Efficacy and safety evaluation of eptacog beta (coagulation factor VIIa [recombinant]-jncw) for the treatment of hemophilia A and B with inhibitors

References

• Iorio A, Stonebraker JS, Chambost H, et al. Establishing the prevalence and prevalence at birth of hemophilia in males: a meta-analytic approach using national registries. Ann Intern Med. 2019 Oct 15;171(8):540–546. doi: 10.7326/M19-1208
   PubMed Web of Science ®Google Scholar
• Srivastava A, Santagostino E, Dougall A, et al. WFH guidelines for the management of hemophilia, 3rd edition. Haemophilia. 2020 Aug;26(Suppl 6):1–158.
   PubMedGoogle Scholar
• Wight J, Paisley S. The epidemiology of inhibitors in haemophilia A: a systematic review. Haemophilia. 2003 Jul;9(4):418–435. doi: 10.1046/j.1365-2516.2003.00780.x
   PubMed Web of Science ®Google Scholar
• DiMichele D. Inhibitor development in haemophilia B: an orphan disease in need of attention. Br J Haematol. 2007 Aug;138(3):305–315. doi: 10.1111/j.1365-2141.2007.06657.x
   PubMed Web of Science ®Google Scholar
• Male C, Andersson NG, Rafowicz A, et al. Inhibitor incidence in an unselected cohort of previously untreated patients with severe haemophilia B: a PedNet study. Haematologica. 2021 Jan 9;106(1):123–129.
   PubMed Web of Science ®Google Scholar
• Oladapo AO, Lu M, Walsh S, et al. Inhibitor clinical burden of disease: a comparative analysis of the CHESS data. Orphanet J Rare Dis. 2018 Nov 9;13(1):198. doi: 10.1186/s13023-018-0929-9
   PubMedGoogle Scholar
• Morfini M, Haya S, Tagariello G, et al. European study on orthopaedic status of haemophilia patients with inhibitors. Haemophilia. 2007 Sep;13(5):606–612. doi: 10.1111/j.1365-2516.2007.01518.x
   PubMed Web of Science ®Google Scholar
• Nuss R, Soucie JM, Evatt B, et al. Changes in the occurrence of and risk factors for hemophilia-associated intracranial hemorrhage. Am J Hematol. 2001 Sep;68(1):37–42. doi: 10.1002/ajh.1146
   PubMed Web of Science ®Google Scholar
• Soucie JM, Cianfrini C, Janco RL, et al. Joint range-of-motion limitations among young males with hemophilia: prevalence and risk factors. Blood. 2004 Apr 1;103(7):2467–2473.
   PubMed Web of Science ®Google Scholar
• Walsh CE, Soucie JM, Miller CH, et al. Impact of inhibitors on hemophilia A mortality in the United States. Am J Hematol. 2015 May;90(5):400–405. doi: 10.1002/ajh.23957
   PubMed Web of Science ®Google Scholar
• Eckhardt CL, Loomans JI, van Velzen AS, et al. Inhibitor development and mortality in non-severe hemophilia A. J Thromb Haemost. 2015 Jul;13(7):1217–1225. doi: 10.1111/jth.12990
   PubMed Web of Science ®Google Scholar
• Gringeri A, Mantovani LG, Scalone L, et al. Cost of care and quality of life for patients with hemophilia complicated by inhibitors: the COCIS study group. Blood. 2003 Oct 1;102(7):2358–2363. doi: 10.1182/blood-2003-03-0941
   PubMed Web of Science ®Google Scholar
