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Lookup NU author(s): Dr Tina Biss, Dr Katherine Talks
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© 2021 John Wiley & Sons Ltd.Introduction: In good risk patients (historic inhibitor peak < 200BU), the International Immune Tolerance Study demonstrated equal efficacy to induce tolerance between high (200iu/kg/day) and low dose (50iu/kg ×3 times/week) immune tolerance induction (ITI) regimens. However, the trial stopped early on account of the excessive bleed rate in the low dose ITI arm. Methods: United Kingdom Haemophilia Centre Doctors’ Organization (UKHCDO) Paediatric and Inhibitor working parties considered available ITI data alongside the bi-phenotypic antibody emicizumab (Hemlibra®) efficacy and safety data to develop a consensus guideline for the future UK ITI guideline. Results: This revision of UKHCDO ITI guidance incorporates the recommendation to use emicizumab as a prophylaxis haemostatic agent to reduce bleeding rates and to facilitate low dose and reduced frequency of FVIII CFC for ITI in the majority of children. Conclusion: This consensus protocol will facilitate future evaluation of ITI outcomes in the evolving landscape of haemophilia therapeutics and ITI strategies.
Author(s): Hart DP, Alamelu J, Bhatnagar N, Biss T, Collins PW, Hall G, Hay C, Liesner R, Makris M, Mathias M, Motwani J, Palmer B, Payne J, Percy C, Richards M, Riddell A, Talks K, Tunstall O, Chalmers E
Publication type: Article
Publication status: Published
Journal: Haemophilia
Year: 2021
Volume: 27
Issue: 6
Pages: 932-937
Print publication date: 01/11/2021
Online publication date: 17/08/2021
Acceptance date: 02/04/2020
ISSN (print): 1351-8216
ISSN (electronic): 1365-2516
Publisher: John Wiley and Sons Inc
URL: https://doi.org/10.1111/hae.14381
DOI: 10.1111/hae.14381
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