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https://www.readbyqxmd.com/read/28220143/venous-thromboembolism-in-children-with-cancer-and-blood-disorders
#1
REVIEW
Richard H Ko, Courtney D Thornburg
Venous thromboembolism (VTE) in children is multifactorial and most often related to a combination of inherited and acquired thrombophilias. Children with cancer and blood disorders are often at risk for VTE due to disease-related factors such as inflammation and abnormal blood flow and treatment-related factors such as central venous catheters and surgery. We will review risk factors for VTE in children with leukemia, lymphoma, and solid tumors. We will also review risk factors for VTE in children with blood disorders with specific focus on sickle cell anemia and hemophilia...
2017: Frontiers in Pediatrics
https://www.readbyqxmd.com/read/28212975/budget-impact-analysis-of-prolonged-half-life-recombinant-fviii-therapy-for-hemophilia-in-the-united-states
#2
Suzanne McMullen, Brieana Buckley, Eric Hall, Jon Kendter, Karissa Johnston
BACKGROUND: Hemophilia A is a factor VIII deficiency, associated with spontaneous, recurrent bleeding episodes. This may lead to comorbidities such as arthropathy and joint replacement, which contribute to morbidity and increased health care expenditure. Recombinant factor VIII Fc fusion protein (rFVIIIFc), a prolonged half-life factor therapy, requires fewer infusions, resulting in reduced treatment burden. OBJECTIVE: Use a budget impact analysis to assess the potential economic impact of introducing rFVIIIFc to a formulary from the perspective of a private payer in the United States...
January 2017: Value in Health: the Journal of the International Society for Pharmacoeconomics and Outcomes Research
https://www.readbyqxmd.com/read/28211222/nasal-hemophilic-pseudotumor-in-a-patient-with-mild-hemophilia-a-and-allergic-rhinitis
#3
Yoshiyasu Ogata, Mikio Monji, Keita Kai, Muneaki Matsuo
Hemophilic pseudotumor is a rare complication, even in patients with severe hemophilia. Herein we report on a case of hemophilic pseudotumor in a patient with mild hemophilia A and allergic rhinitis, initially suspected to be a nasal tumor. The pseudotumor was cured by supplementation with recombinant factor VIII concentrates, and medication for allergic rhinitis. Pseudotumor should always be considered in hemophiliac patients, even in those with only mild deficiency of coagulation factors.
February 2017: Pediatrics International: Official Journal of the Japan Pediatric Society
https://www.readbyqxmd.com/read/28202865/repeated-diffuse-alveolar-hemorrhage-in-a-patient-with-hemophilia-b
#4
Hajime Kasai, Jiro Terada, Hiromasa Hoshi, Takashi Urushibara, Fumiaki Kato, Rintaro Nishimura, Koichiro Tatsumi
Diffuse alveolar hemorrhage (DAH) is a life-threatening complication that occurs in association with various diseases including coagulation disorders. In rare cases, it is caused by hemophilia. A 48-year-old man was admitted to our hospital for a third time due to DAH. Although the cause of DAH could not be identified by bronchoscopy or laboratory tests, a good response to corticosteroids suggested idiopathic DAH with pulmonary capillaritis. The patient was diagnosed with hemophilia B based on the results of a detailed inquiry, a mildly prolonged activated partial thromboplastin time, and low factor IX activity...
2017: Internal Medicine
https://www.readbyqxmd.com/read/28202722/systemic-delivery-of-factor-ix-messenger-rna-for-protein-replacement-therapy
#5
Suvasini Ramaswamy, Nina Tonnu, Kiyoshi Tachikawa, Pattraranee Limphong, Jerel B Vega, Priya P Karmali, Pad Chivukula, Inder M Verma
Safe and efficient delivery of messenger RNAs for protein replacement therapies offers great promise but remains challenging. In this report, we demonstrate systemic, in vivo, nonviral mRNA delivery through lipid nanoparticles (LNPs) to treat a Factor IX (FIX)-deficient mouse model of hemophilia B. Delivery of human FIX (hFIX) mRNA encapsulated in our LUNAR LNPs results in a rapid pulse of FIX protein (within 4-6 h) that remains stable for up to 4-6 d and is therapeutically effective, like the recombinant human factor IX protein (rhFIX) that is the current standard of care...
