haemophilia-a-meta-analysis-of-observational-studies
#4
A Messori, F Peyvandi, D Mengato, P M Mannucci
INTRODUCTION: A few studies have been focused on low-titre inhibitors in patients with haemophilia A. Although several putative factors have been implicated in the development of these inhibitors, solid data are still lacking. AIM: The aim of this study was to perform a proportion meta-analysis on the incidence of low-titre inhibitors in haemophilia A. METHODS: We surveyed the PubMed database to identify studies on de novo development of low-titre inhibitors in haemophilia A patients...
March 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28205285/natural-history-and-clinical-characteristics-of-inhibitors-in-previously-treated-haemophilia-a-patients-a-case-series
#5
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...
March 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
#6
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...
March 2017: British Journal of Haematology
https://www.readbyqxmd.com/read/28182257/inhibitor-screening-in-non-severe-haemophilia-patients-a-major-challenge
#7
EDITORIAL
Anne-Mette Hvas, Lone H Poulsen
No abstract text is available yet for this article.
March 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
#8
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
#9
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...
March 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
#10
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
#11
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
#12
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
#13
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
#14
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...
December 20, 2016: Cochrane Database of Systematic Reviews
https://www.readbyqxmd.com/read/27990785/inhibitor-development-in-haemophilia
#15
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
#16
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
#17
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
#18
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...
March 2017: 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
#19
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
#20
MULTICENTER STUDY
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...
March 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
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