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haemophilia A review

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https://www.readbyqxmd.com/read/28900904/lonoctocog-alfa-a-review-in-haemophilia-a
#1
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...
September 12, 2017: Drugs
https://www.readbyqxmd.com/read/28876509/clot-waveform-analysis-where-do-we-stand-in-2017
#2
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
#3
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
#4
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...
August 24, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28835123/advances-in-gene-therapy-for-haemophilia
#5
Amit C Nathwani, Andrew M Davidoff, Edward G D Tuddenham
Gene therapy provides hope for a cure for patients with haemophilia by establishing continuous endogenous expression of factor VIII or factor IX following transfer of a functional gene copy to replace the haemophilic patient's own defective gene. Haemophilia 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 haemophilia B using adeno-associated viral (AAV) vectors was reported by our group in 2011...
August 23, 2017: Human Gene Therapy
https://www.readbyqxmd.com/read/28826659/haemophilia-b-where-are-we-now-and-what-does-the-future-hold
#6
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
#7
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...
August 6, 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
#8
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...
July 27, 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
#9
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
#10
REVIEW
Hong-Xin Deng, Yuan Wang, Qiu-Rong Ding, Da-Li 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...
July 20, 2017: Gene Therapy
https://www.readbyqxmd.com/read/28646426/eftrenonacog-alfa-a-review-in-haemophilia-b
#11
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
#12
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...
June 21, 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
#13
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...
June 2, 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
#14
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
#15
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
https://www.readbyqxmd.com/read/28429867/health-related-quality-of-life-questionnaires-in-individuals-with-haemophilia-a-systematic-review-of-their-measurement-properties
#16
REVIEW
P F Limperg, C B Terwee, N L Young, V E Price, S C Gouw, M Peters, M A Grootenhuis, V Blanchette, L Haverman
BACKGROUND: The evaluation of health related quality of life (HRQOL) is essential for a full assessment of the influence of an illness on patients' lives. The aim of this paper is to critically appraise and compare the measurement properties of HRQOL questionnaires studied in haemophilia. METHODS: Bibliographic databases (Embase, Medline, Cinahl and PsycInfo) were searched for articles evaluating measurement properties of HRQOL questionnaires in haemophilia. Articles were excluded that did not report HRQOL measurement properties, or when <50% of the study population had haemophilia...
July 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28429865/differential-effects-of-bleeds-on-the-development-of-arthropathy-basic-and-applied-issues
#17
REVIEW
L F D van Vulpen, S C Mastbergen, F P J G Lafeber, R E G Schutgens
Haemophilia is characterized by a spontaneous bleeding tendency, affecting mainly the synovial joints. Recurrent joint bleeds induce a cascade of inflammatory as well as degenerative processes injuring synovium, cartilage and bone. These processes affect each other and may occur in parallel and/or sequentially. Clinically, the effects of joint bleeds are heterogeneous. A marked variability in joint damage is observed in patients with a similar bleeding history. Also late stage effects differ with some patients developing chronic synovitis, and others suffering from osteochondral degeneration called haemophilic arthropathy...
April 21, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28424853/long-term-results-of-ankle-arthrodesis-in-children-and-adolescents-with-haemophilia
#18
Nicolas de l'Escalopier, Alina Badina, Jean Paul Padovani, Annie Harroche, Laurent Frenzel, Philippe Wicart, Christophe Glorion, Chantal Rothschild
INTRODUCTION: Ankle arthrodesis in patients with haemophilia is a joint-non-preserving treatment option often resulting in substantial pain relief and termination of haemarthrosis by osseous consolidating of the joint. However, limited data are available regarding ankle arthrodesis in young patients. AIM: The aim of this study was to evaluate the long-term clinical and radiographic results with a minimum follow-up of ten years in children with haemophilia who underwent ankle arthrodesis...
August 2017: International Orthopaedics
https://www.readbyqxmd.com/read/28420167/treatment-and-prevention-of-bleeds-in-haemophilia-patients-with-inhibitors-to-factor-viii-ix
#19
REVIEW
Angiola Rocino, Massimo Franchini, Antonio Coppola
The development of alloantibodies neutralising therapeutically administered factor (F) VIII/IX (inhibitors) is currently the most severe complication of the treatment of haemophilia. When persistent and at a high titre, inhibitors preclude the standard replacement treatment with FVIII/FIX concentrates, making patients' management challenging. Indeed, the efficacy of bypassing agents, i.e., activated prothrombin complex concentrates (aPCC) and recombinant activated factor VII (rFVIIa), needed to overcome the haemostatic interference of the inhibitor, is not comparable to that of factor concentrates...
April 17, 2017: Journal of Clinical Medicine
https://www.readbyqxmd.com/read/28397753/hip-arthropathy-in-haemophilia
#20
Christian Carulli, Anna Rosa Rizzo, Massimo Innocenti
Hip arthropathy in haemophilic patients is disabling for hip and other common target joints. Even if bleedings in the hip are not frequent, femoroacetabular alterations may affect the functional ability of patients at a very young age. A haematologic prophylaxis combined with an adequate lifestyle and regular and low-traumatic physical activity are the keys to preventing such arthropathy. In the early stages of arthropathy, anti-inflammatory drugs and physical therapy may be sufficient to limit its progression...
April 8, 2017: Journal of Clinical Medicine
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