Care and mobility needs

Care

Early diagnosis and treatment, especially of infection, improves prognosis. Children born in the UK will usually be identified by screening at birth. This means that long term complications can be prevented enabling normal development for many.

The aim of treatment is for the child to lead a normal life, attend school and enter adulthood with few or no complications of the disease. Despite this, up to 8% of children have had strokes by the age of 14 years. A higher number of children are at risk of stroke and will be treated with regular blood transfusions to prevent it.

Ongoing care of children includes -:

• Monitoring the child’s condition and seeking medical help when appropriate to do so.
• Keeping the child warm, well nourished and hydrated to reduce the frequency of crises
• Ensuring child takes medication such as folic acid and penicillin every day as prescribed
• Managing minor crises at home

The frequency of crises and whether they are managed in hospital or at home will be important information to request from the treating specialist. Frequent crises will require more care but supervision will not prevent a crisis from occurring.

Most children will be unwell and in bed for up to week after a crisis. There is likely to be a period of recovery at home after the crisis before the child returns to normal activities and school. Significant care on a daily basis is likely to be given if crises occur more frequently than twice a month on average. Children with frequent crises are also more likely to have developed some of the long-term complications of the condition such as a stroke and epilepsy - see Epilepsy guidance. Children who have infrequent crises are likely to take over the majority of their care in the teenage years. Children who have frequent crises i.e. more than twice a month are likely to continue to require care in relation to their crises.

Care needs related to chelation therapy

Some children will be on transfusion therapy. This means they will have regular blood transfusions at the hospital to prevent the long term complications of Sickle Cell Disease. In order to prevent a life threatening condition called ‘iron overload’ they will need to have iron chelation therapy at home. The chelation treatment is unpleasant and a parent will need to administer the treatment or supervise administration of the treatment to make sure that it is taken as prescribed.

Chelation therapy may be given as an infusion using Desferrioxamine subcutaneously or intravenously. A common regimen is 12 hourly infusions 3-5 times a week.

Another chelation agent called Deferasirox is available in tablet form and this has different but equally severe side effects including -:

• Kidney toxicity and reduced renal function in 30%
• Liver toxicity in 2%
• Severe diarrhoea

An oral chelation agent is available called deferiprone. This is given three times a day 7 days a week in tablet form. 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.

Parental supervision of each dose will be required to ensure children comply with the treatment. Older children may be able to administer their own chelation therapy. Children around the age of 12 to 14 years of age may be expected to take their medication independently but many parents will continue to supervise and monitor this either due to concerns about compliance or due to learning difficulties that their child might have.

Long term complications include -:

• Stroke - Children may have mobility difficulties related to spastic paralysis of one leg or difficulties with balance. Other problems that could be caused by a stroke include epilepsy, cognitive difficulties and behavioural problems all of which may mean more monitoring or care and attention or supervision is required. Cognitive problems after stroke are common in children with Sickle Cell but permanent mobility problems are much rarer.
• Kidney failure – end stage renal failure is likely to require renal dialysis and this may be carried out at home or at the hospital. CAPD (Chronic ambulatory Peritoneal Dialysis) is likely to be done overnight, most nights of the week, at home. The parent will need to set up the dialysis equipment and be available to check it or adjust it during the 8-12 hours it takes during the night. Haemodialysis is done three times a week and takes around 4-6 hours to complete; it can be done at home or in the hospital.
• Arthritis - Avascular necrosis of the shoulder or hip joints can cause severe arthritis – it would be unusual in childhood for this to lead to persistent severe disability as severe arthritis takes some years to develop after the avascular necrosis event. Joint replacement is an option for these children once they have finished growing so disability is not expected to be permanent. Severe arthritis of the hip may limit walking distance. Severe arthritis of the shoulder will restrict range of movement around the shoulder and may make dressing, washing and styling hair more difficult as these activities involve lifting both arms over the head.

Mobility

In the typical case, ability to walk long distances is either normal or reduced by anaemia and breathlessness. Very few children with sickle cell disease have difficulties walking short distances. The complications of the condition that may cause difficulty walking short distances include severe arthritis of the hip due to avascular necrosis and neurological damage following a stroke. Recovery of walking ability is usual after a stroke in children and severe arthritis is treated with joint replacement in severe cases.