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Hemophilia A

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https://www.readbyqxmd.com/read/28105131/immunoglobulin-g4-related-acquired-hemophilia-a-case-report
#1
Xiaoyan Li, Wei Duan, Xiang Zhu, Jianying Xu
Acquired hemophilia A (AHA) is a relatively rare and life-threatening bleeding disorder whose pathogenesis is not completely understood. The present study reports a rare case of immunogubulin (IgG)4-related AHA with multisystemic involvement. A 55-year old male patient presented with symptoms of bronchial asthma and multiple subdermal hematomas. Chest computed tomography showed multiple diffuse nodular lesions with thickening of bronchovascular bundles, and scattered high-density spots in both lung lobes. Laboratory investigations showed increased activated partial prothrombin time (120...
December 2016: Experimental and Therapeutic Medicine
https://www.readbyqxmd.com/read/28103444/efficacy-and-safety-of-a-recombinant-factor-viii-produced-from-a-human-cell-line-simoctocog-alfa
#2
Massimo Franchini, Pier Mannuccio Mannucci
The development of inhibitors against infused factor VIII (FVIII) has a detrimental impact on health and quality of life of patients with hemophilia A. Several observational studies and a recently published randomized trial indicate that the inhibitor risk in previously untreated patients (PUPs) is higher following the use of recombinant FVIII (rFVIII) products compared with plasma-derived FVIII concentrates. There is currently a great interest towards newer rFVIII products that adopt various technological solutions to reduce the inhibitor risk...
January 19, 2017: Expert Opinion on Drug Safety
https://www.readbyqxmd.com/read/28092962/effects-of-selection-and-mutation-on-epidemiology-of-x-linked-genetic-diseases
#3
Francesca Verrilli, Hamed Kebriaei, Luigi Glielmo, Martin Corless, Carmen Del Vecchio
The epidemiology of X-linked recessive diseases, a class of genetic disorders, is modeled with a discrete-time, structured, non linear mathematical system. The model accounts for both de novo mutations (i.e., affected sibling born to unaffected parents) and selection (i.e., distinct fitness rates depending on individual's health conditions). Assuming that the population is constant over generations and relying on Lyapunov theory we found the domain of attraction of model's equilibrium point and studied the convergence properties of the degenerate equilibrium where only affected individuals survive...
June 1, 2017: Mathematical Biosciences and Engineering: MBE
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
#4
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/28079537/successful-total-hip-replacement-with-sequential-administration-of-bypassing-agents-in-an-adolescent-boy-with-hemophilia-a-and-high-inhibitor-titers
#5
Zühre Kaya, Özlem Orhan, Sacit Turanl, İdil Yenicesu, Ülker Koçak, Türkiz Gürsel
There are only a few reports of total hip replacement in patients with hemophilia A and inhibitors. We performed total hip replacement in an 18-year-old adolescent boy who had high inhibitor titers since infancy. Recombinant factor VIIa (NovoSeven) was used as a bypass agent during the surgery. There was no excessive introperative bleeding; however, postsurgical bleeding occurred and was controlled by sequential administration of recombinant factor VIIa and activated prothrombin complex concentrate (FEIBA)...
January 12, 2017: Blood Coagulation & Fibrinolysis: An International Journal in Haemostasis and Thrombosis
https://www.readbyqxmd.com/read/28077675/post-hoc-assessment-of-the-immunogenicity-of-bioengineered-factor-viia-demonstrates-the-use-of-preclinical-tools
#6
Kasper Lamberth, Stine Louise Reedtz-Runge, Jonathan Simon, Ksenia Klementyeva, Gouri Shankar Pandey, Søren Berg Padkjær, Véronique Pascal, Ileana R León, Charlotte Nini Gudme, Søren Buus, Zuben E Sauna
Immunogenicity is an important consideration in the licensure of a therapeutic protein because the development of neutralizing anti-drug antibodies (ADAs) can affect both safety and efficacy. Neoantigens introduced by bioengineering of a protein drug are a particular cause for concern. The development of a bioengineered recombinant factor VIIa (rFVIIa) analog was discontinued after phase 3 trials because of the development of ADAs. The unmodified parent molecule (rFVIIa), on the other hand, has been successfully used as a drug for more than two decades with no reports of immunogenicity in congenital hemophilia patients with inhibitors...
January 11, 2017: Science Translational Medicine
https://www.readbyqxmd.com/read/28077343/disappearance-of-acquired-hemophilia-a-after-complete-remission-in-a-multiple-myeloma-patient
#7
Vanessa Innao, Alessandro Allegra, Rosalba Morreale, Sabina Russo, Caterina Musolino
No abstract text is available yet for this article.
January 12, 2017: Turkish Journal of Haematology: Official Journal of Turkish Society of Haematology
https://www.readbyqxmd.com/read/28073291/future-of-raav-gene-therapy-platform-for-rnai-gene-editing-and-beyond
#8
Paul Valdmanis, Mark A Kay
The use of recombinant adeno-associated viruses (rAAVs) ushered in a new millennium of gene transfer for therapeutic treatment of a number of conditions including congenital blindness, hemophilia, and spinal muscular atrophy (SMA). rAAV vectors have remarkable staying power from a therapeutic standpoint withstanding several ebbs and flows. As new technologies such as clustered regularly interspaced short palindromic repeat (CRISPR) genome editing emerge, it is now the delivery tool - the AAV vector - that is the stalwart...
