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Review

Clinical Utility of Subcutaneous Factor VIII Replacement Therapies in Hemophilia A: A Review of the Evidence

ORCID Icon &
Pages 1031-1036 | Published online: 07 Dec 2021

References

  • Iorio A, Stonebracker 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;171:540–546. doi:10.7326/M19-1208
  • 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;357:535–544. doi:10.1056/NEJMoa067659
  • Gringeri A, Lundin B, von Mackensen S, Mantovani L, Mannucci PM; ESPRIT Study Group. A randomized clinical trial of prophylaxis in children with hemophilia A (the ESPRIT Study). J Thromb Haemost. 2011;9:700–710. doi:10.1111/j.1538-7836.2011.04214.x
  • Mannucci PM. Hemophilia therapy: the future has begun. Haematologica. 2020;105:545–553. doi:10.3324/haematol.2019.232132
  • Mancuso ME, Mahlangu JN, Pipe SW. The changing treatment landscape in haemophilia: from standard half-life clotting factor concentrates to gene editing. Lancet. 2021;397:630–640. doi:10.1016/S0140-6736(20)32722-7
  • Oldenburg J, Hay CRM, Jiménez-Yuste V, et al. Design of a prospective observational study on the effectiveness and real-world usage of recombinant factor VIII Fc (rFVIIIFc) compared with conventional products in haemophilia A: the A-SURE study. BMJ Open. 2019;9(5):e028012. doi:10.1136/bmjopen-2018-028012
  • Pipe SW, Montgomery RR, Pratt KP, Lenting PJ, Lillicrap D. 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. doi:10.1182/blood-2016-04-713289
  • Chhabra ES, Liu T, Kulman J, et al. BIVV001, a new class of factor VIII replacement for hemophilia A that is independent of von Willebrand factor in primates and mice. Blood. 2020;135:1484–1496. doi:10.1182/blood.2019001292
  • Fuller JR, Knockenhauer KE, Leksa NC, Peters RT, Batchelor JD. Molecular determinants of the factor VIII/von Willebrand factor complex revealed by BIVV001 cryo-electron microscopy. Blood. 2021;137:2970–2980. doi:10.1182/blood.2020009197
  • Konkle B, Shapiro AD, Quon DV, et al. BIVV001 fusion protein as factor VIII replacement therapy for hemophilia A. N Engl J Med. 2020;383:1018–1027. doi:10.1056/NEJMoa2002699
  • Oldenburg J, Mahlangu JN, Kim B, et al. Emicizumab prophylaxis in hemophilia A with inhibitors. N Engl J Med. 2017;377:809–818. doi:10.1056/NEJMoa1703068
  • Rodriguez-Merchan EC, Valentino AL. Emicizumab: review of the literature and critical appraisal. Haemophilia. 2019;25:11–20. doi:10.1111/hae.13641
  • Noye J, Rowell JA, Zeissink B, Connolly A, Mason JA. A fixed ‘single vial’ dose of subcutaneous desmopressin (DDAVP) produces adequate biologic responses for persons with mild haemophilia A. Haemophilia. 2021;27(4):e540–e542. doi:10.1111/hae.14287
  • Hashim MA, Yamaguchi A, Vo AT, et al. Orally delivered factor VIII restores hemostasis in a hemophilia A dog. ISTH 2021 Congress. Abstract number: OC 48.4.
  • Bittner B, Richter W, Schmidt J. Subcutaneous administration of biotherapeutics: an overview of current challenges and opportunities. BioDrugs. 2018;32:425–440. doi:10.1007/s40259-018-0295-0
  • Supersaxo A, Steffen WRH, Steffen H. Effect of molecular weight on the lymphatic absorption of water-soluble compounds following subcutaneous administration. Pharm Res. 1990;7:167–169. doi:10.1023/A:1015880819328
  • Lind P, Larsson K, Spira J, et al. Novel forms of B-domain deleted recombinant factor VIII molecules. Construction and biochemical characterization. Eur J Biochem. 1995;232:19–27. doi:10.1111/j.1432-1033.1995.tb20776.x
  • Spira J, Widlund L, Österberg T, et al. Pharmaceutical formulation for subcutaneous, intramuscular or intradermal administration of factor VIII of factor IX. International patent application: US patent US5925739A, European patent EP0772452. International filing. 1995 March 30.
  • Shi Q, Kuether EL, Schroeder JA, Fahs SA, Montgomery RR. Intravascular recovery of VWF and FVIII following intraperitoneal injection and differences from intravenous and subcutaneous injection in mice. Haemophilia. 2012;18:639–646. doi:10.1111/j.1365-2516.2011.02735.x
  • Fatouros A, Liden Y, Sjostrom B. Recombinant factor VIII SQ-stability of FVIII:C in homogenates from porcine, monkey and human subcutaneous tissue. J Pharm Pharmacol. 2000;52:797–805. doi:10.1211/0022357001774651
  • Sadler JE. New concepts in von Willebrand disease. Annu Rev Med. 2005;56:173–191. doi:10.1146/annurev.med.56.082103.104713
  • Solecka-Wituska BA, Wuschko S, Kannicht C. Pharmacokinetics of subcutaneously administered simoctocog alfa with von Willebrand factor fragment candidates ina minipig model. Blood. 2017;130(9):1073. doi:10.1182/blood-2017-07-793356
  • Vollack-Hesse N, Olshko O, Werwitzke S, Solecka-Witulska B, Kannicht C, Tiede A. Recombinant VWF fragments improve bioavailability of subcutaneous factor VIII in hemophilia A mice. Blood. 2021;137:1072–1081. doi:10.1182/blood.2020006468
  • Rode F, Almholt K, Petersen M, et al. Preclinical pharmacokinetics and biodistribution of subcutaneously administered glycoPEGylated recombinant factor VIII (N8-GP) and development of a human pharmacokinetic prediction model. J Thromb Haemost. 2018;16:1141–1152. doi:10.1111/jth.14013
  • Klamroth R, Feistritzer C, Friedrich U, et al. Immunogenicity, safety, pharmacokinetics and preliminary efficacy results from the first human subcutaneous factor VIII trial (subcutaneous turoctocog alfa pegol, SC N8-GP) in previously treated patients with hemophilia A (Alleviate 1). J Thromb Haemost. 2020;18:341–351. doi:10.1111/jth.14660
  • Pittman D, Weston S, Shields K, et al. A novel FVIIa variant with increased potency and duration of effect compared to wildtype FVIIa. A study in a dog model of hemophilia A. Blood. 2011;118:2252. doi:10.1182/blood.V118.21.2252.2252
  • 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:e12576. doi:10.1002/rth2.12576
  • Titapiwatanakun R, Moir C, Pruthi RK, Stavlo PL, Schmidt KA, Rodriguez V. Central venous access devices for paediatric patients with haemophilia: a single-institution experience. Haemophilia. 2009;15:168–174. doi:10.1111/j.1365-2516.2008.01906.x
  • Cadé M, Muñoz-Garcia J, Babuty A, et al. FVIII at the crossroad of coagulation, bone and immune biology: emerging evidence of biological activities beyond hemostasis. Drug Discov Today. 2021. doi:10.1016/j.drudis.2021.07.015