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Hemophilia A and B

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https://www.readbyqxmd.com/read/28079788/the-prevalence-of-factor-viii-and-ix-inhibitors-among-saudi-patients-with-hemophilia-results-from-the-saudi-national-hemophilia-screening-program
#1
Tarek Owaidah, Abdulkareem Al Momen, Hazzaa Alzahrani, Abdulrahman Almusa, Fawaz Alkasim, Ahmed Tarawah, Randa Al Nouno, Fatima Al Batniji, Fahad Alothman, Ali Alomari, Saud Abu-Herbish, Mahmoud Abu-Riash, Khawar Siddiqui, Mansor Ahmed, S Y Mohamed, Mahasen Saleh
Hemophilia A and B are X-linked diseases that predominantly affect male patients. Patients can develop coagulation factor inhibitors, which exponentially increases the treatment cost. However, the prevalence of factor VIII and IX inhibitors in Saudi Arabia is unclear.This study aimed to determine the Saudi prevalence of factor VIII and IX inhibitors.This 4-year, 7-center, cross-sectional study evaluated the Saudi prevalences of hemophilia A and B. We collected the patients' clinical data, evaluated their disease, and tested for factor inhibitors...
January 2017: Medicine (Baltimore)
https://www.readbyqxmd.com/read/28067581/understanding-adherence-to-treatment-and-physical-activity-in-children-with-hemophilia-the-role-of-psychosocial-factors
#2
Sarah Bérubé, Audrey Cloutier-Bergeron, Claudine Amesse, Serge Sultan
The objective of this study was to identify psychosocial factors to explain intentions of children and adolescents with hemophilia to adhere to recommendations for self-care. Twenty-four patients with hemophilia A and B, aged 6-18 years old, and their parents, completed a survey. Measures assessed factors from the theory of planned behavior, physical activity, and medical treatment adherence. The results indicate that past behaviors, attitudes, and subjective norms explained a large proportion of the intention to engage in future nonrecommended physical activity...
January 9, 2017: Pediatric Hematology and Oncology
https://www.readbyqxmd.com/read/28064157/fviii-specific-human-chimeric-antigen-receptor-car-t-regulatory-cells-suppress-t-and-b-cell-responses-to-fviii
#3
Jeongheon Yoon, Anja Schmidt, Ai-Hong Zhang, Christoph Königs, Yong Chan Kim, David W Scott
Replacement therapy with factor VIII (FVIII) is used in hemophilia A patients for treatment of bleeding episodes or for prophylaxis. A common and serious problem with this therapy is the patient's immune response to FVIII, due to a lack of tolerance, leading to the formation of inhibitory antibodies. Development of tolerogenic therapies, other than standard ITI, is an unmet goal. We previously generated engineered antigen- specific regulatory T cells (Tregs), created by transduction of a recombinant T cell receptor (TCR) isolated from a hemophilia A subject's T cell clone...
November 15, 2016: Blood
https://www.readbyqxmd.com/read/28056565/characterization-of-aav-mediated-human-factor-viii-gene-therapy-in-hemophilia-a-mice
#4
Jenny A Greig, Qiang Wang, Amanda L Reicherter, Shu-Jen Chen, Alexandra L Hanlon, Christopher H Tipper, K Reed Clark, Samuel Wadsworth, Lili Wang, James M Wilson
Adeno-associated viral (AAV) vectors are promising vehicles for hemophilia gene therapy, with favorable clinical trial data seen in the treatment of hemophilia B. In an effort to optimize the expression of human coagulation factor VIII (hFVIII) for the treatment of hemophilia A, we performed an extensive study with numerous combinations of liver-specific promoter and enhancer elements with a codon-optimized hFVIII transgene. After generating 42 variants of three reduced-size promoters and three small enhancers, transgene cassettes were packaged within a single AAV capsid, AAVrh10, to eliminate performance differences due to the capsid type...
