References
- Blanchette VS, Key NS, Ljung LR, et al. Definitions in hemophilia: communication from the SSC of the ISTH. J Thromb Haemost. 2014;12:1935–1939.
- Regling K, Callaghan MU, Sidonio R Jr. Managing severe hemophilia A in children: pharmacotherapeutic options. Pediatric Health Med Ther. 2022;13:27–35.
- Marchesini E, Morfini M, Valentino L. Recent advances in the treatment of hemophilia: a review. Biol targ ther. 2021;15:221–235.
- van Vulpen LFD, van Meegeren MER, Roosendaal G, et al. Biochemical markers of joint tissue damage increase shortly after a joint bleed; an explorative human and canine invivo study. Osteoarthritis Cartilage. 2015;23(1):63–69.
- Gringeri A, Lundin B, von Mackensen S, et al. A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT study). J Thromb Haemost. 2011;9(4):700–710.
- Warren BB, Thornhill D, Stein J, et al. Young adult outcomes of childhood prophylaxis for severe hemophilia A: results of the joint outcome continuation study. Blood Adv. 2020;4(11):2451–2459.
- Panicker J, Warrier I, Thomas R, et al. The overall effectiveness of prophylaxis in severe haemophilia. Haemophilia. 2003;9(3):272–278.
- Gouw SC, Marijke Van Den Berg H, Fischer K, et al. Intensity of factor VIII treatment and inhibitor development in children with severe hemophilia A: the RODIN study. Blood. 2013;121(20):4046–4055. Available from: http://ashpublications.org/blood/article-pdf/121/20/4046/531085/4046.pdf
- Gouw SC, van der Bom JG, Marijke Van Den Berg H. Treatment-related risk factors of inhibitor development in previously untreated patients with hemophilia A: the CANAL cohort study. Blood. 2007;109(11):4648–4654. Available from: http://ashpublications.org/blood/article-pdf/109/11/4648/1479075/zh801107004648.pdf
- Peters R, Harris T. Advances and innovations in haemophilia treatment. Nat Rev Drug Discov. 2018;17(7):493–508.
- Pipe SW, Montgomery RR, Pratt KP, et al. Life in the shadow of a dominant partner: the FVIII-VWF association and its clinical implications for hemophilia A. Blood. 2016;128(16):2007–2016. Available from: http://ashpublications.org/blood/article-pdf/128/16/2007/1396232/2007.pdf
- Oldenburg J, Mahlangu JN, Kim B, et al. Emicizumab prophylaxis in hemophilia a with inhibitors. N Engl J Med. 2017;377(9):809–818.
- FDA approves emicizumab-kxwh for hemophilia A with or without factor VIII inhibitors. 2018.
- Hedner U, Erhardtsen E. Review: potential role for rFVIIa in transfusion medicine. Transfusion (Paris). 2022;42(1):114–124.
- Sumner MJ, Geldziler BD, Pedersen M, et al. Treatment of acquired haemophilia with recombinant activated FVII: a critical appraisal. Haemophilia. 2007;13(5):451–461.
- Parameswaran R, Shapiro AD, Gill JC, et al. Dose effect and efficacy of rFVIIa in the treatment of haemophilia patients with inhibitors: analysis from the hemophilia and thrombosis research society registry. Haemophilia. 2005;11(2):100–106.
- Montacir O, Montacir H, Eravci M, et al. Bioengineering of rFVIIa biopharmaceutical and structure characterization for biosimilarity assessment. Bioengineering. 2018;5(1):7.
- Faranoush M, Abolghasemi H, Mahboudi F, et al. A comparison of efficacy between recombinant activated factor VII (Aryoseven) and novoseven in patients with hereditary FVIII deficiency with inhibitor. Clin App Thrombosis/Hemostasis. 2016;22(2):184–190.
- Study on the Dose-response relationship of pharmacodynamic parameters in patients with hemophilia with inhibitors. [cited 2022 Jun 2]. Available from: https://www.clinicaltrials.gov/ct2/show/NCT04789954?term=NCT04789954&draw=2&rank=1
- Prediction of the efficacy of activated recombinant human factor vii in adult congenital haemophilia A or B patients with inhibitors by use of thromboelastography. [cited 2022 Jun 2]. Available from: https://clinicaltrials.gov/ct2/show/record/NCT01561924?term=NCT01561924&draw=2&rank=1
- Bar-Ilan A, Livnat T, Hoffmann M, et al. In vitro characterization of MOD-5014, a novel long-acting carboxy-terminal peptide (CTP)-modified activated FVII. Haemophilia. 2018;24(3):477–486.
