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Inhibitor haemophilia

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https://www.readbyqxmd.com/read/28699675/intracranial-haemorrhage-in-children-and-adolescents-with-severe-haemophilia-a-or-b-the-impact-of-prophylactic-treatment
#1
Nadine G Andersson, Günter Auerswald, Chris Barnes, Manuel Carcao, Amy L Dunn, Karin Fijnvandraat, Marianne Hoffmann, Kaan Kavakli, Gili Kenet, Rainer Kobelt, Karin Kurnik, Ri Liesner, Anne Mäkipernaa, Marilyn J Manco-Johnson, Maria E Mancuso, Angelo C Molinari, Beatrice Nolan, Rosario Perez Garrido, Pia Petrini, Helen E Platokouki, Amy D Shapiro, Runhui Wu, Rolf Ljung
The discussion of prophylactic therapy in haemophilia is largely focused on joint outcomes. The impact of prophylactic therapy on intracranial haemorrhage (ICH) is less known. This study aimed to analyse ICH in children with haemophilia, with a focus on different prophylaxis regimens and sequelae of ICH. We conducted a multicentre retrospective and prospective study that included 33 haemophilia centres from 20 countries. Inclusion criteria were children and adolescents born between 1993 and 2014, with severe haemophilia A or B without inhibitors...
July 12, 2017: British Journal of Haematology
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
#2
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...
July 6, 2017: Thrombosis and Haemostasis
https://www.readbyqxmd.com/read/28686157/the-role-of-recombinant-activated-factor-vii-in-the-haematological-management-of-elective-orthopaedic-surgery-in-haemophilia-a-patients-with-inhibitors
#3
REVIEW
Giancarlo Castaman
The clinical profile and expectations of haemophilic patients with inhibitors have changed over the last three decades, mainly because of the prolongation of life-expectancy, often resulting in an increase of the orthopaedic burden. Recombinant activated factor VII (rFVIIa) is the most frequently used bypassing agent in haemophilia patients with inhibitors during elective orthopaedic surgery. For nearly 30 years, rFVIIa has been successfully used to control haemostasis in several major and minor surgical procedures...
May 16, 2017: Blood Transfusion, Trasfusione del Sangue
https://www.readbyqxmd.com/read/28686156/high-titre-inhibitors-in-previously-untreated-patients-with-severe-haemophilia-a-receiving-recombinant-or-plasma-derived-factor-viii-a-budget-impact-analysis
#4
Andrea Messori, Flora Peyvandi, Sabrina Trippoli, Roberta Palla, Frits R Rosendaal, Pier Mannuccio Mannucci
No abstract text is available yet for this article.
May 15, 2017: Blood Transfusion, Trasfusione del Sangue
https://www.readbyqxmd.com/read/28678027/meta-analysis-on-incidence-of-inhibitors-in-patients-with-haemophilia-a-treated-with-recombinant-factor-viii-products
#5
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/28646426/eftrenonacog-alfa-a-review-in-haemophilia-b
#6
Sheridan M Hoy
Eftrenonacog alfa (Alprolix™) is a recombinant fusion protein comprising human factor IX (FIX) covalently linked to the constant region (Fc) domain of human IgG1 (i.e. rFIXFc). The presence of the Fc domain extends the terminal half-life (t½) of rFIXFc, permitting prolonged treatment intervals. rFIXFc is available for intravenous use for the prophylaxis and treatment of bleeding in patients with haemophilia B. In two multinational, phase III studies in previously treated children, adolescents and adults with severe haemophilia B, rFIXFc prophylaxis resulted in low median annualized bleeding rates (ABRs), and was associated with reductions in median weekly factor consumption and dosing frequency compared with pre-study FIX regimens...
July 2017: Drugs
https://www.readbyqxmd.com/read/28594467/biochemical-characterization-of-lr769-a-new-recombinant-factor-viia-bypassing-agent-produced-in-the-milk-of-transgenic-rabbits
#7
G Chevreux, N Tilly, Y Leblanc, C Ramon, V Faid, M Martin, F Dhainaut, N Bihoreau
BACKGROUND: The bypassing agent factor VII (FVIIa) is a first-line therapy for the treatment of acute bleeding episodes in patients with haemophilia and high-titre inhibitors. FVIIa is a highly post-translationally modified protein that requires eukaryotic expression systems to produce a fully active molecule. A recombinant FVIIa was produced in the milk of transgenic rabbits to increase expression and provide an efficient, safe and affordable product after purification to homogeneity (LR769)...
June 8, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28594458/concizumab-an-anti-tissue-factor-pathway-inhibitor-antibody-induces-increased-thrombin-generation-in-plasma-from-haemophilia-patients-and-healthy-subjects-measured-by-the-thrombin-generation-assay
#8
E K Waters, J Sigh, U Friedrich, I Hilden, B B Sørensen
AIMS: Concizumab, a humanized monoclonal antibody against tissue factor pathway inhibitor (TFPI), is being developed as a subcutaneously (s.c.) administered treatment for haemophilia. It demonstrated a concentration-dependent procoagulant effect in functional TFPI assays; however, global haemostatic assays, such as the thrombin generation assay (TGA), offer a more complete picture of coagulation. We investigated how concizumab affects thrombin generation following ex vivo spiking in plasma from haemophilia patients using the TGA, and if the assay can detect the effect of multiple s...
