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https://www.readbyqxmd.com/read/29899890/pharmacokinetic-based-prediction-of-real-life-dosing-of-extended-half-life-clotting-factor-concentrates-on-hemophilia
#1
REVIEW
Massimo Morfini, Stefano Gherardini
The improvement of clotting factor concentrates (CFCs) has undergone an impressive boost during the last six years. Since 2010, several new recombinant factor (rF)VIII/IX concentrates entered phase I/II/III clinical trials. The improvements are related to the culture of human embryonic kidney (HEK) cells, post-translational glycosylation, PEGylation, and co-expression of the fragment crystallizable (Fc) region of immunoglobulin (Ig)G1 or albumin genes in the manufacturing procedures. The extended half-life (EHL) CFCs allow an increase of the interval between bolus administrations during prophylaxis, a very important advantage for patients with difficulties in venous access...
June 2018: Therapeutic Advances in Hematology
https://www.readbyqxmd.com/read/29895623/a-cross-sectional-analysis-of-cardiovascular-disease-in-the-hemophilia-population
#2
Suman L Sood, Dunlei Cheng, Margaret Ragni, Craig M Kessler, Doris Quon, Amy D Shapiro, Nigel S Key, Marilyn J Manco-Johnson, Adam Cuker, Christine Kempton, Tzu-Fei Wang, M Elaine Eyster, Philip Kuriakose, Annette von Drygalski, Joan Cox Gill, Allison Wheeler, Peter Kouides, Miguel A Escobar, Cindy Leissinger, Sarah Galdzicka, Marshall Corson, Crystal Watson, Barbara A Konkle
Men with hemophilia were initially thought to be protected from cardiovascular disease (CVD), but it is now clear that atherothrombotic events occur. The primary objective of the CVD in Hemophilia study was to determine the prevalence of CVD and CVD risk factors in US older men with moderate and severe hemophilia and to compare findings with those reported in age-comparable men in the Atherosclerosis Risk in Communities (ARIC) cohort. We hypothesized if lower factor levels are protective from CVD, we would see a difference in CVD rates between more severely affected and unaffected men...
June 12, 2018: Blood Advances
https://www.readbyqxmd.com/read/29895509/usefulness-of-chromogenic-assays-for-potency-assignment-and-recovery-of-plasma-derived-fviii-and-fix-concentrates-or-their-recombinant-long-acting-therapeutic-equivalents-with-potential-application-in-treated-pediatric-hemophiliac-patients
#3
REVIEW
Jean Amiral, Jerard Seghatchian
On demand and prophylaxis usage of FVIII/ FIX concentrates for the therapeutic management of hemophilia has greatly changed quality of life, and healthy life span of affected patients. Availability of recombinant therapeutic FVIII and FIX products, and of their long-acting variants, further improves the treatment constraints, and progressively permits to hemophiliacs to have an almost normal way of life. Unlimited amounts of recombinant or engineered substitutive products become available, and open new avenues for extending the benefits of prophylaxis to all hemophiliac patients, not only in economically advanced territories, but also in emerging and developing countries, worldwide...
May 16, 2018: Transfusion and Apheresis Science
https://www.readbyqxmd.com/read/29895176/inherited-bleeding-disorders-experience-of-a-not-for-profit-organization-in-pakistan
#4
Shabneez Hussain, Shahida Baloch, Azra Parvin, Akbar Najmuddin, Farhana Musheer, Mubashra Junaid, Rab Nawaz Memon, Fareeda Bhanbhro, Hayat Ullah, Bushra Moiz
Patient registry is a powerful tool for planning health care and setting groundwork for research. This survey reports a detailed registry of inherited bleeding disorders (IBD) and their management at a not-for-profit organization in a developing country to form the basis for planning development and research. We reviewed medical records of patients with IBD from 8 hemophilia treatment centers of Fatimid Foundation located in various cities. Information collected included sociodemographic data, diagnostic tests, severity of hemophilia A and B, number of bleeding episodes per year, site and frequency of hemarthrosis, and seropositivity for viral diseases...
