Haemophilia A in children: how treatment works, home care and keeping safe

Haemophilia is caused by a lack of a protein called clotting factor, which helps blood clot. The NHS describes two main types. Haemophilia A is caused by a lack of clotting factor 8 (factor VIII), and haemophilia B by a lack of factor 9 (factor IX). The two types have the same symptoms, but the medicines used to treat each type are different, so the type has to be confirmed.

The Haemophilia Society describes haemophilia as a lifelong, inherited bleeding disorder in which one of the clotting factor proteins is either partly or completely missing. People with haemophilia do not bleed faster, but they take longer than normal for bleeding to stop, and they may bleed into joints and muscles without having had an injury. That is why treatment aims to reduce spontaneous bleeding as well as to treat bleeds after injuries.

The NHS says haemophilia is usually caused by an altered gene passed on from parents to a child. Boys and men with the altered gene have symptoms. Women with the altered gene are carriers and can have symptoms, though this is less common and symptoms are less severe. The Haemophilia Society notes that females can also be affected. Symptoms usually begin in early childhood, but sometimes may not be noticed until later.

Diagnosis is by a blood test that measures the amount of clotting factor in the blood. The NHS says that if you or your child are diagnosed, you will be referred to your local haemophilia centre for treatment. Families with a history of haemophilia can be referred to a genetic counsellor to discuss risks and the tests that are available before, during and after pregnancy.

The NHS lists bleeding for a long time after an injury, surgery or having a tooth removed, bruising easily, and joint pain, stiffness and swelling as symptoms. The Haemophilia Society adds that people with haemophilia may bleed into joints and muscles without having had an injury. A child with a painful, swollen joint, or who bleeds for much longer than expected after a minor injury, should be assessed.

Other conditions such as von Willebrand disease can cause similar symptoms, so tests are needed to tell them apart.

The NHS says haemophilia cannot usually be cured but treatment helps control symptoms. The main approach is clotting factor concentrate, which is given by injection to replace the missing factor VIII. It can be given in two main ways.

The NHS says the clotting factor used today is very safe. Much of it is synthetic and has no risk of infection. When it is made from donated blood, it is tested and treated to make sure it is as safe as possible, and the risk of getting an infection from it is very low. For anyone who received a blood transfusion before 1996, the NHS gives separate advice about hepatitis C testing, which is mainly relevant to adults.

Some people develop inhibitors, which are antibodies to the clotting factor that make treatment less effective. If this happens the haemophilia team works with the family to find a more suitable treatment.

The NHS says the haemophilia team will usually show you how to treat bleeding yourself at home with clotting factor injections or other medicines. Treat any bleeding, including bleeding into joints or muscles, as soon as possible, because this reduces pain, recovery time and the risk of permanent damage. When recovering from a joint or muscle bleed, following the PRICE steps for sprains and strains can help. For most bleeds you will also need to get medical help.

Joint bleeds are the typical concern in haemophilia. Repeated bleeds can damage a joint over time, and the NHS lists damage to joints, sometimes needing joint replacement, compartment syndrome and serious bleeding after surgery or injury as possible complications. It adds that treatment reduces the risk of complications.

Children with haemophilia can live active lives. The NHS advises regular exercise to keep joints healthy, with a physiotherapist helping to choose suitable activities, and taking care of teeth and gums with regular dental check-ups. Families should check with the haemophilia team before giving any new medicine and before any surgery or dental treatment, and a medical alert bracelet is recommended.

The NHS says not to take aspirin, ibuprofen or other non-steroidal anti-inflammatory drugs, and not to take part in contact sports such as rugby or boxing unless the haemophilia team has said it is safe. Your team can advise on school, sport, activities and holidays.

• Use the usual medicines straight away if there is any bleeding, and get medical help.

• Wear a medical alert bracelet, as the NHS recommends.

• Check with the haemophilia team before any new medicine, surgery or dental treatment.

• Keep up regular exercise and dental check-ups.

The NHS says to contact your haemophilia team straight away if your child has pain, swelling, tingling or numbness in a joint or muscle, which could be a sign of bleeding; has blood in the urine; is worried about any bleeding; or is bleeding and does not have medicine to treat it. If the haemophilia team is not available, call 111. Where possible, treat bleeding straight away with the usual medicines first.

Call 999 or go to A&E if your child has a head injury, a serious injury such as a deep cut, blood in the poo or dark red or black poo, vomiting blood or coughing up blood, or a sudden bad headache with confusion, sensitivity to light, or nausea and vomiting, which could be a sign of bleeding in the brain. After calling 999, contact the haemophilia team so they can support treatment in hospital.

The NHS says that when a child is diagnosed, the family is referred to their local haemophilia centre for treatment, and the haemophilia team supports them throughout. The care team will pass information on to the National Haemophilia Database, which the NHS says allows care to be planned and helps scientists look for better treatments; you can opt out of research use at any time.

The Haemophilia Society, a charity, provides support and information for people with haemophilia and other bleeding disorders and their families, and the NHS says it may help to get support from other people with haemophilia, or from parents of children with haemophilia. It describes how a new diagnosis can leave families shocked and overwhelmed, and says the outlook is the best it has ever been for people with haemophilia in the UK.