keyword
MENU ▼
Read by QxMD icon Read
search

Hemophilia A

keyword
https://www.readbyqxmd.com/read/29776906/blood-induced-bone-loss-in-murine-hemophilic-arthropathy-is-prevented-by-blocking-the-irhom2-adam17-tnf%C3%AE-pathway
#1
Coline Haxaire, Narine Hakobyan, Tania Pannellini, Camila Carballo, David McIlwain, Tak W Mak, Scott Rodeo, Suchitra Acharya, Daniel Li, Jackie Szymonifka, Xiangqian Song, Sébastien Monette, Alok Srivastava, Jane E Salmon, Carl P Blobel
Hemophilic arthropathy (HA) is a debilitating degenerative joint disease that is a major manifestation of the bleeding disorder Hemophilia A. HA typically begins with hemophilic synovitis (HS) that resembles inflammatory arthritides such as rheumatoid arthritis (RA) and frequently results in bone loss in patients. A major cause of RA is inappropriate release of the pro-inflammatory cytokine tumor necrosis factor α (TNFα) by the TNFα convertase (TACE, also referred to as ADAM17) and its regulator, iRhom2...
May 18, 2018: Blood
https://www.readbyqxmd.com/read/29774375/-successful-treatment-of-acquired-hemophilia-a-with-recombinant-porcine-factor-viii
#2
K Trautmann-Grill, O Tiebel, K Hölig, U Platzbecker
Acquired hemophilia A is a rare, potentially life-threatening disease resulting from autoantibodies against coagulation factor VIII. We report the case of a patient with acquired hemophilia A and severe bleeding after incision of a peritonsillar abscess. Treatment with high dose factor VIII and recombinant activated factor VII failed to control bleeding. However, a single infusion of recombinant porcine factor VIII stopped bleeding efficiently and resulted in measurable factor VIII levels.
May 17, 2018: Medizinische Klinik, Intensivmedizin und Notfallmedizin
https://www.readbyqxmd.com/read/29772615/-a-case-of-renal-pelvic-cancer-with-acquired-hemophilia-and-leukemoid-reaction
#3
Daiki Ikarashi, So Omori, Wataru Izumida, Yasuyuki Nakamura, Masakazu Abe, Takashi Ujiie, Wataru Obara
A 71-year-old man was referred to us with a right renal mass that was discovered by computed tomography (CT) examination for acquired hemophilia and leukemoid reaction. He presented with persistent low-grade fever and purpura on the lower legs caused by acquired hemophilia. Contrastenhanced CT scan showed a right renal tumor 6.0×7.4 cm in diameterwith inhomogeneous enhancement. The result of his urine cytology was negative. After improvement of his coagulation by treatment with immunosuppressants and steroids, he underwent open nephrectomy...
April 2018: Hinyokika Kiyo. Acta Urologica Japonica
https://www.readbyqxmd.com/read/29770199/emerging-therapies-for-hemophilia-controversies-and-unanswered-questions
#4
REVIEW
Valder R Arruda, Bhavya S Doshi, Benjamin J Samelson-Jones
Several new therapies for hemophilia have emerged in recent years. These strategies range from extended half-life factor replacement products and non-factor options with improved pharmacokinetic profiles to gene therapy aiming for phenotypic cure. While these products have the potential to change hemophilia care dramatically, several challenges and questions remain regarding broader applicability, long-term safety, and which option to pursue for each patient. Here, we review these emerging therapies with a focus on controversies and unanswered questions in each category...
2018: F1000Research
https://www.readbyqxmd.com/read/29769259/novel-therapeutics-for-hemophilia-and-other-bleeding-disorders
#5
Michael U Callaghan, Robert Sidonio, Steven W Pipe
Hemophilia and von Willebrand disease are the most common congenital bleeding disorders. Treatment for these disorders has focused on replacement of the missing coagulation factor to prevent or treat bleeding. New technologies and insights into hemostasis have driven the development of many promising new therapies for hemophilia and von Willebrand disease. Emerging bypass agents including zymogen-like factor IXa and Xa molecules are in development and a bispecific antibody, emicizumab, demonstrated efficacy in a phase III trial in people with hemophilia A and inhibitors...
