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https://www.readbyqxmd.com/read/29124682/nonacog-beta-pegol-a-review-in-haemophilia-b
#1
Yahiya Y Syed
Nonacog beta pegol [Refixia(®) (EU)] is an intravenously-administered, glycoPEGylated recombinant factor IX (FIX), with an extended terminal half-life. It is approved in the EU for the treatment and prophylaxis of bleeding in patients aged ≥ 12 years with haemophilia B. The therapeutic efficacy and safety of nonacog beta pegol was demonstrated in the phase 3 Paradigm trials in previously treated adolescents and adults with haemophilia B. In Paradigm 2, nonacog beta pegol showed good haemostatic effects when treating bleeds on-demand, and reduced annualized bleeding rates when used as a once-weekly prophylaxis...
November 9, 2017: Drugs
https://www.readbyqxmd.com/read/29051804/review-of-recombinant-anti-haemophilic-porcine-sequence-factor-viii-in-adults-with-acquired-haemophilia-a
#2
REVIEW
Emma Fosbury, Anja Drebes, Anne Riddell, Pratima Chowdary
Acquired haemophilia A (AHA) is a rare, serious bleeding disorder most often encountered in elderly patients. The mainstay of haemostatic management is with bypassing agents (BPAs) including recombinant activated factor VII (rFVIIa) and activated prothrombin complex concentrates (aPCCs). Their major limitation is incomplete efficacy, potential risk for thrombosis and the lack of routine laboratory assays for monitoring treatment response. Plasma-derived porcine FVIII (pd-pFVIII, Hyate C(®)), first used in the 1950s for the management of congenital haemophilia, has sufficient sequence homology to be haemostatic in humans, but the lack of complete homology facilitates efficacy even in the presence of human allo- and autoantibodies against human FVIII (hFVIII)...
September 2017: Therapeutic Advances in Hematology
https://www.readbyqxmd.com/read/28960809/acquired-haemophilia-in-cancer-a-systematic-and-critical-literature-review
#3
REVIEW
M Napolitano, S Siragusa, S Mancuso, C M Kessler
AIM: There is a paucity of data on the clinical presentation and management of cancer patients with acquired haemophilia (AH), we here report a systematic literature review on acquired haemophilia in the context of cancer. METHODS: Treatment outcomes of AH were defined as complete response (CR), partial response (PR) or no response (NR), based on inhibitor eradication, coagulation factor VIII levels and bleeding control. Reported deaths were either related to cancer or bleeding...
September 27, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28944952/bypassing-agent-prophylaxis-in-people-with-hemophilia-a-or-b-with-inhibitors
#4
REVIEW
Chatree Chai-Adisaksopha, Sarah J Nevitt, Mindy L Simpson, Maissaa Janbain, Barbara A Konkle
BACKGROUND: People with hemophilia A or B with inhibitors are at high risk of bleeding complications. Infusion of bypassing agents, such as recombinant activated FVII (rFVIIa) and plasma-derived activated prothrombin complex concentrate, are suggested as alternative therapies to factor VIII (haemophilia A) or IX (haemophilia B) for individuals who no longer respond to these treatments because they develop inhibitory antibodies. The ultimate goal of treatment is to preserve the individual's joints, otherwise destroyed by recurrent bleeds...
September 25, 2017: Cochrane Database of Systematic Reviews
https://www.readbyqxmd.com/read/28942686/fibrin-glue-for-local-haemostasis-in-haemophilia-surgery
#5
E Carlos Rodriguez-Merchan
INTRODUCTION: Local fibrin glue (FG) appears to be a useful local haemostatic agent for severe haemorrhage in people with haemophilia (PWH) undergoing surgical procedures. AIM: To evaluate the role of local FG in PWH. METHODS: A review of the literature on the topic has been performed. RESULTS: Local FG is not always necessary to achieve haemostasis in all surgical procedures performed in PWH. However, it could be a good adjunct therapy, primarily when a surgical field will bleed more than expected (e...
