Optimizing joint function: new knowledge and novel tools and treatments

Abstract

Progressive joint destruction resulting from intra-articular bleeding is the major morbidity affecting patients with haemophilia (PWH), particularly those with inhibitors. Advances in understanding the detrimental processes set in motion by the exposure of joints to bleeding have shaped current management methods. However, to achieve optimal joint health in PWH, in addition to achieving haemostasis at the bleeding vessel, it may be appropriate to explore experimentally other conceptual frameworks. These include the possibilities that markers might help to identify individuals at the risk of more rapid joint deterioration, that clotting factors may have additional local action within tissues, and that outcomes might be improved with therapies that directly address wound healing and inflammation. Joint assessment tools are important. Conventional radiography is frequently used, but given the possibility of subclinical joint bleeds, accurate non-invasive imaging tools are required to detect soft tissue and cartilage changes. Magnetic resonance imaging and ultrasonography can prove valuable here. New imaging techniques should help to increase understanding of the biological basis of early events in haemophilic arthropathy. The optimal way to measure outcomes in haemophilia is to use several methods - in addition to imaging methods, a 360° approach will use physical, functional and quality-of-life instruments. In PWH, inhibitor development complicates treatment of joint bleeds and increases the risk of developing arthropathy. A new therapeutic approach for joint bleeds in inhibitor patients divides treatment into two phases: bleed control, with bypassing agent therapy until bleeding has definitely ceased, followed by regular dosing to prevent rebleeds until synovial recovery is complete.