138
Views
57
CrossRef citations to date
0
Altmetric
Review

Many factor VIII products available in the treatment of hemophilia A: an embarrassment of riches?

Pages 67-73 | Published online: 15 Jun 2017

References

  • HoyerLWHemophilia AN Engl J Med1994330138478259143
  • CoppolaADi CapuaMDi MinnoMNTreatment of hemophilia: a review of current advances and ongoing issuesJ Blood Med2010118319522282697
  • OrlovaNAKovnirSVVorobievIIGabibovAGVorobievAIBlood clotting factor VIII: from evolution to therapyActa Naturae201352193923819034
  • PipeSWNew therapies for hemophiliaHematology Am Soc Hematol Educ Program20162016165065627913542
  • PipeSWMontgomeryRRPrattKPLentingPJLillicrapDLife in the shadow of a dominant partner: the FVIII-VWF association and its clinical implications for hemophilia ABlood2016128162007201627587878
  • ShapiroADLong-lasting recombinant factor VIII proteins for hemophilia AHematology Am Soc Hematol Educ Program20132013374324319160
  • Bensen-KennedyDBringing new therapy options to the hemophilia communityThromb Res2013131suppl 2S15S1823537722
  • MahlanguJKuliczkowskiKKarimFAEfficacy and safety of rVIII-SingleChain: results of a phase 1/3 multicenter clinical trial in severe hemophilia ABlood2016128563063727330001
  • Pabinger-FaschingIThe story of a unique molecule in hemophilia A: recombinant single-chain factor VIIIThromb Res2016141suppl 3S2S427288063
  • ZollnerSRaquetEClaarPNon-clinical pharmacokinetics and pharmacodynamics of rVIII-SingleChain, a novel recombinant single-chain factor VIIIThromb Res2014134112513124814969
  • ZollnerSBRaquetEMüller-CohrsJPreclinical efficacy and safety of rVIII-SingleChain (CSL627), a novel recombinant single-chain factor VIIIThromb Res2013132228028723830969
  • MiaoHZSirachainanNPalmerLBioengineering of coagulation factor VIII for improved secretionBlood200410393412341914726380
  • SantagostinoEA new recombinant factor VIII: from genetics to clinical useDrug Des Devel Ther2014825072515
  • KavakliKYangRRusenLProphylaxis vs. on-demand treatment with BAY 81-8973, a full-length plasma protein-free recombinant factor VIII product: results from a randomized trial (LEOPOLD II)J Thromb Haemost201513336036925546368
  • SandbergHKannichtCStenlundPFunctional characteristics of the novel, human-derived recombinant FVIII protein product, human-cl rhFVIIIThromb Res2012130580881723010293
  • ViuffDBarrowcliffeTSaugstrupTEzbanMLillicrapDInternational comparative field study of N8 evaluating factor VIII assay performanceHaemophilia201117469570221426445
  • KannichtCRamströmMKohlaGCharacterisation of the post-translational modifications of a novel, human cell line-derived recombinant human factor VIIIThromb Res20121311788823058466
  • TiedeABrandBFischerREnhancing the pharmacokinetic properties of recombinant factor VIII: first-in-human trial of glyco-PEGylated recombinant factor VIII in patients with hemophilia AJ Thromb Haemost201311467067823398640
  • KaufmanRJPowellJSMolecular approaches for improved clotting factors for hemophiliaHematology Am Soc Hematol Educ Program20132013303624319159
  • ChowdaryPFosburyERiddellAMathiasMTherapeutic and routine prophylactic properties of rFactor VIII Fc (efraloctocog alfa, Eloctate(R)) in hemophilia AJ Blood Med2016718719827695377
  • MahlanguJPowellJSRagniMVPhase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia ABlood2014123331732524227821
  • KlukowskaASzczepańskiTVdovinVKnaubSJansenMLiesnerRNovel, human cell line-derived recombinant factor VIII (Human-cl rhFVIII, Nuwiq(®)) in children with severe haemophilia A: efficacy, safety and pharmacokineticsHaemophilia Epub2015914
  • SchmidbauerSWitzelRRobbelLPhysicochemical characterisation of rVIII-SingleChain, a novel recombinant single-chain factor VIIIThromb Res2015136238839526037285
