Considerations During Factor Replacement Therapy

Considerations During Factor Replacement Therapy

Prophylactic use of factor replacement therapy starts when a child with hemophilia is very young, before bleeding and joint damage begin. However, factor replacement products do not last long in the body, meaning that children and adults receiving prophylactic therapy need to get infusions (injections) of replacement factors two to four times a week to prevent bleeding.

Administration of replacement factors also carries some risk. Young children and others may need a central venous access device (port) implanted in their body. The person administering therapy injects the replacement factor into this device so it may enter the vein without the need to puncture the skin. Although this device avoids the pain of repeated injections, it may be become infected or cause blood clots.

Up to one third of people with hemophilia A and up to 5% of those with hemophilia B receiving prophylactic factor replacement therapy develop inhibitors, which are proteins that the body produces to protect itself against foreign substances. At some time during factor replacement therapy, a person with hemophilia could develop inhibitors that make the therapy useless.

In addition, factor replacement therapy is expensive. One study showed that from 2001 to 2007, the average cost of treating hemophilia A over 1 year in the United States was almost $64,000 (over $72,000 in 2014 dollars). Among people who developed inhibitors to factor replacements, the yearly average cost increased to over $271,000 (nearly $306,000 now).

What Does This Mean to People With Hemophilia?

Factor replacement therapy truly is a life-saver. However, it also is associated with some physical risk to the person with hemophilia, inconvenience, and considerable financial costs. Research studies being done now hopefully will result in safer, more affordable replacement products and simpler, more convenient treatment schedules.

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