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https://www.readbyqxmd.com/read/29042366/emicizumab-a-bispecific-antibody-recognizing-coagulation-factors-ix-and-x-how-does-it-actually-compare-to-factor-viii
#1
Peter J Lenting, Cécile V Denis, Olivier D Christophe
During the last decade, the development of improved and novel approaches for the treatment of hemophilia A has expanded tremendously. These approaches include factor VIII (FVIII) with extended half-life (e.g. FVIII-Fc and PEGylated FVIII), monoclonal antibodies targeting tissue factor pathway inhibitor, si-RNA to reduce antithrombin expression and the bispecific antibody ACE910/Emicizumab. Emicizumab is a bispecific antibody recognizing both the enzyme factor IXa (FIXa) and the substrate factor X (FX). By simultaneously binding enzyme and substrate, Emicizumab mimics some part of the function exerted by the original cofactor FVIII, in that it promotes co-localization of the enzyme/substrate complex...
October 17, 2017: Blood
https://www.readbyqxmd.com/read/29039750/modeling-of-body-weight-metrics-for-effective-and-cost-efficient-conventional-factor-viii-dosing-in-hemophilia-a-prophylaxis
#2
Alanna McEneny-King, Pierre Chelle, Severine Henrard, Cedric Hermans, Alfonso Iorio, Andrea N Edginton
The total body weight-based dosing strategy currently used in the prophylactic treatment of hemophilia A may not be appropriate for all populations. The assumptions that guide weight-based dosing are not valid in overweight and obese populations, resulting in overdosing and ineffective resource utilization. We explored different weight metrics including lean body weight, ideal body weight, and adjusted body weight to determine an alternative dosing strategy that is both safe and resource-efficient in normal and overweight/obese adult patients...
October 17, 2017: Pharmaceutics
https://www.readbyqxmd.com/read/29038472/phage-assisted-continuous-evolution-of-proteases-with-altered-substrate-specificity
#3
Michael S Packer, Holly A Rees, David R Liu
Here we perform phage-assisted continuous evolution (PACE) of TEV protease, which canonically cleaves ENLYFQS, to cleave a very different target sequence, HPLVGHM, that is present in human IL-23. A protease emerging from ∼2500 generations of PACE contains 20 non-silent mutations, cleaves human IL-23 at the target peptide bond, and when pre-mixed with IL-23 in primary cultures of murine splenocytes inhibits IL-23-mediated immune signaling. We characterize the substrate specificity of this evolved enzyme, revealing shifted and broadened specificity changes at the six positions in which the target amino acid sequence differed...
October 16, 2017: Nature Communications
https://www.readbyqxmd.com/read/29037559/noninvasive-prenatal-diagnosis-for-x-linked-disease-by-maternal-plasma-sequencing-in-a-family-of-hemophilia-b
#4
Ping Hu, Fengchang Qiao, Yuan Yuan, Ruihong Sun, Yan Wang, Lulu Meng, Ying Lin, Hang Li, Yaoshen Wang, Rui Han, Dong Liang, Dingyuan Ma, Tao Jiang, Hui Jiang, Zhengfeng Xu
OBJECTIVE: To apply a Hidden Markov Model to test Hemophilia B in a fetus by maternal plasma sequencing only employing proband and maternal haplotypes. CASE REPORT: A family at risk for Hemophilia B was recruited in this study. We performed genetic diagnosis on the proband using our targeted capture system (containing F9 gene coding region, highly heterozygous SNPs and a 13-kb chromosome Y specific region), and revealed a causative F9 gene mutation (c.190T>C, p...
October 2017: Taiwanese Journal of Obstetrics & Gynecology
https://www.readbyqxmd.com/read/29033555/treatment-adherence-in-hemophilia
#5
REVIEW
Courtney D Thornburg, Natalie A Duncan
Prophylactic clotting-factor regimens reduce the occurrence of bleeding episodes and maintain joint health in individuals with moderate and severe hemophilia. However, these outcomes are only achieved with adherence to prescribed prophylaxis regimens. There are several types of barriers to adherence related to key patient, condition, treatment, health-care system, and/or socioeconomic variables. Notably, health-care professionals may not prescribe prophylaxis if they perceive that a patient will be nonadherent...
