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haemophilia 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
Zaina T Al-Salama, Lesley J Scott
The article Lonoctocog Alfa: A Review in Haemophilia A, written by Zaina T. Al-Salama and Lesley J. Scott, was originally published Online First without open access. After publication in volume 77, issue 15, pages 1677-1686 CSL Behring GmbH requested that the article be Open Choice to make the article an open access publication. Post-publication open access was funded by CSL Behring GmbH. Further details may be found at . The article is forthwith distributed under the terms of the Creative Commons Attribution-NonCommercial 4...
March 3, 2018: Drugs
C H Miller
Inhibitors are antibodies directed against haemophilia treatment products which interfere with their function. Factor VIII (FVIII) inhibitors in haemophilia A and factor IX (FIX) inhibitors in haemophilia B are significant clinically when they require a change in a patient's treatment regimen. Their persistence may increase morbidity and mortality. Multiple laboratory tests are now available for detecting and understanding inhibitors in haemophilia. Inhibitors are traditionally measured by their interference in clotting or chromogenic factor assays...
February 15, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
M Carcao, A Shapiro, J M Staber, N Hwang, C Druzgal, K Lieuw, M Belletrutti, C D Thornburg, S P Ahuja, J Morales-Arias, J Dumont, G Miyasato, E Tsao, N Jain, S W Pipe
INTRODUCTION: Immune tolerance induction (ITI) is the gold standard for eradication of factor VIII inhibitors in severe haemophilia A; however, it usually requires treatment for extended periods with associated high burden on patients and healthcare resources. AIM: Review outcomes of ITI with recombinant factor VIII Fc fusion protein (rFVIIIFc) in patients with severe haemophilia A and high-titre inhibitors. METHODS: Multicentre retrospective chart review of severe haemophilia A patients treated with rFVIIIFc for ITI...
February 13, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
C Wang, G Young
INTRODUCTION: Although clinical trials have demonstrated extended half-life (EHL) VIII and IX fusion proteins to be safe and efficacious in patients with haemophilia A and B, studies on real-world clinical application have not been performed. AIM: To retrospectively examine the real-world experience of rFVIII Fc and rFIX Fc in patients. METHODS: A retrospective review of existing medical records of patients with haemophilia A or haemophilia B who had been prescribed rFVIII Fc or rFIX Fc was conducted from the Children's Hospital Los Angeles Haemostasis and Thrombosis Centre database...
February 5, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
Bartholomew J Tortella, José Alvir, Margaret McDonald, Dean Spurden, Patrick F Fogarty, Amit Chhabra, Andreas M Pleil
BACKGROUND: Hemophilia B requires replacement therapy with factor IX (FIX) coagulation products to treat and prevent bleeding episodes. A recently introduced extended half-life (EHL) recombinant FIX replacement product provided the opportunity to compare the amount of dispensed factor and expenditures for EHL treatment compared with a standard half-life (SHL) product. OBJECTIVE: To determine factor international units (IUs) dispensed and expenditures associated with switching from nonacog alfa, the most commonly used SHL replacement product, to eftrenonacog alfa, an EHL FIX replacement product...
January 24, 2018: Journal of Managed Care & Specialty Pharmacy
A L Dunn, S P Ahuja, E S Mullins
INTRODUCTION: Prophylaxis with extended half-life factor VIII (FVIII) is approved for haemophilia A, but data regarding routine clinical use are limited. AIM: To assess real-world experience of ADYNOVATE® (Antihemophilic Factor (Recombinant), PEGylated prophylaxis in children and adults with haemophilia A. METHODS: A retrospective chart review was conducted in three US haemophilia treatment centres. Records of all patients who began Adynovate prophylaxis in routine clinical practice were identified...
January 23, 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
Stéphanie Roullet, Emmanuel de Maistre, Brigitte Ickx, Normand Blais, Sophie Susen, David Faraoni, Delphine Garrigue, Fanny Bonhomme, Anne Godier, Dominique Lasne
PURPOSE: Viscoelastic tests (VETs), thromboelastography (TEG®) and thromboelastometry (ROTEM®) are global tests of coagulation performed on whole blood. They evaluate the mechanical strength of a clot as it builds and develops after coagulation itself. The time required to obtain haemostasis results remains a major problem for clinicians dealing with bleeding, although some teams have developed a rapid laboratory response strategy. Indeed, the value of rapid point-of-care diagnostic devices such as VETs has increased over the years...
