How is it treated and managed?

Children require monitoring and treatment throughout their lives to control the effects of crises and reduce complications of the disease. Parents will need to know about and recognise the signs of sickle cell crisis and be prepared to seek immediate medical treatment. They may need to manage minor crises at home.

All children will require the following long-term treatments

A number of measures are undertaken to maintain health and to try to avoid crises. All children will require -:

• Balanced diet, adequate fluid intake and avoidance of cold,
• Regular folic acid supplements to replace loss by a process called haemolysis,
• Continuous oral penicillin or amoxycillin (twice daily) to reduce risk of infection from age 3 months onwards for life,
• Prompt treatment of infections,
• Full courses of vaccinations and flu vaccination.

Children on a programme of blood transfusions will need additional treatment at home. The parent/carer may need to administer iron chelation therapy (a treatment using a substance that is able to bind with excess iron in the body and enable it to be excreted) at home for those children receiving regular blood transfusions.

Some children may require the following long term treatments

• Hydroxycarbamide (hydroxyurea), a drug that affects red cell production may be used to reduce the risk of crises in some children. This is often used in children especially if they have recurrent and severe crises especially acute chest syndrome
• Analgesic drugs e.g. paracetamol, co- codamol, dihydrocodeine, tramadol and anti-inflammatory drugs are prescribed for bone pain and arthritis. Severely arthritic joints may be suitable for artificial joint replacement.
• Treatment of kidney failure including renal transplantation may be necessary. See adult Kidney Disorders guidance.
• Bone marrow transplantation has the potential to cure the condition. However it has a relatively high mortality of 5 – 10% and is only suitable for some people.

Treatment of sickle cell crisis

All but the mildest crises will be treated in hospital. During a crisis a child will need adequate pain relief, to be well hydrated, to keep warm and to rest for a few days. Some will require strong opiates such as morphine or diamorphine for pain relief and these drugs may need to be given by injection. Moderately potent analgesics such as tramadol, dihydrocodeine or co-codamol are used. Intravenous fluids and drugs to control vomiting may be necessary.

Painful crises usually last a few days, and as the acute symptoms settle down children will be encouraged to mobilize and get back to normal activities. Children are likely to receive their acute treatment as a hospital in-patient with further recovery taking place at home. Complete resolution of symptoms is likely to take seven to ten days. There are a small group of children who are very severely affected and these may take longer to recover from crises, for example spending 3-4 weeks in hospital on each occasion.

Some children may be able to manage less severe crises at home with care from parents and advice and/or home nursing from specialist sickle cell teams. Others may routinely be admitted to hospital for a few days. Indications for admission include severe pain, oxygen administration, intravenous fluids and treatment of infections such as pneumonia or septicaemia (generalised blood poisoning). Hospital admission is also needed for severe anaemia requiring blood transfusion.

Some children will require iron chelation therapy at home

Regular blood transfusions (transfusion therapy) are used for children at risk of severe complications such as stroke. The complication of repeated blood transfusions is iron overload, which can be life threatening if it is not treated.

Iron overload is treated with a chelating agent. It is time consuming and has unpleasant side effects. It is likely to be given at home by the parents with outreach support from the treating specialist team.

• Chelation therapy may be given as an infusion using Desferrioxamine subcutaneously or intravenously. Depending on the severity of iron overload the infusion can be given over 12 hours 5-7 days a week or 24 hours a day every day. A common regimen is 12 hourly infusions 3-5 times a week at home. Constant treatment is likely to be given in hospital as part of the treatment of life threatening complications of iron overload such as heart failure. Complications of iron overload such as heart failure can occur in sickle cell anaemia and although rare it is potentially fatal.
• An oral chelation agent is available – deferiprone – this is given three times a day 7 days a week. This treatment has severe side effects including -:
• Neutropenia (reduced white blood cell count) - this can lead to life threatening septicaemia – weekly blood test monitoring is required during treatment.
• Arthropathy and joint destruction. Deferiprone is not licensed for use in sickle cell anaemia but some doctors use it when the alterative treatments have failed or are not available.
• Another oral chelation agent called Deferasirox is also available and this has different but equally severe side effects including -:
• Kidney toxicity and reduced renal function in 30%
• Liver toxicity in 2%
• Severe diarrhoea

Chelation treatment of one sort or another is started usually within one to two years of starting regular transfusions. The amount of iron in the body is monitored regularly by special investigations. It is difficult to comply with these treatments because of side effects, the time-consuming nature of treatment and no immediate and obvious benefit from adhering to them carefully. Later problems with iron overload increase mortality if the young person defaults from chelation therapy.