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https://www.readbyqxmd.com/read/29232720/inhibitors-in-hemophilia-treatment-challenges-and-novel-options
#1
Assaf Arie Barg, Tami Livnat, Gili Kenet
No abstract text is available yet for this article.
December 12, 2017: Seminars in Thrombosis and Hemostasis
https://www.readbyqxmd.com/read/29226208/brucella-arthritis-following-total-knee-arthroplasty-in-a-patient-with-hemophilia-a-case-report
#2
Seyed Mohammad J Mortazavi, Mohammad R Sobhan, Hamed Mazoochy
Total knee arthroplasty (TKA) is a rewarding procedure in patients with hemophilia and end stage knee hemophilic arthropathy. However, this procedure might be associated with complications such as infection. There periprosthetic joint infection in patients with hemophilia is very well known, though we are not aware of any previous report on Brucella infection in this group of patients. Here, we reported a 28-year old man with Brucella infection of total knee replacement who initially underwent a conservative treatment followed bya two-stage revision...
September 2017: Archives of Bone and Joint Surgery
https://www.readbyqxmd.com/read/29225598/innovative-approaches-for-immune-tolerance-to-factor-viii-in-the-treatment-of-hemophilia-a
#3
REVIEW
Alexandra Sherman, Moanaro Biswas, Roland W Herzog
Hemophilia A (coagulation factor VIII deficiency) is a debilitating genetic disorder that is primarily treated with intravenous replacement therapy. Despite a variety of factor VIII protein formulations available, the risk of developing anti-dug antibodies ("inhibitors") remains. Overall, 20-30% of patients with severe disease develop inhibitors. Current clinical immune tolerance induction protocols to eliminate inhibitors are not effective in all patients, and there are no prophylactic protocols to prevent the immune response...
2017: Frontiers in Immunology
https://www.readbyqxmd.com/read/29224506/aav5-factor-viii-gene-transfer-in-severe-hemophilia-a
#4
Savita Rangarajan, Liron Walsh, Will Lester, David Perry, Bella Madan, Michael Laffan, Hua Yu, Christian Vettermann, Glenn F Pierce, Wing Y Wong, K John Pasi
Background Patients with hemophilia A rely on exogenous factor VIII to prevent bleeding in joints, soft tissue, and the central nervous system. Although successful gene transfer has been reported in patients with hemophilia B, the large size of the factor VIII coding region has precluded improved outcomes with gene therapy in patients with hemophilia A. Methods We infused a single intravenous dose of a codon-optimized adeno-associated virus serotype 5 (AAV5) vector encoding a B-domain-deleted human factor VIII (AAV5-hFVIII-SQ) in nine men with severe hemophilia A...
December 9, 2017: New England Journal of Medicine
https://www.readbyqxmd.com/read/29224412/a-cure-for-hemophilia-within-reach
#5
H Marijke van den Berg
No abstract text is available yet for this article.
December 9, 2017: New England Journal of Medicine
https://www.readbyqxmd.com/read/29222310/novel-alternate-hemostatic-agents-for-patients-with-inhibitors-beyond-bypass-therapy
#6
REVIEW
Margaret V Ragni
Inhibitor formation is among the most severe complications of hemophilia treatment. With a cumulative incidence of ∼30% in those with severe hemophilia A and ∼3% in those with severe hemophilia B, inhibitors are caused by a T-cell response directed against infused coagulation factor; these inhibitors neutralize factor VIII or IX activity and disrupt normal hemostasis. Inhibitor patients become unresponsive to standard factor treatment and, as an alternative, use bypass treatment (eg, recombinant factor VIIa or factor VIII inhibitor bypass activity)...
December 8, 2017: Hematology—the Education Program of the American Society of Hematology
https://www.readbyqxmd.com/read/29222309/using-pharmacokinetics-to-individualize-hemophilia-therapy
#7
REVIEW
Alfonso Iorio
Prevention and treatment of bleeding in hemophilia requires that plasma clotting factor activity of the replaced factor exceeds a defined target level. Most clinical decisions in hemophilia are based on implicit or explicit application of pharmacokinetic measures. The large interindividual variability in pharmacokinetics of factor concentrates suggests that relying on the average pharmacokinetic characteristics of factor concentrates would not allow optimizing the treatment of individual patients; for example, adjusting the frequency of infusions and targeting a specific clotting factor activity level on a case-by-case basis...
December 8, 2017: Hematology—the Education Program of the American Society of Hematology
https://www.readbyqxmd.com/read/29222308/hemophilia-gene-therapy-comes-of-age
#8
REVIEW
Lindsey A George
Concurrent with the development of recombinant factor replacement products, the characterization of the F9 and F8 genes over 3 decades ago allowed for the development of recombinant factor products and made the hemophilias a target disease for gene transfer. The progress of hemophilia gene therapy has been announced in 3 American Society of Hematology scientific plenary sessions, including the first "cure" in a large animal model of hemophilia B in 1998, first in human sustained vector-derived factor IX activity in 2011, and our clinical trial results reporting sustained vector-derived factor IX activity well into the mild or normal range in 2016...
