Dunn Amy L
AFLAC Cancer Center and Blood Disorders Service, Department of Pediatrics, Emory University and Children's Healthcare of Atlanta, GA 30322, USA.
Curr Opin Hematol. 2005 Sep;12(5):390-4. doi: 10.1097/01.moh.0000169285.66841.c8.
Degenerative arthropathy resulting from recurrent hemarthrosis remains the largest source of noninfectious morbidity for persons with hemophilia. Optimal treatment to prevent or reduce joint destruction remains controversial.
The most exciting developments in the past year have contributed to our understanding of the pathophysiology behind this destructive process. Particularly interesting is the possible role of protooncogenes and angiogenic factors in the development of hypertrophic synovitis. Arthroscopic synovectomy effectively controls the hypertrophic synovium and resultant bleeding, and can be used safely even in very young patients. Prophylactic factor replacement remains the mainstay to reduce or prevent joint damage. New approaches, chiefly dose escalation strategies, have shown promise instead of standard prophylactic regimens. Additionally aggressive on-demand approaches may be feasible. These techniques allow for fewer indwelling venous access devices in small children, as rates of complications with these devices are shown to be increasingly high. Whether prophylactic therapy can be safely stopped in certain patients is an evolving area of interest. Outcome measures are being validated to assess the impact of interventions on patient quality of life.
As hemophilia care evolves, current on-demand and prophylactic regimens will be optimized. Synovectomy will likely play a larger role in the control of synovitis, and validated quality-of-life scores will allow more consistent communication of outcomes. New understanding of the molecular mechanisms of joint destruction will pave the way for new therapeutic options for persons with hemophilia.