January 5, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28056528/spectrum-of-molecular-defects-in-216-chinese-families-with-hemophilia-a
#5
Zhiping Guo, Linhua Yang, Xiuyu Qin, Xiue Liu, Yaofang Zhang
Hemophilia A (HA) is an X-linked bleeding disorder caused by heterogeneous mutations in the factor VIII gene ( F8). Our aim is to identify the causative mutations in a large HA cohort from China. We studied 216 unrelated HA families. Molecular analyses of F8 were performed using a combination of molecular techniques, including polymerase chain reaction, direct sequencing, and multiplex ligation-dependent probe amplification. The deleterious consequences of the unreported missense mutations were evaluated using various bioinformatics approaches...
January 1, 2017: Clinical and Applied Thrombosis/hemostasis
https://www.readbyqxmd.com/read/28049359/iranian-low-dose-escalating-prophylaxis-regimen-in-children-with-severe-hemophilia-a-and-b
#6
Peyman Eshghi, Elham Sadeghi, S Zahra Tara, Behnaz Habibpanah, Razieh Hantooshzadeh
Establishing an appropriate prophylaxis regimen for children with hemophilia is a critical challenge in developing countries. Barriers including availability and affordability, catheter-related complications, and inhibitor development risks have led to the introduction of new tailored prophylaxis regimens in different countries. This study emphasizes on the benefits of the Iranian low-dose escalating prophylaxis regimen in a Hemophilia Comprehensive Care Center in Iran. Referred patients with hemophilia less than 15 years of age, who were subject to prophylaxis regimen, are studied retrospectively...
January 1, 2017: Clinical and Applied Thrombosis/hemostasis
https://www.readbyqxmd.com/read/28039188/abnormal-joint-and-bone-wound-healing-in-hemophilia-mice-is-improved-by-extending-factor-ix-activity-after-hemarthrosis
#7
Junjiang Sun, Baolai Hua, Eric W Livingston, Sarah Taves, Peter B Johansen, Maureane Hoffman, Mirella Ezban, Dougald M Monroe, Ted A Bateman, Paul E Monahan
Wound healing requires interactions between coagulation, inflammation, angiogenesis, cellular migration, and proliferation. Healing in dermal wounds of hemophilia B mice is delayed when compared to hemostatically normal wild type (WT) mice, with abnormal persistence of iron deposition, inflammation, and neovascularity. We observed healing following induced joint hemorrhage in WT and factor IX (FIX) knockout (FIX(-/-)) mice, examining also parameters previously studied in an excisional skin wound model. Hemostatically normal mice tolerated this joint bleeding challenge, cleared blood from the joint, and healed with minimal pathology, even if additional autologous blood was injected intra-articularly at the time of wounding...
December 30, 2016: Blood
https://www.readbyqxmd.com/read/28025929/hemophilia-management-via-data-collection-and-reporting-initial-findings-from-the-comprehensive-care-sustainability-collaborative
#8
Michael D Tarantino, Vanita K Pindolia
BACKGROUND: Despite being a rare disorder, hemophilia represents a significant driver of health care resource utilization and requires expert hematologic and multidisciplinary services to achieve optimal outcomes. Since their inception nearly 40 years ago, hemophilia treatment centers (HTCs) have served as centers of excellence in providing the intensive care and ancillary services necessary for this unique patient base; however, the full capabilities of these centers may be underused in the current framework of managed care, largely because of a lack of communication and information shared between payers and HTC stakeholders...
January 2017: Journal of Managed Care & Specialty Pharmacy
https://www.readbyqxmd.com/read/27992112/safety-and-efficacy-of-bay-94-9027-a-prolonged-half-life-factor-viii
#9
M T Reding, H J Ng, L H Poulsen, M E Eyster, I Pabinger, H-J Shin, R Walsch, M Lederman, M Wang, M Hardtke, L A Michaels
BACKGROUND: BAY 94-9027 is a B-domain-deleted prolonged-half-life recombinant factor VIII (FVIII) conjugates in a site-specific manner with polyethylene glycol. OBJECTIVE: Assess efficacy and safety of BAY 94-9027 for prophylaxis and treatment of bleeds in patients with severe hemophilia A PATIENTS/METHODS: In this multinational, phase 2/3, partially randomized, open-label trial, males aged 12-65 years with FVIII <1% and ≥150 exposure days to FVIII received BAY 94-9027 for 36 weeks on demand or prophylactically at intervals determined following a 10-week run-in period on 25 IU/kg body weight 2x/wk...
