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Immune tolerance induction in patients with severe hemophilia with inhibitors: expert panel views and recommendations for clinical practice.

Benson, Gary ; Auerswald, Günter ; Elezović, Ivo ; Lambert, Thierry ; Ljung, Rolf LU orcid ; Morfini, Massimo ; Remor, Eduardo and Salek, Silva Zupančić (2012) In European Journal of Haematology 88(5). p.371-379
Abstract
For hemophilia patients with inhibitors, immune tolerance induction (ITI) may help to restore clinical response to Factor (F) VIII or FIX concentrates. Several ITI regimens and protocols exist; however, despite 30 yr of progressive investigation, the ITI evidence base relies mainly on observational data. Expert opinion, experience, and interpretation of the available evidence are therefore valuable to support clinical decision-making. At the Sixth Zürich Haemophilia Forum an expert panel considered recent data and consensus to distill key practice points relating to ITI. The panel supported current recommendations that, where feasible, ITI should be offered early to children and adults (ideally ≤5 yr of inhibitor detection) when inhibitor... (More)
For hemophilia patients with inhibitors, immune tolerance induction (ITI) may help to restore clinical response to Factor (F) VIII or FIX concentrates. Several ITI regimens and protocols exist; however, despite 30 yr of progressive investigation, the ITI evidence base relies mainly on observational data. Expert opinion, experience, and interpretation of the available evidence are therefore valuable to support clinical decision-making. At the Sixth Zürich Haemophilia Forum an expert panel considered recent data and consensus to distill key practice points relating to ITI. The panel supported current recommendations that, where feasible, ITI should be offered early to children and adults (ideally ≤5 yr of inhibitor detection) when inhibitor titers are <10 Bethesda Units, and should be stopped when successful tolerance is achieved. For hemophilia A inhibitor patients, ITI can be founded on recombinant FVIII at high doses. The panel considered that patients with a high bleeding frequency should be offered additional prophylaxis with a bypassing agent. For hemophilia B patients, there may be a benefit to genetic testing to indicate the risk for inhibitors. ITI is often less effective and associated with a greater risk of side effects in these patients. For high-titer inhibitor (≥5 Bethesda Units) hemophilia B patients, the panel advised that bypassing agents could be offered on demand in addition to ITI. Within future ITI regimens there may be a role for additional immunosuppressant therapies. Participants agreed that research is needed to find alternatives to ITI therapy that offer durable and sustained effects and reduced rates of complications. © 2012 John Wiley & Sons A/S. (Less)
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author
; ; ; ; ; ; and
organization
publishing date
type
Contribution to journal
publication status
published
subject
in
European Journal of Haematology
volume
88
issue
5
pages
371 - 379
publisher
Wiley-Blackwell
external identifiers
  • wos:000302612200001
  • pmid:22260405
  • scopus:84859582867
  • pmid:22260405
ISSN
1600-0609
DOI
10.1111/j.1600-0609.2012.01754.x
language
English
LU publication?
yes
id
2938c37f-e481-4cdc-bf01-bf8672eb7685 (old id 2336159)
alternative location
http://www.ncbi.nlm.nih.gov/pubmed/22260405?dopt=Abstract
date added to LUP
2016-04-04 08:14:34
date last changed
2025-04-04 14:39:11
@article{2938c37f-e481-4cdc-bf01-bf8672eb7685,
  abstract     = {{For hemophilia patients with inhibitors, immune tolerance induction (ITI) may help to restore clinical response to Factor (F) VIII or FIX concentrates. Several ITI regimens and protocols exist; however, despite 30 yr of progressive investigation, the ITI evidence base relies mainly on observational data. Expert opinion, experience, and interpretation of the available evidence are therefore valuable to support clinical decision-making. At the Sixth Zürich Haemophilia Forum an expert panel considered recent data and consensus to distill key practice points relating to ITI. The panel supported current recommendations that, where feasible, ITI should be offered early to children and adults (ideally ≤5 yr of inhibitor detection) when inhibitor titers are &lt;10 Bethesda Units, and should be stopped when successful tolerance is achieved. For hemophilia A inhibitor patients, ITI can be founded on recombinant FVIII at high doses. The panel considered that patients with a high bleeding frequency should be offered additional prophylaxis with a bypassing agent. For hemophilia B patients, there may be a benefit to genetic testing to indicate the risk for inhibitors. ITI is often less effective and associated with a greater risk of side effects in these patients. For high-titer inhibitor (≥5 Bethesda Units) hemophilia B patients, the panel advised that bypassing agents could be offered on demand in addition to ITI. Within future ITI regimens there may be a role for additional immunosuppressant therapies. Participants agreed that research is needed to find alternatives to ITI therapy that offer durable and sustained effects and reduced rates of complications. © 2012 John Wiley &amp; Sons A/S.}},
  author       = {{Benson, Gary and Auerswald, Günter and Elezović, Ivo and Lambert, Thierry and Ljung, Rolf and Morfini, Massimo and Remor, Eduardo and Salek, Silva Zupančić}},
  issn         = {{1600-0609}},
  language     = {{eng}},
  number       = {{5}},
  pages        = {{371--379}},
  publisher    = {{Wiley-Blackwell}},
  series       = {{European Journal of Haematology}},
  title        = {{Immune tolerance induction in patients with severe hemophilia with inhibitors: expert panel views and recommendations for clinical practice.}},
  url          = {{http://dx.doi.org/10.1111/j.1600-0609.2012.01754.x}},
  doi          = {{10.1111/j.1600-0609.2012.01754.x}},
  volume       = {{88}},
  year         = {{2012}},
}