• Guh S, Grosse SD, McAlister S, et al. Healthcare expenditures for males with haemophilia and employer-sponsored insurance in the United States, 2008. Haemophilia. 2012 Mar;18(2):268–275. doi: 10.1111/j.1365-2516.2011.02692.x
   PubMed Web of Science ®Google Scholar
• Lindvall K, von Mackensen S, Elmstahl S, et al. Increased burden on caregivers of having a child with haemophilia complicated by inhibitors. Pediatr Blood Cancer. 2014 Apr;61(4):706–711. doi: 10.1002/pbc.24856
   PubMed Web of Science ®Google Scholar
• Erturan G, Guevel B, Alvand A, et al. Over two decades of orthopaedic surgery in patients with inhibitors-quantifying the complication of bleeding. Haemophilia. 2019 Jan;25(1):21–32. doi: 10.1111/hae.13647
   PubMed Web of Science ®Google Scholar
• Rocino A, Franchini M, Coppola A. Treatment and prevention of bleeds in haemophilia patients with inhibitors to factor VIII/IX. J Clin Med. 2017 Apr 17;6(4):46. doi: 10.3390/jcm6040046
   PubMed Web of Science ®Google Scholar
• Young G. How I treat children with haemophilia and inhibitors. Br J Haematol. 2019 Aug;186(3):400–408. doi: 10.1111/bjh.15942
   PubMed Web of Science ®Google Scholar
• Négrier C, Gomperts ED, Oldenburg J. The history of FEIBA: a lifetime of success in the treatment of haemophilia complicated by an inhibitor. Haemophilia. 2006;12(s5):4–13. doi: 10.1111/j.1365-2516.2006.01379.x
   Google Scholar
• Hedner U. Recombinant activated factor VII: 30 years of research and innovation. Blood Rev. 2015 Jun;29(Suppl 1):S4–8.
   PubMedGoogle Scholar
• SEVENFACT® [ Package Insert]. Louisville, KY: HEMA Biologics, LLC; 2022.
   Google Scholar
• Turecek PL, Varadi K, Gritsch H, et al. FEIBA: mode of action. Haemophilia. 2004 Sep;10(Suppl 2):3–9.
   PubMed Web of Science ®Google Scholar
• Abshire T, Kenet G. Recombinant factor VIIa: review of efficacy, dosing regimens and safety in patients with congenital and acquired factor VIII or IX inhibitors. J Thromb Haemost. 2004 Jun;2(6):899–909. doi: 10.1111/j.1538-7836.2004.00759.x
   PubMed Web of Science ®Google Scholar
• Oldenburg J, Mahlangu JN, Kim B, et al. Emicizumab prophylaxis in hemophilia A with inhibitors. N Engl J Med. 2017 Aug 31;377(9):809–818. doi: 10.1056/NEJMoa1703068
   PubMed Web of Science ®Google Scholar
• Wexler M Concizumab approved in Canada for hemophilia B with inhibitors. Hemophilia News Today 2023 Apr 19 [cited Jul 20, 2023]; Available from: https://hemophilianewstoday.com/news/hemophilia-b-therapy-concizumab-be-sold-canada-alhemo/
   Google Scholar
• Astermark J, Donfield SM, DiMichele DM, et al. A randomized comparison of bypassing agents in hemophilia complicated by an inhibitor: the FEIBA NovoSeven Comparative (FENOC) study. Blood. 2007 Jan 15;109(2):546–551. doi: 10.1182/blood-2006-04-017988
   PubMed Web of Science ®Google Scholar
• Gomperts ED, Astermark J, Gringeri A, et al. From theory to practice: applying current clinical knowledge and treatment strategies to the care of hemophilia A patients with inhibitors. Blood Rev. 2008 Feb;22(Suppl 1):S1–11.
