Low Dose Prophylaxis vis-a-vis on-Demand Treatment Strategies for Hemophilia: A Cost Effective and Disability Attenuating Approach.

Low Dose Prophylaxis vis-a-vis on-Demand Treatment Strategies for Hemophilia: A Cost Effective and Disability Attenuating Approach. J Assoc Physicians India. 2019 Nov;67(11):52-55 Authors: Singh A, Mehta S, Goyal LK, Mehta S, Sharma BS Abstract Aim: To assess effect of low dose prophylaxis in hemophilics in terms of bleeding, joint function, QoL and cost-effectiveness. Methods: Analytic study done during one year among 70 adult hemophilics. In observation period (12 weeks), on-demand factor and during prophylaxis (12 weeks), low dose factor was given (Factor VIII 10 IU/KgBW biweekly for haemophilia A and Factor IX 20 IU/KgBW weekly for haemophilia B). Clinical joint assessment was done by Gilbert score and improvement by WFH definitions. Results: Bleed reduced by 68.99% in moderate hemophilics (40 v/s 129) and 64.86% in severe hemophilics (26 v/s74) (p
Source: Journal of the Association of Physicians of India - Category: General Medicine Tags: J Assoc Physicians India Source Type: research

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AbstractHemophilia is an inherited bleeding disorder which causes impaired blood clotting. The severity of disease depends on the type of Hemophilia, level of clotting factor concentrate (CFC), phenotypic heterogeneity and the development of inhibitors. The currently accepted standard of care of this disease is prophylaxis therapy (PT) with CFC. Prophylaxis therapy for Hemophilia is given in developed countries for the last few decades. On the contrary, episodic therapy (ET) is still the mode of treatment in middle and low income countries. ET is documented to have several potential risks such as increased bleeding rate, d...
Source: Indian Journal of Hematology and Blood Transfusion - Category: Hematology Source Type: research
Abstract Hemophilia A is an X-linked recessive hemorrhagic disorder caused by variants in the F8 gene. To identify known and novel causative variants in hemophilia A, we have carried out genetic analysis among Saudi patients. Twenty-one patients, who were negative for inv-1/inv-22, were selected for analysis by next generation sequencing, thereafter confirmed by Sanger sequencing. In addition, the functionality and structural changes in the variant proteins were assessed using Molecular dynamics (MD) simulation and compared with wild-type and native proteins. In the samples we analyzed, we found 10 variants in 12 ...
Source: Acta Biochim Pol - Category: Biochemistry Authors: Tags: Acta Biochim Pol Source Type: research
Conclusions: Current treatments have reduced life-threatening bleeds, chronic joint disease and disability; however, people with haemophilia B continue to have significantly decreased quality of life. This study has shown a single infusion of low dose FLT180a leads to FIX levels of>40%, greatly reducing the risk of spontaneous or traumatic bleeds and raises the prospect of achieving a functional cure by normalising FIX levels with a higher dose. This will reduce the risk of life-threatening bleeds following trauma and surgery, transform quality of life and thus represents a major therapeutic advance for people with haem...
Source: Blood - Category: Hematology Authors: Tags: 322. Disorders of Coagulation or Fibrinolysis: Hemophilia: Emerging Therapies Source Type: research
CONCLUSION: In examining recruits into the present trial, it is clear that inhibitor patients have generally worse functional scores than either of two reference groups. Effective long-term prophylaxis is expected to produce measurable improvement in QOL scores in this hard-to-treat population.DisclosuresBooth: Catalyst Biosciences: Consultancy. Mahlangu: Sanofi: Research Funding, Speakers Bureau; Roche: Consultancy, Research Funding, Speakers Bureau; LFB: Consultancy; NovoNordisk: Consultancy, Research Funding, Speakers Bureau; CSL Behring: Consultancy, Research Funding, Speakers Bureau; Chugai: Consultancy; Catalyst Bios...
Source: Blood - Category: Hematology Authors: Tags: 322. Disorders of Coagulation or Fibrinolysis Source Type: research
Conclusions: The model suggests that prophylaxis treatment of HA with emicizumab can result in a lower number of bleeding events as well as delayed onset of arthropathy and inhibitor development. Findings indicate that prophylaxis with emicizumab leads to better patient outcomes and has a lower financial impact from the US societal perspective.DisclosuresMahajerin: Genentech Inc.: Consultancy. Zhou: Analysis Group, Inc.: Employment; Celgene Corporation: Research Funding. Raimundo: Genentech Inc: Employment, Other: Ownership interests PLC. Patel: Genentech Inc: Employment. Han: Analysis Group Inc.: Research Funding. Ji: Ana...
Source: Blood - Category: Hematology Authors: Tags: 901. Health Services Research-Non-Malignant Conditions: Poster II Source Type: research
Authors: Young G, Callaghan M, Dunn A, Kruse-Jarres R, Pipe S Abstract INTRODUCTION: Hemophilia is a serious bleeding disorder characterized by repeated bleeding episodes often into joints and muscles which can lead to permanent disabilities. Treatment with factor replacement therapy has proven to be effective at preventing these complications, however it can lead to formation of neutralizing antibodies termed inhibitors which significantly complicate the management of the disorder. These inhibitor patients suffer from increased morbidity and mortality and there has been a major unmet need for novel therapeutic app...
Source: Expert Review of Hematology - Category: Hematology Tags: Expert Rev Hematol Source Type: research
This study was initially funded by grants from the Medical Research Council of Canada/Pharmaceutical Manufacturers Association of Canada Partnership Fund and the Bayer/Canadian Blood Services/Hema-Quebec Partnership Fund. Subsequent renewals were funded by Bayer.
Source: The Lancet Haematology - Category: Hematology Source Type: research
This study aims at the detection of subclinical knee-joint involvement by USG, in patients with moderate to severe Haemophilia. 27 patients suffering from moderate and severe Haemophilia and 27 age-matched controls were studied. USG of bilateral knee joints was done to analyse cartilage and synovial thickness, synovial vascularity and resistive index of vascular flow along with synovial collection. The relevant clinical parameters (age at diagnosis and study enrolment, severity of haemophilia A, annualized bleeding rates, total number of joint bleeds, spontaneous and provoked bleed, number of episodes treated with factor V...
Source: Indian Journal of Hematology and Blood Transfusion - Category: Hematology Source Type: research
By ANISH KOKA Frances Oldham Kelsey by all accounts was not mean to have a consequential life.  She was born in Canada in 1914, at a time women were meant to be seen and not heard.  Nonetheless, an affinity for science eventually lead to a masters in pharmacology from the prestigious McGill University.  Her first real break came after she was accepted for PhD level work in the pharmacology lab of a professor at the University of Chicago.  An esteemed professor was starting a pharmacology lab and needed assistants, and the man from Canada seemed to have a perfect resume to fit.  That’s right, ...
Source: The Health Care Blog - Category: Consumer Health News Authors: Tags: Uncategorized Source Type: blogs
Scientists around the world congratulate the team that has made a major advance in finding a cure for the life-threatening blood disorderScientists believe they are on the way to finding a cure for haemophilia A, the bleeding disorder that currently requires sufferers to inject themselves every other day to avoid life-threatening complications.One dose of a gene therapy given experimentally to 13 patients by NHS doctors in the UK has allowed them all to come off treatment. These were men – most sufferers are – who would not only bleed without stopping from an injury but would bleed into their joints even in the...
Source: Guardian Unlimited Science - Category: Science Authors: Tags: Science Health & wellbeing Life and style Society Source Type: news
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