keyword
MENU ▼
Read by QxMD icon Read
search

Hemophilia B

keyword
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
#1
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
#2
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
#3
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
https://www.readbyqxmd.com/read/28799202/immune-tolerance-induced-by%C3%A2-platelet-targeted-factor-viii-gene-therapy-in-hemophilia-a-mice-is-cd4%C3%A2-t-cell-mediated
#4
Y Chen, X Luo, J A Schroeder, J Chen, C K Baumgartner, J Hu, Q Shi
BACKGROUND: Immune responses are a major concern in gene therapy. Our previous studies demonstrated that platelet-targeted FVIII (2bF8) gene therapy together with in vivo drug-selection of transduced cells can rescue the bleeding diathesis and induce immune tolerance in FVIII(null) mice. OBJECTIVE: To investigate whether non-selectable 2bF8 lentiviral vector (LV) for the induction of platelet-FVIII expression is sufficient to induce immune tolerance and how immune tolerance is induced after 2bF8LV gene therapy...
August 11, 2017: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/28793786/hemophilia-gene-therapy-ready-for-prime-time
#5
Thierry VandenDriessche, Marinee K L Chuah
Hemophilia A and B are congenital X-linked bleeding disorders caused by mutations in the genes encoding for the blood clotting factor VIII (FVIII) or factor IX (FIX), respectively. Since the beginning of gene therapy, hemophilia has been considered an attractive disease target that served as a trailblazer for the field at large. Different technologies have been explored to efficiently and safely deliver the therapeutic FVIII and FIX genes into the patients' cells. Currently, the most promising vectors for hemophilia gene therapy are adeno-associated viral vectors (AAV) and lentiviral vectors...
August 10, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28780772/prophylaxis-use-among-males-with-haemophilia-b-in-the-united-states
#6
M Ullman, Q C Zhang, S D Grosse, M Recht, J M Soucie
INTRODUCTION: Prophylaxis is considered the optimal treatment for persons with moderate to severe haemophilia (factor activity between 1-5% of normal and <1% of normal respectively) in countries where safe factor concentrates are available and economically feasible. Historically, prophylactic treatment has not been well studied in the haemophilia B (HB) population due to difficulties in obtaining a sufficiently large sample. AIM: This study examines the prevalence of prophylaxis use among a robust sample of persons with HB in the United States and its association with specific demographic and clinical characteristics...
August 6, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28780767/ageing-successfully-with-haemophilia-a-multidisciplinary-programme
#7
E Boccalandro, M E Mancuso, S Riva, D M Pisaniello, F Ronchetti, E Santagostino, F Peyvandi, L P Solimeno, P M Mannucci, G Pasta
INTRODUCTION: Persons with haemophilia (PWH) born before the middle 1970s have spent a substantial part of their lives without the benefits of replacement therapy, that became available on a relative large scale only during the 1970s. As a consequence, this group of PWH, although still relatively young, suffers from musculoskeletal and functional problems that are typical of old people. METHODS: We report herewith the short-term results of a project based upon a multidisciplinary training programme led by a physiotherapist and an occupational therapist, that was implemented over a period of 12 months in 40 patients with severe or moderate hemophilia A or B born before the middle 1970s and regularly followed-up at a comprehensive haemophilia treatment centre in Italy...
August 6, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28780274/microfluidic-transduction-harnesses-mass-transport-principles-to-enhance-gene-transfer-efficiency
#8
Reginald Tran, David R Myers, Gabriela Denning, Jordan E Shields, Allison M Lytle, Hommood Alrowais, Yongzhi Qiu, Yumiko Sakurai, William C Li, Oliver Brand, Joseph M Le Doux, H Trent Spencer, Christopher B Doering, Wilbur A Lam
