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Current view and outcome of ITI therapy : A change over time?

Holstein, K.; Batorova, A.; Carvalho, M.; Fijnvandraat, K.; Holme, P.; Kavakli, K.; Lambert, T.; Rocino, A.; Jiménez-Yuste, V. and Astermark, J. LU (2016) In Thrombosis Research 148. p.38-44
Abstract

Introduction Inhibitor development in people with haemophilia is a serious complication that may require intensive and costly interventions. The goal of inhibitor management should be permanent inhibitor eradication through immune tolerance induction (ITI), but well-designed studies are lacking and the management of patients is therefore defined by the experience and views of the clinician. Objectives To explore the current clinical practice and outcome of ITI therapy in Europe and how this may have changed over the last decade, as well as to provide consensus recommendations to guide clinicians in their clinical practice. Methods A survey was conducted among 16 European haemophilia comprehensive care centres to evaluate current ITI... (More)

Introduction Inhibitor development in people with haemophilia is a serious complication that may require intensive and costly interventions. The goal of inhibitor management should be permanent inhibitor eradication through immune tolerance induction (ITI), but well-designed studies are lacking and the management of patients is therefore defined by the experience and views of the clinician. Objectives To explore the current clinical practice and outcome of ITI therapy in Europe and how this may have changed over the last decade, as well as to provide consensus recommendations to guide clinicians in their clinical practice. Methods A survey was conducted among 16 European haemophilia comprehensive care centres to evaluate current ITI treatment regimens and success rates in severe and mild/moderate haemophilia A and haemophilia B. In addition, an updated literature review was performed as guidance for providing recommendations. Results We demonstrated successful inhibitor treatment in 86% of severe haemophilia A patients with low responding (LR) and 59% of patients with high responding (HR) inhibitors. Some new trends in the management of patients with inhibitors were identified, including a tendency to use low-dose regimens (< 50 IU/kg/d) in both children and adults with HR inhibitors possibly based on similar success rates demonstrated in the I-ITI study compared to a high-dose protocol. Data on ITI therapy in mild and moderate haemophilia as well as haemophilia B were limited. Conclusions The outcome of ITI therapy seems to be stable over time, and treatment regimens remain heterogeneous. The use of low dose regimens however is considered more frequently.

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author
publishing date
type
Contribution to journal
publication status
published
subject
keywords
Haemophilia, Immune tolerance induction, Immunosuppression, Inhibitors, ITI regimens
in
Thrombosis Research
volume
148
pages
38 - 44
external identifiers
  • scopus:84992153352
ISSN
0049-3848
DOI
10.1016/j.thromres.2016.10.015
language
English
LU publication?
no
id
493a672d-4d63-4773-835a-5426d5dc03bc
date added to LUP
2016-11-02 12:50:10
date last changed
2017-10-16 13:01:38
@article{493a672d-4d63-4773-835a-5426d5dc03bc,
  abstract     = {<p>Introduction Inhibitor development in people with haemophilia is a serious complication that may require intensive and costly interventions. The goal of inhibitor management should be permanent inhibitor eradication through immune tolerance induction (ITI), but well-designed studies are lacking and the management of patients is therefore defined by the experience and views of the clinician. Objectives To explore the current clinical practice and outcome of ITI therapy in Europe and how this may have changed over the last decade, as well as to provide consensus recommendations to guide clinicians in their clinical practice. Methods A survey was conducted among 16 European haemophilia comprehensive care centres to evaluate current ITI treatment regimens and success rates in severe and mild/moderate haemophilia A and haemophilia B. In addition, an updated literature review was performed as guidance for providing recommendations. Results We demonstrated successful inhibitor treatment in 86% of severe haemophilia A patients with low responding (LR) and 59% of patients with high responding (HR) inhibitors. Some new trends in the management of patients with inhibitors were identified, including a tendency to use low-dose regimens (&lt; 50 IU/kg/d) in both children and adults with HR inhibitors possibly based on similar success rates demonstrated in the I-ITI study compared to a high-dose protocol. Data on ITI therapy in mild and moderate haemophilia as well as haemophilia B were limited. Conclusions The outcome of ITI therapy seems to be stable over time, and treatment regimens remain heterogeneous. The use of low dose regimens however is considered more frequently.</p>},
  author       = {Holstein, K. and Batorova, A. and Carvalho, M. and Fijnvandraat, K. and Holme, P. and Kavakli, K. and Lambert, T. and Rocino, A. and Jiménez-Yuste, V. and Astermark, J.},
  issn         = {0049-3848},
  keyword      = {Haemophilia,Immune tolerance induction,Immunosuppression,Inhibitors,ITI regimens},
  language     = {eng},
  month        = {12},
  pages        = {38--44},
  series       = {Thrombosis Research},
  title        = {Current view and outcome of ITI therapy : A change over time?},
  url          = {http://dx.doi.org/10.1016/j.thromres.2016.10.015},
  volume       = {148},
  year         = {2016},
}