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https://www.readbyqxmd.com/read/28726798/gene-therapy-research-in-asia
#1
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/28722832/the-outcomes-of-haemophilia-and-its-treatment-why-we-need-a-core-set
#2
EDITORIAL
B M Feldman
No abstract text is available yet for this article.
July 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28722821/sensory-strategies-of-postural-sway-during-quiet-stance-in-patients-with-haemophilic-arthropathy
#3
C Cruz-Montecinos, C De la Fuente, G Rivera-Lillo, S Morales-Castillo, V Soto-Arellano, F Querol, S Pérez-Alenda
INTRODUCTION: The sensory strategies of postural control in adult haemophilic arthropathy patients are still poorly understood. AIM: To determine sensorial posture-control strategies through postural sway frequency analysis when in a bipedal quiet stance with and without visual stimulus deprivation in healthy subjects and patients with haemophilic arthropathy. Secondarily, to determine the irregularity of postural balance control through sample entropy (SampEn)...
July 19, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28713102/-contemporary-use-of-radiosynoviorthesis-in-chronic-polyarthrtitis
#4
Robert Zwolak, Maria Majdan
Radiosynoviorthesis is used for local treatment of recurrent joint effusions, leads to necrosis of inflamed synovium due to beta radiation energy served after intraarticular radionuclide administration. The aim of the therapy is destruction and fibrosis of abnormal, hypertrophic synovial membrane and then full recovery of its normal function after local corticosteroids and systemic modifying drugs failure. Radiosynoviorthesis is effective in different type of peripheral arthritis like rheumatoid arthritis, inflammatory spondyloarthtropaties, gout, chondrocalcinosis, pigmented villo-nodular synovitis, recurrent knee effusion after total joint replacement, idiopathic knee joint effusion, osteoarthritis and secondary prevention of intraarticular bleeding in haemophilia...
2017: Wiadomości Lekarskie: Organ Polskiego Towarzystwa Lekarskiego
https://www.readbyqxmd.com/read/28705716/occult-hepatitis-b-virus-infection-in-greek-patients-with-congenital-bleeding-disorders
#5
Agoritsa Varaklioti, Anna Kouramba, Panagiota Ioannidou, Olga Katsarou
Occult hepatitis B infection (OBI) is a form of chronic HBV infection characterized by low level HBV DNA, without detectable HBV surface antigen (HBsAg). OBI is frequently associated with the presence of anti-HBc and in some cases also with anti-HBs. Patients, who formerly received non-inactivated factor concentrates, can potentially be considered at high risk for OBI, especially since these patients usually are HIV or HCV co-infected. This study aimed to assess the prevalence of occult HBV infection in Greek patients with hereditary bleeding disorders...
July 10, 2017: Infection, Genetics and Evolution
https://www.readbyqxmd.com/read/28699675/intracranial-haemorrhage-in-children-and-adolescents-with-severe-haemophilia-a-or-b-the-impact-of-prophylactic-treatment
#6
Nadine G Andersson, Günter Auerswald, Chris Barnes, Manuel Carcao, Amy L Dunn, Karin Fijnvandraat, Marianne Hoffmann, Kaan Kavakli, Gili Kenet, Rainer Kobelt, Karin Kurnik, Ri Liesner, Anne Mäkipernaa, Marilyn J Manco-Johnson, Maria E Mancuso, Angelo C Molinari, Beatrice Nolan, Rosario Perez Garrido, Pia Petrini, Helen E Platokouki, Amy D Shapiro, Runhui Wu, Rolf Ljung
The discussion of prophylactic therapy in haemophilia is largely focused on joint outcomes. The impact of prophylactic therapy on intracranial haemorrhage (ICH) is less known. This study aimed to analyse ICH in children with haemophilia, with a focus on different prophylaxis regimens and sequelae of ICH. We conducted a multicentre retrospective and prospective study that included 33 haemophilia centres from 20 countries. Inclusion criteria were children and adolescents born between 1993 and 2014, with severe haemophilia A or B without inhibitors...
July 12, 2017: British Journal of Haematology
https://www.readbyqxmd.com/read/28698745/new-surgical-technologies-could-facilitate-surgical-hemostasis-in-hemophilic-patients
#7
Milcho J Panovski, Igor V Fildishevski, Ljubomir Lj Ognjenovic, Violeta I Dejanova-Ilijevska
BACKGROUND: It's assumed that surgery in haemophilia can be accomplished these days safely. AIM: The aim of this study was to investigate the influence of new surgical technologies in the perioperative management and outcome of surgical procedures in haemophiliacs. METHODS: Two patients with mild haemophilia A underwent surgery (laparoscopic appendectomy and inguinal hernia repair). In both patients, the replacement therapy, with factor VIII, started 30 min before surgery...