  • SchulteSPioneering designs for recombinant coagulation factorsThromb Res2011128suppl 1S9S1222221848
  • DelignatSRepesséYNavarreteAMImmunoprotective effect of von Willebrand factor towards therapeutic factor VIII in experimental haemophilia AHaemophilia201118224825422044692
  • SorvilloNHartholtRBBloemEvon Willebrand factor binds to the surface of dendritic cells and modulates peptide presentation of factor VIIIHaematologica2016101330931826635035
  • BrandBGruppoRWynnTTEfficacy and safety of pegylated full-length recombinant factor VIII with extended half-life for perioperative haemostasis in haemophilia A patientsHaemophilia2016224e251e25827328112
  • WynnTTGumuscuBPotential role of a new PEGylated recombinant factor VIII for hemophilia AJ Blood Med2016712112827382347
  • ScharrerIBrayGLNeutzlingOIncidence of inhibitors in haemophilia A patients – a review of recent studies of recombinant and plasma-derived factor VIII concentratesHaemophilia19995314515410444280
  • VielKRAmeriAAbshireTCInhibitors of factor VIII in black patients with hemophiliaN Engl J Med2009360161618162719369668
  • GunasekeraDEttingerRANakaya FletcherSFactor VIII gene variants and inhibitor risk in African American hemophilia A patientsBlood2015126789590425617427
  • MillerCHBensonJEllingsenDF8 and F9 mutations in US haemophilia patients: correlation with history of inhibitor and race/ethnicityHaemophilia201218337538222103590
  • GouwSCvan der BomJGLjungRFactor VIII products and inhibitor development in severe hemophilia AN Engl J Med2013368323123923323899
  • CalvezTChambostHClaeyssens-DonadelSRecombinant factor VIII products and inhibitor development in previously untreated boys with severe hemophilia ABlood2014124233398340825253771
  • BorelYInduction of immunologic tolerance by a hapten (DNP) bound to a non-immunogenic carrierNat New Biol1971230141804928923
  • GroomesCLGianferanteDMCrouchGDParekhDSScottDWLieuwKReduction of factor VIII inhibitor titers during immune tolerance induction with recombinant factor VIII-Fc fusion proteinPediatr Blood Cancer201663592292426739399
  • MalecLMJourneycakeJRagniMVExtended half-life factor VIII for immune tolerance induction in haemophiliaHaemophilia2016226e552e55427640873
  • IorioAPuccettiPMakrisMClotting factor concentrate switching and inhibitor development in hemophilia ABlood2012120472072722692511
  • IngMGuptaNTeyssandierMImmunogenicity of long-lasting recombinant factor VIII productsCell Immunol2016301404826723503
  • HasanHShaikhOMRassekhSRHowardAFGoddardKComparison of hypersensitivity rates to intravenous and intramuscular PEG-asparaginase in children with acute lymphoblastic leukemia: a meta-analysis and systematic reviewPediatr Blood Cancer2016641818827578304
  • KrishnamoorthySLiuTDragerDRecombinant factor VIII Fc (rFVIIIFc) fusion protein reduces immunogenicity and induces tolerance in hemophilia A miceCell Immunol2015301303926775174
  • PeyvandiFMannucciPMGaragiolaIA randomized trial of factor VIII and neutralizing antibodies in hemophilia AN Engl J Med2016374212054206427223147
  • EttingshausenCEKreuzWRecombinant vs. plasma-derived products, especially those with intact VWF, regarding inhibitor developmentHaemophilia200612suppl 6102106
  • MASACMASAC Recommendation on SIPPET (Survey of Inhibitors in Plasma-Product-Exposed Toddlers): REsults and Recommendations for Treatment Products for Previously Untreated Patients with Hemophilia AMASAC2016
  • ShimaMHanabusaHTakiMFactor VIII-mimetic function of humanized bispecific antibody in hemophilia AN Engl J Med2016374212044205327223146
  • DragerDPatarroyo-WhiteSChaoHRecombinant FVIIIFc-VWF-XTEN demonstrates significant bioavailability following subcutaneous administration in hemophilia A miceBlood20151263492
  • NathwaniACReissUMTuddenhamEGLong-term safety and efficacy of factor IX gene therapy in hemophilia BN Engl J Med2014371211994200425409372