2017: Patient Preference and Adherence
https://www.readbyqxmd.com/read/29033549/reliability-of-patient-reported-outcome-instruments-in-us-adults-with-hemophilia-the-pain-functional-impairment-and-quality-of-life-p-fiq-study
#6
Christine L Kempton, Michael Wang, Michael Recht, Anne Neff, Amy D Shapiro, Amit Soni, Roshni Kulkarni, Tyler W Buckner, Katharine Batt, Neeraj N Iyer, David L Cooper
BACKGROUND: Hemophilia is marked by frequent joint bleeding, resulting in pain and functional impairment. OBJECTIVE: This study aimed to assess the reliability of five patient-reported outcome (PRO) instruments in people with hemophilia (PWH) in a non-bleeding state. METHODS: Adult male PWH of any severity and inhibitor status, with a history of joint pain or bleeding, completed a pain history and five PRO instruments (EQ-5D-5L, Brief Pain Inventory v2 [BPI], International Physical Activity Questionnaire [IPAQ], Short Form 36 Health Survey v2 [SF-36v2], and Hemophilia Activities List [HAL]) during their routine comprehensive care visit...
2017: Patient Preference and Adherence
https://www.readbyqxmd.com/read/29027258/acquired-factor-xi-deficiency-and-therapeutic-plasma-exchange
#7
Geoffrey D Wool, Angela Treml, Jonathan L Miller
Congenital factor XI (FXI) deficiency is associated with a variable bleeding phenotype. Recent reports have documented the use of therapeutic plasma exchange to rapidly and isovolumetrically increase FXI levels before invasive procedures in patients with congenital FXI deficiency. We report a case of acquired FXI deficiency in a pregnant woman with lupus. We proved that the inhibitor was an IgG, therefore potentially capable of crossing the placenta. While immune suppression eliminated detectable circulating inhibitor, the woman's FXI remained quite low...
October 13, 2017: Journal of Clinical Apheresis
https://www.readbyqxmd.com/read/29025913/analyses-of-the-francecoag-cohort-support-immunogenicity-differences-among-one-plasma-derived-and-two-recombinant-factor-viii-brands-in-boys-with-severe-hemophilia-a
#8
Thierry Calvez, Hervé Chambost, Roseline d'Oiron, Vincent Dalibard, Virginie Demiguel, Alexandra Doncarli, Yves Gruel, Yoann Huguenin, Patrick Lutz, Chantal Rothschild, Christine Vinciguerra, Jenny Goudemand
Around one third of boys with severe hemophilia A develop inhibitors (neutralizing antibodies) against their therapeutic factor VIII product. This adverse effect may result in more life-threatening bleeding, disability, impaired quality of life, and costly care. We compared the inhibitor incidence in boys treated with the three factor VIII products most used in France: one plasma-derived (Factane) and two recombinant products (Advate and Kogenate Bayer). A previously untreated patient cohort was created in 1994 to investigate risk factors for inhibitor development...
October 12, 2017: Haematologica
https://www.readbyqxmd.com/read/29025906/comparative-profiling-of-hla-dr-and-hla-dq-associated-factor-viii-peptides-presented-by-monocyte-derived-dendritic-cells
#9
Ivan Peyron, Robin B Hartholt, Laura Pedró-Cos, Floris van Alphen, Anja Ten Brinke, Neubury Lardy, Sander Meijer, Jan Voorberg
The development of anti-factor VIII antibodies represents a major complication in the treatment of patients with hemophilia A. Generation of high affinity anti-factor VIII antibodies is dependent on help provided by CD4+ T cells that recognize factor VIII-derived peptides presented on class II major histocompatibility complex on the surface of antigen presenting cells. In order to identify the immune-dominant epitopes that can be presented to CD4+ T cells, we previously developed a mass-spectrometry based method to identify factor VIII derived peptides that are presented on human leucocyte antigen (HLA)-DR...