January 1, 2018: Clinical and Applied Thrombosis/hemostasis
https://www.readbyqxmd.com/read/29890905/optimized-adeno-associated-virus-mediated-human-factor-viii-gene-therapy-in-cynomolgus-macaques
#5
Jenny Greig, Jayme Nordin, John White, Qiang Wang, Erin Bote, Tamara Goode, Roberto Calcedo, Samuel Wadsworth, Lili Wang, James M Wilson
Hemophilia A is a common hereditary bleeding disorder that is characterized by a deficiency of human blood coagulation factor VIII (hFVIII). Previous studies with adeno-associated viral (AAV) vectors identified two liver-specific promoter and enhancer combinations (E03.TTR and E12.A1AT) that drove high level expression of a codon-optimized, B-domain-deleted hFVIII transgene in a mouse model of the disease. Here, we further evaluated these enhancer/promoter combinations in cynomolgus macaques using two different AAV capsids (AAVrh10 and AAVhu37)...
June 11, 2018: Human Gene Therapy
https://www.readbyqxmd.com/read/29889317/the-socio-economic-burden-of-patients-affected-by-hemophilia-with-inhibitors
#6
REVIEW
L S D'Angiolella, P A Cortesi, A Rocino, A Coppola, H J Hassan, A Giampaolo, P Solimeno, A Lafranconi, M Micale, S Mangano, G Crotti, F Pagliarin, G Cesana, L G Mantovani
Hemophilia is associated with a high financial burden on individuals, healthcare systems and society. The development of inhibitors significantly increases the socio-economic burden of the diseases. This study aimed to review and describe the burden of Hemophilia with inhibitors, providing a reference scenario to assess the impact of new products in the real word. Two systematic literature reviews were performed to collect data on 1) health economics and 2) health-related-quality-of-life evidences in hemophilic patients with inhibitors...
June 11, 2018: European Journal of Haematology
https://www.readbyqxmd.com/read/29888865/in-depth-comparison-of-n-glycosylation-of-human-plasma-derived-factor-viii-and-different-recombinant-products-from-structure-to-clinical-implications
#7
K Canis, J Anzengruber, E Garenaux, M Feichtinger, K Benamara, F Scheiflinger, L-A Savoy, B M Reipert, M Malisauskas
BACKGROUND/OBJECTIVE: Human factor VIII (FVIII) is a plasma glycoprotein, defects of which result in hemophilia A. Current substitution therapy uses FVIII products purified from human plasma or from various cell lines (recombinant FVIII) with different levels of B-domain deletion. Glycosylation is a post-translational protein modification in FVIII that has a substantial influence on its physical, functional, and antigenic properties. Variation in glycosylation is likely the reason that FVIII products differ in their pharmacokinetics, pharmacodynamics, and immunogenicity...
June 11, 2018: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/29888855/in-vitro-studies-show-synergistic-effects-of-a-procoagulant-bispecific-antibody-and-bypassing-agents
#8
R Hartmann, T Feenstra, L Valentino, M Dockal, F Scheiflinger
BACKGROUND: Investigational non-factor products such as emicizumab offer a treatment option for patients with hemophilia and inhibitors. However, their mechanism of action raises questions regarding safety when combined with treatments for breakthrough bleeding. OBJECTIVES: Evaluate in vitro thrombin generation (TG) and clot formation for combinations of activated prothrombin complex concentrate (aPCC), recombinant activated clotting factor VII (rFVIIa), and a sequence-identical analog of emicizumab (SIA)...
June 11, 2018: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/29886751/prophylactic-versus-on-demand-treatments-for-hemophilia-advantages-and-drawbacks
#9
Giancarlo Castaman, Silvia Linari
Early long-term prophylaxis is the standard of care to prevent joint bleeding and chronic arthropathy in patients with severe hemophilia. Areas covered: Despite the obvious prophylaxis advantages upon the clinical outcomes, there are still several drawbacks to be addressed for the optimal patients' compliance. Frequency of treatment due to short half-life of conventional FVIII and FIX concentrates, difficult venous access, adherence to the prescribed therapy and costs may represent significant critical issues...