December 19, 2016: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/27992070/exercise-for-haemophilia
#10
REVIEW
Karen Strike, Kathy Mulder, Rojer Michael
BACKGROUND: Haemophilia is a bleeding disorder associated with haemorrhaging into joints and muscles. Exercise is often used to aid recovery after bleeds, and to improve joint function in the presence of arthropathy. OBJECTIVES: Our objective was to systematically review the available evidence on the safety and effectiveness of exercise for people with haemophilia. SEARCH METHODS: We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group's Coagulopathies Trials Register and electronic databases PubMed, OVID-Embase, and CINAHL...
19, 2016: Cochrane Database of Systematic Reviews
https://www.readbyqxmd.com/read/27971809/reasons-for-switching-treatment-among-severe-hemophilia-a-and-b-adults-in-europe
#11
A Khachatryan, S Walsh, J O'Hara
No abstract text is available yet for this article.
November 2016: Value in Health: the Journal of the International Society for Pharmacoeconomics and Outcomes Research
https://www.readbyqxmd.com/read/27970893/cost-minimization-of-using-activated-prothrombin-complex-concentrate-versus-recombinant-activated-factor-vii-in-patients-with-a-nd-b-hemophilia-in-presence-of-inhibitors
#12
M E Romero Prada, F Gomez de la Rosa, N J Alvis-Zakzuk, M Carrasquilla-Sotomayor, J S Alvis-Zakzuk, J Zakzuk-Sierra
No abstract text is available yet for this article.
November 2016: Value in Health: the Journal of the International Society for Pharmacoeconomics and Outcomes Research
https://www.readbyqxmd.com/read/27942241/once-weekly-prophylactic-dosing-of-recombinant-factor-ix-improves-adherence-in-hemophilia-b
#13
REVIEW
Claudia Djambas Khayat
Regular prophylactic treatment in severe hemophilia should be considered an optimal treatment. There is no general agreement on the optimal prophylaxis regimen, and adherence to prophylaxis is a main challenge due to medical, psychosocial, and cost controversies. Improved approaches in prophylaxis regimen of hemophilia B are needed to make patients' lives easier. There is some evidence to support the efficacy of once-weekly prophylaxis. Longer sampling schedules are required for the determination of pharmacokinetic (PK) properties of factor IX (FIX)...
2016: Journal of Blood Medicine
https://www.readbyqxmd.com/read/27913543/hemophilia-and-inhibitors-current-treatment-options-and-potential-new-therapeutic-approaches
#14
Shannon L Meeks, Glaivy Batsuli
The immune response to infused factor concentrates remains a major source of morbidity and mortality in the treatment of patients with hemophilia A and B. This review focuses on current treatment options and novel therapies currently in clinical trials. After a brief review of immune tolerance regimens, the focus of the discussion is on preventing bleeding in patients with hemophilia and inhibitors. Recombinant factor VIIa and activated prothrombin complex concentrates are the mainstays in treating bleeds in patients with inhibitors...
December 2, 2016: Hematology—the Education Program of the American Society of Hematology
https://www.readbyqxmd.com/read/27898092/transposon-mediated-generation-of-cellular-and-mouse-models-of-splicing-mutations-to-assess-the-efficacy-of-snrna-based-therapeutics
#15
Elena Barbon, Mattia Ferrarese, Laetitia van Wittenberghe, Peggy Sanatine, Giuseppe Ronzitti, Fanny Collaud, Pasqualina Colella, Mirko Pinotti, Federico Mingozzi
Disease-causing splicing mutations can be rescued by variants of the U1 small nuclear RNA (U1snRNAs). However, the evaluation of the efficacy and safety of modified U1snRNAs as therapeutic tools is limited by the availability of cellular and animal models specific for a given mutation. Hence, we exploited the hyperactive Sleeping Beauty transposon system (SB100X) to integrate human factor IX (hFIX) minigenes into genomic DNA in vitro and in vivo. We generated stable HEK293 cell lines and C57BL/6 mice harboring splicing-competent hFIX minigenes either wild type (SChFIX-wt) or mutated (SChFIXex5-2C)...
