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Inhibitor haemophilia

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https://www.readbyqxmd.com/read/29340855/the-relationship-between-target-joints-and-direct-resource-use-in-severe-haemophilia
#1
Jamie O'Hara, Shaun Walsh, Charlotte Camp, Giuseppe Mazza, Liz Carroll, Christina Hoxer, Lars Wilkinson
OBJECTIVES: Target joints are a common complication of severe haemophilia. While factor replacement therapy constitutes the majority of costs in haemophilia, the relationship between target joints and non drug-related direct costs (NDDCs) has not been studied. METHODS: Data on haemophilia patients without inhibitors was drawn from the 'Cost of Haemophilia across Europe - a Socioeconomic Survey' (CHESS) study, a cost assessment in severe haemophilia A and B across five European countries (France, Germany, Italy, Spain, and the United Kingdom) in which 139 haemophilia specialists provided demographic and clinical information for 1285 adult patients...
January 16, 2018: Health Economics Review
https://www.readbyqxmd.com/read/29328905/diagnosis-and-care-of-patients-with-mild-haemophilia-practical-recommendations-for-clinical-management
#2
REVIEW
Gary Benson, Günter Auerswald, Gerry Dolan, Anne Duffy, Cedric Hermans, Rolf Ljung, Massimo Morfini, Silva Zupančić Šalek
Mild haemophilia is defined by factor levels between 0.05 and 0.40 IU/mL and is characterised by traumatic bleeds. Major issues associated with mild haemophilia are that it may not present for many years after birth, and that awareness, even within families, may be low. Methodological problems exist in diagnosis, such as inconsistencies in results obtained from different assays used to measure factor levels in mild haemophilia. Advances in genetic testing provide insight into diagnosis as well as the likelihood of inhibitor development, which is not uncommon in patients with mild or moderate haemophilia and can increase morbidity...
November 14, 2017: Blood Transfusion, Trasfusione del Sangue
https://www.readbyqxmd.com/read/29314552/australian-multicentre-study-of-current-real-world-prophylaxis-practice-in-severe-and-moderate-haemophilia-a-and-b
#3
J A Mason, S Parikh, H Tran, J Rowell, S McRae
INTRODUCTION: With the emergence of novel treatment products for haemophilia and an increasing focus on the benefits of pharmacokinetic driven individualized prophylaxis, robust national data with regard to current patterns of factor consumption and adherence are required. AIM: To characterize current Australian practice with regard to use of prophylactic clotting factor infusions in patients with moderate or severe haemophilia A (HA) and haemophilia B (HB). METHODS: This was a retrospective, non-interventional study utilizing Australian Bleeding Disorder Registry (ABDR) data collected over a 12 month period...
January 3, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29314439/low-incidence-of-factor-viii-inhibitors-in-previously-untreated-patients-with-severe-haemophilia-a-treated-with-octanate%C3%A2-final-report-from-a-prospective-study
#4
A Klukowska, V Komrska, V Vdovin, A Pavlova, M Jansen, S Lowndes, L Belyanskaya, O Walter, P Laguna
INTRODUCTION: Octanate® is a human, plasma-derived, von Willebrand factor-stabilized coagulation factor VIII (FVIII) concentrate with demonstrated haemostatic efficacy in previously treated patients with haemophilia A. AIM: This prospective, open-label study aimed to assess the immunogenicity of octanate® in previously untreated patients (PUPs). METHODS: The study monitored development of FVIII inhibitors in 51 PUPs. Tolerability, viral safety, FVIII recovery and efficacy of octanate® for the prevention and treatment of bleeds and in surgical procedures were also assessed...