- Calo D, Hart G, Hoffman M, et al. Enhancing the longevity and in vivopotency of therapeutic proteins: the power of CTP. Precis Med. 2015;2. 1–8.
- Hart G, Hershkovitz O, Bar-Iilan A, et al. Mod-5014, a Novel Long-Acting FVIIa proposing an improved prophylactic and on demand treatment for hemophilic patients following SC and IV administration – comprehensive In -Vitro and In -Vivo evaluation in preparation for clinical studies. Blood. 2013;122:3578.
- A study of a Long-Acting r-Factor 7a (Factor VIIa) in adult men with hemophilia A or B. [cited 2022 Jun 2]. Available from: https://www.clinicaltrials.gov/ct2/show/NCT02418793?term=NCT02418793&draw=2&rank=1
- A Single-dose, Dose-escalation study of a long-acting MOD-5014 in healthy adult male. [cited 2022 Jun 2]. Available from: https://clinicaltrials.gov/ct2/show/study/NCT02919800
- Chevreux G, Faid V, Scohyers JM, et al. N-/O-glycosylation analysis of human FVIIa produced in the milk of transgenic rabbits. Glycobiology. 2013;23(12):1531–1546.
- Biron-Andreani C, Schved JF. Eptacog beta: a novel recombinant human factor VIIa for the treatment of hemophilia A and B with inhibitors. Expert Rev Hematol. 2019;12(1):21–28.
- A Phase I Safety, Pharmacokinetics and pharmacodynamics study of recombinant factor VIIa in adult patients with hemophilia A or B (rhFVIIa). [cited 2022 Jun 7]. Available from: https://clinicaltrials.gov/ct2/show/NCT01708564?term=NCT01708564&draw=2&rank=1
- Ducore J, Lawrence JB, Simpson M, et al. Safety and dose-dependency of eptacog beta (activated) in a dose escalation study of non-bleeding congenital haemophilia A or B patients, with or without inhibitors. Haemophilia. 2017;23(6):844–851.
- Phase 1 safety, pharmacokinetics and pharmacodynamics study of recombinant factor VIIa variant (813d) in adult subjects with hemophilia. [cited 2022 Jun 7]. Available from: https://clinicaltrials.gov/ct2/show/NCT01439971
- Gruppo RA, Malan D, Kapocsi J, et al. Phase 1, single-dose escalating study of marzeptacog alfa (activated), a recombinant factor VIIa variant, in patients with severe hemophilia. J Thromb Haemost. 2018;16(10):1984–1993.
- Study of coagulation factor viia variant marzeptacog alfa (Activated) in adult subjects with hemophilia A and B. [cited 2022 Jun 7]. Available from: https://clinicaltrials.gov/ct2/show/NCT03407651?term=NCT03407651&draw=2&rank=1
- Mahlangu J, Levy H, Kosinova MV, et al. Subcutaneous engineered factor VIIa marzeptacog alfa (activated) in hemophilia with inhibitors: phase 2 trial of pharmacokinetics, pharmacodynamics, efficacy, and safety. Res Pract Thromb Haemost. 2021;5(6). DOI:10.1002/rth2.12576.
- Figueiredo M. Catalyst halts MarzAA program for hemophilia, despite positive results. Hemophilia News Today. 2021 [cited 2022 Jun 9]. Available from: Hemophilianewstoday.com/2021/11/17/catalyst-halts-marzaa-development-hemophilia-despite-positive-results
- Safety and mode of action of a single dose and multiple doses of long acting activated recombinant human factor VII in patients with haemophilia A and B. [cited 2022 Jun 2]. Available from: https://www.clinicaltrials.gov/ct2/show/study/NCT00922792?term=NCT00922792&draw=2&rank=1
- Mahdi AJ, Obaji SG, Collins PW. Role of enhanced half-life factor VIII and IX in the treatment of haemophilia. Br J Haematol. 2015;169(6):768–776.
- Wynn TT, Gumuscu B. Potential role of a new PEGylated recombinant factor VIII for hemophilia A. J Blood Med. 2016;121. DOI:10.2147/JBM.S82457.
- A Phase II, Muti-Center study of repeat dosing of PEG recombinant human coagulation factor VIII-Fc fusion protein for injection in subjects with severe Hemophilia A. [cited 2022 Jun 7]. Available from: https://clinicaltrials.gov/ct2/show/NCT05265286
- Konkle BA, Shapiro A, Quon D, et al. BIVV001: the first investigational factor VIII therapy to break through the VWF ceiling in Hemophilia A, with potential for extended protection for one week or longer. Blood. 2018;132(Supplement 1):636.
