GTC Biotherapeutics (fka GTCB)

[DewDiligence]

Prophylactic Treatment to Prevent Joint Damage in
Patients With Severe Haemophilia: Costs vs Benefits

http://tinyurl.com/2f37lr

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Ned Tijdschr Geneeskd. 2007 Nov 24;151(47):2606-8.

[Article in Dutch]

Roosendaal G, Lafeber FP.

Universitair Medisch Centrum Utrecht.

A recent publication stated that the cost of prophylactic clotting factor replacement therapy to prevent joint damage in patients with haemophilia is $300,000 per patient per year. Therefore, despite the relatively small number of patients with severe haemophilia, the disease has an important socioeconomic impact.

Joint damage is the most frequent and most debilitating comorbidity of haemophilia and can be prevented by adequate prophylactic treatment. But when and how should prophylaxis be started?

The aforementioned study provides thorough evidence that prophylaxis is clinically beneficial despite its high cost. A reasonable approach is to start individualised prophylactic treatment after the first episode of joint bleeding early in life, because prophylaxis will not prevent progression of established joint disease evident after multiple bleeding episodes.
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