Immune tolerance induction in severe haemophilia A: A UKHCDO inhibitor and paediatric working party consensus update. Issue 6 (17th August 2021)

• Record Type: Journal Article
• Authors: 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
• Is Part Of: Haemophilia. Volume 27:Issue 6(2021)
• Page Start: 932
• Page End: 937
• Publication Date: 2021-08-17
• Subjects: immune tolerance induction -- FVIII -- emicizumab -- severe haemophilia A -- tolerance -- ITI -- PUP; Hemophilia -- Periodicals; 616.1572005
• DOI: 10.1111/hae.14381
• Languages: English
• ISSNs: 1351-8216

Abstract

Abstract: 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.