65 Roses of Yellow and Red by Terri Simpson

Cystic Fibrosis is a genetic condition which affects more than 10,000 people in the UK. However, one in 25 of people carry the gene that causes it, maybe without even knowing it. There is no way to develop CF later in life; you only have it if you are born with it. The gene that is affected by CF controls the movement of salt and water in and out of cells. This results in a build-up of thick mucus in the lungs, digestive system and other organs. This causes a range of difficult symptoms that have an effect on the whole body. People with CF also have a shorter life expectancy than normal. There is currently no cure for CF but new drugs are constantly being developed to make life easier for people affected by Cystic Fibrosis.

Cystic fibrosis can be diagnosed during new-born screening, which is carried out as part of the heel-prick test that is conducted on all babies in the UK, and positive results are followed up with a sweat test.

There are also ways to test for CF during pregnancy, which carry some risks and are only usually carried out in pregnancies with a high chance of cystic fibrosis. If there is a history of CF in a person's family, or they have a partner with CF, their child is more likely to be born with the disease. There are also ways to test for CF during pregnancy, which carry some risks and are only usually carried out in pregnancies with a high chance of cystic fibrosis.

CF can affect many different parts of the body in many different ways, most people with CF will take a number of tablets and treatments a day.

Medications for people with cystic fibrosis are most commonly used for:

  • Lung symptoms
  • Digestive system symptoms
  • Other affected areas

Your Body

Lungs

Medication to treat the lungs can be taken by nebuliser (inhaled into the lungs), orally or intravenously (into the veins). Some examples of treatments include:

  • Bronchodilator drugs which open the airways by relaxing the surrounding muscles, reducing tightness and shortness of breath.
  • Antibiotics treat or control infection
  • Steroids reduce inflammation in the airways
  • Mucolytics such as DNase break down mucus, making it easier to clear from the lungs

Digestive system

Cystic fibrosis affects the pancreas, meaning that many people with the condition require enzyme capsules with meals and snacks. The capsules replace pancreatic enzymes in helping to break down food more effectively.

Bones

A lack of minerals caused by a damaged pancreas can result in osteoporosis (weak or brittle bones). Researchers are examining the benefits of high doses of vitamin D and calcium.

Ear, nose and throat (ENT)

People with CF often experience ENT disorders, particularly ones that affecting  the respiratory system.

  • Nasal steroid sprays like Flixonase help treat-but not cure-rhinitis, a swelling of the nasal lining.
  • Washing through the nose with salty water (nasal douching) and the use of antibiotics may also be advised
  • Nasal douching can control infected nasal discharge as well.
  • Keyhole surgery might be used to treat chronic rhinosinusitis (when thick mucus fills the sinuses and fills the nose with harmful bacteria), or related complications like nasal polyps.

Physiotherapy

Physiotherapy is important and starts very early in the life of a child with cystic fibrosis. How early it begins depends on their symptoms. It covers a range of areas, such as airway clearance to help loosen and remove the mucus that builds up in the lungs, or maintaining good posture to avoid back problems that can have a negatively affect lung function.

Staying active

Exercise is particularly important for people with CF because it can help clear mucus from the lungs, improve physical bulk and strength, and help improve overall health. In particular, children with CF are encouraged to take part in as much physical activity as possible, particularly types of exercise that leave you breathless, such as running.

Nutrition

People with cystic fibrosis need a balanced diet, although many will require more calories than someone without the condition in order to maintain a healthy body weight, which can be challenging. Nutritional needs will vary according to age, weight, height, symptoms, lung function and activity level.

Transplants

Lung transplants are available for eligible cases and can extend the patient's life expectancy by up to 10 years

Mindfulness practices

By practicing mindfulness, some people will find greater peace and calm, even while experiencing uncomfortable symptoms that can make it hard to think clearly.

Medications

Kalydeco

Kalydeco is the first precision medicine for cystic fibrosis to become available on the NHS and is also known by the name ivacaftor.

Kalydeco helps to open 'gates' in the cells of people, allowing chloride to move into and out of the cells and helping to keep the balance of salt and water in the lungs.

Orkambi

Orkambi (ivacaftor/lumacaftor) is the second precision medicine to be licensed for use in the UK by people with cystic fibrosis (CF), although it is not currently provided by the NHS except in rare cases on compassionate grounds.

Orkambi is a combination medicine, made up of ivacaftor and lumacaftor. Lumacaftor helps get more proteins to the surface of cells in the body, and ivacaftor helps the chloride channels in the cells to operate more effectively. The combination of these two things helps to keep a healthy balance of salt and water in the organs – particularly the lungs. Orkambi is manufactured by Vertex Pharmaceuticals.

  • According to the 2014 UK Cystic Fibrosis Registry Report, there are 2,834 people in England, 243 people in Scotland, 118 people in Wales and 101 people in Northern Ireland who could benefit from Orkambi.
  • Orkambi has been licensed for use in the UK for people with CF over the age of two who have two copies of the F508del mutation. However, NICE rejected the use on the NHS.
  • In May 2016, the Scottish Medicines Consortium (SMC) noted that Orkambi was a beneficial treatment.In June 2016, NICE recognised Orkambi as an important treatment.
  • Both the SMC and NICE were unable to recommend Orkambi for general use within the NHS/HSCNI on grounds of cost-effectiveness and a lack of long-term data.
  • Orkambi is currently only prescribed to people on compassionate grounds. Compassionate use means that Vertex Pharmceuticals provide the drug to people who fulfil a number of criteria.
  • Orkambi was licensed for 6-11 year olds by the European Medicines Agency in Europe in January 2018, and was again licensed for 2-5 year olds in January 2019.

Symkevi

Like Orkambi, which combines ivacaftor with lumacaftor, Symkevi combines two drugs in one treatment. Symkevi combines ivacaftor with new drug compound tezacaftor. The treatment has been tested on people with CF with two copies of the F508del mutation and those with one copy of F508del and another mutation that has residual function. There has been positive results.

The European Medicines Agency (EMA) authorised marketing for Symkevi for those aged 12 and over with two copies of F508del or one F508del and a residual function mutation. However, Symkevi is currently not licensed in the UK.  

CF Trusts and Charities

Disclaimer: This content about a medical condition is written by a young person and does not reflect the opinions of Young Scot. Young Scot do not take any responsibility for the information contained in this article, and should anyone have any concerns about their own health or medication, they should contact their doctor or another healthcare professional. The NHS also have online advice about Cystic Fibrosis.