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Hemophilia Treatments

Hemophilia is a rare genetic disorder that prevents a person’s blood from clotting like it should. People suffering from hemophilia often bleed longer and more severely than others after an injury, but they also are more likely to suffer from internal bleeding—which is a much more life-threatening condition than external wounds.

While there is currently no cure for this condition, treating its symptoms and maintaining a healthy lifestyle is still very possible. Here is a look at the most common ways in which hemophilia is treated.

Clotting Factor Replacements

A person with hemophilia has difficulty with clotting because their blood lacks clotting factors—crucial proteins that help initiate the process. There are more than 20 varieties of these proteins, but hemophilia patients are specifically missing either factors VII or VIII. Therefore, the primary purpose of hemophilia treatment is to provide replacement factors for the ones they lack.

There are currently two forms of replacement factors that are used in hemophilia treatments—plasma-derived and recombinant concentrates. As the name implies, plasma-derived factors are harvested from human plasma samples. First, these samples are closely screened for impurities. Then, the specific clotting factor proteins needed are extracted from the sample. Finally, these proteins are then sterilized to completely eliminate the risk of harmful viruses.

On the other hand, recombinant concentrates are completely lab-manufactured. These products are created by injecting animal cells with a human gene responsible for the production of clotting factors. Because of the many similarities between the DNA of humans and other animals, as these cells replicate they actually produce the necessary clotting factors. As with plasma-derived factors, recombinant concentrates are carefully screened and sterilized to prevent the risk of a blood-borne virus.

Administering Clotting Factor Replacements

There is currently only one way to administer clotting factor replacements—via intravenous injections. However, how and when these replacements are injected will vary from person to person.

For those with moderate to severe hemophilia, prophylactic injections are normally recommended. This means that patients receive regular weekly injections of clotting factors in order to prevent uncontrolled bleeding before it starts. The prophylactic method is considered beneficial because it’s been proven to reduce joint damage that’s caused by blood collecting in the joint cavities, and it’s the preferred method for treating children, since they’re more prone to unexpected injuries than adults. However, this approach can get expensive, since the average cost of an injection is anywhere from $1000 to $2000.

For some people with only a mild form of hemophilia, “on demand” injections will normally suffice. This means that they only receive an injection when they are actively bleeding. Although this is a much more affordable way to treat hemophilia than with the prophylactic method, it does put patients at a higher risk of joint problems, since the bleeding is only addressed after it’s begun.

Injection Methods

One of the most common methods for injection is with a simple butterfly needle. This approach is quick, safe, and easy for patients accustomed to the procedure, but it may be difficult to use on children, whose veins are typically smaller and more difficult to find.

Another more kid-friendly approach is using a port and catheter. This involves implanting a small port (about the size of a quarter) underneath the skin on the chest, which is connected to a central vein using a catheter tube. The convenience of using a port is so desirable by some that they continue to use it well into adulthood, when the butterfly needle method becomes an option. While it can be used for many years, the risk of infection at the injection site is higher than with the other approach.

Hemophilia Treatment Centers

After being properly trained by a medical professional, most hemophilia patients can perform clotting factor injections at home by themselves. However, in certain severe cases, inpatient care may be necessary, and in fact, it may be more beneficial than traditional care. A study conducted by the CDC found that people who used hemophilia treatment centers were 40% less likely to be hospitalized than those who opted not to use them.

Treatment centers offer patients a holistic approach to their condition. In addition to hemophilia experts, these centers are also staffed by orthopedists specializing in joint damage, physical therapists, and mental health professionals, who help address the emotional and psychological side of dealing with hemophilia.

Last Updated: February 25, 2016