January 3, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29282815/prevalence-of-and-risk-factors-for-cerebral-microbleeds-among-adult-patients-with-haemophilia-a-or-b
#5
H Husseinzadeh, T Chiasakul, P A Gimotty, B Pukenas, R Wolf, M Kelty, E Chiang, P F Fogarty, A Cuker
INTRODUCTION: Cerebral microbleeds (CMBs) represent clinically silent haemorrhagic events. Cerebral microbleeds (CMBs) portend negative neurovascular and cognitive outcomes in the general population and are associated with cognitive impairment in persons with haemophilia (PWH). Prevalence, patterns, and risk factors for CMBs in PWH have not been directly compared to persons without coagulopathy. AIM: To examine prevalence, patterns, and risk factors for CMBs in PWH vs normal controls...
December 28, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29243367/vaccinations-are-not-associated-with-inhibitor-development-in-boys-with-severe-haemophilia-a
#6
H Platokouki, K Fischer, S C Gouw, A Rafowicz, M Carcao, G Kenet, R Liesner, K Kurnik, G E Rivard, H M van den Berg
BACKGROUND: Inhibitor development in previously untreated patients (PUPs) with severe haemophilia A is a multifactorial event. It is unknown whether paediatric vaccinations given in close proximity to factor VIII (FVIII) are associated with inhibitor development. OBJECTIVE: To assess whether paediatric vaccinations in close proximity to FVIII within the first 75 exposure days (EDs) are associated with inhibitor development in PUPs with severe haemophilia A. METHODS: We included 375 PUPs with severe haemophilia A (<0...
December 15, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29212115/risk-factors-for-the-progression-from-low-to-high-titres-in-260-children-with-severe-haemophilia-a-and-newly-developed-inhibitors
#7
Maria Elisa Mancuso, Kathelijn Fischer, Elena Santagostino, Johannes Oldenburg, Helen Platokouki, Cristoph Königs, Carmen Escuriola-Ettingshausen, George E Rivard, Ana Rosa Cid, Manuel Carcao, Rolf Ljung, Pia Petrini, Carmen Altisent, Gili Kenet, Raina Liesner, Karin Kurnik, Günther Auerswald, Hérvè Chambost, Anne Mäkipernaa, Angelo Claudio Molinari, Mike Williams, H Marijke van den Berg
No abstract text is available yet for this article.
December 2017: Thrombosis and Haemostasis
https://www.readbyqxmd.com/read/29212114/use-of-bypassing-agents-and-risk-of-thromboembolic-events-in-patients-with-haemophilia-and-inhibitors
#8
Katsiaryna Bykov, Rhonda L Bohn, Bruce M Ewenstein, John D Seeger, Jerry Avorn, Brian T Bateman
No abstract text is available yet for this article.
December 2017: Thrombosis and Haemostasis
https://www.readbyqxmd.com/read/29202211/acute-haemarthrosis-in-the-haemophilia-a-rat-generates-a-local-and-systemic-proinflammatory-response
#9
Karin M Lövgren, Kristine R Christensen, Wiktor Majewski, Olga Østrup, Søren Skov, Bo Wiinberg
Background Replacement therapy with coagulation factor VIII (FVIII) concurrent with bleeds (on-demand) in haemophilia A (HA) patients has been hypothesized to increase the risk for antidrug antibodies (inhibitors). A danger signal environment, characterized by tissue damage and inflammation at the site of a bleed, is thought to contribute to the anti-FVIII response. The nature of this inflammatory reaction is, however, not fully known, and new insights will be valuable for both managing inhibitors and understanding arthropathy development...
November 2017: Thrombosis and Haemostasis
https://www.readbyqxmd.com/read/29194852/the-role-of-sclerostin-dickkopf-1-and-receptor-activator-of-nuclear-factor-kb-ligand-osteoprotegerin-signalling-pathways-in-the-development-of-osteoporosis-in-patients-with-haemophilia-a-and-b-a-cross-sectional-study
#10
P Anagnostis, S Vakalopoulou, D Christoulas, S A Paschou, A Papatheodorou, V Garipidou, P Kokkoris, E Terpos
AIM: Haemophilia A and B are associated with reduced bone mineral density (BMD). The aim of this study was to assess circulating sclerostin and dickkopf-1 (Dkk-1), (inhibitors of osteoblastic differentiation), as well as the receptor activator of nuclear factor kB ligand (RANKL)/osteoprotegerin (OPG) system (the major regulator of osteoclastogenesis), in patients with haemophilia (PWH), their possible correlations with clinical risk factors and the effect of ibandronate on these markers...
