Prophylactic treatment for prevention of joint disease in hemophilia--cost versus benefit.

Patients with severe hemophilia number only around 400,000 worldwide, but their lifespan has increased because of improved treatment, and with that the prevalence of the disease is increasing. Despite the relatively small number of patients with severe hemophilia, the disease has an important socioeconomic effect because of its distinctive feature: a level of clotting factor (factor VIII or factor IX) so low that without replacement therapy, frequent hemorrhages occur, most often in the ankle, knee, and elbow joints. These joint hemorrhages culminate in a severe arthropathy, with signs of inflammation (as in rheumatoid arthritis) as well as degenerative damage (as . . .

[1]  Alan R. Cohen,et al.  Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia. , 2007, The New England journal of medicine.

[2]  J. Bijlsma,et al.  Exposure of human cartilage tissue to low concentrations of blood for a short period of time leads to prolonged cartilage damage: an in vitro study. , 2007, Arthritis and rheumatism.

[3]  F. Lafeber,et al.  Pathogenesis of haemophilic arthropathy , 2006, Haemophilia : the official journal of the World Federation of Hemophilia.

[4]  D. Grobbee,et al.  Factor VIII half-life and clinical phenotype of severe hemophilia A. , 2005, Haematologica.

[5]  J. Bijlsma,et al.  Blood-induced joint damage: longterm effects in vitro and in vivo. , 2003, The Journal of rheumatology.

[6]  L. Valentino,et al.  c-myc proto-oncogene expression in hemophilic synovitis: in vitro studies of the effects of iron and ceramide. , 2002, Blood.

[7]  D. Grobbee,et al.  The effects of postponing prophylactic treatment on long-term outcome in patients with severe hemophilia. , 2002, Blood.

[8]  J. Astermark,et al.  Primary prophylaxis in severe haemophilia should be started at an early age but can be individualized , 1999, British journal of haematology.

[9]  J. Bijlsma,et al.  Haemophilic arthropathy resembles degenerative rather than inflammatory joint disease , 1999, Histopathology.

[10]  C. Kessler,et al.  When should prophylactic treatment in patients with haemophilia A and B start?— The German experience , 1998, Haemophilia : the official journal of the World Federation of Hemophilia.

[11]  K. Khair,et al.  The impact of prophylactic treatment on children with severe haemophilia , 1996, British journal of haematology.

[12]  H. Pettersson,et al.  A longitudinal study of orthopaedic outcomes for severe factor‐VIII‐deficient haemophiliacs , 1994, Journal of internal medicine.

[13]  H. Pettersson,et al.  Twenty‐five years' experience of prophylactic treatment in severe haemophilia A and B , 1992, Journal of internal medicine.

[14]  M. Blombäck,et al.  Prophylaxis with factor concentrates in preventing hemophilic arthropathy. , 1991, The American journal of pediatric hematology/oncology.