December 8, 2017: Hematology—the Education Program of the American Society of Hematology
https://www.readbyqxmd.com/read/29220854/inhibitors-in-hemophilia-a-a-pharmacoeconomic-perspective
#9
Andrea Messori
No abstract text is available yet for this article.
December 8, 2017: Seminars in Thrombosis and Hemostasis
https://www.readbyqxmd.com/read/29218759/impact-of-pain-and-functional-impairment-in-us-adults-with-haemophilia-patient-reported-outcomes-and-musculoskeletal-evaluation-in-the-pain-functional-impairment-and-quality-of-life-p-fiq-study
#10
C L Kempton, M Recht, A Neff, M Wang, T W Buckner, A Soni, D Quon, M Witkop, L Boggio, R Z Gut, D L Cooper
INTRODUCTION: Standardized and disease-specific patient-reported outcome (PRO) instruments assessing pain, functional impairment and health-related quality of life (HRQoL) in people with haemophilia (PWH) have been used in studies, but infrequently in comprehensive care settings for individual assessment or treatment planning. AIM: To assess the impact of pain and functional impairment on HRQoL in PWH. METHODS: P-FiQ enrolled 381 adult PWH with a history of joint pain/bleeding and included 5 PROs and a clinical joint evaluation (Hemophilia Joint Health Score v2...
December 8, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/29215814/safety-and-efficacy-of-b-domain-deleted-third-generation-recombinant-factor-viii-greengene-f%C3%A2-in-korean-patients-with-hemophilia-a-data-from-a-post-marketing-surveillance-study
#11
Soon Ki Kim, Ki Young Yoo, Kun Soo Lee, Taiju Hwang, Yong Mook Choi, Eun Jin Choi, Sang Kyu Park
BACKGROUND: New B-domain deleted third generation recombinant factor VIII (FVIII; GreenGene F™, beroctocog alfa) was launched in 2010. We determined safety and efficacy of GreenGene F™ during routine clinical practice in patients with hemophilia A over a period of 12 months. METHODS: From July 2010 to July 2014, a total of 136 hemophilia A patients were enrolled in a post-marketing surveillance (PMS) study. Among them, 134 patients were assessed for drug safety and 114 patients were analyzed for drug efficacy...
January 1, 2018: Journal of Korean Medical Science
https://www.readbyqxmd.com/read/29214439/a-pharmacometric-approach-to-substitute-for-a-conventional-dose-finding-study-in-rare-diseases-example-of-phase-iii-dose-selection-for-emicizumab-in-hemophilia-a
#12
Koichiro Yoneyama, Christophe Schmitt, Naoki Kotani, Gallia G Levy, Ryu Kasai, Satofumi Iida, Midori Shima, Takehiko Kawanishi
BACKGROUND: Emicizumab (ACE910) is a bispecific antibody mimicking the cofactor function of activated coagulation factor VIII. In phase I-I/II studies, emicizumab reduced the bleeding frequency in patients with severe hemophilia A, regardless of the presence of factor VIII inhibitors, at once-weekly subcutaneous doses of 0.3, 1, and 3 mg/kg. METHODS: Using the phase I-I/II study data, population pharmacokinetic and repeated time-to-event (RTTE) modeling were performed to quantitatively characterize the relationship between the pharmacokinetics of emicizumab and reduction in bleeding frequency...
December 6, 2017: Clinical Pharmacokinetics
https://www.readbyqxmd.com/read/29211678/hemophilia-b-gene-therapy-with-a-high-specific-activity-factor-ix-variant
#13
Lindsey A George, Spencer K Sullivan, Adam Giermasz, John E J Rasko, Benjamin J Samelson-Jones, Jonathan Ducore, Adam Cuker, Lisa M Sullivan, Suvankar Majumdar, Jerome Teitel, Catherine E McGuinn, Margaret V Ragni, Alvin Y Luk, Daniel Hui, J Fraser Wright, Yifeng Chen, Yun Liu, Katie Wachtel, Angela Winters, Stefan Tiefenbacher, Valder R Arruda, Johannes C M van der Loo, Olga Zelenaia, Daniel Takefman, Marcus E Carr, Linda B Couto, Xavier M Anguela, Katherine A High
BACKGROUND: The prevention of bleeding with adequately sustained levels of clotting factor, after a single therapeutic intervention and without the need for further medical intervention, represents an important goal in the treatment of hemophilia. METHODS: We infused a single-stranded adeno-associated viral (AAV) vector consisting of a bioengineered capsid, liver-specific promoter and factor IX Padua (factor IX-R338L) transgene at a dose of 5×1011 vector genomes per kilogram of body weight in 10 men with hemophilia B who had factor IX coagulant activity of 2% or less of the normal value...
