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“Your Medicines” discusses cystic fibrosis treatments

Cystic fibrosis, complicated treatment, but compliance is vital

Treatments for cystic fibrosis (CF) are complicated. Cystic fibrosis is one of the most common types of chronic lung disease in children and young adults. It is characterized by the accumulation of thick, sticky mucus in the lungs, pancreas, digestive tract and other areas of the body.

It affects one in every 5,300 newborns in Spain and there are currently around 2,500 patients, of whom 53% are men and 47% women.

CF is an inherited disease, classified as rare, caused by a defective gene.

Mucus clogs the airways and causes a persistent cough, wheezing, shortness of breath, exercise intolerance, recurrent lung infections, inflamed nostrils or nasal congestion. You can also block the pathways that carry digestive enzymes from the pancreas to the small intestine preventing complete absorption of nutrients.

The age of onset of this disease is very variable; some patients begin the clinical picture during infancy, while others may remain asymptomatic until adolescence or young adulthood.

An early diagnosis of CF and a treatment plan can improve both survival and quality of life.

“Your medicines”the video block that EFEsalut and the Viatris Foundation for Health promote to raise awareness and increase therapeutic adherence, analyzes cystic fibrosis.

For this reason, we went to the Ramon i Cajal University Hospital in Madrid, which has a Cystic Fibrosis Unit where doctor and pediatrician Saioa Vicente Santamaría works, attached to Pediatric Gastroenterology, with whom we spoke, and also with Fernando Moreno, patient and president of the Madrid Cystic Fibrosis Association.

Doctor and pediatrician Saioa Vicente Santamaría/EFE

Doctor Saioa Vicente Santamaría: There is a need for interdisciplinary cystic fibrosis units

Doctor, what is cystic fibrosis?

It is a genetic, chronic and potentially lethal disease that worsens the quality of life and survival of those who suffer from it.

The genetics of this disease encode a protein that is defective and is expressed in many organs of the body, primarily in the lungs, pancreas and digestive tract.

In the lungs, this protein makes the secretions thicker and this generates an obstruction in the lung ducts; this means a good culture medium for certain bacteria and that there is a pattern of inflammation and destruction of lung tissue.

It is also expressed in the pancreas where the obstruction of its ducts means that fats are not absorbed well and patients have to take pancreatic enzymes to be able to nourish themselves properly and improve their nutritional situation. And it is also expressed at the digestive level.

Who does this disease affect?

It affects people who have two mutations in the CFTR gene that codes for this protein. It is a rare disease, which affects one in every 4,500 live babies in the Community of Madrid.

Thanks to the neonatal screening program, implemented in recent years, it is possible to diagnose the disease before it shows symptoms.

What are the treatments like?

CF treatments take a lot of time for the patient because the different symptoms of the disease need to be treated. Until a few years ago, treatment was purely symptomatic. If the problem was in the lungs, we apply techniques to improve the fluidization of the lung mucus and remove these mucus to avoid bacteria.

The mainstay of treatment, since most patients have the problem in the pancreas, is pancreatic enzyme treatment, which has improved patient survival from a few months of life to several years.

The treatment also consists of cleaning and clarifying secretions from the lungs with nebulized therapies and respiratory physiotherapy that we teach patients to do at home.

This treatment requires an hour and a half in the morning and an hour and a half in the afternoon. It’s a lot of time for the patient.

Because we start these patients with the prenatal diagnosis, we do it before they feel sick and we see any symptoms of the disease.

It is essential to go ahead, but this makes you less aware of the disease and sometimes relaxes the treatment, which can worsen the prognosis.

You have to be very attentive to the treatment, also with the right diet, the sport they have to do and, in case of more complications, apply antibiotics.

Doctor, what is the challenge of cystic fibrosis?

We are in a very promising and hopeful situation. In recent years, a treatment has come out that improves the function of the protein.

The challenges we have are, firstly, to drive universal neonatal screening programs to diagnose the disease before symptoms, so we are moving forward.

Secondly, guarantee access to treatments for all patients with cystic fibrosis.

And thirdly, to have Interdisciplinary Units that attend to patients, since they have to visit many specialists and the treatments take a long time. We need to coordinate doctors and help patients to make visits easier.

Fernando Moreno, patient and president of the Madrid Association of Cystic Fibrosis/EFE

Fernando Moreno: The treatments are vital and a lifelong commitment

How do patients experience cystic fibrosis and how is their quality of life?

As the doctor said, you need to devote a lot of time to the disease from the first minute it is known.

It’s an almost barracks engagement, you can’t relax for a second. Cystic fibrosis doesn’t go on vacation, and when you feel well, it’s because of the comprehensive treatment we provide, and you can’t quit even when you feel better.

What are the treatments?

It is a lifelong commitment to know that, at least, you must devote three hours a day to treatment, this in the event that the disease is under control.

But in autumn/winter, if there are other complications such as the flu, pneumonia, the treatment worsens and can become even more complex, with intravenous treatments, sometimes reaching the point of having to admit the patient to the hospital several times weeks.

The treatments are a vital commitment of care with three hours a day of dedication and a greatly diminished quality of life.

What is therapeutic adherence?

Cystic fibrosis treatment is very hard, and adherence is not important, it is vital. Either you’re a stickler or you’re going to have a really bad time. Therefore, the sooner treatment is started, the better.

When the diagnosis was late, adherence was much more complicated, but now, with the diagnosis from birth, the parents are the first to learn the treatment, be adherent and then pass it on to their children.

What is important is to make treatment and adherence a routine and to devote the necessary time to it even if it is difficult because, without good therapeutic adherence, even when you are well, the disease becomes very complicated.

From left on the right, the patient Fernando Moreno, Dr. Saioa Vicente Santamaría, the journalist Javier Tovar and the director of the Viatris Foundation for Health, Javier Anitua/EFE

EFEsalut and the Viatris Foundation maintain their common goal of raising awareness in society about the importance of taking medications correctly and improving levels of therapeutic adherence.

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