Treatment of hemophilia B: focus on recombinant factor IX

References

• Bolton-MaggsPHPasiKJHaemophilias A and BLancet200336193711801180912781551
   PubMed Web of Science ®Google Scholar
• MannucciPMTuddenhamEGThe hemophilias: from royal genes to gene therapyN Engl J Med2001344231773177911396445
   PubMed Web of Science ®Google Scholar
• KurachiKKurachiSFurukawaMYaoSNBiology of factor IXBlood Coagul Fibrinolysis1993469539738148489
   PubMed Web of Science ®Google Scholar
• FurieBFurieBCMolecular and cellular biology of blood coagulationN Engl J Med1992326128008061538724
   PubMed Web of Science ®Google Scholar
• MannKGBiochemistry and physiology of blood coagulationThromb Haemost199982216517410605701
   PubMed Web of Science ®Google Scholar
• OldenburgJSchwaabRMolecular biology of blood coagulationSemin Thromb Hemost200127431332411547354
   PubMed Web of Science ®Google Scholar
• WhiteGC2ndRosendaalFAledortLMLusherJMRothschildCIngerslevJFactor VIII and Factor IX Subcommittee. Definitions in hemophilia: recommendation of the scientific subcommittee on factor VIII and factor IX of the scientific and standardization committee of the International Society on Thrombosis and HaemostasisThromb Haemost200185356011307831
   PubMed Web of Science ®Google Scholar
• MakrisMIs VIII worse than IX?Blood2009114475075119628712
   PubMed Web of Science ®Google Scholar
• LoweGDLudlamCALess severe bleeding in hemophilia B than in hemophilia AJ Thromb Haemost20086111982198318752577
   PubMed Web of Science ®Google Scholar
• TagarielloGIorioASantagostinoEItalian Association Hemophilia CentreComparison of the rates of joint arthroplasty in patients with severe factor VIII and IX deficiency: an index of different clinical severity of the 2 coagulation disordersBlood2009114477978419357395
   PubMed Web of Science ®Google Scholar
• FranchiniMFrattiniFCrestaniSBonfantiCHaemophilia B: current pharmacotherapy and future directionsExpert Opin Pharmacother201213142053206322946758
   PubMed Web of Science ®Google Scholar
• MannucciPMBack to the future: a recent history of haemophilia treatmentHaemophilia200814Suppl 3101818510516
   PubMed Web of Science ®Google Scholar
• GaterAThomsonTAStrandberg-LarsenMHaemophilia B: impact on patients and economic burden of diseaseThromb Haemost2011106339840421833450
   PubMed Web of Science ®Google Scholar
• MannucciPMHemophilia: treatment options in the twenty-first centuryJ Thromb Haemost2003171349135512871268
   PubMed Web of Science ®Google Scholar
• NilssonIMBerntorpELöfqvistTPetterssonHTwenty-five years’ experience of prophylactic treatment in severe haemophilia A and BJ Intern Med1992232125321640190
   PubMed Web of Science ®Google Scholar
• AzziACiappiSZakvrewskaKMorfiniMMarianiGMannucciPMHuman parvovirus B19 infection in hemophiliacs first infused with two high-purity, virally attenuated, factor VIII concentratesAm J Hematol19923932282301312304
   PubMed Web of Science ®Google Scholar
• SharpCPLailADonfieldSGompertsEDSimmondsPVirologic and clinical features of primary infection with human parvovirus 4 in subjects with hemophilia: frequent transmission by virally inactivated clotting factor concentratesTransfusion20125271482148922043925
   PubMed Web of Science ®Google Scholar
• PedenAMcCardleLHeadMWVariant CJD infection in the spleen of a neurologically asymptomatic UK adult patient with haemophiliaHaemophilia201016229630420070383
   PubMed Web of Science ®Google Scholar
• LudlamCATurnerMLManaging the risk of transmission of variant Creutzfeldt Jakob disease by blood productsBr J Haematol20061321132416371015
   PubMed Web of Science ®Google Scholar
• KurachiKDavieEWIsolation and characterization of a cDNA coding for human factor IXProc Natl Acad Sci U S A19827921646164646959130
   PubMed Web of Science ®Google Scholar
• ChooKHGouldKGReesDJBrownleeGGMolecular cloning of the gene for human anti-haemophilic factor IXNature198229958791781806287289