February 15, 2017: Proceedings of the National Academy of Sciences of the United States of America
https://www.readbyqxmd.com/read/28196793/specific-factor-ix-mrna-and-protein-features-favor-drug-induced-readthrough-over-recurrent-nonsense-mutations
#6
Alessio Branchini, Mattia Ferrarese, Matteo Campioni, Giancarlo Castaman, Rosella Mari, Francesco Bernardi, Mirko Pinotti
Drug-induced readthrough over premature stop codons (PTCs) is a potentially attractive therapy for genetic disorders but a wide outcome variability has been observed. Through expression studies we investigated the responsiveness to the readthrough-inducing drug geneticin of eleven rationally-selected factor IX (FIX) nonsense mutations, present in 70% (324/469) of Hemophilia B (HB) patients with PTCs. Among the predicted readthrough-permissive TGA variants, only two (p.W240X, p.R384X) responded with a remarkable rescue of FIX activity...
February 14, 2017: Blood
https://www.readbyqxmd.com/read/28187737/evaluation-of-a-web-based-registry-of-inherited-bleeding-disorders-a-descriptive-study-of-the-brazilian-experience-with-hemovidaweb-coagulopatias
#7
Suely Meireles Rezende, Silvia Helena Lacerda Rodrigues, Kelly Neves Pinheiro Brito, Diego Lima Quintino da Silva, Marcos Lázaro Santo, Bárbara de Jesus Simões, Guilherme Genovez, Helder Teixeira Melo, João Paulo Baccara Araújo, Danila Augusta Accioly Varella Barca
BACKGROUND: Inherited bleeding disorders (IBD) consist of a group of rare heterogeneous diseases, which require treatment for life. Management of these disorders is complex and costly. Therefore, good quality data of the affected population is crucial to guide policy planning. The aim of this manuscript is to describe the impact of a national, web-based registry - the Hemovidaweb Coagulopatias (HWC) - in the management of the IBD in Brazil. METHODS: The system was developed in PHP 5...
February 10, 2017: Orphanet Journal of Rare Diseases
https://www.readbyqxmd.com/read/28183693/prophylaxis-usage-bleeding-rates-and-joint-outcomes-of-hemophilia-1999-2010-a-surveillance-project
#8
Marilyn J Manco-Johnson, J Michael Soucie, Joan C Gill
This analysis of the United States Hemophilia Treatment Center Network and the Centers for Disease Control and Prevention surveillance registry assessed trends in prophylaxis use and its impact on key indicators of arthropathy across the life span among participants with severe hemophilia A. Data on demographics, clinical characteristics and outcomes were collected prospectively between 1999 and 2010 at annual clinical visits to 134 hemophilia treatment centers. Trends in treatment and outcomes were evaluated using cross-sectional and longitudinal analyses...
February 9, 2017: Blood
https://www.readbyqxmd.com/read/28174634/acquired-hemophilia-a-in-a-patient-treated-by-ginkgo-dipyridamolum
#9
Jinbo Liu, Hongyu Wang, Guangyun Shang, Lichun Wang, Hongwei Zhao, Huan Liu
Acquired hemophilia A might be caused by Ginkgo-dipyridamolum especially by Ginkgo, and it was successfully treated with hemostasis and immune-suppression therapy including methylprednisolone and cyclophosphamide.
February 2017: Clinical Case Reports
https://www.readbyqxmd.com/read/28173870/an-effective-ex-vivo-approach-for-inducing-endothelial-progenitor-cells-from-umbilical-cord-blood-cd34-cells
#10
Meng Qin, Xin Guan, Huihui Wang, Yu Zhang, Bin Shen, Qingyu Zhang, Wei Dai, Yupo Ma, Yongping Jiang
BACKGROUND: Transplantation of endothelial progenitor cells (EPCs)/endothelial cells (ECs) has been used for the treatment of ischemic diseases and hemophilia A, due to their great capacity for producing factor VIII and for repairing vascular damage. We established an effective approach to stimulate the expansion and differentiation of EPCs for potential therapeutic applications. METHODS: CD34(+) cells isolated from human cord blood were cultured in a two-step system for 21 days...
February 7, 2017: Stem Cell Research & Therapy
https://www.readbyqxmd.com/read/28168417/first-case-report-of-hemophilia-b-leyden-in-japan
#11
Atsuki Yamashita, Chiai Nagae, Mika Mori, Tomoko Ashikaga, Tetsuhito Kojima, Masashi Taki
Hemophilia B Leyden is a unique subtype of hemophilia B, characterized by increasing factor IX activity (FIX:C) after puberty and a lower normal range of FIX:C throughout adulthood. However, to date, no Japanese case has been reported. Here, we report a case of hemophilia B Leyden in a 22-year-old male. He suffered from subgaleal hematoma, and was subsequently diagnosed with hemophilia B (FIX:C 0.2%) in the neonatal period. Both his parents are Japanese. There was no history of hemophilia in his family. FIX:C gradually increased with age (8% at age = 1; 14% at age = 7; 19% at age = 12; 32% at age = 18)...
