• Media type: E-Article
  • Title: Immune tolerance induction in severe haemophilia A: A UKHCDO inhibitor and paediatric working party consensus update
  • Contributor: Hart, Daniel P.; Alamelu, Jayanthi; Bhatnagar, Neha; Biss, Tina; Collins, Peter W.; Hall, Georgina; Hay, Charles; Liesner, Ri; Makris, Michael; Mathias, Mary; Motwani, Jayashree; Palmer, Ben; Payne, Jeanette; Percy, Charles; Richards, Michael; Riddell, Anne; Talks, Kate; Tunstall, Oliver; Chalmers, Elizabeth
  • Published: Wiley, 2021
  • Published in: Haemophilia, 27 (2021) 6, Seite 932-937
  • Language: English
  • DOI: 10.1111/hae.14381
  • ISSN: 1351-8216; 1365-2516
  • Origination:
  • Footnote:
  • Description: <jats:title>Abstract</jats:title><jats:sec><jats:title>Introduction</jats:title><jats:p>In good risk patients (historic inhibitor peak &lt; 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.</jats:p></jats:sec><jats:sec><jats:title>Methods</jats:title><jats:p>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.</jats:p></jats:sec><jats:sec><jats:title>Results</jats:title><jats:p>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.</jats:p></jats:sec><jats:sec><jats:title>Conclusion</jats:title><jats:p>This consensus protocol will facilitate future evaluation of ITI outcomes in the evolving landscape of haemophilia therapeutics and ITI strategies.</jats:p></jats:sec>