Clotting Factor replacement therapy
- Prophylactic (preventative) clotting factor treatment
- Clotting factor replacement ‘on demand’ treatment
- Drugs used in haemophilia and clotting factor deficiency
- Obtaining and storing clotting factors
Prophylactic (preventative) clotting factor treatment
This treatment was introduced throughout the UK in the late 1990’s and it has revolutionised care for those with severe haemophilia. The aim of treatment is to prevent or minimise spontaneous bleeding episodes thereby preventing joint damage, which is the most common cause of disability in the condition. It is most likely to be used in people with very low levels of clotting factors (<1% category).
Treatment involves regular intravenous injection of clotting factors 3 or 4 times a week with dose adjustments and extra doses depending on activities (heavy exercise can use up clotting factors) and monitoring of bleeding tendency and clotting factor levels. The majority of people with haemophilia can and will do this for themselves. A small group of adult patients are unable to self-treat because of severe joint damage in the upper limbs.
Children with severe haemophilia who have had prophylactic treatment since early childhood can achieve adulthood with minimal or no joint damage. The advantage of maintaining normal joints through prophylactic treatment is that normal healthy joints are less likely to bleed spontaneously and although careful management of the bleeding tendency is required, a normal life with no or minimal disability can be achieved. Despite prophylactic treatment, the risk of bleeding persists as low levels of factors will be present just before the next injection. Some joint damage may occur because of this. In those people with poor veins, a ‘Portacath’ (or rarely indwelling Hickman line) for self-administering clotting factors may be used. People born before this treatment was introduced are likely to have joint problems or other difficulties related to bleeds that happened before they started using prophylactic clotting factor replacement.
Clotting factor replacement ‘on demand’ treatment
People with less severe haemophilia or other clotting factor deficiency are likely to use clotting factor replacement only when needed. They will need to administer the clotting factors as described above. Treatment is likely to be intermittent i.e. much less often than in prophylactic treatment. Again the aim is to administer clotting factors as soon as a bleed begins to minimise long term damage to joints. The majority of people with haemophilia who need to do this regularly can and will do this for themselves.
People with mild haemophilia may need clotting factor replacement or other treatment after trauma or to cover them for surgery or tooth extraction. There is no need for people like this to learn how to administer their own clotting factors or to keep supplies at home although some of them may prefer to do this.
Drugs used in haemophilia and clotting factor deficiency
People with low levels of clotting factors can use drug treatments to control bleeding. There are two drugs, which may be used at home to control bleeding and these are -:
- Tranexamic acid – an oral tablet, oral syrup or mouthwash
- Desmopressin (DDAVP) – this drug can be given as an injection or as a nasal spray.
Obtaining and storing clotting factors
Clotting factors are home delivered throughout the UK. They are delivered in powder form and need to be mixed with a ‘carrier’ for injection just prior to use. Supplies should be stored in the fridge but can be kept outside the fridge for up to 3 months making them suitable for carrying around to use at the onset of bleeding. Immediate treatment with clotting factors prevents large and damaging bleeds into joints so minimising damage.
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Amended November 2009