May 16, 2018: Blood
https://www.readbyqxmd.com/read/29766236/ethnicity-specific-impact-of-hla-i-ii-genotypes-on-the-risk-of-inhibitor-development-data-from-korean-patients-with-severe-hemophilia-a
#6
Hyun-Young Kim, Jin-Hee Cho, Hee-Jung Kim, Hae-Sun Chung, Sun-Hee Kim, Ki-O Lee, Soo-Young Jung, Ki-Young Yoo, Hee-Jin Kim
Inhibitor development is the most serious complication in patients with hemophilia. We investigated association of HLA genotypes with inhibitor development in Korean patients with severe hemophilia A (HA). HLA genotyping was done in 100 patients with severe HA including 27 patients with inhibitors. The allele frequencies between inhibitor-positive and inhibitor-negative patients were compared. HLA class I alleles were not associated with the inhibitor status. In HLA class II, DRB1*15 [n = 100, odds ratio (OR) 0...
May 16, 2018: Annals of Hematology
https://www.readbyqxmd.com/read/29765291/the-potential-close-future-of-hemophilia-treatment-gene-therapy-tfpi-inhibition-antithrombin-silencing-and-mimicking-factor-viii-with-an-engineered-antibody
#7
REVIEW
Wolfgang Korte, Lukas Graf
Summary Hemophilia is one of the best researched monogenic diseases. Hemophilia A will affect approximately 1:5,000 male live births. In recent decades, great progress has been made with the introduction of recombinant proteins in the 1990s for therapy and prophylaxis, securing adequate availability and, with the introduction of the prophylaxis concept, reducing the negative impact of hemophilia on morbidity (especially arthropathy). Despite this progress, there are still challenges to overcome to secure adequate prophylaxis and treatment: for the time being, causal pharmacological hemophilia prophylaxis and therapy requires repeated i...
April 2018: Transfusion Medicine and Hemotherapy
https://www.readbyqxmd.com/read/29765290/extended-half-life-factor-viii-and-factor-ix-preparations
#8
REVIEW
Lukas Graf
In the last couple of years, several extended half-life factor VIII and factor IX preparations were intensively studied and gained approval. In order to extend half-lives, techniques like fusion to protein conjugates (Fc part of IgG1 or albumin), chemical modification (PEGylation), and protein sequence modification are implemented. With these techniques, it is possible to extend half-lives of factor IX products 4- to 6- fold, while half-life extension of factor VIII products is limited to 1.5- to 2-fold due to their interaction with von Willebrand factor...
April 2018: Transfusion Medicine and Hemotherapy
https://www.readbyqxmd.com/read/29758989/the-double-hazard-of-bleeding-and-thrombosis-in-hemostasis-from-a-clinical-point-of-view-a-global-assessment-by-rotational-thromboelastometry-rotem
#9
Olga Meltem Akay
Hemostasis is a complex dynamic process involving bleeding and thrombosis as two end-points. Conventional coagulation tests which are measured in plasma examine only isolated portions of the coagulation cascade, thereby giving no information on important interactions essential to the clinical evaluation of hemostatic function. Thromboelastography (TEG), originally described in 1948 has improved over the decades and become a valuable tool of coagulation testing because of the limitations of standard coagulation tests...
January 1, 2018: Clinical and Applied Thrombosis/hemostasis
https://www.readbyqxmd.com/read/29754767/preconception-carrier-screening-by-genome-sequencing-results-from-the-clinical-laboratory
#10
Sumit Punj, Yassmine Akkari, Jennifer Huang, Fei Yang, Allison Creason, Christine Pak, Amiee Potter, Michael O Dorschner, Deborah A Nickerson, Peggy D Robertson, Gail P Jarvik, Laura M Amendola, Jennifer Schleit, Dana Kostiner Simpson, Alan F Rope, Jacob Reiss, Tia Kauffman, Marian J Gilmore, Patricia Himes, Benjamin Wilfond, Katrina A B Goddard, C Sue Richards
Advances in sequencing technologies permit the analysis of a larger selection of genes for preconception carrier screening. The study was designed as a sequential carrier screen using genome sequencing to analyze 728 gene-disorder pairs for carrier and medically actionable conditions in 131 women and their partners (n = 71) who were planning a pregnancy. We report here on the clinical laboratory results from this expanded carrier screening program. Variants were filtered and classified using the latest American College of Medical Genetics and Genomics (ACMG) guideline; only pathogenic and likely pathogenic variants were confirmed by orthologous methods before being reported...
