Hemophilia

The royal disease

In the 19th and 20th centuries, several members of the British royal family suffered from hemophilia – and certain marriages also enabled it to spread to other royal families in Europe. Hence the name "royal disease." For anyone who had the disease at the time, however, the prospects were anything but royal: average life expectancy was less than 20 years.

Women are often the carrier

Hemophiliacs lack an important element in the complex process of blood clotting. People with the most common form of hemophilia produce no – or not enough – coagulation factor VIII. These patients are said to suffer from hemophilia A. A deficiency of factor IX, known as hemophilia B, is rarer. As a rule, these diseases are genetically determined. Although they are transmitted by women, it is men who are most often affected. The number of people suffering from hemophilia worldwide is estimated at about 400,000.

An almost normal life is possible

The consequences of this deficiency can be fatal. Not only external, but also internal bleeding is a major problem. Even at a young age, internal bleeding can cause permanent damage, for example to the joints. At a time when no therapies were available, many sufferers died in childhood. With the preparations that exist today, however, patients can lead an almost normal life.

Replacing what the blood lacks

Although hemophilia remains incurable to this day, what is known as a substitution therapy has existed for several decades. The missing coagulation factor Is supplied from outside by intravenous injection. The manufacturers extract this factor either from the plasma of donated blood, or from genetically modified cell cultures. The latter process became possible after the factor VIII gene was discovered in 1984. Bayer Schering Pharma was one of the first suppliers of such a recombinant factor VIII product. These biotechnologically manufactured products are considered safe because they exclude the risk of infection, which remains a theoretical residual risk in the case of products based on donated plasma – despite the virus-inactivation measures that have been introduced.

• www.bayer-hemophilia-awards.com

Research continues

Diverse clinical studies have proven that the precautionary administration of factor VIII protects the patients from bleeding into joints and therefore from later damage to the joints. However, because the protein is broken down in the body over time, it has to be repeatedly administered intravenously – several times a week. In order to make the therapy more convenient for the patient, therefore, one line of research at Bayer Schering Pharma is looking for a way to prolong the effect of the coagulation factor in the blood in such a way that it need not be injected so frequently.

Advice for patients
Every body reacts differently to medicines. Therefore it is impossible to tell which medicine works best for you. Please consult your physician.