February 7, 2017: International Journal of Hematology
https://www.readbyqxmd.com/read/28166648/characteristics-of-minimally-oversized-adeno-associated-virus-vectors-encoding-human-factor-viii-generated-using-producer-cell-lines-and-triple-transfection
#12
Bindu Nambiar, Cathleen Cornell Sookdeo, Patricia Berthelette, Robert Jackson, Susan Piraino, Brenda Burnham, Shelley Nass, David Souza, Catherine R O'Riordan, Karen A Vincent, Seng H Cheng, Donna Armentano, Sirkka Kyostio-Moore
Several ongoing clinical studies are evaluating recombinant adeno-associated virus (rAAV) vectors as gene delivery vehicles for a variety of diseases. However, the production of vectors with genomes >4.7 kb is challenging, with vector preparations frequently containing truncated genomes. To determine whether the generation of oversized rAAVs can be improved using a producer cell-line (PCL) process, HeLaS3-cell lines harboring either a 5.1 or 5.4 kb rAAV vector genome encoding codon-optimized cDNA for human B-domain deleted Factor VIII (FVIII) were isolated...
February 2017: Human Gene Therapy Methods
https://www.readbyqxmd.com/read/28166609/modulating-immunogenicity-of-factor-ix-by-fusion-to-an-immunoglobulin-fc-domain-a-study-using-hemophilia-b-mouse-model
#13
Ditza Levin, H A Daniel Lagassé, Erin Burch, Scott Strome, Siyuan Tan, Haiyan Jiang, Zuben E Sauna, Basil Golding
BACKGROUND: Fc-fusion is a platform technology used to increase the circulating half-life of protein and peptide therapeutics. However, there are potential immunological consequences with this approach, such as changes in the molecule's immunogenicity as well as possible interactions with a repertoire of Fc-receptors (FcR) which can modulate immune responses. OBJECTIVES/METHODS: Using a mouse hemophilia B (HB) model, we compared the immune responses to infusions of recombinant human Factor IX (hFIX) and hFIX fused to mouse IgG2a-Fc (hFIX-mFc)...
February 6, 2017: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/28166608/safety-efficacy-and-pharmacokinetics-of-rviii-singlechain-in-children-with-severe-hemophilia-a-sesults-of-a-multicenter-clinical-trial
#14
Oleksandra Stasyshyn, Claudia Djambas Khayat, Genadi Iosava, Jeannie Ong, Faraizah Abdul Karim, Kathelijn Fischer, Alex Veldman, Nicole Blackman, Katie St Ledger, Ingrid Pabinger
BACKGROUND: rVIII-SingleChain, is a novel B-domain truncated recombinant Factor VIII (rFVIII) comprised of covalently bonded FVIII heavy and light chains, demonstrating a high binding affinity to von Willebrand Factor. OBJECTIVES: This Phase III study investigated the safety, efficacy and pharmacokinetics of rVIII-SingleChain in previously treated pediatric patients <12 years of age with severe hemophilia A. PATIENTS/METHODS: Patients could be assigned to prophylaxis or on-demand therapy by the investigator...
February 6, 2017: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/28164173/tgf-%C3%AE-1-along-with-other-platelet-contents-augments-treg-cells-to-suppress-anti-fviii-immune-responses-in-hemophilia-a-mice
#15
Dipica Haribhai, Xiaofeng Luo, Juan Chen, Shuang Jia, Linzheng Shi, Jocelyn A Schroeder, Hartmut Weiler, Richard H Aster, Martin J Hessner, Jianda Hu, Calvin B Williams, Qizhen Shi
Platelets are a rich source of many cytokines and chemokines including transforming growth factor β 1 (TGF-β1). TGF-β1 is required to convert conventional CD4(+) T (Tconv) cells into induced regulatory T (iTreg) cells that express the transcription factor Foxp3. Whether platelet contents will affect Treg cell properties has not been explored. In this study, we show that unfractionated platelet lysates (pltLys) containing TGF-β1 efficiently induced Foxp3 expression in Tconv cells. The common Treg cell surface phenotype and in vitro suppressive activity of unfractionated pltLys-iTreg cells were similar to those of iTreg cells generated using purified TGF-β1 (purTGFβ-iTreg) cells...