   PubMedGoogle Scholar
• Berntorp E. Differential response to bypassing agents complicates treatment in patients with haemophilia and inhibitors. Haemophilia. 2009 Jan;15(1):3–10. doi: 10.1111/j.1365-2516.2008.01931.x
   PubMed Web of Science ®Google Scholar
• Schneiderman J, Nugent DJ, Young G. Sequential therapy with activated prothrombin complex concentrate and recombinant factor VIIa in patients with severe haemophilia and inhibitors. Haemophilia. 2004;10(4):347–351. doi: 10.1111/j.1365-2516.2004.00912.x
   PubMed Web of Science ®Google Scholar
• Gringeri A, Fischer K, Karafoulidou A, et al. Sequential combined bypassing therapy is safe and effective in the treatment of unresponsive bleeding in adults and children with haemophilia and inhibitors. Haemophilia. 2011 Jul;17(4):630–635. doi: 10.1111/j.1365-2516.2010.02467.x
   PubMed Web of Science ®Google Scholar
• Teitel J, Berntorp E, Collins P, et al. A systematic approach to controlling problem bleeds in patients with severe congenital haemophilia A and high-titre inhibitors. Haemophilia. 2007 May;13(3):256–263. doi: 10.1111/j.1365-2516.2007.01449.x
   PubMed Web of Science ®Google Scholar
• Ljung R, Karim FA, Saxena K, et al. 40K glycoPEGylated, recombinant FVIIa: 3-month, double-blind, randomized trial of safety, pharmacokinetics and preliminary efficacy in hemophilia patients with inhibitors. J Thromb Haemost. 2013 Jul;11(7):1260–1268. doi: 10.1111/jth.12237
   PubMed Web of Science ®Google Scholar
• Lentz SR, Ehrenforth S, Karim FA, et al. Recombinant factor VIIa analog in the management of hemophilia with inhibitors: results from a multicenter, randomized, controlled trial of vatreptacog alfa. J Thromb Haemost. 2014 Aug;12(8):1244–1253. doi: 10.1111/jth.12634
   PubMed Web of Science ®Google Scholar
• Mahlangu J, Paz P, Hardtke M, et al. TRUST trial: BAY 86-6150 use in haemophilia with inhibitors and assessment for immunogenicity. Haemophilia. 2016 Nov;22(6):873–879. doi: 10.1111/hae.12994
   PubMed Web of Science ®Google Scholar
• Faraj A, Knudsen T, Desai S, et al. Phase III dose selection of marzeptacog alfa (activated) informed by population pharmacokinetic modeling: a novel hemostatic drug. CPT Pharmacometrics Syst Pharmacol. 2022 Dec;11(12):1628–1637. doi: 10.1002/psp4.12872
   PubMedGoogle Scholar
• Catalyst Biosciences, Inc. Catalyst Biosciences announces change in corporate strategy [Press Release]. 2021 Nov 12 [cited 2023 Jul 12]; Available from: https://ir.catalystbiosciences.com/news-releases/news-release-details/catalyst-biosciences-announces-change-corporate-strategy
   Google Scholar
• European Medicines Agency. CEVENFACTA® summary of product characteristics; 2022 [cited May 22, 2023]; Available from: https://www.ema.europa.eu/en/medicines/human/EPAR/cevenfacta
   Google Scholar
• Chevreux G, Faid V, Scohyers JM, et al. N-/O-glycosylation analysis of human FVIIa produced in the milk of transgenic rabbits. Glycobiology. 2013 Dec;23(12):1531–1546. doi: 10.1093/glycob/cwt085
   PubMed Web of Science ®Google Scholar
• Chevreux G, Tilly N, Leblanc Y, et al. Biochemical characterization of LR769, a new recombinant factor VIIa bypassing agent produced in the milk of transgenic rabbits. Haemophilia. 2017 Jul;23(4):e324–e334. doi: 10.1111/hae.13253
   PubMed Web of Science ®Google Scholar
• Vadivel K, Bajaj SP. Structural biology of factor VIIa/tissue factor initiated coagulation. Front Biosci (Landmark Ed). 2012 Jun 1;17(7):2476–2494.
   PubMedGoogle Scholar
• Jurlander B, Thim L, Klausen NK, et al. Recombinant activated factor VII (rFVIIa): characterization, manufacturing, and clinical development. Semin Thromb Hemost. 2001 Aug;27(4):373–384. doi: 10.1055/s-2001-16890
   PubMed Web of Science ®Google Scholar
• Bernardi F, Mariani G. Biochemical, molecular and clinical aspects of coagulation factor VII and its role in hemostasis and thrombosis. Haematologica. 2021 Feb 1;106(2):351–362. doi: 10.3324/haematol.2020.248542
   PubMed Web of Science ®Google Scholar
• Hansson K, Stenflo J. Post-translational modifications in proteins involved in blood coagulation. J Thromb Haemost. 2005 Dec;3(12):2633–2648. doi: 10.1111/j.1538-7836.2005.01478.x
   PubMed Web of Science ®Google Scholar
• Iino M, Foster DC, Kisiel W. Functional consequences of mutations in Ser-52 and Ser-60 in human blood coagulation factor VII. Arch Biochem Biophys. 1998 Apr 15;352(2):182–192. doi: 10.1006/abbi.1998.0595
   PubMed Web of Science ®Google Scholar
• Dale GL. Coated-platelets: an emerging component of the procoagulant response. J Thromb Haemost. 2005 Oct;3(10):2185–2192.
