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Outcome measures for adult and pediatric hemophilia patients with inhibitors

Hermans, Cedric; Auerswald, Günter; Benson, Gary; Dolan, Gerry; Duffy, Anne; Jiménez-Yuste, Victor; Ljung, Rolf LU ; Morfini, Massimo; Lambert, Thierry and Osooli, Mehdi LU , et al. (2017) In European Journal of Haematology 99(2). p.103-111
Abstract

Recent advancements in almost all aspects of hemophilia treatment have vastly improved patient care and management, and new and emerging treatments hold the promise of further progress. However, there remains a scarcity of data on long-term outcomes in hemophilia, particularly among those patients with inhibitors, for whom no validated outcome assessment tools are currently available. At the 15th Zürich Haemophilia Forum, an expert panel reviewed the most important outcome measures in inhibitor patients and considered the challenges associated with assessing outcomes in this population. A framework for outcome assessment in inhibitor patients incorporates traditional hemophilia outcome measures, such as bleed frequency and... (More)

Recent advancements in almost all aspects of hemophilia treatment have vastly improved patient care and management, and new and emerging treatments hold the promise of further progress. However, there remains a scarcity of data on long-term outcomes in hemophilia, particularly among those patients with inhibitors, for whom no validated outcome assessment tools are currently available. At the 15th Zürich Haemophilia Forum, an expert panel reviewed the most important outcome measures in inhibitor patients and considered the challenges associated with assessing outcomes in this population. A framework for outcome assessment in inhibitor patients incorporates traditional hemophilia outcome measures, such as bleed frequency and mortality, alongside measures of health, functioning, disability, social participation, quality of life, and economic considerations. It is important to remember that inhibitor patients differ in their clinical needs, perspectives, and priorities according to age, inhibitor status, degree of joint disease, and activity levels; as a result, the relative importance of different outcome measures will change throughout an inhibitor patient's life. Challenges inherent in measuring long-term outcomes in inhibitor patients include the small number of known patients, the subjective nature of many outcome assessment tools, and the risk of overburdening patients with repeated requests to complete questionnaires or participate in studies. Therefore, there is an urgent need to reach consensus on the most important and appropriate assessment tools for measuring outcomes in this population. These tools should ideally be standardized, easily applied, and internationally applicable in order to collect and generate quality outcome data.

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publication status
published
subject
keywords
adults, children, hemophilia, inhibitors, outcome assessment, treatment outcome
in
European Journal of Haematology
volume
99
issue
2
pages
9 pages
publisher
Wiley-Blackwell
external identifiers
  • scopus:85021727654
  • wos:000404936400001
ISSN
0902-4441
DOI
10.1111/ejh.12881
language
English
LU publication?
yes
id
b464346b-157d-4cee-a120-7465ee0303ed
date added to LUP
2017-07-26 12:43:48
date last changed
2017-09-18 11:39:47
@article{b464346b-157d-4cee-a120-7465ee0303ed,
  abstract     = {<p>Recent advancements in almost all aspects of hemophilia treatment have vastly improved patient care and management, and new and emerging treatments hold the promise of further progress. However, there remains a scarcity of data on long-term outcomes in hemophilia, particularly among those patients with inhibitors, for whom no validated outcome assessment tools are currently available. At the 15<sup>th</sup> Zürich Haemophilia Forum, an expert panel reviewed the most important outcome measures in inhibitor patients and considered the challenges associated with assessing outcomes in this population. A framework for outcome assessment in inhibitor patients incorporates traditional hemophilia outcome measures, such as bleed frequency and mortality, alongside measures of health, functioning, disability, social participation, quality of life, and economic considerations. It is important to remember that inhibitor patients differ in their clinical needs, perspectives, and priorities according to age, inhibitor status, degree of joint disease, and activity levels; as a result, the relative importance of different outcome measures will change throughout an inhibitor patient's life. Challenges inherent in measuring long-term outcomes in inhibitor patients include the small number of known patients, the subjective nature of many outcome assessment tools, and the risk of overburdening patients with repeated requests to complete questionnaires or participate in studies. Therefore, there is an urgent need to reach consensus on the most important and appropriate assessment tools for measuring outcomes in this population. These tools should ideally be standardized, easily applied, and internationally applicable in order to collect and generate quality outcome data.</p>},
  author       = {Hermans, Cedric and Auerswald, Günter and Benson, Gary and Dolan, Gerry and Duffy, Anne and Jiménez-Yuste, Victor and Ljung, Rolf and Morfini, Massimo and Lambert, Thierry and Osooli, Mehdi and Zupančić Šalek, Silva},
  issn         = {0902-4441},
  keyword      = {adults,children,hemophilia,inhibitors,outcome assessment,treatment outcome},
  language     = {eng},
  month        = {08},
  number       = {2},
  pages        = {103--111},
  publisher    = {Wiley-Blackwell},
  series       = {European Journal of Haematology},
  title        = {Outcome measures for adult and pediatric hemophilia patients with inhibitors},
  url          = {http://dx.doi.org/10.1111/ejh.12881},
  volume       = {99},
  year         = {2017},
}