Ex vivo gene therapy using lentiviral vectors (LVs) is a proven approach to treat and potentially cure many hematologic disorders and malignancies but remains stymied by cumbersome, cost-prohibitive, and scale-limited production processes that cannot meet the demands of current clinical protocols for widespread clinical utilization. However, limitations in LV manufacture coupled with inefficient transduction protocols requiring significant excess amounts of vector currently limit widespread implementation...
July 8, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/28772338/qualification-of-a-select-one-stage-activated-partial-thromboplastin-time-based-clotting-assay-and-two-chromogenic-assays-for-the-post-administration-monitoring-of-nonacog-beta-pegol
#9
Stefan Tiefenbacher, Rahul Bohra, Jean Amiral, Annette Bowyer, Steve Kitchen, Anne Lochu, Steffen Rosén, Mirella Ezban
BACKGROUND: Nonacog beta pegol (N9-GP) is an extended half-life, glycoPEGylated recombinant human factor IX (FIX) under development for the prophylaxis and treatment of bleeding episodes in hemophilia B patients. Considerable reagent-dependent variability has been observed when one-stage clotting assays are used to measure the recovery of recombinant FIX products, including N9-GP. OBJECTIVE: To qualify select one-stage clotting and chromogenic FIX activity assays for measuring N9-GP recovery...
August 3, 2017: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/28763308/revascularization-strategies-and-in-hospital-management-in-acute-coronary-syndromes-complicated-by-hemophilia-a-or-hemophilia-b
#10
Matthew J Reilley, Anne Blair, William H Matthai, Rolando Vega, Meghan Buckley, Phyllis A Gimotty, Patrick F Fogarty
: Among adult patients with hemophilia A and hemophilia B the emergent management of acute coronary syndromes (ACSs) is challenging, and exposure to antithrombotic agents and/or revascularization procedures may confer an enhanced risk of bleeding. We sought to identify clinical characteristics and in-hospital outcomes among ACS patients with hemophilia A/hemophilia B, compared with matched noncoagulopathic ACS controls. Case discharges from the Nationwide Inpatient Sample, Healthcare Cost and Utilization Project, Agency for Healthcare Research and Quality (1998-2011) had International Classification of Diseases, 9th Revision codes for hemophilia A/hemophilia B and ACS...
July 29, 2017: Blood Coagulation & Fibrinolysis: An International Journal in Haemostasis and Thrombosis
https://www.readbyqxmd.com/read/28752769/mutation-spectrum-and-genotype-phenotype-analyses-in-a-pakistani-cohort-with-hemophilia-b
#11
Muhammad Tariq Masood Khan, Arshi Naz, Jawad Ahmed, Tahir Shamsi, Shariq Ahmed, Nisar Ahmed, Ayisha Imran, Nazish Farooq, Muhammad Tariq Hamayun Khan, Abid Sohail Taj
This study aimed to (1) identify F9 genetic alterations in patients with hemophilia B (HB) of Pakistani origin and (2) determine the genotype-phenotype relationships in these patients. Diagnosed cases of HB were identified through registries at designated tertiary health-care centers across the country. Consenting patients were enrolled into the study. The factor IX (FIX) coagulation activity (FIX:C) and key clinical features were recorded. Direct sequencing of F9 was carried out in all patients. All the variants identified were analyzed for functional consequences employing in silico analysis tools...
January 1, 2017: Clinical and Applied Thrombosis/hemostasis
https://www.readbyqxmd.com/read/28731872/bleeding-risk-assessment-in-hemophilia-a-carriers-from-dakar-senegal
#12
Moussa Seck, Blaise F Faye, Abibatou Sall, Diariétou Sy, Sokhna A Touré, Nata Dieng, Youssou B Guéye, Macoura Gadji, Awa O Touré, Cathérine Costa, Dominique Lasne, Chantal Rothschild, Saliou Diop