- A Safety, Tolerability, and pharmacokinetics study of a single intravenous injection of recombinant coagulation factor VIII Fc - Von Willebrand factor - XTEN fusion protein. [cited 2022 Jun 7]. Available from: https://clinicaltrials.gov/ct2/show/NCT03205163
- Konkle BA, Shapiro AD, Quon DV, et al. BIVV001 fusion protein as factor VIII replacement therapy for hemophilia A. N Engl J Med. 2020;383(11): 1018–1027.
- Gilbert JC, DeFeo-Fraulini T, Hutabarat RM, et al. First-in-human evaluation of anti-von Willebrand factor therapeutic aptamer ARC1779 in healthy volunteers. Circulation. 2007;116(23):2678–2686.
- Zhou J, Rossi J. Aptamers as targeted therapeutics: current potential and challenges. Nat Rev Drug Discov. 2017;16(3):181–202.
- Zhu S, Gilbert JC, Hatala P, et al. The development and characterization of a long acting anti-thrombotic von Willebrand factor (VWF) aptamer. J Thromb Haemost. 2020;18(5):1113–1123.
- Ay C, Pabinger I, Quehenberger P, et al. BT200 increases von Willebrand factor (VWF), FVIII and platelet counts in patients with von Willebrand disease (VWD) Type IIb [Abstract]. Res Pract Thromb Haemost. 2021 [cited 2022 Sept 8;5. Available from: https://abstracts.isth.org/abstract/bt200-increases-von-willebrand-factor-vwf-fviii-and-platelet-counts-in-patients-with-von-willebrand-disease-vwd-type-iib/
- Kovacevic KD, Grafeneder J, Schörgenhofer C, et al. The von Willebrand Factor A-1 domain binding aptamer BT200 elevates plasma levels of VWF and Factor VIII: a first-in-human trial. Haematologica. 2021. 10.3324/haematol.2021.279948.
- BT200 in hereditary bleeding disorders. [cited 2022 Jun 7]. Available from: https://clinicaltrials.gov/ct2/show/NCT04677803
- Ay C, Derhaschnig U, Jilma B, et al. The VWF-A1 domain binding aptamer BT200 prolongs the half-lives of different factor VIII (FVIII) products in patients with severe hemophilia A and increases FVIII levels in non-severe hemophilia A [Abstract]. Res Pract Thromb Haemost. 2021; 5. [cited 2022 Jun 7]. Available from: https://abstracts.isth.org/abstract/the-vwf-a1-domain-binding-aptamer-bt200-prolongs-the-half-lives-of-different-factor-viii-fviii-products-in-patients-with-severe-hemophilia-a-and-increases-fviii-levels-in-non-severe-hemophilia-a/.
- Louvain-Quintard VB, Bianchini EP, Calmel-Tareau C, et al. Thrombin-activable factor X re-establishes an intrinsic amplification in tenase-deficient plasmas. J Biol Chem. 2005;280(50):41352–41359.
- A safety study of STSP-0601 in adult patients with hemophilia A or B with inhibitor. [cited 2022 Jun 2]. Available from: https://clinicaltrials.gov/ct2/show/NCT04747964
- A safety and efficacy study of STSP-0601 in adult patients with Hemophilia A or B with inhibitor. [cited 2022 Jun 2]. Available from: https://www.clinicaltrials.gov/ct2/show/NCT05027230?term=nct05027230&draw=2&rank=1
- Miller CH, Bean CJ. Genetic causes of haemophilia in women and girls. Haemophilia. 2021;27(2). DOI:10.1111/hae.14186.
- Miller CH, Soucie JM, Byams VR, et al. Women and girls with haemophilia receiving care at specialized haemophilia treatment centres in the United States. Haemophilia. 2021;27(6):1037–1044.
- Olsson A, Hellgren M, Berntorp E, et al. Clotting factor level is not a good predictor of bleeding in carriers of haemophilia A and B. Blood Coagul Fibrin. 2014;25(5):471–475.
- van Galen KPM, d’Oiron R, James P, et al. A new hemophilia carrier nomenclature to define hemophilia in women and girls: communication from the SSC of the ISTH. J Thromb Haemost. 2021;19(8):1883–1887.
- Extended half life factor (EHF) products for heavy menstrual bleeding in Hemophilia carriers. [cited 2022 Jun 2]. Available from: https://www.clinicaltrials.gov/ct2/show/NCT05281718?term=NCT05281718&draw=2&rank=1
- Polderdijk SGI, Adams TE, Ivanciu L, et al. Design and characterization of an APC-specific serpin for the treatment of hemophilia. Blood. 2017;129(1):105–113. Available from: http://ashpublications.org/blood/article-pdf/129/1/105/1398761/blood718635.pdf
- Polderdijk SGI, Baglin TP, Huntington JA. Targeting activated protein C to treat hemophilia. Curr Opin Hematol. 2017;24(5):446–452.
