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Inhibitor haemophilia

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https://www.readbyqxmd.com/read/29777571/optimization-of-pre-analytical-heat-treatment-for-inhibitor-detection-in-haemophilia-a
#1
P Batty, D P Hart, S Platton
INTRODUCTION: Factor VIII (FVIII) antibody formation is the greatest clinical and laboratory challenge within the haemophilia centre. The Nijmegen-Bethesda assay (NBA) is the gold standard for inhibitor quantification, but affected by pre-analytical variables including a patient's FVIII activity (FVIII:C). Pre-analytical heat treatment (PHT) provides a methodology for inhibitor testing when measurable FVIII:C is present. METHODS: We evaluated the effect of different PHT conditions (time/temperature) on FVIII:C as well as on potency of inhibitory activity in samples containing FVIII:C (endogenous pooled plasma and exogenous recombinant FVIII (rFL-FVIII) concentrate) or FVIII inhibitor...
May 18, 2018: International Journal of Laboratory Hematology
https://www.readbyqxmd.com/read/29741299/methodologies-for-data-collection-in-congenital-haemophilia-with-inhibitors-chwi-critical-assessment-of-the-literature-and-lessons-learned-from-recombinant-factor-viia
#2
REVIEW
C M Kessler, S Benchikh El Fegoun, A Worster
AIMS: To systematically review the effectiveness of on-demand treatment with recombinant coagulation factor VIIa (rFVIIa) in congenital haemophilia with inhibitors and, if feasible, perform a meta-analysis of the data. MATERIALS AND METHODS: Publications from Embase® , MEDLINE® , MEDLINE® In-Process and the Cochrane Central Register of Controlled Trials were searched. Selected publications were reviewed for inclusion by two independent expert reviewers. Discrepancies were reconciled by a third independent reviewer...
May 9, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29724628/the-rare-coexistence-of-high-titer-inhibitor-development-and-gastrointestinal-stromal-tumor-in-a-patient-with-severe-hemophilia-a-case-report
#3
Abdulkerim Yıldız, Osman Şahin, Okan Yayar, Çiğdem Pala Öztürk, Hacer Berna Afacan Öztürk, Senem Maral, Harika Okutan, Murat Albayrak
Hemophilia is a hereditary disease with impaired blood coagulation due to a genetic deficiency of blood coagulation factors. The development of inhibitors further complicates the course of the disease and management. The case is here reported of a haemophilia patient who presented with coexisting development of high titer inhibitor with Gastrointestinal Stromal Tumor (GIST) diagnosis and was admitted with upper gastrointestinal system bleeding. The patient had no prior history of inhibitor presence. During all procedures including surgery, excellent hemostasis was achieved with rFVIIa treatment and no hemorrhagic complication was observed...
April 26, 2018: Transfusion and Apheresis Science
https://www.readbyqxmd.com/read/29720192/the-impact-of-severe-haemophilia-and-the-presence-of-target-joints-on-health-related-quality-of-life
#4
Jamie O'Hara, Shaun Walsh, Charlotte Camp, Giuseppe Mazza, Liz Carroll, Christina Hoxer, Lars Wilkinson
BACKGROUND: Joint damage remains a major complication associated with haemophilia and is widely accepted as one of the most debilitating symptoms for persons with severe haemophilia. The aim of this study is to describe how complications of haemophilia such as target joints influence health-related quality of life (HRQOL). METHODS: Data on hemophilia patients without inhibitors were drawn from the 'Cost of Haemophilia across Europe - a Socioeconomic Survey' (CHESS) study, a cost-of-illness assessment in severe haemophilia A and B across five European countries (France, Germany, Italy, Spain, and the UK)...
May 2, 2018: Health and Quality of Life Outcomes
https://www.readbyqxmd.com/read/29703220/european-principles-of-inhibitor-management-in-patients-with-haemophilia
#5
P L F Giangrande, C Hermans, B O'Mahony, P de Kleijn, M Bedford, A Batorova, J Blatný, K Jansone
BACKGROUND: In spite of recent major advances in the understanding and treatment of inhibitor development in patients with haemophilia, multidisciplinary management of many of these patients remains suboptimal and highly heterogenous across Europe. METHODS: Following a series of multidisciplinary meetings and a review of the literature, the European haemophilia community of health professionals and patients jointly defined practical optimum standards for ensuring and harmonizing treatment and care for patients with an inhibitor...