: Hemophilia A carriers have an abnormal X chromosome with a molecular abnormality of FVIII gene. These carriers, long considered to be free of bleeding risk, could have the same symptoms as mild hemophiliacs. This study aim to assess bleeding risk of hemophilia A carriers monitored at the Clinical Hematology of Dakar. This is a prospective study of a period of 6 months including 22 hemophilia A carriers aged between 8 and 48 years. Hemophilia carriers were recruited using the genealogical tree of hemophiliacs followed in the service...
July 20, 2017: Blood Coagulation & Fibrinolysis: An International Journal in Haemostasis and Thrombosis
https://www.readbyqxmd.com/read/28725275/ash-meeting-2016-developments-in-hemostaseology
#13
REVIEW
Clemens Feistritzer, Birgit Mosheimer
During the annual meeting of the American Society of Hematology (ASH) in San Diego/California, novel developments in the field of hemostaseology were presented. Alternative treatment strategies besides factor replacement were discussed for patients with hemophilia. One of the highlights of the meeting in this year's plenary session was the presentation of successful adeno-associated virus mediated gene transfer in patients with hemophilia B leading to sustained elevation of factor IX:C (FIX:c). Other alternative treatment approaches in patients with hemophilia A may include bispecific antibodies mimicking factor VIIIa (FVIIIa) activity or disrupting anticoagulant proteins...
2017: Memo
https://www.readbyqxmd.com/read/28719973/an-exploratory-study-of-autonomic-function-investigations-in-hemophiliacs-on-homoeopathy-medications-using-impedance-plethysmography
#14
Tapas K Kundu, Pradip B Barde, Ghanshyam D Jindal, Farooq F Motiwala
BACKGROUND: Status of autonomic homoeostasis in hemostasic disturbances due to hemophilia needs to be studied. OBJECTIVES: To compare autonomic nervous system markers measured by heart rate variability (HRV) and blood flow variability (BFV) in hemophiliacs and healthy age-matched control population using medical analyzer system. DESIGN: Cross-sectional study. SETTINGS: Motiwala Homoeopathy Medical College, and Hemophilia Clinics, Nashik...
January 1, 2017: Journal of Evidence-based Complementary & Alternative Medicine
https://www.readbyqxmd.com/read/28716211/to-serve-and-protect-the-modulatory-role-of-von-willebrand-factor-on-factor-viii-immunogenicity
#15
REVIEW
Robin B Hartholt, Alice S van Velzen, Ivan Peyron, Anja Ten Brinke, Karin Fijnvandraat, Jan Voorberg
Hemophilia A is a bleeding disorder characterized by the absence or dysfunction of blood coagulation factor VIII (FVIII). Patients are treated with regular infusions of FVIII concentrate. In response to treatment, approximately 30% of patients with severe hemophilia A develop inhibitory antibodies targeting FVIII. Both patient and treatment related risk factors for inhibitor development have been described. Multiple studies comparing the immunogenicity of recombinant and plasma-derived FVIII have yielded conflicting results...
July 4, 2017: Blood Reviews
https://www.readbyqxmd.com/read/28691885/targeting-of-antithrombin-in-hemophilia-a-or-b-with-rnai-therapy
#16
K John Pasi, Savita Rangarajan, Pencho Georgiev, Tim Mant, Michael D Creagh, Toshko Lissitchkov, David Bevan, Steve Austin, Charles R Hay, Inga Hegemann, Rashid Kazmi, Pratima Chowdary, Liana Gercheva-Kyuchukova, Vasily Mamonov, Margarita Timofeeva, Chang-Heok Soh, Pushkal Garg, Akshay Vaishnaw, Akin Akinc, Benny Sørensen, Margaret V Ragni
Background Current hemophilia treatment involves frequent intravenous infusions of clotting factors, which is associated with variable hemostatic protection, a high treatment burden, and a risk of the development of inhibitory alloantibodies. Fitusiran, an investigational RNA interference (RNAi) therapy that targets antithrombin (encoded by SERPINC1), is in development to address these and other limitations. Methods In this phase 1 dose-escalation study, we enrolled 4 healthy volunteers and 25 participants with moderate or severe hemophilia A or B who did not have inhibitory alloantibodies...