December 13, 2016: Blood Adv
https://www.readbyqxmd.com/read/28153454/minimally-invasive-aortic-valve-replacement-in-a-patient-with-severe-hemophilia-a
#16
Christos Chamos, Martin Yates, Steve Austin, Vinayak Bapat
No abstract text is available yet for this article.
November 22, 2016: Journal of Cardiothoracic and Vascular Anesthesia
https://www.readbyqxmd.com/read/28153098/oral-tolerance-induction-in-hemophilia-b-dogs-fed-with-transplastomic-lettuce
#17
Roland W Herzog, Timothy C Nichols, Jin Su, Bei Zhang, Alexandra Sherman, Elizabeth P Merricks, Robin Raymer, George Q Perrin, Mattias Häger, Bo Wiinberg, Henry Daniell
Anti-drug antibodies in hemophilia patients substantially complicate treatment. Their elimination through immune tolerance induction (ITI) protocols poses enormous costs, and ITI is often ineffective for factor IX (FIX) inhibitors. Moreover, there is no prophylactic ITI protocol to prevent anti-drug antibody (ADA) formation. Using general immune suppression is problematic. To address this urgent unmet medical need, we delivered antigen bioencapsulated in plant cells to hemophilia B dogs. Commercial-scale production of CTB-FIX fusion expressed in lettuce chloroplasts was done in a hydroponic facility...
February 1, 2017: Molecular Therapy: the Journal of the American Society of Gene Therapy
https://www.readbyqxmd.com/read/28150393/acquired-hemophilia-a-manifesting-as-plasma-transfusion-uncontrolled-severe-bleeding-2-weeks-after-chorioamnionitis-induced-abortion
#18
Ayumi Matsuoka, Hiromasa Sasaki, Chiharu Sugimori, Shinya Hirabuki, Tsutomu Hoshiba, Hiroshi Fujiwara
Acquired hemophilia A (AHA) is a serious and rare complication of pregnancy, caused by autoantibodies to coagulation factor VIII after delivery. We here report the case of a 36-year-old primigravida woman who developed AHA following chorioamnionitis-caused miscarriage in the second trimester. Thirteen days after abortion, sudden, massive vaginal bleeding occurred with marked prolongation of activated partial thromboplastin time (APTT) in the absence of other abnormal coagulation data. Sequential transfusion of fresh frozen plasma did not achieve normalization of APTT...
February 2, 2017: Journal of Obstetrics and Gynaecology Research
https://www.readbyqxmd.com/read/28133956/development-of-a-novel-automated-screening-method-for-detection-of-fviii-inhibitors
#19
M S Evans, K J Donaldson, M E Eyster
INTRODUCTION: Factor VIII activity is routinely determined by measuring the activated partial thromboplastin time (aPTT) of a patient plasma sample and determining percent activity from a standard curve. To maximize the detection of a clotting factor inhibitor, a subjective assessment of parallelism of a patient curve compared with a standard curve is performed. We developed and validated an automated objective method to assess parallelism as a rapid screening tool for detection of an inhibitor to factor VIII during routine FVIII assays...
January 30, 2017: International Journal of Laboratory Hematology
https://www.readbyqxmd.com/read/28130908/aspartate-transaminase-platelet-ratio-and-fibrosis-4-indices-as-effective-markers-for-monitoring-esophageal-varices-in-hiv-hepatitis-c-virus-co-infected-patients-due-to-contaminated-blood-products-for-hemophilia
#20
Koji Natsuda, Mitsuhisa Takatsuki, Takayuki Tanaka, Akihiko Soyama, Tomohiko Adachi, Shinichiro Ono, Takanobu Hara, Zhassulan Baimakhanov, Hajime Imamura, Satomi Okada, Masaaki Hidaka, Susumu Eguchi
AIM: We examined the feasibility of the aspartate transaminase (AST)-platelet ratio index (APRI) and Fibrosis-4 (FIB4) score, which are well-established markers for liver fibrosis, as indicators for monitoring esophageal varices in patients who were co-infected with HIV and hepatitis C virus (HCV) due to contaminated blood products for hemophilia in Japan. METHODS: Forty-three HIV/HCV co-infected patients were enrolled. All were hemophilic men (median age 41 years; range, 29-66 years)...
January 28, 2017: Hepatology Research: the Official Journal of the Japan Society of Hepatology
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