January 10, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28068306/revision-knee-arthroplasty-in-patients-with-inherited-bleeding-disorders-a-single-center-experience
#9
Andrzej Kotela, Magdalena Wilk-Frańczuk, Piotr Żbikowski, Paweł Łęgosz, Paweł Ambroziak, Ireneusz Kotela
BACKGROUND The results of total knee arthroplasty (TKA) in patients with inherited bleeding disorders (IBDs) are poorer when compared with those in the general population, with a notably higher risk of complications and higher revision rates. Thus, revision procedures are becoming a growing concern in this group of patients. The aim of this study was to evaluate the results of revision TKA in patients with IBD. MATERIAL AND METHODS A retrospective cohort study with longitudinal assessment of hemophilia patients scheduled for revision TKA between January 2010 and September 2015 was performed...
January 9, 2017: Medical Science Monitor: International Medical Journal of Experimental and Clinical Research
https://www.readbyqxmd.com/read/28067581/understanding-adherence-to-treatment-and-physical-activity-in-children-with-hemophilia-the-role-of-psychosocial-factors
#10
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
#11
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
#12
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
#13
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
#14
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/28044933/should-arterial-embolization-in-recurrent-spontaneous-hemophilic-hemarthroses-refractory-to-intensive-prophylaxis-be-the-first-invasive-resort
#15
E Carlos Rodriguez-Merchan
This paper reviews the role of arterial embolization (AE) in recurrent spontaneous hemarthroses (RSH) that are refractory to intensive prophylaxis (RIP) in people with hemophilia (PWH). There are six articles in the literature on the topic. The total number of AEs performed so far is 78 in 69 patients. Four second AEs were required (4/78), and a third AE in one (1/78). No serious adverse effects have been reported except for a pseudoaneurym (1/78) of the femoral artery at the puncture site (that eventually required surgical repair) and for one patient (1/78) that had recurrence of bleeding for whom surgical exploration was required...
December 16, 2016: Cardiovascular & Hematological Disorders Drug Targets
https://www.readbyqxmd.com/read/28039188/abnormal-joint-and-bone-wound-healing-in-hemophilia-mice-is-improved-by-extending-factor-ix-activity-after-hemarthrosis
#16
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/28036020/bispecific-antibodies-as-a-development-platform-for-new-concepts-and-treatment-strategies
#17
REVIEW
Fa Yang, Weihong Wen, Weijun Qin
With the development of molecular cloning technology and the deep understanding of antibody engineering, there are diverse bispecific antibody formats from which to choose to pursue the optimal biological activity and clinical purpose. The single-chain-based bispecific antibodies usually bridge tumor cells with immune cells and form an immunological synapse because of their relatively small size. Bispecific antibodies in the IgG format include asymmetric bispecific antibodies and homodimerized bispecific antibodies, all of which have an extended blood half-life and their own crystalline fragment (Fc)-mediated functions...
December 28, 2016: International Journal of Molecular Sciences
https://www.readbyqxmd.com/read/28035745/one-amino-acid-in-mouse-factor-viia-defines-its-endothelial-protein-c-receptor-binding-and-modulates-its%C3%A2-epcr-dependent-hemostatic-activity-in-vivo
#18
G Pavani, S M Zintner, L Ivanciu, J C Small, K A Stafford, J H Szeto, P Margaritis
BACKGROUND: Human activated Factor VII (hFVIIa), used in hemophilia treatment, binds to the endothelial Protein C (PC) receptor (EPCR) with unclear hemostatic consequences. Interestingly, mice lack the Factor VIIa (FVIIa)-EPCR interaction. Therefore, to investigate the hemostatic consequences of this interaction in hemophilia, we previously engineered a mouse FVIIa (mFVIIa) molecule that bound mouse EPCR (mEPCR) using 3 substitutions from mouse Protein C (mPC, Leu4→Phe, Leu8→Met and Trp9→Arg)...
December 30, 2016: Journal of Thrombosis and Haemostasis: JTH
https://www.readbyqxmd.com/read/28034891/non-neutralizing-antibodies-against-factor-viii-and-risk-of-inhibitor-development-in-patients-with-severe-hemophilia-a
#19
Antonino Cannavò, Carla Valsecchi, Isabella Garagiola, Roberta Palla, Pier Mannuccio Mannucci, Frits R Rosendaal, Flora Peyvandi
The development of anti-factor VIII (FVIII) neutralizing antibodies (inhibitors) is the major complication in hemophilia A. Non-neutralizing antibodies (NNAs) have not only been detected in hemophilia patients but also in unaffected individuals. The aim of this study was to assess the prevalence of NNAs in a cohort of previously untreated or minimally treated patients with hemophilia A, and to evaluate whether their presence is associated with the development of inhibitors. Plasma samples of 237 patients with severe hemophilia A enrolled in the SIPPET trial were collected before any exposure to FVIII concentrates and analyzed for the presence of anti-FVIII NNAs...
December 29, 2016: Blood
https://www.readbyqxmd.com/read/28030484/pharmacokinetics-safety-and-efficacy-of-recombinant-factor-viii-fc-fusion-protein-a-practical-review
#20
Kim Schafer, James Munn, Kate Khair, Neelam Thukral, Angela Tom, Sally McAlister
Prophylaxis for hemophilia A with conventional factor VIII (FVIII) products requires frequent intravenous dosing, which may reduce adherence. Recombinant factor VIII Fc fusion protein (rFVIIIFc) has a prolonged half-life compared with conventional rFVIII, and has demonstrated safety and efficacy for the prevention and treatment of bleeding episodes in phase 3 studies of patients with severe hemophilia A. Most subjects experienced reduced prophylactic dosing frequency with rFVIIIFc compared with prestudy FVIII; the median total weekly prophylactic consumption was comparable...
January 2017: Journal of Infusion Nursing: the Official Publication of the Infusion Nurses Society
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