October 3, 2017: Hospital Practice (Minneapolis)
https://www.readbyqxmd.com/read/28900904/lonoctocog-alfa-a-review-in-haemophilia-a
#6
Zaina T Al-Salama, Lesley J Scott
Lonoctocog alfa (rVIII-SingleChain; Afstyla(®)) is a novel single-chain recombinant factor VIII (FVIII) molecule, with a truncated B-domain and the heavy and light chains covalently linked to form a stable and homogenous drug that binds with high affinity to von Willebrand factor (VWF). Intravenous lonoctocog alfa is approved for the prophylaxis and treatment of bleeding in patients with haemophilia A in several countries worldwide. In two pivotal, multicentre trials, lonoctocog alfa was effective in the treatment of bleeding episodes and as prophylaxis, including for perioperative management in adults, adolescents and children...
October 2017: Drugs
https://www.readbyqxmd.com/read/28876509/clot-waveform-analysis-where-do-we-stand-in-2017
#7
REVIEW
P O Sevenet, F Depasse
Analysis of the optical waveform generated during global coagulation assays, such as activated partial thromboplastin time and prothrombin time, can provide much precious information on the global coagulation state of the plasma sample tested, in addition to a single clotting time. Many studies have been published concerning patient diagnosis and management in haemophilia A, and in the early diagnosis and prognosis of disseminated intravascular coagulation and sepsis. However, many other works have also been published on further potential clinical applications such as lupus anticoagulant diagnosis and anticoagulant monitoring...
September 6, 2017: International Journal of Laboratory Hematology
https://www.readbyqxmd.com/read/28871021/effectiveness-of-two-psychological-interventions-for-pain-management-emotional-regulation-and-promotion-of-quality-of-life-among-adult-portuguese-men-with-haemophilia-psy-haemopeq-study-protocol-for-a-single-centre-prospective-randomised-controlled-trial
#8
Patrícia Ribeiro Pinto, Ana Cristina Paredes, Patrício Costa, Manuela Carvalho, Manuela Lopes, Susana Fernandes, Susana Pedras, Armando Almeida
INTRODUCTION: Haemophilia is a bleeding disorder associated with significant pain, emotional distress, quality of life (QoL) impairment and considerable healthcare costs. Psychosocial health and effective pain management are considered essential end points for optimal haemophilia care, but there is a significant gap in evidence-based treatments targeting these outcomes in people with haemophilia (PWH). Psychological interventions are cost-effective in promoting emotional well-being, QoL and pain control, although these have been scarcely used in haemophilia field...
September 3, 2017: BMJ Open
https://www.readbyqxmd.com/read/28838029/radionuclide-synovectomy-synoviorthesis-rs-in-patients-with-bleeding-disorders-a-review-of-patient-and-procedure-demographics-and-functional-outcomes-in-the-athndataset
#9
C McGuinn, D Cheng, D Aschman, S L Carpenter, R Sidonio, A Soni, M D Tarantino, A P Wheeler, A L Dunn
INTRODUCTION: Radionuclide synovectomy/synoviorthesis (RS) to manage proliferative synovitis in persons with bleeding disorders has been utilized for decades; however, aggregate US results are limited. AIM: To determine the prevalence of RS utilization, patient and procedure related demographics and functional outcomes in United States haemophilia treatment centres (HTCs). The ATHNdataset includes US patients with bleeding disorders who have authorized the sharing of their demographic and clinical information for research...
November 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28835123/advances-in-gene-therapy-for-hemophilia
#10
Amit C Nathwani, Andrew M Davidoff, Edward G D Tuddenham
Gene therapy provides hope for a cure for patients with hemophilia by establishing continuous endogenous expression of factor VIII or factor IX following transfer of a functional gene copy to replace the hemophilic patient's own defective gene. Hemophilia may be considered a "low-hanging fruit" for gene therapy because a small increment in blood factor levels (≥2% of normal) significantly improves the bleeding tendency from severe to moderate, eliminating most spontaneous bleeds. After decades of research, the first trial to provide clear evidence of efficiency after gene transfer in patients with hemophilia B using adeno-associated virus vectors was reported by the authors' group in 2011...