- The safety and tolerability of serpinPC in healthy men and in men with severe blood disorders (Haemophilia A and B). [cited 2022 Jun 2]. Available from: https://clinicaltrials.gov/ct2/show/NCT04073498?term=nct04073498&draw=2&rank=1
- Lenting PJ, Denis V, Christophe OD. Perspective Emicizumab, a bispecific antibody recognizing coagulation factors IX and X: how does it actually compare to factor VIII? Blood. 2017;130:2463–2468.
- Green D, Lechner K. A survey of 215 non-hemophilic patients with inhibitors to Factor VIII. Thromb Haemost. 1981;45(3):200–203.
- Franchini M. Acquired hemophilia A. Hematology. 2006;11(2):119–125.
- Hofbauer CJ, Whelan SFJ, Hirschler M, et al. Affinity of FVIII-specific antibodies reveals major differences between neutralizing and nonneutralizing antibodies in humans. Blood. 2015;125(7):1180–1188. Available from: http://ashpublications.org/blood/article-pdf/125/7/1180/1389064/1180.pdf
- Werwitzke S, Geisen U, Nowak-Göttl U, et al. Diagnostic and prognostic value of factor VIII binding antibodies in acquired hemophilia A: data from the GTH-AH 01/2010 study. J Thromb Haemost. 2016;14(5):940–947.
- Kruse-Jarres R, Kempton CL, Baudo F, et al. Acquired hemophilia A: updated review of evidence and treatment guidance. American Journal of Hematology. 2017;92(7):695–705.
- Knöbl P. Prevention and management of bleeding episodes in patients with acquired hemophilia A. Drugs. 2018;78(18):1861–1872.
- Knoebl P, Thaler J, Jilma P, et al. Emicizumab for the treatment of acquired hemophilia A. Blood. 2021;137(3):410–419. Available from: http://ashpublications.org/blood/article-pdf/137/3/410/1797631/bloodbld2020006315.pdf
- Poston JN, Al-Banaa K, von Drygalski A, et al. Emicizumab for the treatment of acquired Hemophilia A: a multicenter US case series. Blood. 2021;138(Supplement 1):496.
- Emicizumab in acquired hemophilia A. [cited 2022 Jun 2]. Available from: https://www.clinicaltrials.gov/ct2/show/NCT04188639?term=NCT04188639&draw=2&rank=1
- Factor IX as Adjunctive Therapy to Emicizumab (EMIX). [cited 2022 Jun 2]. Available from: https://www.clinicaltrials.gov/ct2/show/NCT05281718?term=NCT05281718&draw=2&rank=1
- Hemlibra® (emicuzmab-kxwh) for US patients and caregivers. [cited 2022 Sept 8]. Available from: https://www.emicizumabinfo.com/content/dam/gene/emicizumabinfo/pdf/archived-reports/reported-fatalities.pdf
- Peyvandi F, Mahlangu J, Pipe S, et al. Application of a hemophilia mortality framework to the emicizumab global safety database. J Thromb Haemost. 2021;19(Suppl 1):32–41.
- Østergaard H, Lund J, Greisen PJ, et al. A factor VIIIa–mimetic bispecific antibody, Mim8,ameliorates bleeding upon severe vascular challenge in hemophilia A mice. Blood. 2021;138(14):1258–1268.
- Kjellev SL, Østergaard H, Greisen PJ, et al. Mim8 - a Next-Generation FVIII mimetic Bi-Specific antibody - potently restores the hemostatic capacity in Hemophilia a settings in vitro and in vivo. Blood. 2019;134(96):96–998.
- Lauritzen B, Bjelke M, Björkdahl O, et al. A novel next-generation FVIIIa mimetic, Mim8, has a favorable safety profile and displays potent pharmacodynamic effects: results from safety studies in cynomolgus monkeys. J Thromb Haemost. 2022;20(6): 1312–1324.
- A research study of how a new medicine NNC0365-3769 (Mim8) works in the body of healthy people. [cited 2022 Jun 7]. Available from: https://clinicaltrials.gov/ct2/show/NCT05127473
- A research study investigating mim8 in people with Haemophilia A (FRONTIER1). [cited 2022 Jun 7]. Available from: https://clinicaltrials.gov/ct2/show/NCT04204408
- Bolliger D, Szlam F, Suzuki N, et al. Heterozygous antithrombin deficiency improves in vivo haemostasis in factor VIII-deficient mice. Thromb Haemost. 2010;103(6):1233–1238.