   PubMed Web of Science ®Google Scholar
• PipeSWRecombinant clotting factorsThromb Haemost200899584085018449413
   PubMed Web of Science ®Google Scholar
• AznarJALucíaFAbad-FranchLHaemophilia in SpainHaemophilia200915366567519432921
   PubMed Web of Science ®Google Scholar
• MonahanPEDi PaolaJRecombinant factor IX for clinical and research useSemin Thromb Hemost201036549850920632248
   PubMed Web of Science ®Google Scholar
• PoonMCLillicrapDHensmanCCardRScullyMFRecombinant factor IX recovery and inhibitor safety: a Canadian post-licensure surveillance studyThromb Haemost200287343143511916075
   PubMed Web of Science ®Google Scholar
• EwensteinBMJoistJHShapiroADMononine Comparison Study GroupPharmacokinetic analysis of plasma-derived and recombinant F IX concentrates in previously treated patients with moderate or severe hemophilia BTransfusion200242219019711896334
   PubMed Web of Science ®Google Scholar
• ShapiroADDi PaolaJCohenAThe safety and efficacy of recombinant human blood coagulation factor IX in previously untreated patients with severe or moderately severe hemophilia BBlood2005105251852515383463
   PubMed Web of Science ®Google Scholar
• BjörkmanSA commentary on the differences in pharmacokinetics between recombinant and plasma-derived factor IX and their implications for dosingHaemophilia201117217918421299739
   PubMed Web of Science ®Google Scholar
• CollinsPWFischerKMorfiniMBlanchetteVSBjörkmanSInternational Prophylaxis Study Group Pharmacokinetics Expert Working GroupImplications of coagulation factor VIII and IX pharmacokinetics in the prophylactic treatment of haemophiliaHaemophilia201117121020731726
   PubMed Web of Science ®Google Scholar
• BjörkmanSShapiroADBerntorpEPharmacokinetics of recombinant factor IX in relation to age of the patient: implications for dosing in prophylaxisHaemophilia20017213313911260271
   PubMed Web of Science ®Google Scholar
• BjörkmanSFolkessonABerntorpEIn vivo recovery of factor VIII and factor IX: intra- and interindividual variance in a clinical settingHaemophilia20071312817212717
   PubMed Web of Science ®Google Scholar
• RothDAKesslerCMPasiKJRupBCourterSGTubridyKLRecombinant Factor IX Study GroupHuman recombinant factor IX: safety and efficacy studies in hemophilia B patients previously treated with plasma-derived factor IX concentratesBlood200198133600360611739163
   PubMed Web of Science ®Google Scholar
• BerntorpEKeelingDMakrisMA prospective registry of European haemophilia B patients receiving nonacog alfa, recombinant human factor IX, for usual useHaemophilia201218450350922044794
   PubMed Web of Science ®Google Scholar
• WhiteGC2ndBeebeANielsenBRecombinant factor IXThromb Haemost19977812612659198163
   PubMed Web of Science ®Google Scholar
• FranchiniMMannucciPMInhibitors of propagation of coagulation (factors VIII, IX and XI): a review of current therapeutic practiceBr J Clin Pharmacol201172455356221204915
   PubMed Web of Science ®Google Scholar
• AstermarkJSantagostinoEKeith HootsWClinical issues in inhibitorsHaemophilia201016Suppl 5546020590857
   PubMed Web of Science ®Google Scholar
• FranchiniMLippiGMontagnanaMAnaphylaxis in patients with congenital bleeding disorders and inhibitorsBlood Coagul Fibrinolysis200920422522919300241
   PubMed Web of Science ®Google Scholar
• KiskerCTEisbergASchwartzBMononine Study GroupProphylaxis in factor IX deficiency product and patient variationHaemophilia20039327928412694518
   PubMed Web of Science ®Google Scholar
• LambertTRechtMValentinoLAReformulated BeneFIX: efficacy and safety in previously treated patients with moderately severe to severe haemophilia BHaemophilia200713323324317498071
   PubMed Web of Science ®Google Scholar
• MonahanPELiesnerRSullivanSTRamirezMEKellyPRothDASafety and efficacy of investigator-prescribed BeneFIX prophylaxis in children less than 6 years of age with severe haemophilia BHaemophilia201016346046820059559