November 29, 2016: Molecular Therapy. Nucleic Acids
https://www.readbyqxmd.com/read/27897038/adeno-associated-virus-gene-therapy-for-liver-disease
#16
Lisa M Kattenhorn, Christopher H Tipper, Lorelei Stoica, Deborah S Geraghty, Teresa L Wright, K Reed Clark, Samuel C Wadsworth
The field of adeno-associated virus (AAV) gene therapy has progressed rapidly over the past decade, with the advent of novel capsid serotype and organ-specific promoters, and an increasing understanding of the immune response to AAV administration. In particular, liver-directed therapy has made remarkable strides, with a number of clinical trials currently planned and ongoing in hemophilia A and B, as well as other liver disorders. This review focuses on liver-directed AAV gene therapy, including historic context, current challenges, and future developments...
December 2016: Human Gene Therapy
https://www.readbyqxmd.com/read/27885871/continuous-prophylaxis-with-recombinant-factor-ix-fc-fusion-protein-and-conventional-recombinant-factor-ix-products-comparisons-of-efficacy-and-weekly-factor-consumption
#17
Alfonso Iorio, Sangeeta Krishnan, Karl-Johan Myrén, Stefan Lethagen, Nora McCormick, Sander Yermakov, Paul Karner
BACKGROUND: Continuous prophylaxis for patients with hemophilia B requires frequent injections that are burdensome and that may lead to suboptimal adherence and outcomes. Hence, therapies requiring less-frequent injections are needed. In the absence of head-to-head comparisons, this study compared the first extended-half-life-recombinant factor IX (rFIX) product-recombinant factor IX Fc fusion protein (rFIXFc)-with conventional rFIX products based on annualized bleed rates (ABRs) and factor consumption reported in studies of continuous prophylaxis...
December 21, 2016: Journal of Medical Economics
https://www.readbyqxmd.com/read/27868337/enhanced-uptake-of-blood-coagulation-factor-viii-containing-immune-complexes-by-antigen-presenting-cells
#18
R B Hartholt, A Wroblewska, E Herczenik, I Peyron, A Ten Brinke, T Rispens, M A Nolte, E Slot, J W Claassens, F Nimmerjahn, J S Verbeek, J Voorberg
: Essentials Anti-factor (F) VIII antibody formation is a major complication in the treatment of hemophilia A. We investigated uptake of FVIII and FVIII immune complex by bone marrow derived dendritic cells. Immune complex formation increased uptake of FVIII 3-4 fold in a Fcγ receptor dependent manner. FVIII immune complex binding to Fcγ receptors may modulate immune tolerance induction. SUMMARY: Background A major complication in the treatment of hemophilia A is the development of inhibitory antibodies targeting coagulation factor VIII (FVIII)...
November 21, 2016: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/27863665/biodegradable-hydrophilic-carriers-for-the-oral-delivery-of-hematological-factor-ix-for-hemophilia-b-treatment
#19
Sarena D Horava, Katie J Moy, Nicholas A Peppas
Current protein replacement therapies for hemophilia B, a genetic bleeding disorder caused by a deficiency in coagulation factor IX, rely on IV injections and infusions. Oral delivery of factor IX is a desirable needle-free option, especially for prophylaxis. We have developed a biodegradable, pH-responsive hydrogel microcarrier system based on the poly(methacrylic acid)-grafted-poly(ethylene glycol) [P(MAA-g-EG)]. Incorporation of an enzymatically degradable peptide crosslinking agent allows for site-specific degradation by trypsin in the small intestine...
November 30, 2016: International Journal of Pharmaceutics
https://www.readbyqxmd.com/read/27834949/direct-interaction-of-human-serum-proteins-with-aav-virions-to-enhance-aav-transduction-immediate-impact-on-clinical-applications
#20
M Wang, J Sun, A Crosby, K Woodard, M L Hirsch, R J Samulski, C Li
Recent hemophilia B clinical trials using adeno-associated virus (AAV) gene delivery have demonstrated much lower coagulation factor IX (FIX) production in patients compared with the high levels observed in animal models and AAV capsid-specific cytotoxic T lymphocyte response elicited at high doses of AAV vectors. These results emphasize the necessity to explore effective approaches for enhancement of AAV transduction. Initially, we found that incubation of all AAV vectors with human serum enhanced AAV transduction...
December 1, 2016: Gene Therapy
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