Search results for "FVIII"

showing 8 items of 8 documents

Recombinant factor VIII: past, present and future of treatment of hemophilia A.

2018

The development of recombinant factor VIII (rFVIII) was initially driven by the necessity to treat hemophilia A (HA) patients with FVIII concentrates without the risk of transmitting infectious agents. Over the last three decades the safety of rFVIII has been further improved by completely removing animal or human proteins from the manufacturing process, so that patients would not be exposed to known or emerging pathogens. Recent efforts have concentrated on improving the expression of rFVIII, reducing its immunogenicity and enhancing its pharmacokinetic (PK) behavior. These new goals have been possible thanks to the develop-ment of biotechnology and a better knowledge of the function and s…

0301 basic medicine030204 cardiovascular system & hematologyPharmacologyStandard half-life FVIIIHemophilia ARecombinant factor viiiHemostaticslaw.inventionCoagulation factor disorder03 medical and health sciences0302 clinical medicineHemostaticlawExtended half-life FVIIIMedicineHumansPharmacology (medical)Mode of actionPharmacologyFactor VIIIbiologybusiness.industryManufacturing processImmunogenicityHuman cellRecombinant ProteinRecombinant FVIIIRecombinant Proteins030104 developmental biologyTreatment OutcomeConsumer Product Safetybiology.proteinRecombinant DNAPEGylationAntibodybusinessDrug ContaminationHumanHalf-LifeDrugs of today (Barcelona, Spain : 1998)
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The important impact of dental care on haemostatic treatment burden in patients with mild haemophilia

2022

Background: Mild haemophilia (MH) is mainly characterized by haemorrhages secondary to surgery/invasive procedures or trauma. Haemostatic treatment in MH ranges from on demand to short prophylaxis according to the type of bleeding events and the basal clotting factor level. Oral surgery and dental extractions can represent a frequent haemostatic challenge in MH requiring appropriate treatment. However, only few studies on limited numbers of patients are available in the literature regarding the implications of dental management in patients with MH. Objectives: The purpose of the study was to evaluate the impact of dental care on the burden of haemostatic treatment in patients affected by MH…

AdultMaleAged 80 and overFactor VIIIAdolescentHemorrhageHematologyGeneral MedicineMiddle AgedHemophilia Amild haemophilia dental care FVIII FIX haemophilia treatmentHemostaticsAntifibrinolytic AgentsBlood Coagulation FactorsYoung AdultHumansDental CareGenetics (clinical)Aged
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Distribution, genetic and cardiovascular determinants of FVIII:c - Data from the population-based Gutenberg Health Study

2015

Background: Elevated levels of FVIII:c are associated with risk for both venous and arterial thromboembolism. However, no population-based study on the sex-specific distribution and reference ranges of plasma FVIII: c and its cardiovascular determinants is available. Methods: FVIII:c was analyzed in a randomly selected sample of 2533 males and 2440 females from the Gutenberg Health Study in Germany. Multivariable regression analyses for FVIII:c were performed under adjustment for genetic determinants, cardiovascular risk factors and cardiovascular disease. Results and conclusions: Females (126.6% (95% CI: 125.2/128)) showed higher FVIII:c levels than males (121.2% (119.8/122.7)). FVIII:c le…

AdultMalecongenital hereditary and neonatal diseases and abnormalitiesmedicine.medical_specialtyLinkage disequilibriumGenotypeanimal diseasesPopulationFVIII:c reference valuesSingle-nucleotide polymorphismDiseaseAge DistributionVon Willebrand factorGermanyThromboembolismhemic and lymphatic diseasesInternal medicineVenous thrombosisHumansMedicineGenetic Predisposition to DiseaseProspective StudiesSex DistributioneducationAgededucation.field_of_studyEpidemiological studiesFactor VIIIPolymorphism Geneticbiologybusiness.industryIncidenceC-reactive proteinArterial thrombosisDNAMiddle AgedNomogrammedicine.diseaseVenous thrombosisPopulation SurveillanceImmunologybiology.proteinFemaleCardiology and Cardiovascular MedicinebusinessFollow-Up Studies
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Tailoring haemophilia A prophylaxis with BAY 81-8973: A case series

2020

BAY 81-8973 is an unmodified, full-length third generation recombinant factor VIII (rFVIII) which offers a more favorable pharmacokinetic (PK) profile, compared to its predecessor sucrose-formulated rFVIII (rFVIII-FS). We here report on a retrospective case series of nine patients affected by hemophilia A (HA), with variable disease severity, bleeding phenotype and comorbidities, to underline our clinical practice on prophylaxis with a recently introduced standard hall-life recombinant Factor VIII. The current case series highlights how the current clinical management of hemophilia is able to personalize treatment in several specific conditions like concomitant illnesses with thrombotic ris…

AdultMalemedicine.medical_specialtyAdolescentHemophilia A HemarthrosisHemophilic arthropathyrFVIIIPharmacokineticProphylaxisHaemophilia A030204 cardiovascular system & hematologyHemophilia ARecombinant factor viii03 medical and health sciencesYoung Adult0302 clinical medicinehemic and lymphatic diseasesInternal medicineMedicineHumansChildVariable disease severityAgedRetrospective StudiesThrombotic riskFactor VIIIbusiness.industryHematologyHemarthrosisMiddle Agedmedicine.diseaseThird generationClinical PracticeTreatment OutcomeConcomitantbusiness030215 immunology
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Can we compare haemophilia carriers with clotting factor deficiency to male patients with mild haemophilia?

