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Inhibitors in haemophilia: what have we learned from registries? A systematic review.

Osooli, Mehdi LU and Berntorp, Erik LU (2015) In Journal of Internal Medicine 277(1). p.1-15
Abstract
Congenital haemophilia A and B are genetic disorders affecting factor VIII and factor IX production, respectively. Factor replacement is the only effective treatment for these deficiencies, but a patient's immune system can develop inhibitory antibodies which bind and interfere with the function of the replaced factor in a variety of ways. The main treatment goal for patients with inhibitors is to induce immune tolerance to the injected factor. If not successful, a different treatment termed bypass therapy is needed to treat bleeds. The goal of this review is to demonstrate the usefulness of haemophilia registries as information sources to supplement available evidence regarding predictors of inhibitor development and immune tolerance... (More)
Congenital haemophilia A and B are genetic disorders affecting factor VIII and factor IX production, respectively. Factor replacement is the only effective treatment for these deficiencies, but a patient's immune system can develop inhibitory antibodies which bind and interfere with the function of the replaced factor in a variety of ways. The main treatment goal for patients with inhibitors is to induce immune tolerance to the injected factor. If not successful, a different treatment termed bypass therapy is needed to treat bleeds. The goal of this review is to demonstrate the usefulness of haemophilia registries as information sources to supplement available evidence regarding predictors of inhibitor development and immune tolerance induction (ITI) outcomes. In this systematic review, relevant keywords were used to search online academic databases during February 2014. Inclusion criteria were original publication and data obtained from a haemophilia or ITI registry with a minimum of 30 patients. A data collection form was created to extract information from selected manuscripts. Titles, abstracts and then full texts were screened to determine the eligibility of reports for this review. Eleven manuscripts from nine registries were determined eligible and included in the study. Registries have reported on some core variables, but are inconsistent in reporting less practiced predicting variables. Variables that may affect inhibitor and ITI outcomes were each divided into two categories: patient characteristics (such as age and family history) and treatment-related variables (including exposure days, treatment duration and dose). It is recommended that, in addition to exploratory hypothesis testing, a minimum set of variables should be collected and reported by registries. International collaboration and well-designed prospective registries are of major importance to advance this field in order to determine inhibitor risks and ITI outcomes and facilitate the development of new treatments. This article is protected by copyright. All rights reserved. (Less)
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author
organization
publishing date
type
Contribution to journal
publication status
published
subject
in
Journal of Internal Medicine
volume
277
issue
1
pages
1 - 15
publisher
Wiley-Blackwell Publishing Ltd
external identifiers
  • pmid:25169114
  • wos:000346652000001
  • scopus:84917738258
ISSN
1365-2796
DOI
10.1111/joim.12301
language
English
LU publication?
yes
id
6ea237b0-6d77-4532-be39-e0fb57f77bc3 (old id 4612737)
alternative location
http://www.ncbi.nlm.nih.gov/pubmed/25169114?dopt=Abstract
date added to LUP
2014-09-09 19:29:57
date last changed
2017-10-01 03:12:05
@article{6ea237b0-6d77-4532-be39-e0fb57f77bc3,
  abstract     = {Congenital haemophilia A and B are genetic disorders affecting factor VIII and factor IX production, respectively. Factor replacement is the only effective treatment for these deficiencies, but a patient's immune system can develop inhibitory antibodies which bind and interfere with the function of the replaced factor in a variety of ways. The main treatment goal for patients with inhibitors is to induce immune tolerance to the injected factor. If not successful, a different treatment termed bypass therapy is needed to treat bleeds. The goal of this review is to demonstrate the usefulness of haemophilia registries as information sources to supplement available evidence regarding predictors of inhibitor development and immune tolerance induction (ITI) outcomes. In this systematic review, relevant keywords were used to search online academic databases during February 2014. Inclusion criteria were original publication and data obtained from a haemophilia or ITI registry with a minimum of 30 patients. A data collection form was created to extract information from selected manuscripts. Titles, abstracts and then full texts were screened to determine the eligibility of reports for this review. Eleven manuscripts from nine registries were determined eligible and included in the study. Registries have reported on some core variables, but are inconsistent in reporting less practiced predicting variables. Variables that may affect inhibitor and ITI outcomes were each divided into two categories: patient characteristics (such as age and family history) and treatment-related variables (including exposure days, treatment duration and dose). It is recommended that, in addition to exploratory hypothesis testing, a minimum set of variables should be collected and reported by registries. International collaboration and well-designed prospective registries are of major importance to advance this field in order to determine inhibitor risks and ITI outcomes and facilitate the development of new treatments. This article is protected by copyright. All rights reserved.},
  author       = {Osooli, Mehdi and Berntorp, Erik},
  issn         = {1365-2796},
  language     = {eng},
  number       = {1},
  pages        = {1--15},
  publisher    = {Wiley-Blackwell Publishing Ltd},
  series       = {Journal of Internal Medicine},
  title        = {Inhibitors in haemophilia: what have we learned from registries? A systematic review.},
  url          = {http://dx.doi.org/10.1111/joim.12301},
  volume       = {277},
  year         = {2015},
}