October 12, 2017: Haematologica
https://www.readbyqxmd.com/read/29025717/impacting-inhibitor-development-in-hemophilia-a
#10
Barbara A Konkle
No abstract text is available yet for this article.
October 12, 2017: Blood
https://www.readbyqxmd.com/read/29019885/two-incision-laparoscopic-appendectomy-for-a-severe-hemophilia-a-child-patient-with-coagulation-factor-vii-deficiency-case-report-and-review-of-literature
#11
Jin Peng He, Jie Xiong Feng
RATIONALE: The main complication of patients with severe hemophilia is recurrent bleeding events that usually affected musculoskeletal contractures. And replacement therapy methods were continuously improved to minimize adverse impacts brought by those complications. However, only several cases reported about the appendectomy for hemophilia A. We report a case of acute appendicitis treated by two-incision laparoscopy in a boy with hemophilia A and coagulation factor VII deficiency for the first time...
October 2017: Medicine (Baltimore)
https://www.readbyqxmd.com/read/29018078/novel-approaches-to-hemophilia-therapy-successes-and-challenges
#12
Valder R Arruda, Bhavya S Doshi, Benjamin J Samelson-Jones
New therapies for hemophilia A and hemophilia B will likely continue to change clinical practice. Ranging from extended half-life to non-factor products and gene therapy, these innovative approaches have the potential to enhance the standard of care by decreasing infusion frequency to increase compliance, promoting prophylaxis, offering alternatives to inhibitor patients, and easing route of administration. Each category has intrinsic challenges that may limit the broader application of these promising therapies...
October 10, 2017: Blood
https://www.readbyqxmd.com/read/28994581/a-sulfur-doped-graphene-based-immunological-biosensing-platform-for-multianalysis-of-cancer-biomarkers
#13
Xiang Ren, Hongmin Ma, Tong Zhang, Yong Zhang, Tao Yan, Bin Du, Qin Wei
The accurate tumor marker detection at early stage can prevent people from getting cancer to a great extent. Herein, a novel tri-antibody dual-channel biosensing strategy is applied in multianalysis of carcino-embryonic antigen (CEA) and nuclear matrix protein 22 (NMP22). In this immunosensor fabrication process, graphene oxide/polyaniline nanostructures are used as matrix and mesoporous NKF-5-3 is used as labels. Two kind antigens can be obtained from the signals of neutral red and toluidine blue, respectively, which are modified on the labels...
October 10, 2017: ACS Applied Materials & Interfaces
https://www.readbyqxmd.com/read/28992766/health-related-quality-of-life-and-association-with-arthropathy-in-greek-patients-with-hemophilia
#14
Agoritsa Varaklioti, Nick Kontodimopoulos, Dimitris Niakas, Anna Kouramba, Olga Katsarou
Health-related quality of life (HRQoL) is increasingly implicated in contemporary hemophilia management. This study focuses on the assessment of HRQoL in Greek patients with hemophilia and the comparison with normative data from the general population, as well as on the extent arthropathy may affect the patients' HRQoL. One hundred and nine adult patients completed the Greek social functioning (SF-36) and Haem-A-QoL questionnaires. Arthropathy was assessed by both the World Federation of Hemophilia clinical score and Pettersson radiological score...
January 1, 2017: Clinical and Applied Thrombosis/hemostasis
https://www.readbyqxmd.com/read/28983056/catalytically-inactive-gla-domainless-factor-xa-binds-to-tfpi-and-restores-ex-vivo-coagulation-in-hemophilia-plasma
#15
Atanur Ersayin, Aline Thomas, Landry Seyve, Nicole Thielens, Mathieu Castellan, Raphaël Marlu, Benoît Polack, Marie-Claire Dagher
No abstract text is available yet for this article.
