Potential role of a new PEGylated recombinant factor VIII for hemophilia A

References

• HoyerLWHemophilia AN Engl J Med199433038458259143
   PubMed Web of Science ®Google Scholar
• MannucciPMTuddenhamEGDThe hemophilias from royal genes to gene therapyN Engl J Med20013441773177911396445
   PubMed Web of Science ®Google Scholar
• AntonarakisSERossiterJPYoungMFactor VIII inversions in severe hemophilia A: results from an international consortiumBlood199586220622127662970
   PubMed Web of Science ®Google Scholar
• PaolaJDMontgomeryRRGillJCFloodVHemophilia and Von Willebrand diseaseOrkinSHFisherDGinsburgDLookATLuxSENathanDGNathan and Oski’s Hematology and Oncology of Infancy and Childhood8th edPhiladelphia, PAElsevier201510281039
   Google Scholar
• RagniMVTargeting antithrombin to treat hemophiliaN Engl J Med201537338939126200986
   PubMed Web of Science ®Google Scholar
• HowardTEYanoverCMahlanguJHaemophilia management: time to get personal?Haemophilia20111772172821649795
   PubMed Web of Science ®Google Scholar
• TooleJJKnopfJLWozneyJMMolecular cloning of a cDNA encoding human antihaemophilic factorNature19843123423476438528
   PubMed Web of Science ®Google Scholar
• Medical and Scientific Advisory Council (MASAC) Recommendations Concerning Prophylaxis Medical bulletin: 193New YorkNational Hemophilia Foundation1994
   Google Scholar
• Manco-JohnsonMJAbshireTCShapiroADProphylaxis versus episodic treatment to prevent joint disease in boys with severe hemophiliaN Engl J Med2007357653554417687129
   PubMed Web of Science ®Google Scholar
• OladapoAOEpsteinJDWilliamsEItoDGringeriAValentinoLAHealth-related quality of life assessment in haemophilia patients on prophylaxis therapy: a systematic review of results from prospective clinical trialsHaemophilia2015215e34435826390060
   PubMed Web of Science ®Google Scholar
• GouwSCvan den BergHMFischerKIntensity of factor VIII treatment and inhibitor development in children with severe hemophilia A: the RODIN studyBlood2013121204046405523553768
   PubMed Web of Science ®Google Scholar
• FischerKLassilaRPeyvandiFEUHASS participantsInhibitor development in haemophilia according to concentrate. Four-year results from the European Haemophilia Safety Surveillance (EUHASS) projectThromb Haemost20151135911115725854290
   PubMed Web of Science ®Google Scholar
• HayCRDiMicheleDMThe principal results of the International Immune Tolerance Study: a randomized dose comparisonBlood201211961335134422101900
   PubMed Web of Science ®Google Scholar
• RocinoACortesiPAScaloneLMantovaniLGCreaRGringeriAImmune tolerance induction in patients with haemophilia a and inhibitors: effectiveness and cost analysis in an European Cohort (The ITER Study)Haemophilia20162219610226278414
   PubMed Web of Science ®Google Scholar
• FayPJChavinSISchroederPurification and characterization of a highly purified human factor VIII consisting of a single type of polypeptide chainProc Natl Acad Sci U S A198279720072046818542
   PubMed Web of Science ®Google Scholar
• GitschierJWoodWIGoralkaCharacterization of the human factor VIII geneNature19843123263306438525
   PubMed Web of Science ®Google Scholar
• WoodWICaponDJSimonsenCCExpression of active human factor VIII from recombinant DNA clonesNature19843123303376438526
   PubMed Web of Science ®Google Scholar
• TantravahiUMurtyVVJhanwarSCPhysical mapping of the factor VIII gene proximal to two polymorphic DNA probes in human chromosome band Xq28: implications for factor VIII gene segregation analysisCytogenet Cell Genet19864275793013509
   PubMedGoogle Scholar
• LentingPJVan MourikJMertensKThe life cycle of coagulation factor VIII in view of its structure and functionBlood199892398339969834200
   PubMed Web of Science ®Google Scholar
• KumaranVBentenDFollenziAJosephBSarkarRGuptaSTransplantation of endothelial cells corrects the phenotype in hemophilia A miceJ Thromb Haemost2005392022203116102109
   PubMed Web of Science ®Google Scholar
• FollenziABentenDNovikoffPFaulknerLRautSGuptaSTransplanted endothelial cells repopulate the liver endothelium and correct the phenotype of hemophilia A miceJ Clin Invest2008118393594518274668