December 1, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29170896/modern-treatments-of-haemophilia-review-of-cost-effectiveness-analyses-and-future-directions
#11
REVIEW
Paolo A Cortesi, Lucia S D'Angiolella, Alessandra Lafranconi, Mariangela Micale, Giancarlo Cesana, Lorenzo G Mantovani
BACKGROUND: Cost is currently one of the most important aspects in haemophilia care. Factor concentrates absorb more than 90% of healthcare direct costs of haemophilia care, and the debate regarding the high cost of haemophilia treatments and their different use across different countries is increasing. OBJECTIVE: The objective of this study was to review cost-effectiveness analyses conducted on treatment options in haemophilia, focusing on their results and their strengths and limitations; to highlight the possible issues associated with economic evaluations of new treatment options...
November 23, 2017: PharmacoEconomics
https://www.readbyqxmd.com/read/29149550/a-promising-on-demand-treatment-option-for-bleeding-events-in-haemophilia-patients-with-inhibitors
#12
Amy D Shapiro
No abstract text is available yet for this article.
November 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29135476/thrombogenicity-evaluation-in-221-patients-with-haemophilia-b-treated-with-nonacog-alfa
#13
Pablo Rendo, Janice Lamb, Lynne Smith, Joanne Fuiman, Joan M Korth-Bradley
: Risk for thrombotic events with factor IX replacement therapy in patients with haemophilia B remains a concern for patients, those who treat them, and regulatory agencies, based on experience with early use of prothrombin complex concentrates. The current post hoc analysis assessed the incidence of thrombotic events and changes in prothrombin fragment 1 + 2, thrombin-antithrombin complex, and D-dimer in 221 patients with haemophilia B who received nonacog alfa in clinical studies. Thrombotic event and coagulation marker data were collected from 8 interventional studies utilizing on-demand, prophylactic, and preventive regimens in patients with haemophilia B...
November 10, 2017: Blood Coagulation & Fibrinolysis: An International Journal in Haemostasis and Thrombosis
https://www.readbyqxmd.com/read/29124682/nonacog-beta-pegol-a-review-in-haemophilia-b
#14
Yahiya Y Syed
Nonacog beta pegol [Refixia(®) (EU)] is an intravenously-administered, glycoPEGylated recombinant factor IX (FIX), with an extended terminal half-life. It is approved in the EU for the treatment and prophylaxis of bleeding in patients aged ≥ 12 years with haemophilia B. The therapeutic efficacy and safety of nonacog beta pegol was demonstrated in the phase 3 Paradigm trials in previously treated adolescents and adults with haemophilia B. In Paradigm 2, nonacog beta pegol showed good haemostatic effects when treating bleeds on-demand, and reduced annualized bleeding rates when used as a once-weekly prophylaxis...
November 9, 2017: Drugs
https://www.readbyqxmd.com/read/29094008/total-joint-replacement-in-inhibitor-positive-haemophilia-long-term-outcome-analysis-in-fifteen-patients
#15
Heidi Danielson, Riitta Lassila, Pekka Ylinen, Timo Yrjönen
AIM: To collect data from joint replacement in inhibitor patients, evaluate haemostatic and patient outcomes, and analyse the costs. METHODS: We report our 21-year, single-centre cumulative experience of 15 joint arthroplasties in six inhibitor patients. RESULTS: Two low responder inhibitor patients were in the early days treated with FVIII, whereas bypassing agents were used in the rest of the high responder patients. The primary haemostatic outcome was good in 8/15, fair in 4/15 and poor in 3/15 operations...