- Sehgal A, Barros S, Ivanciu L, et al. An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia. Nat Med. 2015;21(5):492–497.
- An open-label extension study of an investigational drug, fitusiran, in patients with moderate or severe Hemophilia A or B. [cited 2022 Jun 7]. Available from: https://clinicaltrials.gov/ct2/show/NCT02554773
- Pasi KJ, Lissitchkov T, Mamonov V, et al. Targeting of antithrombin in hemophilia A or B with investigational siRNA therapeutic fitusiran—Results of the phase 1 inhibitor cohort. J Thromb Haemost. 2021;19(6): 1436–1446.
- Pasi KJ, Georgiev P, Mant T, et al. Fitusiran, an investigational RNAi therapeutic targeting antithrombin for the treatment of hemophilia: updated results from a Phase 1 and Phase 1/2 extension study in patients with inhibitors. Blood. 2016;128(22):1397.
- Alnylam Reports Patient Death in Fitusiran Clinical Study. National Hemophilia foundation. 2017 [cited 2022 Sept 8]. Available from: https://www.hemophilia.org/news/alnylam-reports-patient-death-in-fitusiran-clinical-study
- Update- FDA lifts suspension of Fitusiran trial. World Federation of Hemophilia. 2017 [cited 2022 Jun 2]. Available from: https://news.wfh.org/update-fda-lifts-suspension-fitusiran-trial/
- Taylor N. Sanofi plans 2021 restart for pediatric hemophilia trial hit by fallout from thrombotic events. Fierce Biotech. 2021 [cited 2022 Jun 2]. Available from: https://www.fiercebiotech.com/biotech/sanofi-plans-2021-restart-for-pediatric-hemophilia-trial-hit-by-fallout-from-thrombotic
- Fitusiran prophylaxis in male pediatric subjects Aged 1 to less than 12 years with Hemophilia A or B (ATLAS-PEDS). [cited 2022 Jun 7]. Available from: https://clinicaltrials.gov/ct2/show/NCT03974113
- Sanofi: data from two Phase 3 studies demonstrating fitusiran significantly reduced bleeds in people with hemophilia A or B, with or without inhibitors. ASH plenary and late-breaking session. 2021.
- Kato H. Tissue factor pathway inhibitor; its structure, function and clinical significance. Pol J Pharmacol. 1996;48:67–72.
- Mast AE. Tissue factor pathway inhibitor: multiple anticoagulant activities for a single protein. Arterioscler Thromb Vasc Biol. 2016;36(1):9–14.
- Hedner U, Ezban M. Tissue factor and factor VIIa as therapeutic targets in disorders of hemostasis. Annu Rev Med. 2008;59(1):29–41.
- Safety of NNC 0172-0000-2021 in healthy male subjects and subjects with Haemophilia A or B (Explorer 1). [cited 2022 Jun 7]. Available from: https://clinicaltrials.gov/ct2/show/NCT01228669
- Chowdary P, Lethagen S, Friedrich U, et al. Safety and pharmacokinetics of anti- TFPI antibody (concizumab) in healthy volunteers and patients with hemophilia: a randomized first human dose trial. J Thromb Haemost. 2015;13(5):743–754.
- Trial Investigating Safety, Pharmacokinetics and pharmacodynamics of concizumab administered subcutaneously to haemophilia A subjects. [cited 2022 Jun 7]. Available from: https://clinicaltrials.gov/ct2/show/NCT02490787
- Eichler H, Angchaisuksiri P, Kavakli K, et al. A randomized trial of safety, pharmacokinetics and pharmacodynamics of concizumab in people with hemophilia A. J Thromb Haemost. 2018;16(11):2184–2195.
- Eichler H, Angchaisuksiri P, Kavakli K, et al. Concizumab restores thrombin generation potential in patients with haemophilia: pharmacokinetic/pharmacodynamic modelling results of concizumab phase 1/1b data. Haemophilia. 2019;25(1):60–66.
- Shapiro AD, Angchaisuksiri P, Astermark J, et al. Subcutaneous concizumab prophylaxis in hemophilia A and hemophilia A/B with inhibitors: phase 2 trial results. 2019 Available from: www.clinicaltrials.gov.