June 15, 2017: Open Access Macedonian Journal of Medical Sciences
https://www.readbyqxmd.com/read/28698538/warfarin-should-not-be-used-for-thromboprohylaxis-in-elective-major-orthopaedic-surgery-a-croatian-perspective
#8
Robert Kolundžić, Marijana Šimić Jovičić, Maja Ðinkić, Tadija Petrović, Tomislav Crnković, Vladimir Trkulja
Aim To identify modes of venous thromboembolism (VTE) prophylaxis in patients undergoing elective major orthopaedic surgery (total hip or knee arthroplasty, THA/TKA) at a single university-associated hospital in Croatia. Methods A retrospective analysis of consecutive patients subjected to THA or TKA over a two-year period (2014-2015) with a focus on anticoagulation during the first 15 post-surgical days (period of highest VTE risk). Results Of 603 identified patients three (0.5%) were not anticoagulated (haemophilia) and others received perioperative doses of low molecular weight heparins (LMWH)...
August 1, 2017: Medicinski Glasnik
https://www.readbyqxmd.com/read/28692108/safety-and-efficacy-of-a-glycopegylated-rfviii-turoctocog-alpha-pegol-n8-gp-in-paediatric-patients-with-severe-haemophilia-a
#9
Sandrine Meunier, Jayanthi Alamelu, Silke Ehrenforth, Hideji Hanabusa, Faraizah Abdul Karim, Kaan Kavakli, Melanie Khodaie, Janice Staber, Oleksandra Stasyshyn, Donald L Yee, Lina Rageliene
Turoctocog alfa pegol (N8-GP, Novo Nordisk, Bagsværd, Denmark), an extended half-life glycoPEGylated recombinant factor VIII (rFVIII), is being developed for prophylaxis and treatment of bleeds in haemophilia A patients. pathfinder™5 is a multinational, open-label, single-arm trial to assess safety, efficacy and pharmacokinetics of N8-GP in paediatric (<12 years), previously treated patients. Boys with severe haemophilia A (<1 % FVIII), no history of inhibitors and previously treated with FVIII products (>50 exposure days [ED] for patients aged 0-5 years [younger cohort]; >150 ED for patients aged 6-11 years [older cohort]) were included...
July 6, 2017: Thrombosis and Haemostasis
https://www.readbyqxmd.com/read/28689372/deficits-of-ankle-muscle-strength-not-found-in-children-adolescents-and-young-adults-with-haemophilic-ankle-arthropathy
#10
S Lobet, J-L Croisier, A-C Lantin, C Hermans, K Peerlinck, J Vandesande, J-B Pialat, K Deschamps
OBJECTIVES: Adequate management of haemophilia patients requires early detection of joint impairment in relatively asymptomatic patients. This study sought to quantify the impact of the ankle's structural impairment on muscle strength in children, adolescent and young adults with haemophilia (CAAwH). METHODS: Twenty-three CAAwH underwent bilateral magnetic resonance imaging (MRI) assessing the anatomical status of tibiotalar joint (TTJ) and subtalar joint (STJ) using the International Prophylaxis Study Group MRI scale...
July 9, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28686157/the-role-of-recombinant-activated-factor-vii-in-the-haematological-management-of-elective-orthopaedic-surgery-in-haemophilia-a-patients-with-inhibitors
#11
REVIEW
Giancarlo Castaman
The clinical profile and expectations of haemophilic patients with inhibitors have changed over the last three decades, mainly because of the prolongation of life-expectancy, often resulting in an increase of the orthopaedic burden. Recombinant activated factor VII (rFVIIa) is the most frequently used bypassing agent in haemophilia patients with inhibitors during elective orthopaedic surgery. For nearly 30 years, rFVIIa has been successfully used to control haemostasis in several major and minor surgical procedures...
May 16, 2017: Blood Transfusion, Trasfusione del Sangue
https://www.readbyqxmd.com/read/28686156/high-titre-inhibitors-in-previously-untreated-patients-with-severe-haemophilia-a-receiving-recombinant-or-plasma-derived-factor-viii-a-budget-impact-analysis
#12
Andrea Messori, Flora Peyvandi, Sabrina Trippoli, Roberta Palla, Frits R Rosendaal, Pier Mannuccio Mannucci
No abstract text is available yet for this article.