May 3, 2018: American Journal of Human Genetics
https://www.readbyqxmd.com/read/29751951/biochemical-characterization-and-immunogenicity-of-neureight-a-recombinant-full-length-factor-viii-produced-by-fed-batch-process-in-disposable-bioreactors
#11
Sandrine Delignat, Ivan Peyron, Maria El Ghazaly, Srinivas V Kaveri, Jan Rohde, Frank Mueller, Sebastien Lacroix-Desmazes
Hemophilia A is a X-linked recessive bleeding disorder consecutive to the lack of circulating pro-coagulant factor VIII (FVIII). The most efficient strategy to treat or prevent bleeding in patients with hemophilia A relies on replacement therapy using exogenous FVIII. Commercially available recombinant FVIII are produced using an expensive perfusion technology in stainless steel fermenters. A fed-batch fermentation technology was recently developed to produce 'Neureight', a full-length recombinant human FVIII, in Chinese hamster ovary (CHO) cells...
May 5, 2018: Cellular Immunology
https://www.readbyqxmd.com/read/29746288/bypassing-agents-for-bleeding-prophylaxis-in-hemophilia-b-a-clinical-conundrum
#12
Akshay M Khatri, George Jolly, Arun Kumar
No abstract text is available yet for this article.
May 1, 2018: American Journal of Therapeutics
https://www.readbyqxmd.com/read/29742662/patterns-of-von-willebrand-disease-screening-in-girls-and-adolescents-with-heavy-menstrual-bleeding
#13
Amanda E Jacobson, Sara K Vesely, Terah Koch, Janis Campbell, Sarah H OʼBrien
OBJECTIVE: To estimate the frequency of von Willebrand disease screening and factors that affect screening frequency in a national sample of girls and adolescents with heavy menstrual bleeding. METHODS: In this retrospective cohort study, we used a national claims database for privately and publicly insured patients between 2011 and 2013 for girls aged 10-17 years. Diagnostic criteria of heavy menstrual bleeding were the presence of one inpatient or two outpatient International Classification of Diseases, 9th Revision codes for heavy menstrual bleeding...
May 7, 2018: Obstetrics and Gynecology
https://www.readbyqxmd.com/read/29741261/new-challenges-and-best-practices-for-the-laboratory-monitoring-of-factor-viii-and-factor-ix-replacement
#14
REVIEW
D Van den Bossche, K Peerlinck, M Jacquemin
Several recombinant factor VIII and factor IX concentrates with extended half-life (EHL) have recently been validated by clinical studies. The availability of these novel concentrates is expected to significantly facilitate the treatment of patients with hemophilia A and B. However, the modification applied to these molecules has introduced variations in their activity measurement in routine coagulation assays. Depending on the assays, underestimations of up to 10-fold or overestimations of up to approximately 30-fold in the measurements of the recovery have been reported in some factor concentrates...
May 2018: International Journal of Laboratory Hematology
https://www.readbyqxmd.com/read/29739073/a-case-of-acquired-hemophilia-a-usefulness-of-various-methods-for-judging-mixing-test-results-for-monitoring-the-effect-of-immunosuppressive-therapy
#15
Ryosuke Moriai, Nozomi Yanagihara, Akemi Endoh, Satoru Yamada, Maki Mochizuki, Takashi Kondo, Teruo Endoh, Koichi Asanuma, Satoshi Takahashi
BACKGROUND: Measurement of FVIII inhibitor (FVIII INH) levels is important for determining the effect of immunosuppressive therapy on acquired hemophilia A (AHA). However, FVIII INH can only be measured at a limited number of laboratories, which means that there are delays in obtaining the results at many sites. METHODS: A series of mixing tests were carried out in a case of AHA, followed by comparison of various methods for judging the obtained results in association with a change of FVIII INH...
April 1, 2018: Clinical Laboratory
https://www.readbyqxmd.com/read/29735454/-prophylactic-treatment-with-low-and-intermediate-dose-factor-viii-in-children-with-severe-hemophilia-a-comprehensive-evaluation-of-joint-outcomes-and-correlation-analysis
#16
Jin-Mu Zhuang, Xue-Yan Sun, Xuan Zhou, Zhu-Qin Liu, Jing Sun
OBJECTIVE: To study the effect of low- and intermediate-dose factor VIII (FVIII) for prophylactic treatment of severe hemophilia A in children by comprehensively evaluating the outcomes of the joints. METHODS: Forty-seven children with severe hemophilia A (FVIII activity ≤2%) were enrolled in this study. Eighteen of the children received prophylactic treatment with low-dose FVIII (10 U/kg, 2-3 times a week), 20 received prophylactic treatment with intermediate-dose FVIII (15-30 U/kg, 3 times a week), and 9 received on-demand treatment with FVIII infusion when bleeding occurred according to the Chinese Expert Consensus on the Diagnosis and Treatment of Hemophilia...
