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https://www.readbyqxmd.com/read/28088606/choice-of-factor-viii-ix-regimen-in-adolescents-and-young-adults-with-severe-or-moderately-severe-haemophilia-a-french-national-observational-study-orthem-15-25
#1
Sandrine Meunier, Roseline d'oiron, Hervé Chambost, Edita Dolimier, Benoît Guillet
INTRODUCTION: The value and challenges of long-term prophylaxis (LTP) in adolescents and young adults need further characterisation. AIM: To determine the proportions of adolescents and young adults with severe or moderately severe haemophilia in France under LTP and treatment on demand (OD). METHODS: Patients 15 to 25years old with haemophilia A or B, factor VIII/IX ≤2% and no current inhibitor could be included if they had been under factor VIII/IX treatment at least 12months and kept a treatment and bleeding diary...
December 28, 2016: Thrombosis Research
https://www.readbyqxmd.com/read/28073995/a-pericentric-inversion-of-chromosome-x-disrupting-f8-and-resulting-in-haemophilia-a
#2
Yu Xin, Jingyi Zhou, Qiulan Ding, Changming Chen, Xi Wu, Xuefeng Wang, Hongli Wang, Xiaofeng Jiang
AIMS: The frequency of X chromosome pericentric inversion is much less than that of autosome chromosome. We hereby characterise a pericentric inversion of X chromosome associated with severe factor VIII (FVIII) deficiency in a sporadic haemophilia A (HA) pedigree. METHODS: PCR primer walking and genome walking strategies were adopted to identify the exact breakpoints of the inversion. Copy number variations (CNVs) of the F8 and the whole chromosomes were detected by AccuCopy and Affymetrix CytoScan High Definition (HD) assays, respectively...
January 10, 2017: Journal of Clinical Pathology
https://www.readbyqxmd.com/read/28026074/measurement-properties-of-the-haem-a-qol-in-haemophilia-clinical-trials
#3
S von Mackensen, A Eldar-Lissai, P Auguste, S Krishnan, R von Maltzahn, R Yu, K W Wyrwich
INTRODUCTION: Patients with haemophilia on long-acting prophylactic treatment may experience an improvement in health-related quality of life (HRQoL) through reductions in breakthrough bleeds and associated complications, including long-term joint damage, compared with episodic treatment. AIM: This analysis examined clinical trial data to understand the psychometric characteristics (reliability, validity and sensitivity to change over time) of the Haem-A-QoL Questionnaire in adult males with haemophilia...
December 27, 2016: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28026073/use-of-the-ukhcdo-database-for-a-postmarketing-surveillance-study-of-different-doses-of-recombinant-factor-viia-in-haemophilia
#4
C R M Hay, T Sharpe, G Dolan
INTRODUCTION: Recombinant factor VIIa (rFVIIa) is recommended in Europe at standard (3 × 90 μg kg(-1) ) or high (1 × 270 μg kg(-1) ) doses. When granting the license for the high dose, the European Medicines Agency (EMA) requested postmarketing surveillance for thrombosis. This was conducted by the United Kingdom National Haemophilia Database (NHD) on behalf of Novo Nordisk and the EMA. AIM: To assess the use and safety of rFVIIa utilizing prospective data collected by the NHD (1 January 2008 to 30 June 2011)...
December 27, 2016: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/28007939/single-synonymous-mutation-in-factor-ix-alters-protein-properties-and-underlies-haemophilia-b
#5
Vijaya L Simhadri, Nobuko Hamasaki-Katagiri, Brian C Lin, Ryan Hunt, Sujata Jha, Sandra C Tseng, Andrew Wu, Amber A Bentley, Ran Zichel, Qi Lu, Lily Zhu, Darón I Freedberg, Dougald M Monroe, Zuben E Sauna, Robert Peters, Anton A Komar, Chava Kimchi-Sarfaty
BACKGROUND: Haemophilia B is caused by genetic aberrations in the F9 gene. The majority of these are non-synonymous mutations that alter the primary structure of blood coagulation factor IX (FIX). However, a synonymous mutation c.459G>A (Val107Val) was clinically reported to result in mild haemophilia B (FIX coagulant activity 15%-20% of normal). The F9 mRNA of these patients showed no skipping or retention of introns and/or change in mRNA levels, suggesting that mRNA integrity does not contribute to the origin of the disease in affected individuals...
