The Extravagances of ‘The Royal Disease’ Hemophilia

2010-04-19 | |
Last updated: 2010-04-19

One of the first medical conditions that typically come to mind when we think of blood disorders is the disease Hemophilia. Though most of us will not personally know anyone with this rare condition, we will have learned about the disease from school history lessons about 19th and 20th century Europe.

Hemophilia Blood Cells

Once called “the royal disease”, Queen Victoria of England passed the condition onto two daughters who went on to marry into and introduce the disorder to the royal families of Germany, Spain and Russia. In particular, the condition became well known because of the Russian royal family and the Siberian mystic Rasputin who was thought to have saved the life of the hemophiliac child of the last tsar of Russia.

Amongst the ranks of the common man, Hemophilia affects roughly 1 in 5000 males and rarely appears in females though they may be carriers. World wide, the disease affects some 400,000 people. Though one of the most well known of the blood and clotting disorders, it is not the most frequently occurring and Von Willebrand Disease (VWD) is actually a more common condition.

Rather than being a single condition, Hemophilia is actually a collection of genetic disorders of the blood that prevent production of the material needed for clotting or coagulation. This means that external wounds or internal broken blood vessels will take a significant amount of time to stop bleeding. Until recently, this meant that even relatively minor injuries could be debilitating or even deadly.

In fact, before the introduction of treatment in the 1960’s, those with the condition had a life expectancy of only 11 years. In contrast, for individuals now receiving treatment, the average lifespan is only 10 years less than for those without the condition.

Another lesser known, but common aspect of the disease is joint damage caused by bleeding into the joint. When not properly treated, this bleeding can actually cause harm or even destruction of the joint making this effect of the disease a significant contributor to disability associated with the disease.

The modern form of treatment for Hemophilia involves either regular preventative infusion of the missing clotting agent into the blood every few days or administering the clotting material only when needed. Though receiving the clotting material regularly is the best option for preventing joint damage (6 times better) and limiting the risks of uncontrolled bleeding, doing so has three important cons.

The first is that such preventative treatment is expensive, having been estimated to be US $300,000 annually per patient prior to 2007. Much of this figure is associated with the high costs of creating the clotting protein. This cost is by no means something that individuals can bear, but at the same time without it, the risk of disability and death for patients is greatly increased.

Secondly, regular infusions can be thwarted by a patient’s very own immune system. In 25% of patients, the immune system prevents the clotting agents from working, resulting in patients receiving no further significant benefit from the transfusions. Furthermore, in 4% of patients, the immune system more actively fights the foreign clotting protein and patients can develop a severe allergic reaction that can be life threatening.

Thirdly, because of the risks of introducing the blood coagulant, patients must receive the treatment in a hospital or clinical setting. In addition to the costs mentioned above, this introduces significant demands on individuals with the condition and the hospitals providing the service making the treatment effort in controlling the disease high for both the patient and health systems.

With these still significant problems associated with the current forms of treatment, Hemophelia is a disease that continues to be untamed. Because of its high costs, its often-fickle response to treatment and its demanding use of resources, it is very much a royal disease even today. Add to this that the admirable successes achieved so far mean more individuals with the disease are alive and we have a compounding problem of more people needing such treatment just to maintain a decent quality of life.

Clearly, the financial and personal costs of the disease must be reduced, so in the next article, some current efforts to reduce the impact of the disease will be explored.

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