June 9, 2018: Expert Review of Hematology
https://www.readbyqxmd.com/read/29884997/shifting-landscape-of-hemophilia-therapy-implications-for-current-clinical-laboratory-coagulation-assays
#10
REVIEW
Hanny Al-Samkari, Stacy E Croteau
Clinical coagulation assays are an integral part of diagnosing and managing patients with hemophilia; however, in this new era of bioengineered factor products and non-factor therapeutics, problems have arisen with use of traditional coagulation tests for the quantification of several of these new products. Discussion over the use of one-stage clotting assays versus chromogenic substrate assays for clinical decision making and potency labeling has been ongoing for many years. Emerging factor concentrates have heightened concern over assay selection and availability...
June 8, 2018: American Journal of Hematology
https://www.readbyqxmd.com/read/29881193/risk-factors-for-postoperative-complications-of-orthopedic-surgery-in-patients-with-hemophilia-second-report
#11
Jun Hirose, Hideyuki Takedani, Masanori Nojima, Tomohiko Koibuchi
This study was conducted to investigate the incidence in patients with hemophilia of postoperative complications and risk factors for these complications. Overall, 12 (6.5%) patients developed a postoperative infection. There were 6 (3.4%) postoperative surgical site infections. The presence of an inhibitor was the only risk factor for surgical site infection. Risk factors for delayed wound healing were older age, higher preoperative serum albumin level and procedures other than joint replacement or arthroscopy...
June 2018: Journal of Orthopaedics
https://www.readbyqxmd.com/read/29879771/perioperative-management-of-patients-with-hemophilia-during-spinal-surgery
#12
Kazuyoshi Kobayashi, Shiro Imagama, Kei Ando, Kenyu Ito, Mikito Tsushima, Masayoshi Morozumi, Satoshi Tanaka, Masaaki Machino, Kyotaro Ota, Yoshihiro Nishida, Naoki Ishiguro
STUDY DESIGN: Single-center retrospective study. PURPOSE: To optimize the perioperative management of patients with hemophilia who are undergoing spinal surgery. OVERVIEW OF LITERATURE: Hemophilia is a rare disease in which there is a tendency of bleeding because of a congenital deficiency in blood coagulation factor activity. There has been no previous report on spinal surgery in patients with hemophilia. METHODS: The subjects were five patients (all males) with hemophilia who underwent spinal surgery at Nagoya University Hospital...
June 2018: Asian Spine Journal
https://www.readbyqxmd.com/read/29879570/risk-factors-for-inhibitor-development-in-severe-hemophilia-a
#13
REVIEW
Isabella Garagiola, Roberta Palla, Flora Peyvandi
Although significant advances in hemophilia treatment have improved patient outcomes and quality of life, one of the greatest complications in severe hemophilia A is the development of anti-Factor VIII (FVIII) antibodies that inhibit FVIII activity in almost 30% of previously untreated patients (PUPs). Inhibitors make very difficult the management of patients and increase their morbidity and mortality reducing drastically their quality of life. Numerous studies have investigated the mechanisms leading to the development of FVIII inhibitors...
May 25, 2018: Thrombosis Research
https://www.readbyqxmd.com/read/29879294/human-platelets-express-endothelial-protein-c-receptor-which-can-be-utilized-to-enhance-localization-of-factor-viia-activity
#14
A M Fager, K R Machlus, M Ezban, M Hoffman
High-dose factor VIIa (FVIIa) is routinely used as an effective bypassing agent to treat hemophilia patients with inhibitory antibodies that compromise factor replacement. However, the mechanism by which FVIIa binds activated platelets to promote hemostasis is not fully understood. FVIIa-DVQ is an analog of FVIIa with enhanced tissue factor (TF)-independent activity and hemostatic efficacy relative to FVIIa. Our previous studies have shown that FVIIa-DVQ exhibits greater platelet binding, thereby suggesting that features in addition to lipid composition contribute to platelet-FVIIa interactions OBJECTIVES: Endothelial cell protein C receptor (EPCR) also functions as a receptor for FVIIa on endothelial cells...
