Extending the life of clotting factors may improve quality of life for people with haemophilia
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For the parents of a child born with haemophilia, the diagnosis comes with both good and bad news. The good news is that the child, at least if he (or rarely, she) is born in the developed world, can expect a near-normal lifespan, up from a mere 20 years in 1970. The bad is that the parents must teach themselves to find their child's veins, insert a needle and infuse him with a clotting factor to replace what he lacks. Parents must infuse a toddler as often as every other day, and children with haemophilia will have to continue that treatment for the rest of their lives.
But treatment is getting easier. Down the road, gene therapy and other approaches look likely to bring longer-term treatments for patients with the rare bleeding disorder. For now, improvement in treatment lies in the emergence of new, longer-lasting replacements for the blood-clotting factors missing from the blood of people with the condition. These therapies could stretch the time between infusions to days or even weeks. The first two such treatments were approved by the US Food and Drug Administration (FDA) earlier this year, and more are in the pipeline, with some expected to be approved in 2015. As these therapies emerge, dealing with haemophilia will become less troublesome (see below). This could increase compliance with treatment, reduce complications — and perhaps even allow some people to live almost as if they were free of the disease.
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