- A trial evaluating the efficacy and safety of prophylactic administration of concizumab in Haemophilia A and B patients with inhibitors (explorerTM4). [cited 2022 Jun 7]. Available from: https://clinicaltrials.gov/ct2/show/NCT03196284
- A trial evaluating efficacy and safety of prophylactic administration of concizumab in patients with severe Haemophilia A without inhibitors (explorerTM5). [cited 2022 Jun 7]. Available from: https://clinicaltrials.gov/ct2/show/NCT03196297
- Ray F. Novo nordisk resumes Phase 3 trials of concizumab in Hemophilia A and B. Hemophilia News Today. 2020 [cited 2022 Jun 9]. Available from: hemophilianewstoday.com/2020/08/14/novo-nordisk-resumes-phase-3-trials-of-concizumab-in-hemophilia-a-and-b
- Yegneswaran S, Evans V, Jiang X, et al. BAY 1093884 binds to the Kunitz 1 and 2 domain interface of tissue factor pathway inhibitor and inhibits its function . Berlin Germany: XXVI International Society on haemostasis and Thrombosis Congress; 2017.
- Gu JM, Zhao XY, Schwarz T, et al. Mechanistic modeling of the pharmacodynamic and pharmacokinetic relationship of tissue factor pathway Inhibitor-Neutralizing antibody (BAY 1093884) in cynomolgus monkeys. AAPS J. 2017;19(4):1186–1195.
- A single escalating dose and multiple dose study of BAY 1093884 in subjects with severe Hemophilia Types A or B, with or without inhibitors. [cited 2022 Jun 7]. Available from: https://clinicaltrials.gov/ct2/show/NCT02571569
- Chowdary P, Lissitchkov TJ, Willmann S, et al. Pharmacodynamics, pharmacokinetics and safety of Bay 1093884, an antibody directed against human TFPI, in patients with factor VIII or IX deficiency (with and without inhibitors)- A Phase 1 study. Blood. 2018;132(Supplement 1):1176.
- A study to assess pharmacokinetics and pharmacodynamics following administration of BAY1093884 in patients with severe hemophilia. [cited 2022 Jun 2]. Available from: https://clinicaltrials.gov/ct2/show/NCT03481946?term=nct03481946&draw=2&rank=1
- Mancuso ME, Ingham SJM, Kunze M Befovacimab, an anti‐tissue factor pathway inhibitor antibody: early termination of the multiple‐dose, dose‐escalating Phase 2 study due to thrombosis. Haemophilia. 2022;28(5):702–712. Available from: https://onlinelibrary.wiley.com/doi/10.1111/hae.14595
- Patel-Hett S, Martin EJ, Mohammed BM, et al. Marstacimab, a tissue factor pathway inhibitor neutralizing antibody, improves coagulation parameters of ex vivo dosed haemophilic blood and plasmas. Haemophilia. 2019;25(5):797–806.
- Parng C, Singh P, Pittman DD, et al. Translational pharmacokinetic/pharmacodynamic characterization and Target-Mediated drug disposition modeling of an Anti–Tissue factor pathway inhibitor antibody, PF-06741086. J Pharm Sci. 2018;107(7):1995–2004.
- Dose escalation study of PF-06741086 in healthy subjects. [cited 2022 Jun 7]. Available from: https://clinicaltrials.gov/ct2/show/NCT02531815
- Cardinal M, Kantaridis C, Zhu T, et al. A first-in-human study of the safety, tolerability, pharmacokinetics and pharmacodynamics of PF-06741086, an anti-tissue factor pathway inhibitor mAb, in healthy volunteers. J Thromb Haemost. 2018;16(9):1722–1731.
- Study to evaluate safety and tolerability of a single dose of PF-06741086 in Chinese adult participants with severe hemophilia. [cited 2022 Jun 2]. Available from: https://clinicaltrials.gov/ct2/show/NCT04878731?term=nct04878731&draw=2&rank=1
- Mahlangu J, Lamas J, Morales J, et al. A Phase 1b/2 study of the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of PF-06741086, an anti-TFPI monoclonal antibody, in patients with severe Hemophilia A or B. Res Pract Thromb Haemost. 2019;3(1):85–86.
- PF-06741086 long-term treatment in severe Hemophilia. [cited 2022 Jun 7]. Available from: https://clinicaltrials.gov/ct2/show/NCT03363321
- PF-06741086 multiple dose study in severe hemophilia. [cited 2022 Jun 7]. Available from: https://clinicaltrials.gov/ct2/show/NCT02974855
- Mahlangu J, Jl L, Morales JC, et al. Long-term safety and efficacy of the Anti-TFPI monoclonal antibody marstacimab in patients with severe Haemophilia A or B. Res Pract Thromb Haemost. 2021;5. Available from: https://abstracts.isth.org/abstract/long-term-safety-and-efficacy-of-the-anti-tfpi-monoclonal-.