May 15, 2017: Blood Transfusion, Trasfusione del Sangue
https://www.readbyqxmd.com/read/28678027/meta-analysis-on-incidence-of-inhibitors-in-patients-with-haemophilia-a-treated-with-recombinant-factor-viii-products
#13
Matteo Rota, Paolo A Cortesi, Katharina N Steinitz-Trost, Armin J Reininger, Alessandro Gringeri, Lorenzo G Mantovani
: Recent cohort studies showed differences in inhibitor incidence in previously untreated patients (PUPs) with haemophilia A treated with recombinant factor VIII (rFVIII) concentrates. We carried out a systematic literature search and meta-analysis for all randomized clinical trials and observational studies published from 1 January 1988 to 31 August 2015, to assess the incidence of inhibitor development and the relationship with rFVIII product used in PUPs and minimally treated patients (MTPs, ≤5 previous exposure days), with severe haemophilia...
July 3, 2017: Blood Coagulation & Fibrinolysis: An International Journal in Haemostasis and Thrombosis
https://www.readbyqxmd.com/read/28677841/unexpected-haemophilia-despite-pre-natal-testing-a-combined-haemophilia-a-and-haemophilia-b-family
#14
Noémi B A Roy, Nicola Curry, David Keeling
No abstract text is available yet for this article.
July 5, 2017: British Journal of Haematology
https://www.readbyqxmd.com/read/28646426/eftrenonacog-alfa-a-review-in-haemophilia-b
#15
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/28646206/crispr-cas9-mediated-genome-editing-via-postnatal-administration-of-aav-vector-cures-haemophilia-b-mice
#16
Tsukasa Ohmori, Yasumitsu Nagao, Hiroaki Mizukami, Asuka Sakata, Shin-Ichi Muramatsu, Keiya Ozawa, Shin-Ichi Tominaga, Yutaka Hanazono, Satoshi Nishimura, Osamu Nureki, Yoichi Sakata
Haemophilia B, a congenital haemorrhagic disease caused by mutations in coagulation factor IX gene (F9), is considered an appropriate target for genome editing technology. Here, we describe treatment strategies for haemophilia B mice using the clustered regularly interspaced short palindromic repeat (CRISPR)/Cas9 system. Administration of adeno-associated virus (AAV) 8 vector harbouring Staphylococcus aureus Cas9 (SaCas9) and single guide RNA (sgRNA) to wild-type adult mice induced a double-strand break (DSB) at the target site of F9 in hepatocytes, sufficiently developing haemophilia B...
June 23, 2017: Scientific Reports
https://www.readbyqxmd.com/read/28636264/desmopressin-in-haemophilia-the-need-for-a-standardised-clinical-response-and-individualised-test-regimen
#17
S C M Stoof, L M Schütte, F W G Leebeek, M H Cnossen, M J H A Kruip
INTRODUCTION: Due to interindividual variation in desmopressin response, non-severe haemophilia A patients require desmopressin testing prior to therapeutic treatment. However, adequate response or frequency of blood sampling is not standardised in international guidelines. Consequently, various definitions and blood sampling protocols are currently applied. Interestingly, sustainability of desmopressin response is not incorporated into these definitions. AIM: To study desmopressin response rates in a cohort of non-severe haemophilia A patients using currently accepted desmopressin response definitions...
June 21, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28636084/co-morbidities-and-bleeding-in-elderly-patients-with-haemophilia-a-survey-of-the-german-austrian-and-swiss-society-of-thrombosis-and-haemostasis-research-gth
#18
W Miesbach, S-E Reitter-Pfoertner, R Klamroth, F Langer, H-H Wolf, A Tiede, B Siegmund, U Scholz, P R Müller, H Eichler, I Pabinger
BACKGROUND: Nowadays patients with haemophilia survive longer due to improvements in haemophilia care. It has been hypothesized that the bleeding type and frequency may vary with age and are influenced by co-morbidities and co-medication in elderly patients. OBJECTIVES: To investigate a large group of patients older than 60 years of age with haemophilia concerning haemophilia treatment, bleeding pattern changes, co-morbidities, co-medication, bleeding sites and patient mortality...
June 21, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
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
#19
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/28621454/adult-males-with-haemophilia-have-a-different-macrovascular-and-microvascular-endothelial-function-profile-compared-with-healthy-controls
#20
H Sun, M Yang, M Fung, S Chan, M Jawi, T Anderson, M-C Poon, S Jackson
INTRODUCTION: Endothelial function has been identified as an independent predictor of cardiovascular risk in the general population. It is unclear if the haemophilia population has a different endothelial function profile compared to the healthy population. AIM: This prospective study aims to assess if there is a difference in endothelial function between haemophilia patients and healthy controls, and the impact of endothelial function on vascular outcomes in the haemophilia population...
June 16, 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
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