October 5, 2017: Haematologica
https://www.readbyqxmd.com/read/28979328/challenges-to-design-and-develop-of-dna-aptamers-for-protein-targets-ii-development-of-the-aptameric-affinity-ligands-specific-to-human-plasma-coagulation-factor-viii-using-sec-selex
#16
Hossein Vahidi, Nastaran Nafissi-Varcheh, Bahram Kazemi, Reza Aboofazeli, Soraya Shahhosseini, Maryam Tabarzad
Protein specific aptamers are highly applicable affinity ligands in different fields of research and clinical applications. They have been developed against various targets, in particular, bio-macromolecules such as proteins. Among human proteins, the coagulation factors are the most attractive targets for aptamer selection and their specific aptamers have valuable characteristics in therapeutic and analytical applications. In this study, a plasma derived coagulation factor VIII was considered as the protein target for DNA aptamer selection using size exclusion chromatography-SELEX...
2017: Iranian Journal of Pharmaceutical Research: IJPR
https://www.readbyqxmd.com/read/28978851/future-prospects-of-hemostatic-treatment-for-hemophilia-patients
#17
Keiji Nogami
In the treatment of hemophilia patients, factor (F) VIII or FIX product prophylaxis results in arthropathy prevention and quality of life (QOL) improvement. Serious issues concerning hemostatic treatment of hemophilia include frequent intravenous administration of products, inhibitor development, and hemostatic treatment of patients with inhibitors. To overcome these challenges, products with extended half-life were developed. Furthermore, alternative products based on new concepts of hemostatic therapy were developed...
2017: [Rinshō Ketsueki] the Japanese Journal of Clinical Hematology
https://www.readbyqxmd.com/read/28978569/marginal-zone-b-cells-are-critical-to-factor-viii-inhibitor-formation-in-mice-with-hemophilia-a
#18
Patricia E Zerra, Courtney Cox, W Hunter Baldwin, Seema R Patel, Connie M Arthur, Pete Lollar, Shannon L Meeks, Sean R Stowell
While factor VIII (FVIII) replacement therapy can be life saving for patients with hemophilia A, neutralizing alloantibodies to FVIII, known as inhibitors, develop in a significant number of patients and actively block FVIII activity, making bleeding difficult to control and prevent. Although a variety of downstream immune factors likely regulate inhibitor formation, the identification and subsequent targeting of key initiators in inhibitor development may provide an attractive approach to prevent inhibitor formation before amplification of the FVIII immune response occurs...
October 4, 2017: Blood
https://www.readbyqxmd.com/read/28971697/the-benefit-of-low-dose-prophylaxis-in-the-treatment-of-hemophilia-a-focus-on-china
#19
Runhui Wu, Koon Hung Luke
Currently full dose prophylaxis is the standard of care in the treatment of hemophilia (World Federation of Hemophilia). However, the high costs prevent the use of standard or intermediate dose prophylaxis in China and other developing countries. Low dose prophylaxis would be a viable alternative treatment. At present global research data on the use of low dose prophylaxis is limited. Areas covered: Since 2007, China has been developing low dose prophylaxis as a high priority (90 % of moderate and severe hemophilia boys suffer joint disease by age 6 - 9)...
October 3, 2017: Expert Review of Hematology
https://www.readbyqxmd.com/read/28970713/chronic-hepatitis-c-do-generics-work-as-well-as-branded-drugs
#20
REVIEW
Madhumita Premkumar, Gagandeep S Grover, Radha K Dhiman
India has a large share of the hepatitis C virus (HCV) burden of the world. Unsafe medical practices and blood transfusions are the leading modes of transmission of HCV in India. The commonest HCV genotype in India is genotype 3 followed by genotype 1. While directly acting antivirals (DAAs) agents have become available at reasonable rates in India, cost of therapy remains a major barrier for control of HCV in India. Generic DAAs have been proven to be cost-saving in prior studies. We examined data from various studies in India and elsewhere using generic DAAs, and evaluated whether they are equally efficacious as the branded drugs...
September 2017: Journal of Clinical and Experimental Hepatology
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