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

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.

journal_name

Thromb Res

journal_title

Thrombosis research

authors

Lambert T,Auerswald G,Benson G,Hedner U,Jiménez-Yuste V,Ljung R,Morfini M,Remor E,Santagostino E,Zupančić Šalek S

doi

10.1016/j.thromres.2014.02.015

subject

Has Abstract

pub_date

2014-06-01 00:00:00

pages

967-71

issue

eissn

0049-3848

issn

1879-2472

pii

S0049-3848(14)00094-2

journal_volume

133

pub_type

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