- Open-Label extension study of marstacimab in Hemophilia participants with or without inhibitors. Open-Label extension study of marstacimab in Hemophilia participants with or without inhibitors. [cited 2022 Jun 2].
- Study of the efficacy and safety PF-06741086 in adult and teenage participants with severe Hemophilia A or moderately severe to severe hemophilia B. [cited 2022 Jun 2]. Available from: https://clinicaltrials.gov/ct2/show/NCT03938792?term=NCT03938792&draw=2&rank=1
- Kwak H, Lee S, Jo S, et al. MG1113, a specific anti–tissue factor pathway inhibitor antibody, rebalances the coagulation system and promotes hemostasis in hemophilia. Res Pract Thromb Haemost. 2020;4(8):1301–1312.
- young KE, ju KM, Hyun PJ, et al. Target-mediated drug disposition modeling of an anti-TFPI antibody (MG1113) in cynomolgus monkeys to predict human pharmacokinetics and pharmacodynamics. J Thromb Haemost. 2021;19(6):1425–1435.
- A study to investigate the safety, tolerability, PK and PD of MG1113 in healthy subjects and Hemophilia patients. [cited 2022 Jun 2]. Available from: https://clinicaltrials.gov/ct2/show/NCT03855696?term=nct03855696&draw=2&rank=1
- Borker A, Choudhary NR. Rituximab. Indian Pediatr. 2011;48(8):627–632.
- Ng KP, Cambridge G, Leandro MJ, et al. B cell depletion therapy in systemic lupus erythematosus: long-term follow-up and predictors of response. Ann Rheum Dis. 2007;66(9):1259–1262.
- Franchini M, Mengoli C, Lippi G, et al. Immune tolerance with rituximab in congenital haemophilia with inhibitors: a systematic literature review based on individual patients’analysis. Haemophilia. 2008;14(5):903–912.
- Giulino LB, Bussel JB, Neufeld EJ. Treatment with rituximab in benign and malignant hematologic disorders in children. J Paediatr. 2007;150(4):338–344.e1.
- Rituximab to treat severe Hemophilia A (RICH). [cited 2022 Jun 7]. Available from: https://clinicaltrials.gov/ct2/show/NCT00331006
- Leissinger C, Josephson CD, Granger S, et al. Rituximab for treatment of inhibitors in haemophilia a: a phase II study. Thromb Haemost. 2014;112(9):445–458.
- Lisowski L, Staber JM, Wright JF, et al. The intersection of vector biology, gene therapy, and hemophilia. Res Pract Thromb Haemost. 2021;5(6). DOI:10.1002/rth2.12586.
- Doshi BS, Arruda VR. Gene therapy for hemophilia: what does the future hold? Ther Adv Hematol. 2018;9(9):273–293.
- High KA, Roncarolo MG. Gene therapy. N Engl J Med. 2019;381(5):455–464. Available from: http://www.nejm.org/doi/10.1056/NEJMra1706910
- Inagaki K, Piao C, Kotchey NM, et al. Frequency and spectrum of genomic integration of recombinant Adeno-Associated virus Serotype 8 vector in neonatal mouse liver. J Virol. 2008;82(19):9513–9524.
- Mcintosh J, Lenting PJ, Rosales C, et al. Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant. Blood. 2013;121(17):3335–3344. Available from: http://ashpublications.org/blood/article-pdf/121/17/3335/1365452/3335.pdf
- Gene Therapy for Haemophilia A. (GO–8). [cited 2022 Jun 2]. Available from: https://www.clinicaltrials.gov/ct2/show/NCT03001830?term=nct03001830&draw=2&rank=1
- Study to test the safety and how well patients with severe Hemophilia A respond to treatment with BAY 2599023 (DTX 201). [cited 2022 Jun 7]. Available from: https://clinicaltrials.gov/ct2/show/NCT03588299
- ASC618 gene therapy in hemophilia A patients. [cited 2022 Jun 2]. Available from: https://www.clinicaltrials.gov/ct2/show/NCT04676048?term=nct04676048&draw=2&rank=1
- Veselinovic M, Gilam A, Ross A, et al. Preclinical-Development-of-ASC-618, an advanced human factor VIII gene therapy vector for the treatment of Hemophilia A: results from FRG-KO humanized liver mice, C57BI/6 mice and cynomolgus monkeys. In: ASGCT 23rd Annual Meeting Abstract Presentation, Boston, MA; 2020.
