Treatment and Management

• Haemophilia & Clotting Factor deficiency
• Clotting Factor replacement therapy
• Treatment of acute bleeding episodes
• Treatment of neurological effects
• Von Willebrand’s disease

Haemophilia & Clotting Factor deficiency

There is no cure for haemophilia or other bleeding disorders and the need for monitoring and treatment is life-long.

Anyone with a bleeding disorder of any severity has access to a Haemophilia centre and / or Haemophilia Comprehensive Care Centre. Anyone who requires regular treatment or who has joint damage is likely to receive all of their medical care via the local centre. There are various aspects to care required and these are co-ordinated through the centres. Examples of care include -:

• Management of bleeding tendency and clotting factor replacement
• Management of complications of bleeds
• Specialist physiotherapy assessment and treatment
• Joint surgery
• Psychological and social care
• Genetic counselling

The aims of management are to -:

• Control bleeding tendency
• Treat bleeding episodes
• Prevent joint damage and neurological complications
• Manage joint damage
• Enable normal growth and development during childhood and support normal life in adulthood

Clotting Factor replacement therapy

The cornerstone of treatment is the replacement of clotting factors to enable normal blood clotting and to prevent complications of bleeds. Much of this is self administered with help, support and guidance from the centre. The clotting factor used will vary depending on which factor is deficient -:

• Haemophilia A – clotting factor VIII
• Haemophilia B – clotting factor IX
• Other clotting factor deficiencies are named for the deficient factor e.g. factor II, factor X.

There are two different regimens used and these are -:

• Prophylactic (preventative) clotting factor treatment
• Clotting factor replacement as needed to treat bleeding

Choice of regimen will depend on frequency of bleeding without treatment and levels of clotting factors.

Treatment of acute bleeding episodes

Minor cuts and bruises are easily managed at home with pressure, first aid and administration of clotting factors if necessary. There are no long term consequences from these injuries.

Anything more than minor trauma may require hospital attendance to check for internal bleeding e.g. banging head on cupboard door. This is particularly important for those with moderate and severe clotting factor deficiency.

The most common problem in terms of bleeding for someone with severe clotting factor deficiency will be bleeding in to joints. Obviously as joint damage is the most common cause of mobility problems for them, minimising harm from bleeding into joints is a very important. Most people will be able to identify bleeding at a very early stage and administer clotting factor treatment. This prevents the large joint haematomas which were so characteristic of this condition, from forming. Because bleeding is stopped at an early stage ‘splintage’ and joint aspiration are not required and people can manage their bleeds at home. Home management consists of -:

• Administration of clotting factor
• Rest
• Ice
• Elevation
• Wrapping of affected limb.
• Gentle mobilisation can begin the following day.

Recovery from a bleed like this will take 2-3 days during which a person will be mobile around the home.

People with inhibitor are at risk from large haematomas and may not be able to adequately manage their bleeding at home. They may need to attend hospital as some bleeds will require admission for ‘splintage’ and strict bed rest. Recovery from an acute bleed in this situation is likely to take up to two weeks.

Treatment of neurological events

The principles of treatment are to stop bleeding by replacing clotting factors and provide and to reduce resulting permanent disability. Such treatment will take place in hospital and may include neurosurgery.

Treatment of inhibitor

Replacement of clotting factors to treat bleeding will be less effective and such treatment may be given in hospital. Bleeds into joints are likely to require splinting and occasionally aspiration. Recovery from individual episodes is likely to take up to 2 weeks. Intensive treatment to remove inhibitor may be given using a portocath or Hickmann line for 18 months to 2 years. If this is not effective, inhibitor will still be present afterwards.

Small joints of the hands are rarely affected by arthritis and the majority of adults will be able to administer their own treatment for haemophilia at home in-between bleeds. If a ‘fixed flexion’ deformity of the elbow or limited mobility of the wrists is present or if the veins are difficult to access then help with treatment may be required.

Von Willebrand’s disease

The majority of cases are mild and any bleeding can be controlled with DDAVP or tranexamic acid as in mild haemophilia.

When severe, this condition may require clotting factor replacement. This is rare. No recombinant factor is suitable and human derived plasma products contain clotting factor are used. People with severe Von Willebrand’s disease tend to be on self-treatment at home including preventative treatment as for those with severe haemophilia.

Click on the link for details of -:

• Clotting factor replacement therapy
• Tests for bleeding disorders
• Complications of Clotting Factor replacement therapy

Amended November 2009