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.
| Original language | English |
|---|---|
| Pages (from-to) | 967-971 |
| Number of pages | 5 |
| Journal | Thrombosis Research |
| Volume | 133 |
| Issue number | 6 |
| DOIs | |
| Publication status | Published - 2014 |
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Keywords
- Arthropathy
- Haemophilia
- Inhibitors
- Joint score
- On-demand treatment
- Prophylaxis
ASJC Scopus subject areas
- Hematology
- Medicine(all)
Cite this
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; Jiménez-Yuste, Victor; Ljung, Rolf; Morfini, Massimo; Remor, Eduardo; Santagostino, Elena; Zupančić Šalek, Silva.
In: Thrombosis Research, Vol. 133, No. 6, 2014, p. 967-971.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - Joint disease, the hallmark of haemophilia
T2 - What issues and challenges remain despite the development of effective therapies?
AU - Lambert, Thierry
AU - Auerswald, Günter
AU - Benson, Gary
AU - Hedner, Ulla
AU - Jiménez-Yuste, Victor
AU - Ljung, Rolf
AU - Morfini, Massimo
AU - Remor, Eduardo
AU - Santagostino, Elena
AU - Zupančić Šalek, Silva
PY - 2014
Y1 - 2014
N2 - 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.
AB - 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.
KW - Arthropathy
KW - Haemophilia
KW - Inhibitors
KW - Joint score
KW - On-demand treatment
KW - Prophylaxis
UR - http://www.scopus.com/inward/record.url?scp=84900486542&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=84900486542&partnerID=8YFLogxK
U2 - 10.1016/j.thromres.2014.02.015
DO - 10.1016/j.thromres.2014.02.015
M3 - Article
C2 - 24613700
AN - SCOPUS:84900486542
VL - 133
SP - 967
EP - 971
JO - Thrombosis Research
JF - Thrombosis Research
SN - 0049-3848
IS - 6
ER -