- A Gene Transfer Study for Hemophilia A. Previous study | return to list | next study A gene transfer study for Hemophilia A. [cited 2022 Jun 2].
- Dose-finding study of SPK-8016 gene therapy in patients with Hemophilia A to support evaluation in individuals with FVIII inhibitors. dose-finding study of SPK-8016 gene therapy in patients with Hemophilia A to support evaluation in individuals with FVIII inhibitors. [cited 2022 Jun 2].
- Spark Announces Preliminary Data from Investigational Gene Therapy Trial. National Hemophilia Foundation. 2021. [cited 2022 Aug 2]. Available from: https://www.hemophilia.org/news/spark-announces-preliminary-data-from-investigational-gene-therapy-trial
- A study of BAX 888 in male adults with severe Hemophilia A. [cited 2022 Jun 2]. Available from: https://www.clinicaltrials.gov/ct2/show/NCT03370172?term=NCT03370172&draw=2&rank=1
- Gene therapy study in severe Haemophilia A patients (270-201). [cited 2022 Jun 7]. Available from: https://www.clinicaltrials.gov/ct2/show/NCT02576795
- Long BR, Veron P, Kuranda K, et al. Early phase clinical immunogenicity of valoctocogene roxaparvovec, an AAV5-Mediated gene therapy for Hemophilia A. Mol Ther. 2021;29(2): 597–610.
- Safety, tolerability, and efficacy study of valoctocogene roxaparvovec in Hemophilia A with active or prior inhibitors. [cited 2022 Jun 2]. Available from: https://clinicaltrials.gov/ct2/show/NCT04684940?term=Valoctocogene+roxaparvovec&draw=2&rank=3
- Gene therapy study in severe Hemophilia A patients with antibodies against AAV5 (27–203). [cited 2022 Jun 2]. Available from: https://clinicaltrials.gov/ct2/show/NCT03520712?term=Valoctocogene+roxaparvovec&draw=2&rank=5
- A study of recombinant AAV2:6 human factor 8 gene therapy SB-525 (PF-07055480) in subjects with severe Hemophilia A. [cited 2022 Jun 2]. Available from: https://clinicaltrials.gov/ct2/show/NCT03061201?term=SB-525+%28PF-07055480%29&draw=2&rank=1
- Hematopoietic stem cell transplantation gene therapy for treatment of severe Hemophilia A (NCT05265767). [cited 2022 Jun 2]. Available from: https://www.clinicaltrials.gov/ct2/show/NCT05265767?term=NCT05265767&draw=2&rank=1
- Hematopoietic stem cell transplantation gene therapy for treatment of severe Hemophilia A (NCT04418414). [cited 2022 Jun 2]. Available from: https://www.clinicaltrials.gov/ct2/show/NCT04418414?term=nct04418414&draw=2&rank=1
- Lentiviral FVIII Gene Therapy. [cited 2022 Jun 2]. Available from: https://www.clinicaltrials.gov/ct2/show/NCT03217032?term=nct03217032&draw=2&rank=1
- Gene therapy trial for platelet derived factor VIII production in Hemophilia A. [cited 2022 Jun 2]. Available from: https://www.clinicaltrials.gov/ct2/show/NCT03818763?term=nct03818763&draw=2&rank=1
- Nathwani AC. Gene therapy for hemophilia. Hematol Am SocHematol Educ Progr. 2019;1:1–8.
- Gollomp KL, Doshi BS, Arruda VR. Gene therapy for hemophilia: progress to date and challenges moving forward. Transfus Apheresis Sci. 2019;59(5):602–612.
- Manco-Johnson MJ, Abshire TC, Shapiro AD, et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe Hemophilia. N Engl J Med. 2007;357(6):535–579. Available from: www.nejm.org
- Hassan E, Lancashire J, Motwani J. Emicizumab in previously untreated and minimally treated infants. Haemophilia. 2022;28(2):61–63.
- Escuriola Ettingshausen C, Sidonio RF. Design of an international investigator-initiated study on MOdern Treatment of Inhibitor-positiVe pATiEnts with haemophilia A (MOTIVATE). Ther Adv Hematol. 2021;12:1–12.
- Batsuli G, Zimowski KL, Tickle K, et al. Immune tolerance induction in paediatric patients with haemophilia A and inhibitors receiving emicizumab prophylaxis. Haemophilia. 2019;25(5):789–796.
- Carcao M, Mancuso ME, Young G, et al. Key questions in the new hemophilia era: update on concomitant use of FVIII and emicizumab in hemophilia A patients with inhibitors. Expert Rev Hematol. 2021;14(2):143–148.