November 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28826659/haemophilia-b-where-are-we-now-and-what-does-the-future-hold
#11
REVIEW
Gerry Dolan, Gary Benson, Anne Duffy, Cedric Hermans, Victor Jiménez-Yuste, Thierry Lambert, Rolf Ljung, Massimo Morfini, Silva Zupančić Šalek
Research has been lacking on the natural history, complications, and treatment of haemophilia B, which is less common than haemophilia A and was recognized as a distinct clinical entity in 1947. Although the two diseases share the same clinical manifestations, they differ in causative mutation, risk of inhibitor development, and patient quality of life. Frequently debated is whether haemophilia B is as clinically severe as haemophilia A, with much of the published data on overall and haemophilia-specific health outcomes suggesting that haemophilia B may have a less severe clinical phenotype...
August 16, 2017: Blood Reviews
https://www.readbyqxmd.com/read/28780770/subcutaneous-diphtheria-and-tetanus-vaccines-in-children-with-haemophilia-a-pilot-study-and-review-of-the-literature
#12
B A Schaefer, R A Gruppo, E S Mullins, C Tarango
INTRODUCTION: Subcutaneous (SQ) vaccination has emerged as standard of care in children with severe bleeding disorders to reduce unnecessary factor exposure and avoid provoking an intramuscular bleed, but little is known about comparative immunogenicity to intramuscular (IM) vaccination. AIM: To confirm immunogenicity of Diphtheria Tetanus acellular Pertussis (DTaP) vaccines administered SQ to individuals <6 years old with haemophilia. METHODS: We performed a retrospective and prospective pilot study of tetanus and diphtheria antibody titres among patients evaluated at our Haemophilia Treatment Centre between 2015-2016...
November 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28752639/perioperative-management-of-haemophilia-b-a-critical-appraisal-of-the-evidence-and-current-practices
#13
REVIEW
E J Neufeld, L Solimeno, D Quon, C Walsh, S Seremetis, D Cooper, N N Iyer, C S Hoxer, P Giangrande
BACKGROUND: While there is substantial literature addressing the principles of general management of haemophilia, literature on perioperative management of haemostasis is scarce. OBJECTIVE: The aim of this study was to better understand perioperative management among congenital haemophilia B patients (without inhibitors) and to gain insights into real-world surgical practices. METHOD: A systematic literature review, with an emphasis on haemophilia B, was conducted using EMBASE(®) , Medline(®) and the Cochrane Library...
November 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28748763/induced-pluripotent-stem-cell-technology-a-paradigm-shift-in-medical-science-for-drug-screening-and-disease-modeling
#14
Meera Nair, Sardul Singh Sandhu, Anil Kumar Sharma
BACKGROUND: Induced Pluripotent Stem Cell (IPSC) Technology is the most advanced research as it offers an attractive alternative for establishing patient-specific IPSCs to recapitulate phenotypes of not only monogenic diseases (viz. Thalassaemia, Sickle cell anemia, Haemophilia, Tay-Sachs disease), but also late-onset polygenic diseases (viz. Parkinson's disease, Alzheimer's disease, schizophrenia). Over the hindsight, numerous studies of the past and current scientists have led to the production, maturation and understanding of induced pluripotent stem cell technology and its use in basic and clinical research...
July 27, 2017: Current Medicinal Chemistry
https://www.readbyqxmd.com/read/28726798/gene-therapy-research-in-asia
#15
REVIEW
H-X Deng, Y Wang, Q-R Ding, D-L Li, Yu-Quan Wei
Gene therapy has shown great potential for the treatment of diseases that previously were either untreatable or treatable but not curable with conventional schemes. Recent progress in clinical gene therapy trials has emerged in various severe diseases, including primary immunodeficiencies, leukodystrophies, Leber's congenital amaurosis, haemophilia, as well as retinal dystrophy. The clinical transformation and industrialization of gene therapy in Asia have been remarkable and continue making steady progress...
