keyword
MENU ▼
Read by QxMD icon Read
search

hemophilia review

keyword
https://www.readbyqxmd.com/read/28332238/outcome-measures-for-adult-and-pediatric-hemophilia-patients-with-inhibitors
#1
REVIEW
Cedric Hermans, Günter Auerswald, Gary Benson, Gerry Dolan, Anne Duffy, Victor Jiménez-Yuste, Rolf Ljung, Massimo Morfini, Thierry Lambert, Mehdi Osooli, Silva Zupančić Šalek
Recent advancements in almost all aspects of hemophilia treatment have vastly improved patient care and management, and new and emerging treatments hold the promise of further progress. However, there remains a scarcity of data on long-term outcomes in hemophilia, particularly among those patients with inhibitors, for whom no validated outcome assessment tools are currently available. At the 15(th) Zürich Haemophilia Forum, an expert panel reviewed the most important outcome measures in inhibitor patients and considered the challenges associated with assessing outcomes in this population...
March 22, 2017: European Journal of Haematology
https://www.readbyqxmd.com/read/28323492/regulatory-and-exhausted-t-cell-responses-to-aav-capsid
#2
Gwladys Gernoux, James M Wilson, Christian Mueller
Recombinant adeno-associated viruses are quickly becoming the preferred viral vector for viral gene delivery for the treatment of a wide variety of genetic disorders. However, since their use in a clinical trial targeting Hemophilia B patients 10 years ago, immune responses to AAV capsid appear to have hampered some of the early clinical gene transfer efficacy. Indeed, AAV-based gene transfer has been shown to reactivate capsid-specific memory T cells which have correlated with a decline in AAV transduced tissue in some patients...
March 21, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28321438/a-case-of-hemophilia-a-presenting-in-a-neonate-and-a-review-of-the-literature
#3
Esther Kisseih, Neeraja Yerrapotu, Deepak Yadav, Melissa February
No abstract text is available yet for this article.
2017: Global Pediatric Health
https://www.readbyqxmd.com/read/28298793/perioperative-considerations-in-a-patient-with-hemophilia-a-a-case-report-and-review-of-literature
#4
Tuhin Mistry, Neelam Dogra, Kanchan Chauhan, Jigyasa Shahani
Classic hemophilia or hemophilia A is a congenital bleeding diathesis in which the affected individual may present with spontaneous hemorrhage or persistent bleeding even after minor trauma. Knowledge about the disease process, multidisciplinary team approach, and timely management can lead to favorable outcome in these patients. We report management of a child with hemophilia A for suturing of lacerated upper lip mucosa following trauma. A review of literature with recommendations for perioperative management, especially in the setting of emergency surgery, is also provided...
January 2017: Anesthesia, Essays and Researches
https://www.readbyqxmd.com/read/28295573/therapeutic-plasma-exchange-for-perioperative-management-of-patients-with-congenital-factor-xi-deficiency
#5
Mohamed S Alsammak, Aneel A Ashrani, Jeffrey L Winters, Rajiv K Pruthi
BACKGROUND: Factor XI (FXI) deficiency (hemophilia C [HEM-C]) is a bleeding disorder with unpredictable severity that correlates poorly with FXI coagulation activity (FXI:C). It poses a perioperative hemostatic management challenge. For US patients with severe disease, fresh frozen plasma (FFP) or, in current use, thawed plasma is the most readily available option but comes with risk of volume overload. We report our experience of using therapeutic plasma exchange (TPE) as an alternative perioperative management strategy...
March 15, 2017: Journal of Clinical Apheresis
https://www.readbyqxmd.com/read/28286423/oligonucleotides-targeting-coagulation-factor-mrnas-use-in-thrombosis-and-hemophilia-research-and-therapy
#6
REVIEW
Marco Heestermans, Bart J M van Vlijmen
Small interfering (si) RNAs and antisense oligonucleotides (ASOs; here for simplicity reasons, both referred to as oligonucleotides) are small synthetic RNA or DNA molecules with a sequence complementary to a (pre)mRNA. Although the basic mechanisms of action between siRNAs and ASO are distinct, a sequence-specific interaction of the both oligonucleotides with the target (pre)mRNA alters the target's fate, which includes highly effective sequence-specific blockade of translation and consequently depletion of the corresponding protein...
2017: Thrombosis Journal
https://www.readbyqxmd.com/read/28277160/production-of-recombinant-coagulation-factors-are-humans-the-best-host-cells
#7
Kamilla Swiech, Virgínia Picanço-Castro, Dimas Tadeu Covas
The main treatment option for Hemophilia A/B patients involves the administration of recombinant coagulation factors on-demand or in a prophylactic approach. Despite the safety and efficacy of this replacement therapy, the development of antibodies against the coagulation factor infused, which neutralize the procoagulant activity, is a severe complication. The production of recombinant coagulation factors in human cell lines is an efficient approach to avoid such complication. Human cell lines can produce recombinant proteins with post translation modifications more similar to their natural counterpart, reducing potential immunogenic reactions...
