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Review
. 2018 Sep 6;9(9):295-308.
doi: 10.1177/2040620718796429. eCollection 2018 Sep.

Practical aspects of extended half-life products for the treatment of haemophilia

Thierry Lambert  1 , Gary Benson  2 , Gerry Dolan  3 , Cedric Hermans  4 , Victor Jiménez-Yuste  5 , Rolf Ljung  6 , Massimo Morfini  7 , Silva Zupančić-Šalek  8 , Elena Santagostino  9
Affiliations
Review

Practical aspects of extended half-life products for the treatment of haemophilia

Thierry Lambert et al. Ther Adv Hematol. .

Abstract

Haemophilia A and haemophilia B are congenital X-linked bleeding disorders caused by deficiency of coagulation factor VIII (FVIII) and IX (FIX), respectively. The preferred treatment option for patients with haemophilia is replacement therapy. For patients with severe disease, prophylactic replacement of coagulation factor is the treatment of choice; this has been shown to reduce arthropathy significantly, reduce the frequency of bleeds and improve patients' quality of life. Prophylaxis with standard recombinant factor requires regular intravenous infusion at least two (FIX) to three (FVIII) times a week. Recombinant FVIII and FIX products with an extended half-life are in development, or have been recently licensed. With reported mean half-life extensions of 1.5-1.8 times that of standard products for FVIII and 3-5 times that of standard products for FIX, these products have the potential to address many of the unmet needs of patients currently treated with standard factor concentrates. For example, they may encourage patients to switch from on-demand treatment to prophylaxis and improve the quality of life of patients receiving prophylaxis. Indeed, extended half-life products have the potential to reduce the burden of frequent intravenous injections, reducing the need for central venous lines in children, promote adherence, improve outcomes, potentially allow for more active lifestyles and, depending on the dosing regimen, increase factor trough levels. Members of the Zürich Haemophilia Forum convened for their 19th meeting to discuss the practicalities of incorporating new treatments into the management of people with haemophilia. This review of extended half-life products considers their introduction in haemophilia treatment, including the appropriate dose and schedule of infusions, laboratory monitoring, patient selection, safety considerations, and the economic aspects of care.

Keywords: extended half-life products; factor IX; factor VIII; haemophilia; practical considerations; prophylaxis.

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Conflict of interest statement

Conflict of interest statement: TL has received reimbursement for attending symposia/congresses and/or honoraria for consulting and/or funds for research from Baxter (Baxalta), Bayer, CSL Behring, LFB, Novo Nordisk, Octapharma, Pfizer, Roche, and Biogen and Sobi. GB has received honoraria from Novo Nordisk for speaking and participating on advisory boards. GD has received honoraria from Novo Nordisk for speaking and participating on advisory boards. CH has acted as a consultant and been a board member for Baxter (Baxalta), Bayer, CAF-DCF, CSL Behring, LFB, Octapharma, Novo Nordisk, Pfizer, and Biogen and Sobi, and has received grants from Baxter (Baxalta), Bayer, and Pfizer. VJ-Y has received reimbursement for attending symposia/congresses and/or honoraria for speaking and/or consulting and/or funds for research from Shire, Bayer, CSL Behring, Grifols, Novo Nordisk, Octapharma, and Pfizer. RL has received consultancy or speaker fees from Baxter (Baxalta), Bayer, Biogen, Novo Nordisk, and Octapharma. MM has acted as a paid consultant to Baxter (Baxalta), Bayer, and Novo Nordisk, and has served as a consultant on Pfizer advisory boards; has received speaker fees from Bayer, CSL Behring, Novo Nordisk, and Octapharma, and has received unrestricted research grants from Baxter (Baxalta), Bayer, and Pfizer. SZ-S has received reimbursement for attending symposia and congresses, and honoraria payment for speaking, from Baxter (Baxalta), Novo Nordisk, Octapharma, and Biogen and Sobi. ES has received speaker fees for meetings organized by Bayer, Bioverativ, CSL Behring, Grifols, Kedrion, Novo Nordisk, Octapharma, Roche, Shire, Biogen and Sobi, and Pfizer; has acted as a paid consultant for Bayer, Bioverativ, CSL Behring, Grifols, Kedrion, Novo Nordisk, Pfizer, Roche, Shire, and Biogen and Sobi.

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