   PubMed Web of Science ®Google Scholar
• Riedl J, Ay C, Pabinger I. Platelets and hemophilia: a review of the literature. Thromb Res. 2017 Jul;155:131–139. doi: 10.1016/j.thromres.2017.05.013
   PubMed Web of Science ®Google Scholar
• Hoffman M, Monroe DM 3rd, Roberts HR. Activated factor VII activates factors IX and X on the surface of activated platelets: thoughts on the mechanism of action of high-dose activated factor VII. Blood Coagul Fibrinolysis. 1998 Mar;9(Suppl 1):S61–5.
   PubMed Web of Science ®Google Scholar
• Monroe DM. Further understanding of recombinant activated factor VII mode of action. Semin Hematol. 2008 Apr;45(2 Suppl 1):S7–S11.
   PubMed Web of Science ®Google Scholar
• Keshava S, Sundaram J, Rajulapati A, et al. Factor VIIa interaction with EPCR modulates the hemostatic effect of rFVIIa in hemophilia therapy: mode of its action. Blood Adv. 2017 Jun 27;1(15):1206–1214.
   PubMed Web of Science ®Google Scholar
• Dahlbäck B, Villoutreix BO. The anticoagulant protein C pathway. FEBS Lett. 2005 Jun 13;579(15):3310–3316. doi: 10.1016/j.febslet.2005.03.001
   PubMed Web of Science ®Google Scholar
• Das K, Keshava S, Ansari SA, et al. Factor VIIa induces extracellular vesicles from the endothelium: a potential mechanism for its hemostatic effect. Blood. 2021 Jun 17;137(24):3428–3442. doi: 10.1182/blood.2020008417
   PubMed Web of Science ®Google Scholar
• Monroe DM, Hoffman M, Oliver JA, et al. Platelet activity of high-dose factor VIIa is independent of tissue factor. Br J Haematol. 1997 Dec;99(3):542–547. doi: 10.1046/j.1365-2141.1997.4463256.x
   PubMed Web of Science ®Google Scholar
• Ruf W, Rehemtulla A, Morrissey JH, et al. Phospholipid-independent and -dependent interactions required for tissue factor receptor and cofactor function. J Biol Chem. 1991 Aug 25;266(24):16256. doi: 10.1016/S0021-9258(18)98544-3
   PubMedGoogle Scholar
• Maroney SA, Haberichter SL, Friese P, et al. Active tissue factor pathway inhibitor is expressed on the surface of coated platelets. Blood. 2007 Mar 1;109(5):1931–1937. doi: 10.1182/blood-2006-07-037283
   PubMed Web of Science ®Google Scholar
• Mast AE. Tissue factor pathway inhibitor: multiple anticoagulant activities for a single protein. Arterioscler Thromb Vasc Biol. 2016 Jan;36(1):9–14. doi: 10.1161/ATVBAHA.115.305996
   PubMed Web of Science ®Google Scholar
• Vatsyayan R, Kothari H, Mackman N, et al. Inactivation of factor VIIa by antithrombin in vitro, ex vivo and in vivo: role of tissue factor and endothelial cell protein C receptor. Plos One. 2014;9(8):e103505. doi: 10.1371/journal.pone.0103505
   PubMed Web of Science ®Google Scholar
• Olson ST, Richard B, Izaguirre G, et al. Molecular mechanisms of antithrombin-heparin regulation of blood clotting proteinases. A paradigm for understanding proteinase regulation by serpin family protein proteinase inhibitors. Biochimie. 2010 Nov;92(11):1587–1596.