February 23, 2017: Bioengineered
https://www.readbyqxmd.com/read/28264199/factor-activity-assays-for-monitoring-extended-half-life-fviii-and-factor-ix-replacement-therapies
#8
Steve Kitchen, Stefan Tiefenbacher, Robert Gosselin
The advent of modified factor VIII (FVIII) and factor IX (FIX) molecules with extended half-lives (EHLs) compared with native FVIII and FIX represents a major advance in the field of hemophilia care, with the potential to reduce the frequency of prophylactic injections and/or to increase the trough level prior to subsequent injections. Monitoring treatment through laboratory assays will be an important part of ensuring patient safety, including any tailoring of prophylaxis. Several approaches have been used to extend half-lives, including PEGylation, and fusion to albumin or immunoglobulin...
March 6, 2017: Seminars in Thrombosis and Hemostasis
https://www.readbyqxmd.com/read/28262242/an-extra-x-does-not-prevent-acquired-hemophilia-pregnancy-associated-acquired-hemophilia-a
#9
Assaf A Barg, Tami Livnat, Gili Kenet
Acquired hemophilia A (AHA) is a severe bleeding disorder caused by autoantibodies against clotting factor VIII (FVIII). With an estimated annual incidence of 1.3 to 1.5 per million, AHA is a rare disease. An extremely rare form of AHA has been described among women in the peripartum period, and may present with peripartum hemorrhage. Notably, although hemorrhagic symptoms commonly present 1-4 months around delivery, they may occur up to 1 year after parturition. When caring for a mother with AHA it is important to note that Factor VIII inhibitor may be transferred via the placenta from the mother to the fetus...
March 2017: Thrombosis Research
https://www.readbyqxmd.com/read/28255432/response-to-treatment-and-adverse-events-associated-with-use-of-recombinant-activated-factor-vii-in-children-a-retrospective-cohort-study
#10
James D Cooper, Arthur K Ritchey
BACKGROUND: Recombinant activated factor VII (rFVIIa) is United States (US) Food and Drug Administration (FDA)-approved for patients with hemophilia with inhibitors or congenital factor VII deficiency. Initial reports of off-label use highlighted its efficacy, though newer reports have not repeated these findings. In both types of publication, though, secondary thromboses have been seen in adult patients. The data in children are less clear. METHODS: This study analyzed all rFVIIa use at a large children's hospital for characteristics and outcomes...
February 2017: Therapeutic Advances in Drug Safety
https://www.readbyqxmd.com/read/28238287/genome-editing-for-inborn-errors-of-metabolism-advancing-towards-the-clinic
#11
REVIEW
Jessica L Schneller, Ciaran M Lee, Gang Bao, Charles P Venditti
Inborn errors of metabolism (IEM) include many disorders for which current treatments aim to ameliorate disease manifestations, but are not curative. Advances in the field of genome editing have recently resulted in the in vivo correction of murine models of IEM. Site-specific endonucleases, such as zinc-finger nucleases and the CRISPR/Cas9 system, in combination with delivery vectors engineered to target disease tissue, have enabled correction of mutations in disease models of hemophilia B, hereditary tyrosinemia type I, ornithine transcarbamylase deficiency, and lysosomal storage disorders...
February 27, 2017: BMC Medicine
https://www.readbyqxmd.com/read/28220143/venous-thromboembolism-in-children-with-cancer-and-blood-disorders
#12
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/28114794/change-of-coagulation-after-novoseven%C3%A2-use-for-bleeding-during-cardiac-surgery
#13
Emi Tomita, Hajime Takase, Keiichi Tajima, Yoshihiro Suematsu
Objectives Recombinant activated factor VII has been used for the treatment of hemophilia, factor VII deficiency, and Glanzmann's thrombasthenia. Off-label uses have recently been increasing, and there are reports that recombinant activated factor VII is effective for the treatment of excessive bleeding during or after cardiovascular surgery. We retrospectively reviewed the effectiveness of recombinant activated factor VII and its influence on the coagulation system as a treatment for uncontrollable bleeding during cardiovascular surgery...
February 2017: Asian Cardiovascular & Thoracic Annals
https://www.readbyqxmd.com/read/28103444/efficacy-and-safety-of-a-recombinant-factor-viii-produced-from-a-human-cell-line-simoctocog-alfa
#14
REVIEW
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...
March 2017: Expert Opinion on Drug Safety
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/28036020/bispecific-antibodies-as-a-development-platform-for-new-concepts-and-treatment-strategies
#16
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/28030484/pharmacokinetics-safety-and-efficacy-of-recombinant-factor-viii-fc-fusion-protein-a-practical-review
#17
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
https://www.readbyqxmd.com/read/28025929/hemophilia-management-via-data-collection-and-reporting-initial-findings-from-the-comprehensive-care-sustainability-collaborative
#18
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/27995035/clinical-and-imaging-analysis-of-subclinical-hemophilia-combined-with-coxarthrosis-case-report-and-literature-review
#19
Cheng Liu, Jun Guo, Qiu Cui, Dingfeng Li, Yanjun Zeng
INTRODUCTION: The incidence of subclinical hemophilic arthritis is low, with this case reports and literature review, we hope clinicians could pay more attention to the diagnosis of subclinical hemophilic arthritis and prevent the misdiagnosis and mistreatment. CASE PRESENTATION: We analyzed the imaging feature, and therapy of a subclinical hemophilia case with hip lesion by reporting its clinical manifestations, imaging features and therapy procedure, and reviewing literatures...
2016: SpringerPlus
https://www.readbyqxmd.com/read/27992070/exercise-for-haemophilia
#20
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...
December 19, 2016: Cochrane Database of Systematic Reviews
keyword
keyword
97529
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"