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Hemophilia B

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https://www.readbyqxmd.com/read/29025906/comparative-profiling-of-hla-dr-and-hla-dq-associated-factor-viii-peptides-presented-by-monocyte-derived-dendritic-cells
#1
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/29018078/novel-approaches-to-hemophilia-therapy-successes-and-challenges
#2
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/28978851/future-prospects-of-hemostatic-treatment-for-hemophilia-patients
#3
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
#4
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/28964439/economic-burden-of-illness-among-persons-with-hemophilia-b-from-hugs-vb-examining-the-association-of-severity-and-treatment-regimens-with-costs-and-annual-bleed-rates
#5
MULTICENTER STUDY
Christina X Chen, Judith R Baker, Michael B Nichol
OBJECTIVES: To determine US societal burden of illness, including direct and indirect costs and annual bleed rate (ABR), for persons with hemophilia B (HB), a rare and debilitating genetic disorder, and to examine associations of hemophilia severity and treatment regimens with costs and ABR. METHODS: From 2009 to 2014, the Hemophilia Utilization Group Studies Part Vb collected prospective data from 10 US hemophilia treatment centers. Participants with HB completed initial surveys on sociodemographic characteristics, clinical characteristics, and treatment patterns...
September 2017: Value in Health: the Journal of the International Society for Pharmacoeconomics and Outcomes Research
https://www.readbyqxmd.com/read/28956740/genetic-correction-and-hepatic-differentiation-of-hemophilia-b-specific-human-induced-pluripotent-stem-cells
#6
Qiong He, Hui-Hui Wang, Tao Cheng, Wei-Ping Yuan, Yu-Po Ma, Yong-Ping Jiang, Zhi-Hua Ren
Objective To genetically correct a disease-causing point mutation in human induced pluripotent stem cells (iPSCs) derived from a hemophilia B patient. Methods First, the disease-causing mutation was detected by sequencing the encoding area of human coagulation factor IX (F IX) gene. Genomic DNA was extracted from the iPSCs, and the primers were designed to amplify the eight exons of F IX. Next, the point mutation in those iPSCs was genetically corrected using CRISPR/Cas9 technology in the presence of a 129-nucleotide homologous repair template that contained two synonymous mutations...
September 27, 2017: Chinese Medical Sciences Journal, Chung-kuo i Hsüeh K'o Hsüeh Tsa Chih
https://www.readbyqxmd.com/read/28944952/bypassing-agent-prophylaxis-in-people-with-hemophilia-a-or-b-with-inhibitors
#7
REVIEW
Chatree Chai-Adisaksopha, Sarah J Nevitt, Mindy L Simpson, Maissaa Janbain, Barbara A Konkle
BACKGROUND: People with hemophilia A or B with inhibitors are at high risk of bleeding complications. Infusion of bypassing agents, such as recombinant activated FVII (rFVIIa) and plasma-derived activated prothrombin complex concentrate, are suggested as alternative therapies to factor VIII (haemophilia A) or IX (haemophilia B) for individuals who no longer respond to these treatments because they develop inhibitory antibodies. The ultimate goal of treatment is to preserve the individual's joints, otherwise destroyed by recurrent bleeds...
September 25, 2017: Cochrane Database of Systematic Reviews
https://www.readbyqxmd.com/read/28921441/purification-and-autoactivation-method-for-recombinant-coagulation-factor-vii
#8
Vladimir Granovski, Marcela C C Freitas, Mario Soares Abreu-Neto, Dimas T Covas
Recombinant coagulation factor VII is a very important and complex protein employed for treatment of hemophiliac patients (hemophilia A/B) who develop inhibitors antibodies to conventional treatments (FVIII and FIX). The rFVII is a glycosylated molecule and circulates in plasma as zymogen of 50 kDa. When activated the molecule is cleaved to 20-30 kDa and has a half-life of about 3 h, needing to be processed fast and efficiently until freeze-drying. Here, we describe a very simple and fast purification sequence for rFVII using affinity FVII Select resin and a dialysis system that can be easily scaled up...
2018: Methods in Molecular Biology
https://www.readbyqxmd.com/read/28920105/the-impact-of-von-willebrand-factor-on-factor-viii-memory-immune-responses
#9
Juan Chen, Jocelyn A Schroeder, Xiaofeng Luo, Qizhen Shi
Immune tolerance induction (ITI) with aggressive infusion of factor VIII (FVIII) is the current strategy used to eradicate FVIII inhibitors and restore normal FVIII pharmacokinetics in inhibitor patients. Whether the use of FVIII products containing von Willebrand factor (VWF) will affect the efficacy of ITI is still controversial. In this study, we explored the impact of VWF on FVIII memory immune responses in hemophilia A (HA) mice. A T-cell proliferation assay and cytokine profile analysis were used to study FVIII-primed CD4(+) T cells...
