Real-life experience in switching to new extended half-life products at European haemophilia centres

Flora Peyvandi, Isabella Garagiola, Marco Boscarino, Aislin Ryan, Cedric Hermans, Michael Makris, Flora Peyvandi, Isabella Garagiola, Marco Boscarino, Aislin Ryan, Cedric Hermans, Michael Makris

Abstract

The concept of replacement therapy in haemophilia is changing significantly thanks to the switch from standard products to extended half-life products. These novel drugs are showing beneficial effects overcoming current prophylaxis limitations by reducing the infusion frequency, maintaining a higher trough level to ensure a lower risk of bleeding, and making treatment significantly less distressing to patients by improving the quality of life. Real-life data on the efficacy of novel drugs and their impact on routine management of haemophilia A and B patients are still limited. This manuscript reports the results of a European survey conducted by the European Association for Haemophilia and Allied Disorders (EAHAD) at the beginning of 2018 on the clinical management of patients using extended half-life recombinant FVIII and FIX fusion products, since at the time of the survey none of the PEGylated products were available yet. We report data on the efficacy of these novel drugs by 33 European haemophilia centres that have already switched to extended half-life fusion products, showing a significant reduction in the number of infusions and a satisfactory trough levels in the clinical care of haemophilia patients, with a greater impact for haemophilia B.

Keywords: extended half-life products; hemophilia; infusion; treatment; trough level.

© 2019 John Wiley & Sons Ltd.

