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Rationale for individualizing haemophilia care.

Sørensen, Benny; Auerswald, Günter; Benson, Gary; Elezović, Ivo; Felder, Markus; Lambert, Thierry; Morfini, Massimo; Remor, Eduardo; Salaj, Peter and Santagostino, Elena, et al. (2015) In Blood Coagulation and Fibrinolysis 26(8). p.849-857
Abstract
Owing to the heterogeneity in the clinical phenotype of haemophilia A and B, it is now recognized that disease severity (based on factor VIII/IX activity) may no longer be the most appropriate guide for treatment and that a 'one-size-fits-all' approach is unlikely to achieve optimal therapy. Based on the present literature and consensus views of a group of experts in the field, this article highlights key gaps in the understanding of the diverse relationships between bleeding phenotype and factors such as joint health, genetic susceptibility, laboratory parameters, quality of life and management of pain. Early prophylaxis is a potential 'gold standard' therapy and issues surrounding inhibitor development, variations in its clinical use and... (More)
Owing to the heterogeneity in the clinical phenotype of haemophilia A and B, it is now recognized that disease severity (based on factor VIII/IX activity) may no longer be the most appropriate guide for treatment and that a 'one-size-fits-all' approach is unlikely to achieve optimal therapy. Based on the present literature and consensus views of a group of experts in the field, this article highlights key gaps in the understanding of the diverse relationships between bleeding phenotype and factors such as joint health, genetic susceptibility, laboratory parameters, quality of life and management of pain. Early prophylaxis is a potential 'gold standard' therapy and issues surrounding inhibitor development, variations in its clinical use and long-term outcomes are discussed. Comprehensive treatment should be individualized for all patients (including those with mild or moderate haemophilia and carriers). Wherever possible all patients should be given prophylaxis. However, adult patients with a milder haemophilia phenotype may be candidates for ceasing prophylaxis and switching to on-demand treatment. Regardless, all treatment (on-demand and prophylaxis) should be tailored towards both the patient's personal needs and their clinical profile. In addition, as the associations between risk factors (psychosocial, condition-related and treatment-related) and clinical features are unique to each patient, an individualized approach is required to enable patients to alter their behaviour in response to them. The practical methodologies needed to reach this goal of individualized haemophilia care, and the health economic implications of this strategy, are ongoing topics for discussion. (Less)
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published
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Blood Coagulation and Fibrinolysis
volume
26
issue
8
pages
849 - 857
publisher
Lippincott Williams and Wilkins
external identifiers
  • pmid:25396764
  • wos:000364279300001
  • scopus:84947126707
ISSN
1473-5733
DOI
10.1097/MBC.0000000000000225
language
English
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yes
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8caafeba-aecf-4173-a16e-bd0a9b399037 (old id 4816657)
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http://www.ncbi.nlm.nih.gov/pubmed/25396764?dopt=Abstract
date added to LUP
2014-12-02 17:26:28
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2017-10-01 03:08:00
@article{8caafeba-aecf-4173-a16e-bd0a9b399037,
  abstract     = {Owing to the heterogeneity in the clinical phenotype of haemophilia A and B, it is now recognized that disease severity (based on factor VIII/IX activity) may no longer be the most appropriate guide for treatment and that a 'one-size-fits-all' approach is unlikely to achieve optimal therapy. Based on the present literature and consensus views of a group of experts in the field, this article highlights key gaps in the understanding of the diverse relationships between bleeding phenotype and factors such as joint health, genetic susceptibility, laboratory parameters, quality of life and management of pain. Early prophylaxis is a potential 'gold standard' therapy and issues surrounding inhibitor development, variations in its clinical use and long-term outcomes are discussed. Comprehensive treatment should be individualized for all patients (including those with mild or moderate haemophilia and carriers). Wherever possible all patients should be given prophylaxis. However, adult patients with a milder haemophilia phenotype may be candidates for ceasing prophylaxis and switching to on-demand treatment. Regardless, all treatment (on-demand and prophylaxis) should be tailored towards both the patient's personal needs and their clinical profile. In addition, as the associations between risk factors (psychosocial, condition-related and treatment-related) and clinical features are unique to each patient, an individualized approach is required to enable patients to alter their behaviour in response to them. The practical methodologies needed to reach this goal of individualized haemophilia care, and the health economic implications of this strategy, are ongoing topics for discussion.},
  author       = {Sørensen, Benny and Auerswald, Günter and Benson, Gary and Elezović, Ivo and Felder, Markus and Lambert, Thierry and Morfini, Massimo and Remor, Eduardo and Salaj, Peter and Santagostino, Elena and Salek, Silva Z and Ljung, Rolf},
  issn         = {1473-5733},
  language     = {eng},
  number       = {8},
  pages        = {849--857},
  publisher    = {Lippincott Williams and Wilkins},
  series       = {Blood Coagulation and Fibrinolysis},
  title        = {Rationale for individualizing haemophilia care.},
  url          = {http://dx.doi.org/10.1097/MBC.0000000000000225},
  volume       = {26},
  year         = {2015},
}