Search results for "haemophilia"
showing 10 items of 86 documents
Lonoctocog alfa (rVIII-SingleChain) for the treatment of haemophilia A
2017
Introduction: The administration of factor VIII (FVIII) concentrates on-demand or on long-term prophylaxis is the effective and safe standard of care of patients with hemophilia A (HA). Development of neutralizing antibodies against exogenous FVIII and the short half-life of the current available products remain major challenges. There is currently a great interest towards newer FVIII products with the goal of reducing the inhibitor risk and increasing the half-life. Area covered: In this review, the authors describe the efficacy and safety of rVIII-SingleChain (Lonoctocog alfa), the first and only single chain recombinant FVIII (rFVIII) molecule developed for the prevention and treatment o…
A systematic overview of the first pasteurised VWF/FVIII medicinal product, Haemate P/Humate -P: history and clinical performance
2008
Patients with von Willebrand disease (VWD) and haemophilia A (HA) lack, to varying degrees, the von Willebrand factor (VWF) and coagulation factor VIII (FVIII) that are critical for normal haemostasis. These conditions in turn make patients prone to uncontrolled bleeding. Historically, patients with severe forms of VWD or HA were crippled before adulthood and their life expectancy was significantly reduced. Over the past decades, specific coagulation factor replacement therapies including Haemate P, have been developed to help patients achieve and maintain normal haemostasis. Haemate P is a human, plasma-derived VWF/FVIII medicinal product, which was first licensed in Germany in 1981 for th…
Results of a Prospective, Open-Label Trial to Assess the Efficacy, Safety and Immunogenicity of Wilate® in Children Under 6 Years of Age with Inherit…
2011
Abstract Abstract 3330 Introduction: Wilate® is a new-generation plasma-derived concentrate of von Willebrand factor (VWF) and factor VIII (FVIII) developed for treatment of patients with von Willebrand disease (VWD) and haemophilia A. The objective of this study was to assess efficacy, safety and immunogenicity of Wilate in children below 6 years of age with inherited VWD. Methods: The prospective, multi-centre trial included patients with inherited VWD of any type below 6 years of age with insufficient response to DDAVP. Any clinical event requiring VWF/FVIII concentrate treatment within 1 year was treated with Wilate. Efficacy was recorded by using a 4-point VRS and in vivo recovery of F…
Diagnosis and treatment of chronic synovitis in patients with haemophilia: consensus statements from the Italian Association of Haemophilia Centres
2022
Although synovitis is recognized as a marker of joint disease activity, its periodic assessment is not included in routine clinical surveillance of patients with haemophilia (PwH). In order to evaluate the current knowledge and to identify controversial issues, a preliminary literature search by the Musculoskeletal Committee of the Italian Association of Haemophilia Centres (AICE) has been conducted. Statements have been established and sent to the Italian AICE members to collect their level of agreement or disagreement by a Delphi process. Thirty-seven consensus recommendations have been drafted. We found a general agreement on the indication to consider the presence of synovitis as a mark…
NEURAL CONTROL OF GAIT IN PEOPLE WITH HAEMOPHILIC ARTHROPATHY
2022
La hemofilia es un trastorno hemorrágico causado por una deficiencia de los factores VIII o IX de la coagulación. Las personas con hemofilia grave pueden tener hemorragias espontáneas o hemorragias en respuesta a traumatismos menores; la mayoría de los eventos ocurren en las articulaciones y los músculos. La manifestación clínica más frecuente es la artropatía hemofílica, que resulta del sangrado intraarticular repetitivo y de la membrana sinovial inflamada, lo que puede resultar en dolor crónico y deterioro articular. El objetivo general de mi tesis fue investigar el control neural de la marcha en personas con artropatía hemofílica (PCAH). La hipótesis de mi tesis fue que control neural de…
Kinesiotaping for the Treatment of Haematomas in Haemophiliacs: a Case Control Study
2022
Introduction. Intramuscular hemorrhages are the second most common type of bleeding in patients with haemophilia. In haemophilic patients, muscle bleeding causes pain and postural defects and requires a long healing time. Aim. The purpose of this study is to evaluate the effectiveness of kinesiotaping, in combination with functional rehabilitation and CO2 laser therapy, in the healing process of MH in patients with haemophilia. Methods. We recruited 39 haemophilia patients who presented with a muscle hematoma. The patients were divided into 2 groups: treatment group treated with kinesiotaping, functional re-education and CO2 laser therapy, and a control group treated with functional re-educ…
Combined use of antifibrinolytics and activated prothrombin complex concentrate (aPCC) is not related to thromboembolic events in patients with acqui…
2019
Antifibrinolytics combined with aPCC are not routinely administered to patients with acquired hemophilia A due to increased thrombotic risk. This association normalizes clot stability, and improves the efficacy of therapy, but can increase the risk of severe side effects. Due to these premises it has always raised doubts and perplexities in the clinics. We now report the data of the "FEIBA® on acquired haemophilia A Italian Registry (FAIR Registry)", a retrospective-prospective study that included 56 patients. This is the first study that assessed the clinical response of the combination of aPCC and antifibrinolytic agents in patients with acquired haemophilia A. A total of 101 acute bleeds…
The inhibitors - a challenge for the management of patients with hereditary haemophilia A.
2018
Abstract Introduction. Our research strategy was aimed at evaluating the possible implication of the type of factor VIII product administered as substitution treatment to haemophilia A patients in the occurrence of inhibitors and their consequences on the management. Methods. Scientific articles from July 2015 to July 2017 were searched using the PubMed and PubMed Central databases. The used search terms included “haemophilia A”, “inhibitors”, “plasma-derived factor VIII” and “recombinant factor VIII”. Results. The risk factors for inhibitors occurrence may be patients-related (genetic and nongenetic) and treatment-related. The possibility of a correlation between the increased purity of fa…
Comparison of physical activity and sedentary behaviours between young haemophilia A patients and healthy adolescents
2011
In recent studies, adolescent haemophilia A patients and healthy adolescents have been encouraged to participate in physical activity (PA) based on its many established health benefits. However, none of the studies to date has used objective measures of PA and sedentary behaviour. The aims of the current study included: (i) to determine the amount and intensity of habitual PA among haemophilia A and healthy adolescents, and in haemophilia A patients with and without bleeding episodes in the previous year, and (ii) to identify the type and determine the time spent in sedentary activities in which both groups participate to obtain a broadened view of their daily activities. A total of 41 adol…
The orthopaedic status of severe haemophiliacs in Spain
2000
Summary. This paper provides an outline of the results obtained in a cross-sectional study conducted primarily with the aim of ascertaining orthopaedic complications in a group of young severe A and B haemophiliacs, the effects which these complications have on the medical resources used with these patients, and the impact of severe haemophilia on their quality of life. Its secondary aim was to link their current orthopaedic status to the type of treatment they had received prior to the study. Eleven Spanish hospitals took part in this study, monitoring 70 severe haemophiliacs (FVIII:C 6 months). In 40 cases (57.1%) the patients underwent one or more periods of prophylaxis. Thirty-three pat…