January 17, 2018: Anaesthesia, Critical Care & Pain Medicine
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
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
C L Venema, R E G Schutgens, K Fischer
Introduction Non-severe haemophilia A is characterized by coagulation Factor VIII activity (FVIII:C) levels of 1 to 40 IU/dL. It has been reported that strenuous exercise increases the plasma FVIII:C in haemophilia A patients. This review highlights current knowledge about the pathophysiological mechanisms of endogenous FVIII release following strenuous exercise. Methods A literature search was performed to include relevant studies with data on pathophysiological mechanisms of FVIII release following strenuous exercise in haemophilia...
December 2017: Thrombosis and Haemostasis
R Kulkarni
Advances in technology such as telemedicine (TM) have made access to cost-effective, quality health care feasible for remote patients. TM is especially well suited for patients with chronic disorders such as haemophilia and related haemostatic disorders that benefit not only from more frequent interaction with care providers at a specialized haemophilia treatment center but also from consultations with other specialists. Telehealth refers to a broader application of TM and includes non-clinical services such as education, provider training, administrative meetings etc...
January 2018: Haemophilia: the Official Journal of the World Federation of Hemophilia
J Staber, S E Croteau, J Davis, E F Grabowski, P Kouides, R F Sidonio
Although hemophilia B affects 1 in 25,000 males there may be 3 female hemophilia B carriers per affected male. This clinical review highlights the unique challenges faced by hemophilia B carriers including the under-recognition of bleeding symptoms associated with and without FIX deficiency, discrepancies in correlation between genotype and bleeding phenotype and therapeutic considerations utilizing clinical vignettes of common scenarios.
November 27, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
M A Timmer, S C Gouw, B M Feldman, A Zwagemaker, P de Kleijn, M F Pisters, R E G Schutgens, V Blanchette, A Srivastava, J A David, K Fischer, J van der Net
INTRODUCTION: Monitoring clinical outcome in persons with haemophilia (PWH) is essential in order to provide optimal treatment for individual patients and compare effectiveness of treatment strategies. Experience with measurement of activities and participation in haemophilia is limited and consensus on preferred tools is lacking. AIM: The aim of this study was to give a comprehensive overview of the measurement properties of a selection of commonly used tools developed to assess activities and participation in PWH...
November 27, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
Neha Bhatnagar, Georgina W Hall
In this review, we outline the standard of care for children in the UK with the most common major bleeding disorder, haemophilia, and how exciting new developments in therapy have the potential for further improvements in quality of life and clinical outcome. The combination of comprehensive specialist medical care, safer factor concentrates, earlier introduction of prophylaxis and patient-specific education has allowed the current generation of patients with haemophilia to grow into adulthood with excellent joint function, pursuing full-time employment with a good quality of life...
November 25, 2017: Archives of Disease in Childhood
E Carlos Rodriguez-Merchan
INTRODUCTION: Intra-articular (IA) corticosteroid (CS) injections are commonly used in the treatment of osteoarthritis. However, they are rarely utilized in haemophilic arthropathy. In fact, the efficacy of this method in haemophilic arthropathy is frequently discussed and debated in clinical practice. AIM: To investigate the effectiveness of IA CS injections in patients with painful haemophilic arthropathy. METHODS: A review of the literature on the topic was performed...
February 2018: Hospital Practice (Minneapolis)
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
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...
December 2017: Drugs
Emma Fosbury, Anja Drebes, Anne Riddell, Pratima Chowdary
Acquired haemophilia A (AHA) is a rare, serious bleeding disorder most often encountered in elderly patients. The mainstay of haemostatic management is with bypassing agents (BPAs) including recombinant activated factor VII (rFVIIa) and activated prothrombin complex concentrates (aPCCs). Their major limitation is incomplete efficacy, potential risk for thrombosis and the lack of routine laboratory assays for monitoring treatment response. Plasma-derived porcine FVIII (pd-pFVIII, Hyate C(®)), first used in the 1950s for the management of congenital haemophilia, has sufficient sequence homology to be haemostatic in humans, but the lack of complete homology facilitates efficacy even in the presence of human allo- and autoantibodies against human FVIII (hFVIII)...
September 2017: Therapeutic Advances in Hematology
M Napolitano, S Siragusa, S Mancuso, C M Kessler
AIM: There is a paucity of data on the clinical presentation and management of cancer patients with acquired haemophilia (AH), we here report a systematic literature review on acquired haemophilia in the context of cancer. METHODS: Treatment outcomes of AH were defined as complete response (CR), partial response (PR) or no response (NR), based on inhibitor eradication, coagulation factor VIII levels and bleeding control. Reported deaths were either related to cancer or bleeding...
September 27, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
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