September 7, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28646426/eftrenonacog-alfa-a-review-in-haemophilia-b
#16
Sheridan M Hoy
Eftrenonacog alfa (Alprolix™) is a recombinant fusion protein comprising human factor IX (FIX) covalently linked to the constant region (Fc) domain of human IgG1 (i.e. rFIXFc). The presence of the Fc domain extends the terminal half-life (t½) of rFIXFc, permitting prolonged treatment intervals. rFIXFc is available for intravenous use for the prophylaxis and treatment of bleeding in patients with haemophilia B. In two multinational, phase III studies in previously treated children, adolescents and adults with severe haemophilia B, rFIXFc prophylaxis resulted in low median annualized bleeding rates (ABRs), and was associated with reductions in median weekly factor consumption and dosing frequency compared with pre-study FIX regimens...
July 2017: Drugs
https://www.readbyqxmd.com/read/28636076/prevalence-and-impact-of-obesity-in-people-with-haemophilia-review-of-literature-and-expert-discussion-around-implementing-weight-management-guidelines
#17
REVIEW
S Kahan, A Cuker, R F Kushner, J Maahs, M Recht, T Wadden, T Willis, S Majumdar, D Ungar, D Cooper
Obesity affects more than 35% of Americans, increasing the risk of more than 200 comorbid conditions, impaired quality of life and premature mortality. This review aimed to summarize literature published over the past 15 years regarding the prevalence and impact of obesity in people with haemophilia (PWH) and to discuss implementing general guidelines for weight management in the context of the haemophilia comprehensive care team. Although few studies have assessed the effects of obesity on haemophilia-specific outcomes, existing evidence indicates an important impact of weight status on lower extremity joint range of motion and functional disability, with potentially important effects on overall quality of life...
November 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28574216/imaging-of-haemophilic-arthropathy-in-growing-joints-pitfalls-in-ultrasound-and-mri
#18
REVIEW
M Soliman, P Daruge, S S J Dertkigil, E De Avila Fernandes, J R Negrao, S de Aguiar Vilela Mitraud, E T I Sakuma, A R C Fernandes, N Zhang, A Huo, Y-J Li, F Zhou, B M Rodrigues, A Mohanta, V S Blanchette, A S Doria
The purpose of this review was to summarize the current knowledge on the utilization of magnetic resonance imaging (MRI) and ultrasound (US) for assessing arthropathy in children and adolescents with haemophilia and to recognize the limitations of each imaging modality and pitfalls in the diagnosis of soft tissue and osteochondral abnormalities. Awareness of MRI and US limitations and pitfalls in the assessment of joints in persons with haemophilia is essential for accurate diagnosis and optimal management of haemophilic arthropathy...
September 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28567541/gene-therapy-for-monogenic-liver-diseases-clinical-successes-current-challenges-and-future-prospects
#19
Julien Baruteau, Simon N Waddington, Ian E Alexander, Paul Gissen
Over the last decade, pioneering liver-directed gene therapy trials for haemophilia B have achieved sustained clinical improvement after a single systemic injection of adeno-associated virus (AAV) derived vectors encoding the human factor IX cDNA. These trials demonstrate the potential of AAV technology to provide long-lasting clinical benefit in the treatment of monogenic liver disorders. Indeed, with more than ten ongoing or planned clinical trials for haemophilia A and B and dozens of trials planned for other inherited genetic/metabolic liver diseases, clinical translation is expanding rapidly...
July 2017: Journal of Inherited Metabolic Disease
https://www.readbyqxmd.com/read/28499509/ten-year-study-of-postoperative-complications-following-dental-extractions-in-patients-with-inherited-bleeding-disorders
#20
J-T Hsieh, K Klein, M Batstone
Dental extractions challenge the body's haemostatic mechanism. Postoperative bleeding from dental extraction can be prolonged, or even life threatening in patients with inherited bleeding disorders. Pre- and postoperative clotting factor replacements or systemic desmopressin (ddAVP) have been advocated at our institution to prevent bleeding complications in these patients. This study aimed to assess the postoperative bleeding rate in patients with inherited bleeding disorders that underwent dental extractions at our institution between 2003 and 2012...
September 2017: International Journal of Oral and Maxillofacial Surgery
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