October 18, 2017: World Journal of Orthopedics
https://www.readbyqxmd.com/read/29080391/timing-and-severity-of-inhibitor-development-in-recombinant-versus-plasma-derived-factor-viii-concentrates-a-sippet-analysis
#16
F Peyvandi, A Cannavò, I Garagiola, R Palla, P M Mannucci, F R Rosendaal
Essentials Recombinant factor VIII (rFVIII) was contrasted with plasma-derived FVIII (pdFVIII). In previously untreated patients with hemophilia A, rFVIII led to more inhibitors than pdFVIII. Inhibitors with rFVIII developed earlier, and the peak rate was higher than with pdFVIII. Inhibitors with rFVIII were more severe (higher titre) than with pdFVIII. SUMMARY: Background The development of neutralizing antibodies (inhibitors) against factor VIII (FVIII) is the most severe complication in the early phases of treatment of severe hemophilia A...
January 2018: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/29080389/sippet-insights-into-factor-viii-immunogenicity
#17
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/29048712/efficacy-and-safety-of-nuwiq-%C3%A2-human-cl-rhfviii-in-patients-with-severe-haemophilia-a-undergoing-surgical-procedures
#18
N Zozulya, C M Kessler, A Klukowska, M von Depka, K Hampton, C R M Hay, M Jansen, J Bichler, S Knaub, S Rangarajan
INTRODUCTION: Haemophilia A patients are at a high risk of excess bleeding during surgeries. The aim of haemostatic therapy during the perioperative period is to normalize FVIII level perioperatively and postoperatively to maintain normal haemostasis until wound healing is complete. AIMS/METHODS: To examine the efficacy of Nuwiq(®) (simoctocog alfa, human-cl rhFVIII), a 4(th) generation recombinant FVIII produced in a human cell line, for surgical prophylaxis in patients with severe haemophilia A...
October 19, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29044825/pattern-of-bleeding-in-a-large-prospective-cohort-of-haemophilia-a-patients-a-three-year-follow-up-of-the-ahead-advate-in-haemophilia-a-outcome-database-study
#19
K Khair, M G Mazzucconi, R Parra, E Santagostino, D A Tsakiris, C Hermans, J Oldenburg, G Spotts, K Steinitz-Trost, A Gringeri
INTRODUCTION: Outcome data on treatment of patients with haemophilia A spanning several years of real-world evidence collection are currently very limited. AIM AND METHODS: The global prospective long-term Advate(®) Haemophilia A Outcome Database (AHEAD) cohort study collects real-world data from patients with severe and moderate haemophilia. We report an interim data read-out after three years of observation. RESULTS: A total of 522 patients were enrolled from 21 countries: 334 completed year 1 follow-up, 238 completed year 2 and 136 completed year 3, with an overall follow-up of 811 patient-years...
October 17, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28984010/safety-and-dose-dependency-of-eptacog-beta-activated-in-a-dose-escalation-study-of-non-bleeding-congenital-haemophilia-a-or-b-patients-with-or-without-inhibitors
#20
J Ducore, J B Lawrence, M Simpson, L Boggio, A Bellon, J Burggraaf, J Stevens, M Moerland, J Frieling, J Reijers, M Wang
INTRODUCTION: Varying initial doses of activated eptacog beta (recombinant human FVIIa, rhFVIIa) may provide therapeutic options when treating bleeding in patients with congenital haemophilia who have developed inhibitory antibodies to factor VIII (FVIII) or factor IX (FIX). This study evaluated escalated doses of a new rhFVIIa product as a prelude to selecting the doses for clinical efficacy evaluation in haemophilia patients. AIM: To assess the safety, pharmacokinetics, and laboratory pharmacodynamics of 3 doses of rhFVIIa in non-bleeding patients with congenital haemophilia A or B with or without inhibitors...
November 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
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