July 10, 2017: New England Journal of Medicine
https://www.readbyqxmd.com/read/28691557/emicizumab-prophylaxis-in-hemophilia-a-with-inhibitors
#17
Johannes Oldenburg, Johnny N Mahlangu, Benjamin Kim, Christophe Schmitt, Michael U Callaghan, Guy Young, Elena Santagostino, Rebecca Kruse-Jarres, Claude Negrier, Craig Kessler, Nancy Valente, Elina Asikanius, Gallia G Levy, Jerzy Windyga, Midori Shima
Background Emicizumab (ACE910) bridges activated factor IX and factor X to restore the function of activated factor VIII, which is deficient in persons with hemophilia A. This phase 3, multicenter trial assessed once-weekly subcutaneous emicizumab prophylaxis in persons with hemophilia A with factor VIII inhibitors. Methods We enrolled participants who were 12 years of age or older. Those who had previously received episodic treatment with bypassing agents were randomly assigned in a 2:1 ratio to emicizumab prophylaxis (group A) or no prophylaxis (group B)...
July 10, 2017: New England Journal of Medicine
https://www.readbyqxmd.com/read/28688133/in-silico-evaluation-of-limited-blood-sampling-strategies-for-individualized-recombinant-factor-ix-prophylaxis-in-hemophilia-b-patients
#18
T Preijers, H C A M Hazendonk, K Fijnvandraat, F W G Leebeek, M H Cnossen, R A A Mathôt
BACKGROUND: Patients with severe hemophilia B regularly administer prophylactic intravenous doses of clotting factor IX (FIX) concentrate to maintain a trough level of at least 0.01 IUmL(-1) in order to prevent joint bleeds. Assessment of individual pharmacokinetic (PK) parameters allows individualization of the recombinant FIX (rFIX) dose. AIM: This in silico study aims to evaluate the predictive performance of limited sampling strategies (LSSs) with 1 to 3 samples to estimate individual PK parameters of rFIX...
July 8, 2017: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/28685500/rituximab-for-treating-inhibitors-in-people-with-inherited-severe-hemophilia
#19
REVIEW
Lucan Jiang, Yi Liu, Lingli Zhang, Cristina Santoro, Armando Rodriguez
BACKGROUND: Hemophilia A and B are inherited coagulation disorders characterized by a reduced or absent level of factor VIII or factor IX respectively. The severe form is characterized by a factor level less than 0.01 international units (IU) per milliliter. The development of inhibitors in hemophilia is the main complication of treatment, because the presence of these antibodies, reduces or even nullifies the efficacy of replacement therapy, making it very difficult to control the bleeding...
July 7, 2017: Cochrane Database of Systematic Reviews
https://www.readbyqxmd.com/read/28677246/factor-xi-deficient-mice-exhibit-increased-bleeding-after-injury-to-the-saphenous-vein
#20
C Ay, Y Hisada, B Cooley, N Mackman
BACKGROUND: Factor XI (FXI) is a key component of the intrinsic pathway of coagulation. It can be activated by either FXIIa or thrombin and amplifies thrombin generation during clot formation. Congenital FXI deficiency in humans (known as hemophilia C) is associated with bleeding after hemostatic challenge. However, to date there are no reports of excess bleeding in FXI deficient mice. OBJECTIVES: To determine if the absence of FXI in mice prolongs bleeding in different models...
July 4, 2017: Journal of Thrombosis and Haemostasis: JTH
keyword
keyword
6655
1
2
Fetch more papers »
Fetching more papers... Fetching...
Read by QxMD. Sign in or create an account to discover new knowledge that matter to you.
Remove bar
Read by QxMD icon Read
×

Search Tips

Use Boolean operators: AND/OR

diabetic AND foot
diabetes OR diabetic

Exclude a word using the 'minus' sign

Virchow -triad

Use Parentheses

water AND (cup OR glass)

Add an asterisk (*) at end of a word to include word stems

Neuro* will search for Neurology, Neuroscientist, Neurological, and so on

Use quotes to search for an exact phrase

"primary prevention of cancer"
(heart or cardiac or cardio*) AND arrest -"American Heart Association"