Extrait

Background and study aims Sickle cell disease (SCD) is an inherited blood disorder where the red blood cells do not develop normally. Instead of being flexible and disc-shaped like normal blood cells, they are rigid and shaped like a crescent (or sickle). They also contain defective haemoglobin, the iron-rich protein that enables red blood cells to carry oxygen. This results in lower than normal oxygen levels in the tissues and organs of the body and can cause people to feel lethargic (lack of energy), tired and breathless, particularly after exercise. Many complications of the condition are thought to be caused by low daytime and night time oxygen levels. These can be made worse if the patient also suffers from obstructive sleep apnoea (OSA) where the muscles and soft tissues in the windpipe relax and collapse during sleep, causing a total blockage of the airway; this results in an extra dip in oxygen levels during sleep. Overnight oxygen is already used in patients with chronic lung disease who suffer from low oxygen levels during sleep. Automatic positive airway pressure (APAP) machines, which work by blowing air at a pressure that keeps the windpipe open, are used successfully for OSA patients of all ages. It is possible that these treatments may also help people suffering from SCD. Early studies have shown that SCD patients do find overnight oxygen safe and easy to use. There is also some evidence that APAP can be beneficial to children with SCD, resulting in improved attention span and fewer crises. However, further work is needed to decide whether the inconvenience of these treatments outweigh any benefits from using them for treating dips in night time oxygen levels for SCD patients. The aim of this small scale (pilot) study is to find out which intervention (the overnight oxygen or APAP) is the most acceptable for patients by asking them to use them for one week each and then have an interview describing their experience. The most acceptable intervention will then be used for further study. Who can participate? English speaking SCD patients aged over 8 years and who are able to (or their parents are able to) use a smart phone. What does the study involve? Participants are first asked to fill in a daily pain diary for a week. They are then are randomly allocated to receive one of the two interventions. A sleep physiotherapist sets up the intervention and shows the participant how to use it. The participant them uses the intervention for a week, filling in their pain diary daily and will have phone interviews to discuss any symptoms suffered. After the seven day treatment, the intervention is stopped and the participant attends hospital for a review, which includes having blood and urine tests taken, and having their lung function and oxygen levels measured. Afterwards, the participant continues to fill in their pain diary for another week before repeating the procedure with the second intervention. What are the possible benefits and risks of participating? Participants may find that their condition improves from one or both interventions given in this trial. If this proves to be the case, we will leave the machine that provides the most benefit with the patient (with approval from their doctor). As regards to risks, some people may find the tests to check lung function uncomfortable and may make them feel dizzy. Blood tests can also be slightly painful. Overnight APAP and oxygen therapies may also be uncomfortable and inconvenient to the participant. There is evidence that higher doses of oxygen can stop new red blood cells from being made, but not with the doses and timing of the oxygen being used for this study. Where is the study run from? University Hospital, Southampton (UK) Guy's and St Thomas' NHS Foundation Trust (UK) UCL Institute of Child Health (UK) When is the study starting and how long is it expected to run for? July 2014 to December 2014 Who is funding the study? National Institute for Health Research (UK) Who is the main contact? Professor Fenella Kirkham [email protected]

Critère d'inclusion

• Sickle Cell Disease

Liens

• ictrp
• isrctn