   PubMed Web of Science ®Google Scholar
• RechtMPollmannHTagliaferriAMussoRJancoRNeumanWRA retrospective study to describe the incidence of moderate to severe allergic reactions to factor IX in subjects with haemophilia BHaemophilia201117349449921518148
   PubMed Web of Science ®Google Scholar
• IvensIABaumannAMcDonaldTAHumphriesTMichaelsLAMathewPPEGylated therapeutic proteins for haemophilia treatment: a review for haemophilia caregiversHaemophilia2013191112022913770
   PubMed Web of Science ®Google Scholar
• ElmTOstergaardHTranholmMDose response and prolonged effect of 40K PEG-FIX on bleeding in hemophilia B mice [abstract]J Thromb Haemost20097Suppl 2OC-MO-084
   Google Scholar
• NegrierCKnobeKTiedeAGiangrandePMøssJEnhanced pharmacokinetic properties of a glycoPEGylated recombinant factor IX: a first human dose trial in patients with hemophilia BBlood2011118102695270121555744
   PubMed Web of Science ®Google Scholar
• CollinsPWMøssJKnobeKGrothAColbergTWatsonEPopulation pharmacokinetic modeling for dose setting of nonacog beta pegol (N9-GP), a glycoPEGylated recombinant factor IXJ Thromb Haemost Epub September 23, 2012
   Web of Science ®Google Scholar
• ValentinoLARecombinant FIXFc: a novel therapy for the royal disease?Expert Opin Biol Ther201111101361136821780945
   PubMed Web of Science ®Google Scholar
• PetersRTLowSCKamphausGDProlonged activity of factor IX as a monomeric Fc fusion proteinBlood2010115102057206420056791
   PubMed Web of Science ®Google Scholar
• ShapiroADRagniMVValentinoLARecombinant factor IX-Fc fusion protein (rFIXFc) demonstrates safety and prolonged activity in a phase 1/2a study in hemophilia B patientsBlood2012119366667222110246
   PubMed Web of Science ®Google Scholar
• Biogen IdecStudy of recombinant factor IX Fc fusion protein (rFIXFc) in subjects with hemophilia BClinicalTrials.gov [website on the Internet]Bethesda, MDUS National Library of Medicine2009 [updated October 11, 2012]. Available from: http://clinicaltrials.gov/ct2/show/NCT01027364. NLM identifier: NCT01027364Accessed December 18, 2012
   Google Scholar
• Biogen IdecLong-term safety and efficacy of recombinant human coagulation factor IX fusion protein (rFIXFc) in the prevention and treatment of bleeding episodes in previously treated subjects with hemophilia BClinicalTrials.gov [website on the Internet]Bethesda, MDUS National Library of Medicine2011 [updated July 12, 2012]. Available from: http://clinicaltrials.gov/ct2/show/NCT01425723. NLM identifier: NCT01425723Accessed December 18, 2012
   Google Scholar
• MetznerHJWeimerTKronthalerULangWSchulteSGenetic fusion to albumin improves the pharmacokinetic properties of factor IXThromb Haemost2009102463464419806248
   PubMed Web of Science ®Google Scholar
• SantagostinoENegrierCKlamrothRSafety and pharmacokinetics of a novel recombinant fusion protein linking coagulation factor IX with albumin (rFIX-FP) in hemophilia B patientsBlood2012120122405241122859609
   PubMed Web of Science ®Google Scholar
• CSL BehringA safety and efficacy study of a recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in patients with hemophilia BClinicalTrials.gov [website on the Internet]Bethesda, MDUS National Library of Medicine2011 [updated November 28, 2012]. Available from: http://clinicaltrials.gov/ct2/show/NCT01496274. NLM identifier: NCT01496274Accessed January 30, 2013
   Google Scholar
• GompertsEDLeeMNicholsTGriffithMIB1001, a new recombinant factor IX preparation: initial safety and characterization [abstract]J Thromb Haemost20097Supplement 2 Abstract OC-MO-086
   Google Scholar
• MartinowitzUShapiroAQuonDVPharmacokinetic properties of IB1001, an investigational recombinant factor IX, in patients with haemophilia B: repeat pharmacokinetic evaluation and sialylation analysisHaemophilia201218688188722764744
   PubMed Web of Science ®Google Scholar