   PubMed Web of Science ®Google Scholar
• Clark CA, Vatsyayan R, Hedner U, et al. Endothelial cell protein C receptor-mediated redistribution and tissue-level accumulation of factor VIIa. J Thromb Haemost. 2012 Nov;10(11):2383–2391. doi: 10.1111/j.1538-7836.2012.04917.x
   PubMed Web of Science ®Google Scholar
• Magisetty J, Pendurthi UR, Madhunapantula SV, et al. Increased accumulation and retention of rhFVIIa (eptacog beta) in knee joints of hemophilia A mice compared to wild-type mice. Thromb Haemost. 2019 Aug;119(8):1283–1294. doi: 10.1055/s-0039-1688907
   PubMed Web of Science ®Google Scholar
• Sutkeviciute I, Mistiniene E, Sereikaite J, et al. The influence of different glycosylation patterns on factor VII biological activity. Biochimie. 2009 Sep;91(9):1123–1130. doi: 10.1016/j.biochi.2009.05.015
   PubMed Web of Science ®Google Scholar
• Klausen NK, Bayne S, Palm L. Analysis of the site-specific asparagine-linked glycosylation of recombinant human coagulation factor VIIa by glycosidase digestions, liquid chromatography, and mass spectrometry. Mol Biotechnol. 1998 Jun;9(3):195–204.
   PubMed Web of Science ®Google Scholar
• Thim L, Bjoern S, Christensen M, et al. Amino acid sequence and posttranslational modifications of human factor VIIa from plasma and transfected baby hamster kidney cells. Biochemistry. 1988 Oct 4;27(20):7785–7793. doi: 10.1021/bi00420a030
   PubMed Web of Science ®Google Scholar
• Grandoni J, Perret G, Forier C. Kinetic analysis and binding studies of a new recombinant human factor VIIa for treatment of haemophilia. Haemophilia. 2017 Mar;23(2):300–308. doi: 10.1111/hae.13110
   PubMed Web of Science ®Google Scholar
• Ghosh S, Pendurthi UR, Steinoe A, et al. Endothelial cell protein C receptor acts as a cellular receptor for factor VIIa on endothelium. J Biol Chem. 2007 Apr 20;282(16):11849–11857. doi: 10.1074/jbc.M609283200
   PubMed Web of Science ®Google Scholar
• Rao LVM, Esmon CT, Pendurthi UR. Endothelial cell protein C receptor: a multiliganded and multifunctional receptor. Blood. 2014 Sep 4;124(10):1553–1562. doi: 10.1182/blood-2014-05-578328
   PubMed Web of Science ®Google Scholar
• Sen P, Gopalakrishnan R, Kothari H, et al. Factor VIIa bound to endothelial cell protein C receptor activates protease activated receptor-1 and mediates cell signaling and barrier protection. Blood. 2011 Mar 17;117(11):3199–3208. doi: 10.1182/blood-2010-09-310706
   PubMed Web of Science ®Google Scholar
• 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 Nov;23(6):844–851. doi: 10.1111/hae.13357
   PubMed Web of Science ®Google Scholar
• NOVOSEVEN® RT [Package Insert]. Bagsværd, Denmark: Novo Nordisk A/S; 2020.
   Google Scholar
• Wang M, Lawrence JB, Quon DV, et al. PERSEPT 1: a phase 3 trial of activated eptacog beta for on-demand treatment of haemophilia inhibitor-related bleeding. Haemophilia. 2017 Nov;23(6):832–843. doi: 10.1111/hae.13301
   PubMed Web of Science ®Google Scholar
• Escobar M, Castaman G, Boix SB, et al. The safety of activated eptacog beta in the management of bleeding episodes and perioperative haemostasis in adult and paediatric haemophilia patients with inhibitors. Haemophilia. 2021 Nov;27(6):921–931. doi: 10.1111/hae.14419
   PubMed Web of Science ®Google Scholar
• Journeycake JM, Hermans C, Ducore JM, et al. Single 225µg/kg dose treatment with eptacog beta (factor VIIa, recombinant) results in rapid hemostasis in joint bleeds for persons with hemophilia A or B with inhibitors: a PERSEPT1 subset analysis [abstract]. Blood. 2020;136(Supplement 1):2–3.
