AI Article Synopsis

  • The International Immune Tolerance Study found that both high and low doses of immune tolerance induction (ITI) were equally effective in good risk patients, although the trial was halted due to excessive bleeding in the low dose group.
  • The UKHCDO used available ITI and emicizumab (Hemlibra®) efficacy data to create new guidelines for ITI in the UK.
  • The updated guidance recommends using emicizumab to lower bleeding rates, allowing for lower doses and less frequent administration of FVIII CFC in most children with hemophilia.

Article 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.

Download full-text PDF

Source
http://dx.doi.org/10.1111/hae.14381DOI Listing

Publication Analysis

Top Keywords

immune tolerance
4
tolerance induction
4
induction severe
4
severe haemophilia
4
haemophilia ukhcdo
4
ukhcdo inhibitor
4
inhibitor paediatric
4
paediatric working
4
working party
4
party consensus
4

Similar Publications

Want AI Summaries of new PubMed Abstracts delivered to your In-box?

Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!