June 8, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28594432/involvement-of-the-ige-basophil-system-and-mild-complement-activation-in-haemophilia-b-with-anti-factor-ix-neutralizing-antibodies-and-anaphylaxis
#9
M Cugno, M E Mancuso, A Tedeschi, E Santagostino, M Lorini, V Carbonelli, F Peyvandi, P M Mannucci
INTRODUCTION: Patients with haemophilia B who develop factor IX (FIX) neutralizing antibodies (inhibitors) after FIX infusion are at high risk of hypersensitivity reactions upon FIX re-exposure, but the underlying mechanisms are incompletely understood. AIM: To investigate biomechanisms of FIX hypersensitivity. METHODS: A cellular antigen stimulation test (CAST) was employed to evaluate leukotriene C4 (LTC4) release from basophils stimulated by FIX in three treated children with haemophilia B, one of whom developed FIX inhibitor and experienced anaphylaxis following FIX re-exposure...
June 8, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28574229/the-effects-of-joint-disease-inhibitors-and-other-complications-on-health-related-quality-of-life-among-males-with-severe-haemophilia-a-in-the-united-states
#10
J M Soucie, S D Grosse, A-E-A Siddiqi, V Byams, J Thierry, M M Zack, A Shapiro, N Duncan
INTRODUCTION: Health-related quality of life (HRQoL) is reduced among persons with haemophilia. Little is known about how HRQoL varies with complications of haemophilia such as inhibitors and joint disease. Estimates of preference-based HRQoL measures are needed to model the cost-effectiveness of prevention strategies. AIM: We examined the characteristics of a national sample of persons with severe haemophilia A for associations with two preference-based measures of HRQoL...
June 2, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28574205/first-line-immune-tolerance-induction-for-children-with-severe-haemophilia-a-a-protocol-from-the-uk-haemophilia-centre-doctors-organisation-inhibitor-and-paediatric-working-parties
#11
P Collins, E Chalmers, J Alamelu, C Hay, R Liesner, M Makris, M Mathias, J Payne, S Rangarajan, M Richards, K Talks, O Tunstall, M Williams, D P Hart
No abstract text is available yet for this article.
June 2, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28544163/the-impact-of-clinical-practice-on-the-outcome-of-central-venous-access-devices-in-children-with-haemophilia
#12
K Khair, S Ranta, A Thomas, K Lindvall
INTRODUCTION: Central venous access devices facilitate home treatment in boys with haemophilia. These are usually fully implanted lines, referred to as ports. Caregivers are taught to manage the port using sterile techniques and maintaining patency by flushing with saline or heparin solution. National and international guidelines for the home care of ports are lacking. AIM: To evaluate if infection or occlusion rates differ between home care regimens used for ports in children with haemophilia...
May 24, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28499509/ten-year-study-of-postoperative-complications-following-dental-extractions-in-patients-with-inherited-bleeding-disorders
#13
J-T Hsieh, K Klein, M Batstone
Dental extractions challenge the body's haemostatic mechanism. Postoperative bleeding from dental extraction can be prolonged, or even life threatening in patients with inherited bleeding disorders. Pre- and postoperative clotting factor replacements or systemic desmopressin (ddAVP) have been advocated at our institution to prevent bleeding complications in these patients. This study aimed to assess the postoperative bleeding rate in patients with inherited bleeding disorders that underwent dental extractions at our institution between 2003 and 2012...
May 9, 2017: International Journal of Oral and Maxillofacial Surgery
https://www.readbyqxmd.com/read/28492697/cd32-inhibition-and-high-dose-of-rhfviii-suppress-murine-fviii-specific-recall-response-by-distinct-mechanisms-in-vitro
#14
Nadine Vollack, Julia Friese, Sabine Bergmann, Andreas Tiede, Sonja Werwitzke
Development of neutralising antibodies (inhibitors) against factor VIII (FVIII) is a frequent and severe complication of replacement therapy in haemophilia A. Previous data from haemophilia A mouse model demonstrates that both CD32 inhibition and high doses of rhFVIII prevent the differentiation of FVIII-specific memory B cells (MBCs) into antibody secreting cells (ASCs). Here, cellular targets responsible for the suppression of ASC formation by means of CD32 inhibition and high dose of rhFVIII were analysed...