   Google Scholar
• Reding MT, Mancuso ME, Acharya S, et al. Eptacog beta efficacy in treating mild or moderate bleeds in target joints of individuals with hemophilia A or B and inhibitors in PERSEPT 1 [abstract]. Blood. 2022;140(Supplement 1):5597–5599. doi: 10.1182/blood-2022-156110
   Google Scholar
• Boggio L, Mancuso ME, Acharya S, et al. Eptacog beta efficacy at 24 hours post-infustion for mild or moderate bleeds in individuals with hemophilia A or B and inhibitors [abstract]. Am J Hematol. 2023 Feb;98:E13.
   Web of Science ®Google Scholar
• Pipe SW, Hermans C, Chitlur M, et al. Eptacog beta efficacy and safety in the treatment and control of bleeding in paediatric subjects (<12 years) with haemophilia A or B with inhibitors. Haemophilia. 2022;28:548–556. doi: 10.1111/hae.14563
   PubMed Web of Science ®Google Scholar
• Young G, Shapiro AD, Walsh CE, et al. Patient/caregiver-reported recombinant factor VIIa (rFVIIa) dosing: home treatment of acute bleeds in the Dosing Observational Study In Hemophilia (DOSE). Haemophilia. 2012 May;18(3):392–399. doi: 10.1111/j.1365-2516.2011.02704.x
   PubMed Web of Science ®Google Scholar
• Valentino LA, Walsh CE, Reding MT, et al. Patient- and caregiver-reported bleeding symptoms and reasons for starting and stopping treatment with recombinant factor VIIa: analysis of the Dosing Observational Study in Haemophilia (DOSE). Haemophilia. 2012 Jul;18(4):554–560. doi: 10.1111/j.1365-2516.2012.02762.x
   PubMed Web of Science ®Google Scholar
• Amby LK, Seremetis S, Obergfell A, et al. Challenges of defining reliable clinical surrogate end points in haemophilia trials: a critical review. Blood Coagul Fibrinolysis. 2009 Oct;20(7):488–493. doi: 10.1097/MBC.0b013e32832c8803
   PubMed Web of Science ®Google Scholar
• Gruppo RA, Kessler CM, Neufeld EJ, et al. Assessment of individual dose utilization vs. physician prescribing recommendations for recombinant activated factor VII (rFVIIa) in paediatric and adult patients with congenital haemophilia and alloantibody inhibitors (CHwI): the Dosing Observational Study In Hemophilia (DOSE). Haemophilia. 2013 Jul;19(4):524–532. doi: 10.1111/hae.12113
   PubMed Web of Science ®Google Scholar
• Coppola A, Windyga J, Tufano A, et al. Treatment for preventing bleeding in people with haemophilia or other congenital bleeding disorders undergoing surgery. Cochrane Database Syst Rev. 2015 Feb;9(2):CD009961. doi: 10.1002/14651858.CD009961.pub2
   Google Scholar
• Mingot-Castellano ME, Alvarez-Roman MT, Lopez-Fernandez MF, et al. Spanish consensus guidelines on prophylaxis with bypassing agents for surgery in patients with haemophilia and inhibitors. Eur J Haematol. 2016 May;96(5):461–474. doi: 10.1111/ejh.12730
   PubMed Web of Science ®Google Scholar
• Escobar M, Luck J, Averianov Y, et al. PERSEPT 3: a phase 3 clinical trial to evaluate the haemostatic efficacy of eptacog beta (recombinant human FVIIa) in perioperative care in subjects with haemophilia A or B with inhibitors. Haemophilia. 2021 Nov;27(6):911–920. doi: 10.1111/hae.14418
   PubMed Web of Science ®Google Scholar
• Ju HY, Jang HL, Park YS. The efficacy of bypassing agents in surgery of hemophilia patients with inhibitors. Blood Res. 2015 Sep;50(3):173–178. doi: 10.5045/br.2015.50.3.173
   PubMedGoogle Scholar
• Bossard D, Carrillon Y, Stieltjes N, et al. Management of haemophilic arthropathy. Haemophilia. 2008;14(s4):11–19. doi: 10.1111/j.1365-2516.2008.01734.x
   PubMedGoogle Scholar
• Levy GG, Asikanius E, Kuebler P, et al. Safety analysis of rFVIIa with emicizumab dosing in congenital hemophilia A with inhibitors: experience from the HAVEN clinical program. J Thromb Haemost. 2019 May 24;17(9):1470–1477. doi: 10.1111/jth.14491
   PubMed Web of Science ®Google Scholar
• Maria A, Alessandro C, Declan N, et al. Hemorrhagic and thrombotic adverse events associated with emicizumab and extended half-life factor VIII replacement drugs: EudraVigilance data of 2021. J Thromb Haemost. 2023 Mar;21(3):546–552.
