Costly clot drug stems joint woes in hemophiliacs

By Gene Emery

BOSTON (Reuters) - Regular infusions of a protein called factor VIII can prevent joint problems in boys who lack it because they suffer severely from the bleeding disease hemophilia, researchers said on Wednesday.

But the good news carries a stiff price tag. Regularly giving factor VIII, a protein necessary for blood clotting, would cost about $300,000 per year.

The research team, led by Dr. Marilyn Manco-Johnson of the Children's Hospital in Denver, had hoped the protein would only need to be given when a child had joint bleeding, which can lead to swelling, inflammation, pain and, eventually, destruction of the joint.

However, the benefits of regular infusions were far more dramatic, they reported in this week's issue of the New England Journal of Medicine.

Among the 32 boys who got factor VIII treatment every other day beginning before the age of 2-1/2, 93 percent had normal joints by their sixth birthday, compared to just 55 percent of the 33 youngsters who got factor VIII after occurrence of joint bleeding.

Overall, there was an 83 percent reduction in the risk of joint damage in the "prophylaxis" group.

Three boys in the group that got factor VIII on an as-needed basis developed life-threatening bleeding.

About 400,000 males worldwide have severe hemophilia. The inability of their blood to clot properly often produces bleeding into the knee, ankle and elbow joints.