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Joint disease, the hallmark of haemophilia: What issues and challenges remain despite the development of effective therapies?

Lambert, Thierry; Auerswald, Günter; Benson, Gary; Hedner, Ulla LU ; Jiménez-Yuste, Victor; Ljung, Rolf LU ; Morfini, Massimo; Remor, Eduardo; Santagostino, Elena and Zupančić Šalek, Silva (2014) In Thrombosis Research 133(6). p.967-971
Abstract
Although effective therapies for haemophilia have been available for decades, the prevention and treatment of joint disease remain major clinical concerns for all haemophilia patients. Early identification of joint disease is vital to initiate or modify treatment, and prevent arthropathy. However, there remains a need for more sensitive and accurate methods, which may also detect improvement in patient outcome with new therapies or different prophylaxis regimens. These topics were explored at the Ninth Zürich Haemophilia Forum. A summary of our shared views on the limitations of current assessment methods, and the potential advantages of more recently developed tools, is provided. Ultrasonography enables more frequent routine monitoring... (More)
Although effective therapies for haemophilia have been available for decades, the prevention and treatment of joint disease remain major clinical concerns for all haemophilia patients. Early identification of joint disease is vital to initiate or modify treatment, and prevent arthropathy. However, there remains a need for more sensitive and accurate methods, which may also detect improvement in patient outcome with new therapies or different prophylaxis regimens. These topics were explored at the Ninth Zürich Haemophilia Forum. A summary of our shared views on the limitations of current assessment methods, and the potential advantages of more recently developed tools, is provided. Ultrasonography enables more frequent routine monitoring and the early detection of joint disease. In addition, serological markers may provide suitable biomarkers of early arthropathy. To prevent arthropathy, in our opinion, prophylaxis is key to prevent joint bleeds and subsequent initiation of the 'vicious circle of joint disease'. However, issues remain, including when prophylaxis should be started, stopped, and if it is efficacious for inhibitor patients. Once joint bleeding has occurred, enhanced on-demand treatment should be considered. For more advanced stages of joint disease, the issues regarding the treatment options available are explored. Radiosynovectomy should be performed to treat chronic synovitis, and may prevent the need for elective orthopaedic surgery (EOS). Ultimately, however, EOS can be considered once all other treatment options have been explored. While, bypassing agents have facilitated the use of EOS in inhibitor patients, a multidisciplinary approach and careful surveillance is required for good patient outcome. (Less)
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author
organization
publishing date
type
Contribution to journal
publication status
published
subject
in
Thrombosis Research
volume
133
issue
6
pages
967 - 971
external identifiers
  • pmid:24613700
  • wos:000335910900006
  • scopus:84900486542
ISSN
1879-2472
DOI
10.1016/j.thromres.2014.02.015
language
English
LU publication?
yes
id
b947d27d-45d6-48c0-8af3-3d371e579854 (old id 4383510)
alternative location
http://www.ncbi.nlm.nih.gov/pubmed/24613700?dopt=Abstract
date added to LUP
2014-04-02 21:40:00
date last changed
2017-02-12 03:12:39
@article{b947d27d-45d6-48c0-8af3-3d371e579854,
  abstract     = {Although effective therapies for haemophilia have been available for decades, the prevention and treatment of joint disease remain major clinical concerns for all haemophilia patients. Early identification of joint disease is vital to initiate or modify treatment, and prevent arthropathy. However, there remains a need for more sensitive and accurate methods, which may also detect improvement in patient outcome with new therapies or different prophylaxis regimens. These topics were explored at the Ninth Zürich Haemophilia Forum. A summary of our shared views on the limitations of current assessment methods, and the potential advantages of more recently developed tools, is provided. Ultrasonography enables more frequent routine monitoring and the early detection of joint disease. In addition, serological markers may provide suitable biomarkers of early arthropathy. To prevent arthropathy, in our opinion, prophylaxis is key to prevent joint bleeds and subsequent initiation of the 'vicious circle of joint disease'. However, issues remain, including when prophylaxis should be started, stopped, and if it is efficacious for inhibitor patients. Once joint bleeding has occurred, enhanced on-demand treatment should be considered. For more advanced stages of joint disease, the issues regarding the treatment options available are explored. Radiosynovectomy should be performed to treat chronic synovitis, and may prevent the need for elective orthopaedic surgery (EOS). Ultimately, however, EOS can be considered once all other treatment options have been explored. While, bypassing agents have facilitated the use of EOS in inhibitor patients, a multidisciplinary approach and careful surveillance is required for good patient outcome.},
  author       = {Lambert, Thierry and Auerswald, Günter and Benson, Gary and Hedner, Ulla and Jiménez-Yuste, Victor and Ljung, Rolf and Morfini, Massimo and Remor, Eduardo and Santagostino, Elena and Zupančić Šalek, Silva},
  issn         = {1879-2472},
  language     = {eng},
  number       = {6},
  pages        = {967--971},
  series       = {Thrombosis Research},
  title        = {Joint disease, the hallmark of haemophilia: What issues and challenges remain despite the development of effective therapies?},
  url          = {http://dx.doi.org/10.1016/j.thromres.2014.02.015},
  volume       = {133},
  year         = {2014},
}