December 22, 2016: Journal of Medical Genetics
https://www.readbyqxmd.com/read/28004057/long-term-safety-and-efficacy-of-extended-interval-prophylaxis-with-recombinant-factor-ix-fc-fusion-protein-rfixfc-in-subjects-with-haemophilia-b
#6
K John Pasi, Kathelijn Fischer, Margaret Ragni, Beatrice Nolan, David J Perry, Roshni Kulkarni, Margareth Ozelo, Johnny Mahlangu, Amy D Shapiro, Ross I Baker, Carolyn M Bennett, Christopher Barnes, Johannes Oldenburg, Tadashi Matsushita, Huixing Yuan, Alejandra Ramirez-Santiago, Glenn F Pierce, Geoffrey Allen, Baisong Mei
The safety, efficacy, and prolonged half-life of recombinant factor IX Fc fusion protein (rFIXFc) were demonstrated in the Phase 3 B-LONG (adults/adolescents ≥12 years) and Kids B-LONG (children <12 years) studies of subjects with haemophilia B (≤2 IU/dl). Here, we report interim, long-term safety and efficacy data from B-YOND, the rFIXFc extension study. Eligible subjects who completed B-LONG or Kids B-LONG could enrol in B-YOND. There were four treatment groups: weekly prophylaxis (20-100 IU/kg every 7 days), individualised prophylaxis (100 IU/kg every 8-16 days), modified prophylaxis (further dosing personalisation to optimise prophylaxis), and episodic (on-demand) treatment...
December 22, 2016: Thrombosis and Haemostasis
https://www.readbyqxmd.com/read/27996087/gene-therapy-for-haemophilia
#7
REVIEW
Akshay Sharma, Manu Easow Mathew, Vasumathi Sriganesh, Ulrike M Reiss
BACKGROUND: Haemophilia is a genetic disorder characterized by spontaneous or provoked, often uncontrolled, bleeding into joints, muscles and other soft tissues. Current methods of treatment are expensive, challenging and involve regular administration of clotting factors. Gene therapy has recently been prompted as a curative treatment modality. This is an update of a published Cochrane Review. OBJECTIVES: To evaluate the safety and efficacy of gene therapy for treating people with haemophilia A or B...
20, 2016: Cochrane Database of Systematic Reviews
https://www.readbyqxmd.com/read/27995727/kinetic-analysis-and-binding-studies-of-a-new-recombinant-human-factor-viia-for-treatment-of-haemophilia
#8
J Grandoni, G Perret, C Forier
INTRODUCTION/AIM: LR769 is a new second-generation recombinant human Factor VIIa (rhFVIIa) developed for haemophilia treatment. We determined enzymatic properties of LR769 and its interaction with antithrombin, tissue factor, platelets and endothelial protein C receptor (EPCR), compared with NovoSevenRT. METHODS: Kinetic enzyme assays and active site titration were used for enzymatic studies. Surface Plasmon Resonance (SPR) was used for determination of binding constants...
December 19, 2016: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/27995680/bay-81-8973-a-full-length-recombinant-factor-viii-manufacturing-processes-and-product-characteristics
#9
REVIEW
S Garger, J Severs, L Regan, A Hesslein, J Ignowski, P Wu, E Long, S Gupta, S Liu, W Wang
BAY 81-8973 (Kovaltry(®) , Bayer, Berkeley, CA, USA) is an unmodified, full-length recombinant human factor VIII (FVIII) approved for prophylaxis and on-demand treatment of bleeding episodes in patients with haemophilia A. The BAY 81-8973 manufacturing process is based on the process used for sucrose-formulated recombinant FVIII (rFVIII-FS), with changes and enhancements made to improve production efficiency, further augment pathogen safety, and eliminate animal- and human-derived raw materials from the production processes...
December 19, 2016: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/27992070/exercise-for-haemophilia
#10
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...
19, 2016: Cochrane Database of Systematic Reviews
https://www.readbyqxmd.com/read/27990785/inhibitor-development-in-haemophilia
#11
F Peyvandi, M Makris
No abstract text is available yet for this article.
January 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/27990784/new-findings-on-inhibitor-development-from-registries-to-clinical-studies
#12
REVIEW
F Peyvandi, C E Ettingshausen, J Goudemand, V Jiménez-Yuste, E Santagostino, M Makris
The high incidence of inhibitors against factor VIII (FVIII) concentrates in patients with haemophilia A has encouraged debate as to whether product-type plays a role. There is debate in the literature as to whether rFVIII concentrates are associated with a higher incidence of inhibitors compared to pdFVIII products. The management of haemophilia in patients with inhibitors includes on-demand/prophylaxis treatment with bypassing agents, and/or immune tolerance induction (ITI). However, these options create an economic and emotional burden on patients, their families and healthcare practitioners...