April 20, 2018: Nan Fang Yi Ke da Xue Xue Bao, Journal of Southern Medical University
https://www.readbyqxmd.com/read/29732985/treatment-of-chronic-articular-pain-in-adults-with-hemophilia
#17
E Carlos Rodriguez-Merchan
INTRODUCTION: Adult people with hemophilia (PWH) mainly use Factor VIII/Factor IX (FVIII/FIX) to lessen chronic articular pain, followed in frequency by nonsteroidal anti-inflammatory drugs. Analgesics are used by only one-third of adult PWH. Limitations in activities of daily living are encountered in the large majority of PWH, and most describe pain as affecting their state of mind. A review of the literature on their treatment is important because chronic pain in adult PWH is often undertreated or wrongly treated, causing psychological problems for these patients...
May 3, 2018: Cardiovascular & Hematological Disorders Drug Targets
https://www.readbyqxmd.com/read/29731369/tailored-frequency-escalated-primary-prophylaxis-for-severe-haemophilia-a-results-of-the-16-year-canadian-hemophilia-prophylaxis-study-longitudinal-cohort
#18
Brian M Feldman, Georges E Rivard, Paul Babyn, John K M Wu, MacGregor Steele, Man-Chiu Poon, Robert T Card, Sara J Israels, Nicole Laferriere, Kulwant Gill, Anthony K Chan, Manuel Carcao, Robert J Klaassen, Stephanie Cloutier, Victoria E Price, Saunya Dover, Victor S Blanchette
BACKGROUND: Severe haemophilia A has high morbidity, and treatment, while effective, is very expensive. We report the 16-year follow-up of the Canadian Hemophilia Prophylaxis Study, which examined the effectiveness of tailored frequency-escalated primary prophylaxis with a focus on health outcomes within the domains of body structures and functions, and activities and participation (according to the WHO International Classification of Functioning, Disability and Health [WHO-ICF] framework) and a view to reducing consumption of costly clotting factor, which accounts for more than 90% of the cost of care of severe haemophilia...
May 3, 2018: Lancet Haematology
https://www.readbyqxmd.com/read/29724628/the-rare-coexistence-of-high-titer-inhibitor-development-and-gastrointestinal-stromal-tumor-in-a-patient-with-severe-hemophilia-a-case-report
#19
Abdulkerim Yıldız, Osman Şahin, Okan Yayar, Çiğdem Pala Öztürk, Hacer Berna Afacan Öztürk, Senem Maral, Harika Okutan, Murat Albayrak
Hemophilia is a hereditary disease with impaired blood coagulation due to a genetic deficiency of blood coagulation factors. The development of inhibitors further complicates the course of the disease and management. The case is here reported of a haemophilia patient who presented with coexisting development of high titer inhibitor with Gastrointestinal Stromal Tumor (GIST) diagnosis and was admitted with upper gastrointestinal system bleeding. The patient had no prior history of inhibitor presence. During all procedures including surgery, excellent hemostasis was achieved with rFVIIa treatment and no hemorrhagic complication was observed...
April 26, 2018: Transfusion and Apheresis Science
https://www.readbyqxmd.com/read/29723894/anxiety-related-bleeding-and-thrombosis
#20
Silvia Hoirisch-Clapauch
Anxiety, a normal response to stressful situations, is characterized by increased levels of factor VIII, fibrinogen, and von Willebrand factor, and by enhanced platelet aggregability. One would expect acute anxiety to be a prothrombotic state, but since acute mental stress induces tissue plasminogen activator (tPA) release from endothelial and chromaffin cells, fibrinolysis counteracts procoagulant stimuli. It could be said that procoagulant changes accompanying the fight-or-flight response reduce the risk of bleeding in case of potential injuries, while activation of fibrinolysis counteracts activation of hemostasis to prevent intravascular thrombus formation before injuries occur...
May 3, 2018: Seminars in Thrombosis and Hemostasis
keyword
keyword
6654
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"