April 27, 2018: Orphanet Journal of Rare Diseases
https://www.readbyqxmd.com/read/29665204/factor-viii-products-and-inhibitor-development-in-previously-treated-patients-with-severe-or-moderately-severe-haemophilia-a-a-systematic-review
#6
Shermarke Hassan, Antonino Cannavò, Samantha Gouw, Frits R Rosendaal, Johanna van der Bom
BACKGROUND: Patients with severe haemophilia A who have been treated extensively with factor VIII (FVIII) products face a low but potentially serious risk of inhibitor development. It is unknown why these patients break immunological tolerance and data on product-related immunogenicity is scarce. AIMS: To summarize the currently available evidence on the relationship between inhibitor development and recombinant FVIII product type in previously treated patients with severe haemophilia A...
April 17, 2018: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/29652676/immune-tolerance-induction-rescue-with-turoctocog-alfa-in-a-poor-risk-haemophilia-a-inhibitor-young-child-the-history-of-a-success
#7
Ezio Zanon, Marta Milan, Samantha Pasca
: The development of alloantibodies against the replacement of Factor VIII (FVIII) is the major complication in haemophilia A treatment. The gold standard to eradicate inhibitors is the immune tolerance induction (ITI), but in some cases it fails requiring another immune tolerance, defined ITI rescue (ITI-R), using a different concentrate, even though it is still debated. We report a successful case of a poor risk (titre of inhibitor at start of ITI > 10 BU/ml, peak titre on ITI > 200 BU/ml, >2 years since the inhibitor diagnosis) haemophilia A child treated with a high-dose regimen (200 UI/kg/day) turoctocog-alfa after a failed first-line ITI with octocog-alfa lasting 29 months...
April 12, 2018: Blood Coagulation & Fibrinolysis: An International Journal in Haemostasis and Thrombosis
https://www.readbyqxmd.com/read/29626368/patient-centred-value-framework-for-haemophilia
#8
B O'Mahony, G Dolan, D Nugent, C Goodman
INTRODUCTION: Growing budgetary demands have led to increased scrutiny of healthcare spending for rare diseases, leading to a unified goal within the haemophilia community to define objectively patient-centred value in haemophilia care. AIM: To develop a patient-centred outcomes framework with global applicability for assessing value in haemophilia healthcare. METHODS: An international, multidisciplinary panel of experts convened to identify the range of patient impacts of haemophilia health care and organize these into a three-tiered, patient-centred outcomes framework based on Porter's model for assessing value...
April 6, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29611182/factor-f-viii-viia-enhances-global-haemostatic-function-in-the-co-presence-of-bypassing-agents-and-fviii-among-patients-with-haemophilia-a-with-inhibitor
#9
Keiji Nogami, Tomoko Matsumoto, Koji Yada, Kenichi Ogiwara, Shoko Furukawa, Yasuaki Shida, Masahiro Takeyama, Midori Shima
Bypassing therapy is essential for the haemostatic management of patients with haemophilia A with inhibitor (PWHA-inh), but the therapeutic effects are inconsistent. We previously reported that activated prothrombin complex concentrates (aPCC) activated factor (F)VIIIin vitro, and was mediated mainly by the activated FVII (FVIIa) contained in aPCC. We have extended those studies to assess global coagulation in whole blood from 18 PWHA-inh in the co-presence of aPCC and FVIII using Ca2+ -triggered rotational thromboelastometry...
April 2, 2018: British Journal of Haematology
https://www.readbyqxmd.com/read/29582525/clinical-experience-with-moroctocog-alfa-af-cc-in-younger-paediatric-patients-with-severe-haemophilia-a-two-open-label-studies
#10
REVIEW
L Rusen, K Kavakli, J Korth-Bradley, F Huard, P Rendo, J Fuiman, J A Baumann, L Smith, C Alvey, J Rupon
INTRODUCTION: The pharmacokinetics (PK), efficacy and safety of moroctocog alfa (AF-CC) have been demonstrated in haemophilia A patients aged ≥6 years. AIM: These studies aimed to further describe moroctocog alfa (AF-CC) experience in paediatric patients (<12 years) with severe haemophilia A (FVIII:C < 1%). METHODS: Two prospective, open-label studies enrolled patients aged <12 years: one study with 37 previously treated patients (PTPs) and another with 23 previously untreated patients (PUPs)...