References

REFERENCES

    1. Mannucci PM, Tuddenham EG. The haemophilias-from royal genes to gene therapy. N Engl J Med. 2001;344:1773-1779.
    1. Peyvandi F, Garagiola I, Young G. The past and future of haemophilia: diagnosis, treatments, and its complications. Lancet. 2016;388:187-197.
    1. Manco-Johnson MJ, Abshire TC, Shapiro AD, et al. Prophylaxis versus episodic treatment to prevent joint disease in boys with severe haemophilia. N Engl J Med. 2007;357:535-544.
    1. Shapiro A, Chaudhury A, Jain N, et al. Real-world data on the use of rFIXFc in subjects with hemophilia B for Up to 3.7 years demonstrates improved bleed control and adherence with reduced treatment burden. Blood. 2018;132;(Suppl. 1):2493.
    1. Peyvandi F, Garagiola I, Seregni S. Future of coagulation factor replacement therapy. J Thromb Haemost. 2013;11(Suppl 1):84-98.
    1. Ragni M. New and emerging agents for the treatment of hemophilia: focus on extended half-life recombinant clotting proteins. Drugs. 2015;75:1587-1600.
    1. Peyvandi F, Garagiola I. Treatment of haemophilia in the near future. Semin Thromb Hemost. 2015;41:838-848.
    1. Powell JS. Longer-acting clotting factor concentrates for hemophilia. J Thromb Haemost. 2015;13(Suppl 1):S167-S175.
    1. Peyvandi F, Garagiola I, Biguzzi E. Advances in the treatment of bleeding disorders. J Thromb Haemost. 2016;14:2095-2106.
    1. Berntorp E, Andersson NG. Prophylaxis for hemophilia in the era of extended half-life factor VIII/Factor IX products. Semin Thromb Hemost. 2016;42:518-525.
    1. Mahlangu J, Powell JS, Ragni MV, et al. Phase 3 study of recombinant factor VIII Fc fusion protein in severe haemophilia A. Blood. 2014;123:317-325.
    1. Santagostino E, Martinowitz U, Lissitchkov T, et al. PROLONG-9FP Investigators Study Group. Long acting recombinant coagulation factor IX albumin fusion protein (rIX-FP) in haemophilia B: results of a phase 3 trial. Blood. 2016;127:1761-1769.
    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;169:768-776.
    1. Lyseng-Williamson KA. Coagulation factor IX (Recombinant), albumin fusion protein (Albutrepenonacog Alfa; Idelvion®): a review of its use in haemophilia B. Drugs. 2017;77:97-106.
    1. Oldenburg J, Carcao M, Lentz SR, et al. Once-weekly prophylaxis with 40 IU/kg nonacog beta pegol (N9-GP) achieves trough levels of >15% in patients with haemophilia B: pooled data from the paradigm™ trials. Haemophilia. 2018;24:911-920.
    1. Carcao M, Lambert T, Leissinger C, et al. Prophylaxis re-visited: the potential impact of novel factor and non-factor therapies on prophylaxis. Haemophilia. 2018;24:845-848.
    1. Wyrwich KW, Krishnan S, Auguste P, et al. Changes in health-related quality of life with treatment of longer-acting clotting factors: results in the A-LONG and B-LONG clinical studies. Haemophilia. 2016;22:866-872.
    1. von Mackensen S, Shah J, Seifert W, Kenet G. Health-related quality of life in paediatric haemophilia B patients treated with rIX-FP. Haemophilia. 2019;25:45-53.
    1. Peyvandi F, Rosendaal FR, O’Mahony B, Mannucci PM. Pediatric requirements in Europe stymie help for hemophilia. Nat Med. 2014;20:466.
    1. Dunn AL, Ahuja SP, Mullins ES. Real-world experience with use of antihemophilic factor (Recombinant), PEGylated for prophylaxis in severe haemophilia A. Haemophilia. 2018;24(3):e84-e92.
    1. Keepanasseril A, Stoffman J, Bouskill V, et al. Switching to extended half-life products in Canada - preliminary data. Haemophilia. 2017;23(4):e365-e367.
    1. Wang C, Young G. Clinical use of recombinant factor VIII Fc and recombinant factor IX Fc in patients with haemophilia A and B. Haemophilia. 2018;24:414-419.
    1. Tagliaferri A, Quintavalle G, Riccardi F, et al. Benefits of switch to FVIII-FC: Experience of prophylaxis in eight patients. Haemophilia. 2018;24(Suppl 1):55.
    1. Jimenez R, Nuñez R, Jimenez P, Rodriguez-Martorell FJ, Perez-Simon JA. Switch to extended half-life recombinant factor VIII FC in severe haemophilia A patients under prophylaxis. Experience in one centre. Haemophilia. 2018;24(Suppl 1):117.
    1. Prunty K, Brady B, Kelly I, et al. Real world experience of Elocta in paediatric patients with severe haemophilia A in Ireland. WFH 2018 World Congress. Haemophilia. 2018;2018(S5):27.
    1. Scott M, Xiang H, Collins PW, Hay C. The effect of switching to rFVIIIFc on treatment patterns and annualized bleed rate before and after: A within-patient comparison from the UK National Haemophilia Database. WFH 2018 World Congress. Haemophilia. 2018;2018(S5):91.
    1. Pouplard C, Sattler L, Ryman A, et al. Multicenter Pharmacokinetic Evaluation of rFVIII-Fc (Elocta) in a real life and comparison with non-extended half-life FVIII concentrates. Blood. 2018;132(Suppl. 1):1196.
    1. Chalmers E, Bagot C, Campbell Tait R, et al Scottish experience introducing extended half-life factor concentrates in Haemophilia A and Haemophilia B patients. WFH 2018 World Congress. Haemophilia. 2018;2018(S5):27.
    1. Phillott A, Stanley J, Alamelu J. Single centre experience of switching from standard half-life to extended half-life products in patients with severe haemophilia A and B. WFH 2018 World Congress. Haemophilia. 2018;2018(S5):32.
    1. Peyvandi F, Makris M, Collins P, et al. Subcommittee on factor VIII, factor IX and rare coagulation disorders. Minimal dataset for post-registration surveillance of new drugs in hemophilia: communication from the SSC of the ISTH. J Thromb Haemost. 2017;15:1878-1881.
    1. Ivens IA, Achanzar W, Baumann A, et al. PEGylated biopharmaceuticals: current experience and considerations for nonclinical development. Toxicol Pathol. 2015;43:959-983.
    1. Turecek PL, Bossard MJ, Schoetens F, Ivens IA. PEGylation of biopharmaceuticals: a review of chemistry and nonclinical safety information of approved drugs. J Pharm Sci. 2016;105:460-475.
    1. Aledort LM, Jain N, Li N, Krishnan S, Hagberg B, Su J. Changing the paradigm in hemophilia care: extended half-life products (rFVIIIFc and rFIXFc). Blood. 2018;132;(Suppl. 1):3514.
    1. Malec LM, Witmer CM, Jaffray J, et al. Real world use of extended half-life products and the impact on bleeding events and joint health in the United States. Blood. 2018;132(Suppl. 1):1195.
    1. O’Connell N, Lavin M, Byrne M, et al. An en-masse switch to extended half life recombinant factor IX replacement therapy in Irish adults with haemophilia B. WFH 2018 World Congress. Haemophilia. 2018;2018(S5):29.
    1. Ragni M, Kulkarni R, Pasi KJ, et al. B-YOND final results confirm established safety, sustained efficacy, and extended dosing interval for up to 4 years of treatment with rFIXFc in previously treated subjects with severe hemophilia B. Blood. 2018;132(Suppl. 1):1214.

Source: PubMed

3
Subscribe