2020

Introduction Certain haemophilia carriers demonstrate an increased bleeding tendency, mainly related to clotting factor deficiency. No study has so far formally compared the bleeding phenotype of women and girls with mild FVIII or FIX deficiency and associated management with that of male patients affected by mild haemophilia A and B. Material and methods We retrospectively evaluated 44 women and girls with mild FVIII or FIX deficiency (FVIII or FIX 0.05-0.5 IU/mL) and 77 male patients with mild haemophilia A or B and compared them with respect to clotting factor level, age at and trigger for diagnosis, as well as treatment modalities. Results After excluding gender-related haemorrhagic sym…

FVIIImild haemophiliaAdultMalePediatricsmedicine.medical_specialtycongenital hereditary and neonatal diseases and abnormalitiesHeterozygoteAdolescentMucocutaneous zonecarriersPlasma factorAge at diagnosis030204 cardiovascular system & hematologyHaemophiliaHemophilia AHemostatics03 medical and health sciencesYoung Adult0302 clinical medicinecarrierhemic and lymphatic diseasesmedicineHumansDeamino Arginine VasopressinClotting factor deficiencyChildGenetics (clinical)AgedClotting factorAged 80 and overbusiness.industryFIXHematologyGeneral MedicineMiddle Agedmedicine.diseaseBlood Coagulation Factorsbleeding phenotypebleeding phenotype carriers FIX FVIII mild haemophiliaMale patientChild PreschoolMild haemophilia AFemalebusiness030215 immunologyHaemophilia : the official journal of the World Federation of HemophiliaREFERENCES
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Emergency management in patients with haemophilia A and inhibitors on prophylaxis with emicizumab: AICE practical guidance in collaboration with SIBi…

2020

Emicizumab has been approved in several countries for regular prophylaxis in patients with congenital haemophilia A and FVIII inhibitors because it substantially reduces their bleeding risk and improves quality of life. However, although significantly less frequent, some breakthrough bleeds may still occur while on emicizumab, requiring treatment with bypassing or other haemostatic agents. Thrombotic complications have been reported with the associated use of activated prothrombin complex concentrates. In addition, when surgery/invasive procedures are needed while on emicizumab, their management requires multidisciplinary competences and direct supervision by experts in the use of this agen…

Factor VIIIFVIII inhibitorSettore BIO/12Antibodies Bispecific Antibodies Monoclonal Humanized Factor VIII Hemophilia A Hemorrhage Hemostatics Humans Italy Quality of LifeFVIII inhibitorsHemorrhageAntibodies Monoclonal HumanizedHemophilia AAntibodiesHemostaticsbypassing agents; emergency; emicizumab; FVIII inhibitors; haemophilia AItalyhemic and lymphatic diseasesMonoclonalEmergencyHaemophilia AAntibodies BispecificQuality of LifeHumansBispecificBypassing agentsEmicizumabHumanizedBypassing agentHaemostasis
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Status of Recombinant Factor VIII Concentrate Treatment for Hemophilia A in Italy: Characteristics and Clinical Benefits

2019

The current interest in recombinant factor VIII (rFVIII) products stems from the fact that they offer a technological solution to prolonging the half-life of and reducing the risk of formation of alloantibodies (inhibitors) against FVIII in treated patients with hemophilia A (HA). The Italian health care system has authorized the use of a wide range of rFVIII concentrates of the first, second, and third generation, as well as new innovative rFVIII preparates with an extended half-life (EHL) (Kogenate FS®-Bayer, belonging to the second generation and replaced since 2017 by a product consisting of the same modified molecule; because it is only available until the end of the current year, it w…

Review030204 cardiovascular system & hematologyPharmacologyHemophilia ARecombinant factor viii03 medical and health sciences0302 clinical medicineVon Willebrand factorhemic and lymphatic diseasesinhibitorsMoroctocog alfaEHL-rFVIIIMedicineHemophilia A recombinant Factor VIII products pharmacokinetics inhibitors EHL-rFVIIIlcsh:R5-920biologybusiness.industryvirus diseasesrecombinant Factor VIII productsGeneral MedicineTuroctocog alfaThird generationPharmacodynamicsbiology.proteinMedicineSimoctocog alfabusinesslcsh:Medicine (General)pharmacokinetics030215 immunologyClearanceFrontiers in Medicine
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Patient preferences in the treatment of hemophilia A: impact of storage conditions on product choice

2018

Bernd Tischer,1 Renato Marino,2 Mariasanta Napolitano3 1Kantar Health, Munich, Germany; 2Haemophilia and Thrombosis Centre, University Hospital of Bari, Apulia, Italy; 3University of Palermo, Reference Regional Center for Thrombosis and Hemostasis Hematology Unit, Palermo, Italy Objectives: To gain insights into the usage of factor VIII (FVIII) products by patients diagnosed with moderate/severe hemophilia A, and to assess the impact and perceived importance of product storage.Methods: In this study, 200 patients diagnosed with moderate or severe hemophilia A across seven countries participated. Data were collected via a 30-minute, face-to-face interview in six countries and via a web-based…

medicine.medical_specialtycongenital hereditary and neonatal diseases and abnormalitieshemophilia A recombinant FVIII plasma-derived FVIII storage reconstitution stabilityTreatment Adherence haemophiliaActivities of daily livingMedicine (miscellaneous)030204 cardiovascular system & hematologySevere hemophilia AHaemophiliaHemophilia Astorage03 medical and health sciences0302 clinical medicinePatient satisfactionrecombinant FVIIIplasma-derived FVIIIhemic and lymphatic diseasesMedicine030212 general & internal medicineProduct (category theory)Pharmacology Toxicology and Pharmaceutics (miscellaneous)Original Researchlcsh:R5-920business.industryHealth Policystabilitymedicine.diseasePatient preferenceConjoint analysisProduct choicePatient Preference and AdherenceEmergency medicinereconstitutionbusinesslcsh:Medicine (General)Social Sciences (miscellaneous)Patient Preference and Adherence
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