March 26, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29582516/long-term-tolerability-immunogenicity-and-efficacy-of-nuwiq-%C3%A2-human-cl-rhfviii-in-children-with-severe-haemophilia-a
#11
A Klukowska, T Szczepański, V Vdovin, S Knaub, J Bichler, M Jansen, I Dzhunova, R J Liesner
INTRODUCTION: Nuwiq® (human-cl rhFVIII, simoctocog alfa) is a 4th generation recombinant human FVIII, without chemical modification or fusion with any other protein, produced in a human cell line. AIM/METHODS: This study (GENA-13) was an extension of the GENA-03 study in which previously treated children aged 2-12 years with severe haemophilia A received Nuwiq® prophylaxis for ≥6 months. GENA-13 examined long-term tolerability, immunogenicity and efficacy of Nuwiq® prophylaxis in children...
March 26, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29554701/human-dendritic-cells-synergistically-activated-by-fviii-plus-lps-induce-activation-of-autologous-cd4-t-cells
#12
Lilija Miller, Eva Ringler, Klaus Maximilian Kistner, Zoe Waibler
The most severe side effect in haemophilia A (HA) treatment is the development of anti-factor VIII antibodies, also called inhibitors. Why inhibitors develop in a proportion of treated HA patients and how this can be prevented remains largely unanswered. Among numerous theories, the presence of immunological danger signals, associated with events such as surgery or infection, has been proposed to play a role. In this study, we demonstrate that human dendritic cells (DC) synergistically activated by a combination of factor VIII (FVIII) concentrate plus the bacterial danger signal lipopolysaccharide (LPS) induce a significantly stronger activation of autologous CD4+ T cells than DC pretreated with FVIII or LPS alone...
March 19, 2018: Thrombosis and Haemostasis
https://www.readbyqxmd.com/read/29543371/immune-tolerance-induction-what-have-we-learned-over-time
#13
REVIEW
H-H Brackmann, G C White, E Berntorp, T Andersen, C Escuriola-Ettingshausen
Development of inhibitory antibodies to infused factor VIII (FVIII) concentrates continues to be the most serious complication of haemophilia A management. Induction of immune tolerance by administering high doses of FVIII concentrate (antigen) and prothrombin complex concentrates to control bleeding was originated in the 1970s in Bonn, Germany, by Dr Hans-Hermann Brackmann, and became known as the Bonn protocol. ITI transformed the life of the index patient, who was 19 years of age when he began treatment, and dramatically improved the medical landscape for all patients with haemophilia and inhibitors...
April 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29537116/in-vitro-characterization-of-mod-5014-a-novel-long-acting-carboxy-terminal-peptide-ctp-modified-activated-fvii
#14
A Bar-Ilan, T Livnat, M Hoffmann, L Binder, M Zakar, R Guy, Y Felikman, L Moschcovich, B Shenkman, D Monroe, O Hershkovitz, G Kenet, G Hart
INTRODUCTION: Recombinant FVIIa (rFVIIa) is an effective treatment for haemophilia through frequent administration. However, the short half-life of rFVIIa decreases its prophylactic ability to reduce bleeding. Carboxy-terminal peptide (CTP)-modified FVIIa (MOD-5014) is a long-acting rFVIIa developed for on-demand treatment of haemophilia using either an intravenous or subcutaneous injection with the aim of less frequent administrations, as well as for prophylactic use. AIM: The comprehensive evaluation of the activity MOD-5014 vs commercially available rhFVIIa, as well as their interaction with cofactors and inhibitors...
March 14, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29532595/factor-xa-and-viia-inhibition-by-tissue-factor-pathway-inhibitor-is-prevented-by-a-monoclonal-antibody-to-its-kunitz-1-domain
#15
C Augustsson, A Svensson, B Kjaer, T-Y Chao, X Wenjuan, B O Krogh, J Breinholt, J T Clausen, I Hilden, H H Petersen, L C Petersen
Essentials Activated FVII (FVIIa) and FX (FXa) are inhibited by tissue factor pathway inhibitor (TFPI). A monoclonal antibody, mAb2F22, was raised against the N-terminal fragment of TFPI (1-79). mAb2F22 bound exclusively to the K1 domain of TFPI (KD ∼1 nm) and not to the K2 domain. mAb2F22 interfered with inhibition of both FVIIa and FXa activities and restored clot formation. SUMMARY: Background Initiation of coagulation is induced by binding of activated factor VII (FVIIa) to tissue factor (TF) and activation of factor X (FX) in a process regulated by tissue factor pathway inhibitor (TFPI)...