August 22, 2017: Blood Advances
https://www.readbyqxmd.com/read/28919830/identification-of-patients-with-congenital-hemophilia-in-a-large-electronic-health-record-database
#10
Michael Wang, Anissa Cyhaniuk, David L Cooper, Neeraj N Iyer
BACKGROUND: Electronic health records (EHRs) are an important source of information with regard to diagnosis and treatment of rare health conditions, such as congenital hemophilia, a bleeding disorder characterized by deficiency of factor VIII (FVIII) or factor IX (FIX). OBJECTIVE: To identify patients with congenital hemophilia using EHRs. DESIGN: An EHR database study. SETTING: EHRs were accessed from Humedica between January 1, 2007, and July 31, 2013...
2017: Journal of Blood Medicine
https://www.readbyqxmd.com/read/28905885/long-term-correction-of-hemophilia-a-mice-following-lentiviral-mediated-delivery-of-an-optimized-canine-factor-viii-gene
#11
J M Staber, M J Pollpeter, C-G Anderson, M Burrascano, A L Cooney, P L Sinn, D T Rutkowski, W C Raschke, P B McCray
Current therapies for hemophilia A include frequent prophylactic or on-demand intravenous factor treatments which are costly, inconvenient and may lead to inhibitor formation. Viral vector delivery of factor VIII (FVIII) cDNA has the potential to alleviate the debilitating clotting defects. Lentiviral-based vectors delivered to murine models of hemophilia A mediate phenotypic correction. However, a limitation of lentiviral-mediated FVIII delivery is inefficient transduction of target cells. Here, we engineer a feline immunodeficiency virus (FIV) -based lentiviral vector pseudotyped with the baculovirus GP64 envelope glycoprotein to mediate efficient gene transfer to mouse hepatocytes...
September 14, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28901996/intracranial-hemorrhage-in-congenital-bleeding-disorders
#12
Shadi Tabibian, Hoda Motlagh, Majid Naderi, Akbar Dorgalaleh
: Intracranial hemorrhage (ICH), as a life-threatening bleeding among all kinds of congenital bleeding disorders (CBDs), is a rare manifestation except in factor XIII (FXIII) deficiency, which is accompanied by ICH, early in life, in about one-third of patients. Most inherited platelet function disorders (IPFDs) are mild to moderate bleeding disorders that can never experience a severe bleeding as in ICH; however, Glanzmann's thrombasthenia, a common and severe inherited platelet function disorder, can lead to ICH and occasional death...
September 9, 2017: Blood Coagulation & Fibrinolysis: An International Journal in Haemostasis and Thrombosis
https://www.readbyqxmd.com/read/28895852/gene-therapy-for-hemophilia
#13
REVIEW
Amit C Nathwani, Andrew M Davidoff, Edward G D Tuddenham
The best currently available treatments for hemophilia A and B (factor VIII or factor IX deficiency, respectively) require frequent intravenous infusion of highly expensive proteins that have short half-lives. Factor levels follow a saw-tooth pattern that is seldom in the normal range and falls so low that breakthrough bleeding occurs. Most hemophiliacs worldwide do not have access to even this level of care. In stark contrast, gene therapy holds out the hope of a cure by inducing continuous endogenous expression of factor VIII or factor IX following transfer of a functional gene to replace the hemophilic patient's own defective gene...
October 2017: Hematology/oncology Clinics of North America
https://www.readbyqxmd.com/read/28895303/coronary-angiography-with-or-without-percutaneous-coronary-intervention-in-patients-with-hemophilia-systematic-review
#14
Christian Boehnel, Hans Rickli, Lukas Graf, Micha T Maeder
OBJECTIVES: We aimed to summarize the evidence for periprocedural and long-term strategies to both minimize the bleeding risk and ensure sufficient anticoagulation and antiaggregation in hemophilia patients undergoing coronary angiography with or without percutaneous coronary interventions (PCI). BACKGROUND: Hemophilia patients undergoing coronary angiography and PCI are at risk of bleeding due to deficiency of the essential clotting factors VIII or IX combined with the need of peri-interventional anticoagulation and antiaggregation and dual antiplatelet therapy (DAPT) after PCI...
September 12, 2017: Catheterization and Cardiovascular Interventions
https://www.readbyqxmd.com/read/28852852/hemophilic-pseudotumor-of-the-mandible-in-a-patient-with-hemophilia-b
#15
Probodh K Chattopadhyay, Shakil Ahmed Nagori, Rahul P Menon, T Balasundaram
INTRODUCTION: Hemophilic pseudotumor is a rare complication occurring in patients with hemophilia, frequently seen in the femur, tibia, pelvic bones, iliac bones, or rarely in the maxillofacial region. CASE REPORT: A 7-year-old male reported with a spontaneous extra-oral swelling that was managed with pre-operative transfusion of factor IX along with curettage of the lesion. Our report presents only the fourth case in literature wherein this tumor presented in a patient with hemophilia B...