December 7, 2017: New England Journal of Medicine
https://www.readbyqxmd.com/read/29211662/closing-in-on-treatment-for-hemophilia-b
#14
EDITORIAL
Matthew Porteus
New England Journal of Medicine, Volume 377, Issue 23, Page 2274-2275, December 2017.
December 7, 2017: New England Journal of Medicine
https://www.readbyqxmd.com/read/29204261/recombinant-human-factor-viia-rfviia-in-hemophilia-mode-of-action-and-evidence-to-date
#15
REVIEW
Muriel Giansily-Blaizot, Jean-François Schved
Recombinant activated factor VII (rFVIIa) is a bypassing agent widely used both in the treatment and prevention of hemorrhagic complications due to hemophilia with inhibitor. In such cases, antihemophilic factors cannot be used. The normal physiology of factor VII/ factor VIIa (FVII/FVIIa) in the hemostatic process requires the presence of tissue factor (TF) that links to FVII leading to a FVIIa-TF complex which activates both factor X and factor IX. The therapeutic use of rFVIIa requires high amount of FVIIa...
December 2017: Therapeutic Advances in Hematology
https://www.readbyqxmd.com/read/29197156/identification-of-aggregates-in-therapeutic-formulations-of-recombinant-full-length-factor-viii-products-by-sedimentation-velocity-analytical-ultracentrifugation
#16
J F Healey, E T Parker, P Lollar
BACKGROUND: The development of inhibitory anti-factor VIII (fVIII) antibodies is the most serious complication in the management of patients with hemophilia A. Studies have suggested that recombinant full-length fVIII is more immunogenic than plasma-derived fVIII and that among recombinant fVIII products, Kogenate is more immunogenic than Advate. Aggregates in biopharmaceutical products are considered a risk factor for the development of anti-drug antibodies. OBJECTIVE: To evaluate recombinant full-length fVIII products for the presence of aggregates...
December 2, 2017: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/29195742/preclinical-models-to-assess-the-immunogenicity-of-aav-vectors
#17
Hildegund C J Ertl
Although gene transfer using adeno-associated virus (AAV) vectors has made tremendous progress in recent years, challenges remain due to vector-specific adaptive immune responses. Specifically, AAV-neutralizing antibodies reduce AAV-transduction rates, while CD8+ T cells directed to AAV capsid antigens cause rejection of AAV-transduced cells. This has been addressed clinically by excluding humans with pre-existing AAV-neutralizing antibodies from gene transfer trials or by using immunosuppression or reduced doses of vectors expressing improved transgene products to blunt or circumvent destructive T cell responses...
November 23, 2017: Cellular Immunology
https://www.readbyqxmd.com/read/29181141/correlation-to-fviii-c-in-two-thrombin-generation-tests-tga-cat-and-innovance-etp
#18
Marcus Ljungkvist, Maria Berndtsson, Margareta Holmström, Danijela Mikovic, Ivo Elezovic, Jovan P Antovic, Eva Zetterberg, Erik Berntorp
Introduction: Several thrombin-generation tests are available, but few have been directly compared. Our primary aim was to investigate the correlation of two thrombin generation tests, thrombin generation assay-calibrated automated thrombogram (TGA-CAT) and INNOVANCE ETP, to factor VIII levels (FVIII:C) in a group of patients with hemophilia A. The secondary aim was to investigate inter-laboratory variation for the TGA-CAT method. Methods: Blood samples were taken from 45 patients with mild, moderate and severe hemophilia A...
2017: Mediterranean Journal of Hematology and Infectious Diseases
https://www.readbyqxmd.com/read/29181011/from-igg-fusion-proteins-to-engineered-specific-human-regulatory-t-cells-a-life-of-tolerance
#19
REVIEW
David W Scott
Recent efforts have concentrated on approaches to expand and "specify" human regulatory T cells (Tregs) and to apply them to modulate adverse immune responses in autoimmunity and hemophilia. We have used retroviral transduction of specific T-cell receptor, single chain Fv, or antigen domains in Tregs to achieve this goal. Each of these approaches have advantages and disadvantages. Results with these engineered T cells and evolution of the research developments and paths that led to the development of specific regulatory approaches for tolerance are summarized...
2017: Frontiers in Immunology
https://www.readbyqxmd.com/read/29178638/living-with-a-rare-disorder-a-systematic-review-of-the-qualitative-literature
#20
REVIEW
Charlotte von der Lippe, Plata S Diesen, Kristin B Feragen
BACKGROUND: Individuals with rare diseases may face challenges that are different from those experienced in more common medical conditions. A wide range of different rare conditions has resulted in a myriad of studies investigating the specificities of the diagnosis in focus. The shared psychological experiences of individuals with a rare condition, however, have not been reviewed systematically. METHODS: We performed a systematic review, including qualitative studies on adults, published between 2000 and 2016...
November 2017: Molecular Genetics & Genomic Medicine
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