May 2018: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/29517971/non-factor-replacement-therapy-for-haemophilia-a-current-update
#16
REVIEW
Massimo Franchini, Pier Mannuccio Mannucci
One of the most challenging issues facing us in the treatment of haemophilia is the development of alloantibodies against infused factor VIII (FVIII) or factor IX (FIX). Inhibitors render factor replacement therapy ineffective, exposing patients to an unacceptably high risk of morbidity and mortality. Besides the well-known bypassing agents (i.e. activated prothrombin complex concentrate and recombinant activated factor VII) used to treat or prevent bleeding in haemophilia patients with inhibitors, there is growing interest in a new class of therapeutic agents which act by enhancing coagulation (i...
February 14, 2018: Blood Transfusion, Trasfusione del Sangue
https://www.readbyqxmd.com/read/29493848/long-term-clinical-and-economic-outcomes-in-previously-untreated-paediatric-patients-with-severe-haemophilia-a-a-nationwide-real-world-study-with-700-person-years
#17
K Vepsäläinen, P Riikonen, R Lassila, M Arola, P Huttunen, P Lähteenmäki, M Möttönen, T Selander, J Martikainen
AIM: For previously untreated patients (PUPs) with severe haemophilia A in Finland for the past 2 decades, the standard practice has been to start early primary prophylaxis. We evaluated the long-term clinical outcomes and costs of treatment with high-dose prophylaxis in PUPs from birth to adolescence, including immune tolerance induction (ITI). METHODS: From the medical records of all PUPs born between June 1994 and May 2013 in Finland, we retrospectively extracted data on clinical outcomes and healthcare use...
March 1, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29474202/a-decade-long-clinical-experience-on-the-prophylactic-use-of-activated-prothrombin-complex-concentrate-in-acquired-haemophilia-a-a-case-series-from-a-tertiary-care-centre
#18
Anita Árokszállási, Katalin Rázsó, Péter Ilonczai, Zsolt Oláh, Zsuzsanna Bereczky, Zoltán Boda, Ágota Schlammadinger
: In acquired haemophilia A (AHA), risk for recurrent bleeding exists until the inhibitor is detectable. Thus, patients with persisting inhibitor may benefit from prophylaxis with activated prothrombin complex concentrate (aPCC). Potential thromboembolic complications and cost are also factors to consider. Today, no high level evidence or clear recommendations are available on aPCC prophylaxis in AHA. Recently, a small prospective study demonstrated a favourable outcome with short-term, daily administered aPCC infusion...
April 2018: Blood Coagulation & Fibrinolysis: An International Journal in Haemostasis and Thrombosis
https://www.readbyqxmd.com/read/29465806/principles-of-haemophilia-care-the-asia-pacific-perspective
#19
REVIEW
S Dunkley, J C M Lam, M J John, R S M Wong, H Tran, R Yang, S C Nair, M Shima, A Street, A Srivastava
Optimal haemophilia care is best established and implemented through a well-coordinated plan guided by clearly defined principles and priorities. A document which enunciates those details is therefore important. A successful example of this approach is the definition of principles of haemophilia care (PHC) outlined by the European Association for Haemophilia and Associated Disorders (EAHAD) and also the World Federation of Hemophilia. A similar document applicable to the Asia-Pacific region must take into account not only the highly varied healthcare systems but also the tremendous socio-economic and cultural diversities which impact provision of such care...
February 21, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29461004/effects-of-pre-analytical-heat-treatment-in-factor-viii-fviii-inhibitor-assays-on-fviii-antibody-levels
#20
B Boylan, C H Miller
INTRODUCTION: The use of pre-analytical heat treatment (PHT) with the Nijmegen-Bethesda assay (NBA) for inhibitors to factor VIII (FVIII) can remove/destroy infused or endogenous FVIII from patient plasma samples, allowing testing of recently infused patients with haemophilia. Two PHT methods have been described as follows: heating to 56°C for 30 minutes and heating to 58°C for 90 minutes. Data examining the effects of PHT on anti-FVIII IgG4 , the antibodies known to correlate most closely with the presence of FVIII inhibitors, are limited...
February 20, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
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