January 2017: Haemophilia: the Official Journal of the World Federation of Hemophilia
https://www.readbyqxmd.com/read/27988873/coagulation-factor-ix-recombinant-albumin-fusion-protein-albutrepenonacog-alfa-idelvion-%C3%A2-a-review-of-its-use-in-haemophilia-b
#13
Katherine A Lyseng-Williamson
Albutrepenonacog alfa (Idelvion(®)), a fusion protein that genetically fuses recombinant factor IX (rFIX) with recombinant human albumin (rAlbumin), is indicated in the treatment of haemophilia B. This narrative review discusses the pharmacological properties and clinical data related to the use of this novel fusion protein, hereafter referred to as rIX-FP. The fusion of rFIX to rAlbumin prolongs the elimination half-life of rIX-FP in the circulation, allowing routine prophylaxis to be administered once every 7-14 days...
December 17, 2016: Drugs
https://www.readbyqxmd.com/read/27976591/efficacy-of-pulsed-high-intensity-laser-therapy-on-pain-functional-capacity-and-gait-in-children-with-haemophilic-arthropathy
#14
Shamekh Mohamed El-Shamy, Ashraf Abdelaal Mohamed Abdelaal
AIM: The aim of this study was to evaluate the effects of pulsed high-intensity laser therapy (HILT) on pain, functional capacity, and gait in children with haemophilia. METHODS: Thirty children with haemophilia type A with ages ranging from 9 to 13 years were selected for this study. They were assigned randomly, into two equal treatment groups. The laser group received the traditional physical therapy programme plus active laser (total energy of 1500 J through three phases/3 sessions/week), whereas the placebo group received the same physical therapy programme plus placebo laser over three consecutive months...
December 15, 2016: Disability and Rehabilitation
https://www.readbyqxmd.com/read/27971804/review-of-national-health-technology-assessment-reports-on-treatment-of-severe-haemophilia-a
#15
I Odnoletkova
No abstract text is available yet for this article.
November 2016: Value in Health: the Journal of the International Society for Pharmacoeconomics and Outcomes Research
https://www.readbyqxmd.com/read/27971789/cost-effectiveness-of-prophylaxis-compared-to-on-demand-treatment-in-severe-haemophilia-a-systematic-review
#16
I Odnoletkova
No abstract text is available yet for this article.
November 2016: Value in Health: the Journal of the International Society for Pharmacoeconomics and Outcomes Research
https://www.readbyqxmd.com/read/27971756/the-impact-of-haemophilia-complications-on-health-related-quality-of-life-for-adults-with-severe-haemophilia
#17
G Mazza, J O'Hara, L Carroll, C Camp, C Stentoft Hoxer, L Wilkinson
No abstract text is available yet for this article.
November 2016: Value in Health: the Journal of the International Society for Pharmacoeconomics and Outcomes Research
https://www.readbyqxmd.com/read/27971747/health-status-in-patients-with-severe-haemophilia-a-according-to-treatment-regimen-and-age
#18
C Hirst, J Black, A Elnoursi, J O'Hara
No abstract text is available yet for this article.
November 2016: Value in Health: the Journal of the International Society for Pharmacoeconomics and Outcomes Research
https://www.readbyqxmd.com/read/27971742/cost-utility-of-prophylaxis-vs-on-demand-treatment-in-severe-haemophilia-a-patients
#19
Z Gharibnaseri, M Davari, A Cheraghali, P Eshghi, R Ravanbod, R Spendar, J Khedmati
No abstract text is available yet for this article.
November 2016: Value in Health: the Journal of the International Society for Pharmacoeconomics and Outcomes Research
https://www.readbyqxmd.com/read/27971734/a-cost-effectiveness-analysis-of-pharmacokinetic-pk-driven-prophylaxis-vs-standard-prophylaxis-in-haemophilia-a
#20
P Cortesi, S Iannazzo, R Crea, K Steinitz, A Gringeri, L G Mantovani
No abstract text is available yet for this article.
November 2016: Value in Health: the Journal of the International Society for Pharmacoeconomics and Outcomes Research
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