   PubMed Web of Science ®Google Scholar
• Kemball-CookGGomezKMolecular basis of hemophilia ALeeCABerntorpEEKeith HootsWTextbook of HemophiliaHoboken, NJWiley Blackwell20142331
   Google Scholar
• GouwSCVan der BomJGLjungRFactor VIII products and inhibitor development in severe hemophilia AN Engl J Med201336823123923323899
   PubMed Web of Science ®Google Scholar
• AstermarkJFVIII inhibitors: pathogenesis and avoidanceBlood2015125132045205125712994
   PubMed Web of Science ®Google Scholar
• MahdiAJObajiSGCollinsPWRole of enhanced half-life factor VIII AND ix in the treatment of haemophiliaBr J Haematol201516976877625754016
   PubMed Web of Science ®Google Scholar
• MahlanguJPowellJSRagniMVPhase 3 study of recombinant factor VIII Fc fusion protein in severe haemophilia ABlood2014123331732524227821
   PubMed Web of Science ®Google Scholar
• ZollnerSRaquetEClaarPNon-clinical pharmacokinetics and pharmacodynamics of rVIII-SingleChain, a novel recombinant single-chain factor VIIIThromb Res2014134112513124814969
   PubMed Web of Science ®Google Scholar
• KonkleBAStasyshynOChowdaryPPegylated, full-length, recombinant factor VIII for prophylactic and on-demand treatment of severe hemophilia ABlood201512691078108526157075
   PubMed Web of Science ®Google Scholar
• TiedeABrandBFischerREnhancing the pharmacokinetic properties of recombinant factor VIII: first-in-human trial of glycoPEGylated recombinant factor VIII in patients with hemophilia AJ Thromb Haemost201311467067823398640
   PubMed Web of Science ®Google Scholar
• CoyleTERedingMTLinJCMichaelsLAShahAPowellJPhase I study of BAY 94-9027, a pegylated B-domain-deleted recombinant factor VIII with an extended half-life, in subjects with hemophilia AJ Thromb Haemost20141248849624843882
   PubMed Web of Science ®Google Scholar
• KrishnanSVietriJFurlanRDuncanNAdherence to prophylaxis is associated with better outcomes in moderate and severe haemophilia: results of a patient surveyHaemophilia2015211647025470071
   PubMed Web of Science ®Google Scholar
• ShapiroADDonfieldSMLynnHSDefining the impact of hemophilia: the Academic Achievement in Children with Hemophilia StudyPediatrics20011086E10511731632
   PubMed Web of Science ®Google Scholar
• DuncanNShapiroAYeXEpsteinJLuoMPTreatment patterns, health-related quality of life and adherence to prophylaxis among haemophilia A patients in the United StatesHaemophilia201218576076522548353
   PubMed Web of Science ®Google Scholar
• MannucciPMGringeriAPeyvandiFSantagostinoEFactor VIII products and inhibitor development: the SIPPET study (survey of inhibitors in plasma-product exposed toddlers)Haemophilia200713Suppl 5S65S68
   PubMed Web of Science ®Google Scholar
• PeyvandiFMannucciPMGaragiolaISource of factor VIII replacement (plasmatic or recombinant) and incidence of inhibitory alloantibodies in previously untreated patients with severe hemophilia a: The Multicenter Randomized Sippet StudyPresented at: ASH 57th Annual Meeting Plenary SessionDecember 6, 2015Orlando, FL
   Google Scholar
• OldenburgJJiménez-YusteVPeiró-JordánRAledortLMSantagostinoEPrimary and rescue immune tolerance induction in children and adults: a multicentre international study with a VWF-containing plasma-derived FVIII concentrateHaemophilia2014201839124354480
   PubMed Web of Science ®Google Scholar
• KrishnamoorthySLiuTDragerDRecombinant factor VIII Fc (rFVIIIFc) fusion protein reduces immunogenicity and induces tolerance in hemophilia A miceCell Immunol2016301303926775174
   PubMed Web of Science ®Google Scholar
• GroomesCLGianferanteDMCrouchGDParekhDSScottDWLiewuKReduction of factor VIII inhibitor titers during immune tolerance induction with recombinant factor VIII-Fc fusion proteinPediatr Blood Cancer20166392292426739399
   PubMed Web of Science ®Google Scholar
• BlanchetteVSShapiroADLiesnerRJPlasma and albumin-free recombinant factor VIII: pharmacokinetics, efficacy and safety in previously treated pediatric patientsJ Thromb Haemost2008681319132618503631
   PubMed Web of Science ®Google Scholar