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Mastering Hemophilia – Winning Freedom
Having hemophilia used to be a terrible burden. It’s still a burden, but one that can be overcome. Nowadays people with hemophilia can lead a practically normal life thanks to major medical progress. And thanks to modern drugs which, although not able to cure this hereditary disease, can make it bearable.
There is only one component missing from the blood of those with hemophilia – but it is a vital one. These people have too little of a certain coagulation factor in their blood – or none at all. This means that they bleed for considerably longer than healthy individuals.
Doctors distinguish between two forms: hemophilia A and B. Type A is far more common and accounts for 80 percent of hemophilia patients. They are unable to produce enough coagulation factor VIII in their blood. Hemophilia B is a deficiency of Factor IX. Both factors are vital components of the blood coagulation process.
If a healthy person is injured, blood platelets (thrombocytes) accumulate in the wound and start to close it. A network of cells forms in the wound, visible from the outside as a scab. The wound closure process is regulated by 13 different coagulation factors, with one factor triggering the next. This process does not work in those with hemophilia. The coagulation cascade is interrupted because one factor – a protein – is missing.
Nowadays genetic engineering techniques allow this vital Factor VIII to be produced industrially. It is a giant protein molecule made up of 2,232 amino acid building blocks. Production is a tremendous technological challenge, involving some 600 isolation, purification and testing steps to create an active substance in pure form. But the effort is worthwhile – because the safety of patients has top priority at Bayer HealthCare.
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