June 7, 2018: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/29877292/development-of-an-fviii-inhibitor-in-a-mild-hemophilia-patient-with-a-phe595cys-mutation
#15
Jun Yamanouchi, Daiki Tokumoto, Yuichi Ikeda, Masaki Maruta, Masahiko Kaneko, Takaaki Hato, Masaki Yasukawa
Mild hemophilia A is caused by a missense mutation in the FVIII gene that is responsible for a decrease in the FVIII:C to between 5% and 40%. The development of FVIII inhibitors has been reported in 3%-13% of patients with mild hemophilia. Genetic risk factors for the development of inhibitors in mild hemophilia have been investigated. In the present study, we encountered a case of mild hemophilia with an FVIII inhibitor and identified the mutation responsible: a novel Phe595Cys mutation in the FVIII gene. In addition, this study showed that the inhibitor recognized exogenous wild-type FVIII and autologous mutant FVIII...
June 6, 2018: Internal Medicine
https://www.readbyqxmd.com/read/29877044/modified-clot-waveform-analysis-to-measure-plasma-coagulation-potential-in-the-presence-of-the-anti-factor-ixa-factor-x-bispecific-antibody-emicizumab-comment
#16
Takeshi Matsumoto, Hideo Wada, Hidemi Toyoda, Masahiro Hirayama, Yoshiki Yamashita, Naoyuki Katayama
A patient with hemophilia A had severe coagulation factor VIII (FVIII) deficiency and required injection with FVIII concentrates two or three times a week. Although the extended-half-life (EHL) [1] FVIII products and emicizumab [2, 3], a bispecific antibody for FX and FIX can reduce the injection frequency for FVIII products, monitoring the FVIII activity is difficult using the activated partial thromboplastin time (APTT) assay. The development of an APTT waveform including first and second derivatives can provide new information on hemostatic abnormalities [4]...
June 7, 2018: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/29875973/-sickle-cell-trait-and-haemophilia-a-rare-association
#17
Hayat El Maataoui, Amina Fahi, Bouchra Oukkache
In this paper we analyze the combination of HbAS disease and haemophilia A must be exceedingly rare. Because of this rarity we report the case of two brothers with sickle cell trait and major haemophilia A. We conclude that it is about a post-circumcision bleeding due to major hemophilia A associated to sickle cell AS, this association was a systematic discovery.
2018: Pan African Medical Journal
https://www.readbyqxmd.com/read/29871018/management-of-hcv-related-liver-disease-in-hemophilia-and-thalassemia
#18
Maria Grazia Rumi, Vito Di Marco, Massimo Colombo
Chronic infection with the hepatitis C virus (HCV) has long been the dominant complication of substitution therapy in patients with inherited blood disorders and the cause of anticipated death due to end-stage liver disease. In hemophilia, transmission of HCV with clotting factors concentrates started to be curbed in the mid-1980s following the adoption of procedures of virus inactivation of concentrates based on heat, whereas in the 1990s treatment of HCV infection with interferon monotherapy was attempted, however, with little success...
May 2018: Seminars in Liver Disease
https://www.readbyqxmd.com/read/29869648/recombinant-factor-viii-past-present-and-future-of-treatment-of-hemophilia-a
#19
S Raso, C Hermans
The development of recombinant factor VIII (rFVIII) was initially driven by the necessity to treat hemophilia A (HA) patients with FVIII concentrates without the risk of transmitting infectious agents. Over the last three decades the safety of rFVIII has been further improved by completely removing animal or human proteins from the manufacturing process, so that patients would not be exposed to known or emerging pathogens. Recent efforts have concentrated on improving the expression of rFVIII, reducing its immunogenicity and enhancing its pharmacokinetic (PK) behavior...
April 2018: Drugs of Today
https://www.readbyqxmd.com/read/29866493/the-evolving-understanding-of-factor-viii-binding-sites-and-implications-for-the-treatment-of-hemophilia-a
#20
REVIEW
Gary E Gilbert
Hemophilia A is caused by decreased or dysfunctional blood coagulation factor VIII (FVIII). Recent developments in the understanding of FVIII biology, in particular the nature of FVIII binding sites on platelets, may provide new insight into the limitations of current assays. Recent data suggest that the phospholipid vesicles, which represent nonphysiologic membranes of high phosphatidylserine (PS) content, poorly reflect functional FVIII binding sites critical to coagulation. This narrative review describes the function of FVIII in clotting and discusses our evolving understanding of FVIII binding sites and their clinical implications...
May 24, 2018: Blood Reviews
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