August 30, 2017: Oral and Maxillofacial Surgery
https://www.readbyqxmd.com/read/28834196/advanced-cell-based-modeling-of-the-royal-disease-characterization-of-the-mutated-f9-mrna
#16
L Martorell, E Luce, J L Vazquez, Y Richaud-Patin, S Jimenez-Delgado, I Corrales, N Borras, S Casacuberta-Serra, A Weber, R Parra, C Altisent, A Follenzi, A Dubart-Kupperschmitt, A Raya, F Vidal, J Barquinero
Essentials The Royal disease (RD) is a form of hemophilia B predicted to be caused by a splicing mutation. We generated an iPSC-based model of the disease allowing mechanistic studies at the RNA level. F9 mRNA analysis in iPSC-derived hepatocyte-like cells showed the predicted abnormal splicing. Mutated F9 mRNA level was very low but we also found traces of wild type transcripts. SUMMARY: Background The royal disease is a form of hemophilia B (HB) that affected many descendants of Queen Victoria in the 19th and 20th centuries...
August 21, 2017: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/28828393/neonatal-gene-therapy-for-hemophilia-b-by-a-novel-adenovirus-vector-showing-reduced-leaky-expression-of-viral-genes
#17
Shunsuke Iizuka, Fuminori Sakurai, Masashi Tachibana, Kazuo Ohashi, Hiroyuki Mizuguchi
Gene therapy during neonatal and infant stages is a promising approach for hemophilia B, a congenital disorder caused by deficiency of blood coagulation factor IX (FIX). An adenovirus (Ad) vector has high potential for use in neonatal or infant gene therapy for hemophilia B due to its superior transduction properties; however, leaky expression of Ad genes often reduces the transduction efficiencies by Ad protein-mediated tissue damage. Here, we used a novel Ad vector, Ad-E4-122aT, which exhibits a reduction in the leaky expression of Ad genes in liver, in gene therapy studies for neonatal hemophilia B mice...
September 15, 2017: Molecular Therapy. Methods & Clinical Development
https://www.readbyqxmd.com/read/28820407/-evaluation-of-parameters-of-hemostasis-in-the-third-trimester-in-pregnant-women-with-congen%C3%A4-tal-hemorrhagic-diathesis
#18
L Rzaguliyeva, G Afandiyeva, N Malikqasımova
The aim is to study the coagulogram indices in the III trimester of pregnancy in women with hemophilia and von Willebrand disease. Twenty-seven pregnant women with hereditary coagulopathies of mild and moderate form (mean age 27.9±3.3 years) in the third trimester of pregnancy were examined. Of the examined 12 women - carriers of hemophilia A and B, 15 - with Willebrand disease (BV). Clinico-laboratory examination, clinical and biochemical blood tests were carried out. Coagulologic examination was performed on 17 parameters...
July 2017: Georgian Medical News
https://www.readbyqxmd.com/read/28819614/acute-compartment-syndrome-after-an-olecranon-fracture-in-a-patient-with-mild-hemophilia-b
#19
John M Reynolds, Christy Christophersen, Mary K Mulcahey
INTRODUCTION: Compartment syndrome is a serious condition characterized by compartmental pressures within 20 mmHg of diastolic blood pressure, or clinical signs of pain, paresthesia, pallor, and lack of pulses. Often a surgical intervention is necessary. Increased surveillance for compartment syndrome is important when a patient with a bleeding disorder sustains a traumatic injury. CASE REPORT: We present a case of forearm compartment syndrome in a patient with mild hemophilia B who sustained an olecranon fracture...
March 2017: Journal of Orthopaedic Case Reports
https://www.readbyqxmd.com/read/28802375/post-tonsillectomy-hemorrhagic-outcomes-in-children-with-bleeding-disorders-at-a-single-institution
#20
Priyesh N Patel, Alexandra M Arambula, Allison P Wheeler, Edward B Penn
OBJECTIVE: To report on the post-tonsillectomy bleeding outcomes and factors associated with hemorrhage among children with pre- or post-operatively diagnosed bleeding disorders treated with an institutional protocol. METHODS: Retrospective cohort study of patients with hematologic disorders who underwent tonsillectomy between 2003 and 2016 and were treated with perioperative desmopressin or factor replacement and/or aminocaproic acid. Postoperative outcomes were compared to controls matched for age, sex, and indication for surgery...
September 2017: International Journal of Pediatric Otorhinolaryngology
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