   PubMed Web of Science ®Google Scholar
• Grandoni J, Duretz V, Bonzo D, et al. Exploratory in vitro evaluation of thrombin generation of eptacog beta (recombinant human fviia) and emicizumab in congenital haemophilia A plasma. Haemophilia. 2021 Mar;27(2):321–328. doi: 10.1111/hae.14253
   PubMed Web of Science ®Google Scholar
• Medical and Scientific Advisory Council (MASAC). MASAC recommendations concerning products licensed for the treatment of hemophilia and other bleeding disorders. MASAC Document #272; (NY), NY: National Hemophilia Foundation; 2022.
   Google Scholar
• Ciolek AM, Arnall J, Moore DC, et al. Eptacog beta for bleeding treatment and prevention in congenital hemophilia A and B with inhibitors: a review of clinical data and implications for clinical practice. Ann Pharmacother. 2022 Jul;56(7):831–838. doi: 10.1177/10600280211049394
   PubMed Web of Science ®Google Scholar
• Alexander WA, Jensen I, Hathway J, et al. Bleeding in patients with hemophilia who have inhibitors: modeling US medical system utilization and cost avoidance between recombinant factor VIIa products with different clinical dosing requirements. J Manag Care Spec Pharm. 2022 May;28(5):518–527. doi: 10.18553/jmcp.2022.21197
   PubMed Web of Science ®Google Scholar
• Youkhana K, Abajas Y. Successful use of eptacog beta prophylaxis in an adolescent with severe hemophilia B with high-titer inhibitor resulting in decreased bleeding episodes and cost of treatment [abstract]. Am J Hematol. 2023 Feb;98:E122.
   PubMed Web of Science ®Google Scholar
• Berntorp E, Shapiro AD. Modern haemophilia care. Lancet. 2012 Apr 14;379(9824):1447–1456. doi: 10.1016/S0140-6736(11)61139-2
   PubMed Web of Science ®Google Scholar
• Pipe SW. The promise and challenges of bioengineered recombinant clotting factors. J Thromb Haemost. 2005 Aug;3(8):1692–1701. doi: 10.1111/j.1538-7836.2005.01367.x
   PubMed Web of Science ®Google Scholar
• Berntorp E, Fischer K, Hart DP, et al. Haemophilia. Nat Rev Dis Primers. 2021 Jun 24;7(1):45. doi: 10.1038/s41572-021-00278-x
   PubMed Web of Science ®Google Scholar
• Pipe SW, Leebeek FWG, Recht M, et al. Gene therapy with etranacogene dezaparvovec for hemophilia B. N Engl J Med. 2023 Feb 23;388(8):706–718. doi: 10.1056/NEJMoa2211644
   PubMed Web of Science ®Google Scholar
• von Drygalski A, Chowdary P, Kulkarni R, et al. Efanesoctocog alfa prophylaxis for patients with severe hemophilia A. N Engl J Med. 2023 Jan 26;388(4):310–318.
   PubMed Web of Science ®Google Scholar
• Linari S, Castaman G. Concomitant use of rFVIIa and emicizumab in people with hemophilia A with inhibitors: current perspectives and emerging clinical evidence. Ther Clin Risk Manag. 2020;16:461–469.
   PubMed Web of Science ®Google Scholar