May 11, 2017: Thrombosis and Haemostasis
https://www.readbyqxmd.com/read/28475272/recombinant-factor-viia-addition-to-haemophilic-blood-perfused-over-collagen-tissue-factor-can-sufficiently-bypass-the-factor-ixa-viiia-defect-to-rescue-fibrin-generation
#15
R Li, K A Panckeri, P F Fogarty, A Cuker, S L Diamond
INTRODUCTION: Factor VIII (FVIII) or factor IX (FIX)-deficient haemophilic patients display deficits in platelet and fibrin deposition under flow detectable in microfluidics. Compared to fibrin generation, decreased platelet deposition in haemophilic blood flow is more easily rescued with recombinant factor VIIa (rFVIIa), whereas rFVIIa requires FXIIa participation to generate fibrin when tissue factor (TF) is absent. AIMS: Perfusion of haemophilic whole blood (WB) over collagen/TF surfaces was used to determine whether rFVIIa/TF was sufficient to bypass poor FIXa/FVIIIa function in blood from patients with haemophilia A and B...
May 5, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28470911/platelet-rich-plasma-for-chronic-synovitis-treatment-in-patients-with-haemophilia
#16
H Caviglia, M E Landro, C Daffunchio, G Galatro, A L Douglas Price, P Salgado, D Neme
INTRODUCTION: Haemophilic synovitis is caused by chronic accumulation of blood in the joint. Conservative treatment is insufficient to solve this pathology. Platelet-rich plasma (PRP) has a high concentration of growth factors (GFs) that play a key role in regulation and stimulation of healing processes. The aim of this study was to describe the effect of PRP injection in chronic synovitis of the joints in patients with haemophilia (PWH). PATIENTS AND METHODS: Nineteen patients with 28 joints were treated at our centre in Buenos Aires, Argentina between December 2014 and December 2015...
May 4, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28452151/pk-guided-personalized-prophylaxis-with-nuwiq-%C3%A2-human-cl-rhfviii-in-adults-with-severe-haemophilia-a
#17
T Lissitchkov, L Rusen, P Georgiev, J Windyga, R Klamroth, L Gercheva, L Nemes, A Tiede, J Bichler, S Knaub, L Belyanskaya, O Walter, K J Pasi
INTRODUCTION: Nuwiq(®) (human-cl rhFVIII) is a 4(th) generation recombinant human FVIII, without chemical modification or protein fusion, produced in a human cell-line. AIMS/METHODS: This study (NuPreviq) was a prospective, open-label, multicentre, phase IIIb study of the efficacy and safety of personalized prophylaxis with Nuwiq(®) in 66 previously treated adults with severe haemophilia A. NuPreviq had three phases: (i) a 72-h pharmacokinetic (PK) phase; (ii) a 1-3 month standard prophylaxis phase; and (iii) a 6-month personalized prophylaxis phase...
April 27, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28440004/safety-and-effectiveness-of-room-temperature-stable-recombinant-factor-viia-in-patients-with-haemophilia-a-or-b-and-inhibitors-results-of-a-multinational-prospective-observational-study
#18
K Kavakli, F Demartis, M Karimi, P Eshghi, D Neme, H Chambost, L Sommer, M Zak, G Benson
INTRODUCTION: A room temperature stable formulation of recombinant activated factor VII (NovoSeven(®) ), allowing convenient storage and therefore improved treatment access, has been developed. Bioequivalence to the previous NovoSeven(®) was demonstrated in healthy humans, leading to European approval (2008). Although no confirmed cases of neutralising antibodies to rFVIIa in patients with haemophilia A or B have been observed with the original formulation, changes in formulation or storage condition may alter immunogenicity...
April 24, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28438758/diabetes-mellitus-and-acquired-haemophilia-new-association
#19
Carlos Tavares Bello, Yuliya Vasylenko, José Esteves, Carlos Augusto Vasconcelos
Diabetes mellitus encompasses a group of highly prevalent carbohydrate metabolic disorders with an increasing incidence. Some subtypes are thought to be associated with other immune-mediated diseases. Acquired haemophilia on the other hand is a quite rare autoimmune disease that is thought to be secondary to the emergence of inhibiting anticoagulation factor VIII antibodies (inhibitors) in patients with previously normal haemostatic function. More recently, numerous different diseases have been associated with acquired haemophilia namely immune-mediated diseases, drugs and solid and haematologic neoplasms...
April 24, 2017: BMJ Case Reports
https://www.readbyqxmd.com/read/28420167/treatment-and-prevention-of-bleeds-in-haemophilia-patients-with-inhibitors-to-factor-viii-ix
#20
REVIEW
Angiola Rocino, Massimo Franchini, Antonio Coppola
The development of alloantibodies neutralising therapeutically administered factor (F) VIII/IX (inhibitors) is currently the most severe complication of the treatment of haemophilia. When persistent and at a high titre, inhibitors preclude the standard replacement treatment with FVIII/FIX concentrates, making patients' management challenging. Indeed, the efficacy of bypassing agents, i.e., activated prothrombin complex concentrates (aPCC) and recombinant activated factor VII (rFVIIa), needed to overcome the haemostatic interference of the inhibitor, is not comparable to that of factor concentrates...
April 17, 2017: Journal of Clinical Medicine
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