Corresponding author: Maria Becheva ( olivier@abv.bg ) Academic editor: Plamen Peikov
© 2021 Maria Becheva, Petar Atanasov.
This is an open access article distributed under the terms of the Creative Commons Attribution License (CC BY 4.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original author and source are credited.
Citation:
Becheva M, Atanasov P (2021) Therapeutic guidelines for the treatment of cystic fibrosis. Pharmacia 68(1): 151-154. https://doi.org/10.3897/pharmacia.68.e49247
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Cystic fibrosis (CF) is a complex, systemic autosomal recessive disease that affects the functions of the respiratory system, the digestive tract and all exocrine glands. The frequency for Europe averages 1: 2500 to 1: 3500 live births. The total number of patients with cystic fibrosis in Bulgaria is about 180. About 10% of the patients are diagnosed at birth. About 60–70% of patients are diagnosed before they reach one year of age. Respiratory symptoms predominate in the clinical picture in patients with cystic fibrosis and determine the prognosis in more than 90% of the patients. The treatment of patients with cystic fibrosis is strictly individualized, pharmacological and non-pharmacological and requires a comprehensive therapeutic approach. The complex therapy also includes bronchodilators, NSAIDs, corticosteroids, respiratory rehabilitation in combination with general body massage. Continued courses of broad-spectrum antibiotics are required to suppress chronic infection. With the progression of the disease, complications such as atelectasis, pneumothorax and pulmonary hemorrhages are observed. The establishment of specialized centers with trained and experienced professionals is essential in order to provide optimal patient care. These include frequent clinical evaluations, follow-up of complications, and early interventions for the treatment of patients with cystic fibrosis.
The aim of the article is to familiarize the audience with the therapeutic measures applied in the treatment of patients with cystic fibrosis.
Complications, cystic fibrosis, treatment
Cystic fibrosis (CF) is a complex, systemic autosomal recessive disease that affects the functions of the respiratory system, digestive tract and all exocrine glands (O’Sullivan et al. 2009). The disease is due to a mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene for the transmembrane transport regulator. The frequency for Europe averages 1: 2500 to 1: 3500 live births. It is most commonly found in Caucasian representatives in Europe, North America, Australia. The frequency of the disease is variable depending on ethnicity and geography (Mihov et al. 1997)
The total number of patients with cystic fibrosis in Bulgaria is about 180. About 10% of the patients are diagnosed at birth. About 60–70% of the patients are diagnosed before one year of age. Late diagnosis is associated with insufficient expression and poor progression of clinical symptoms. Such patients are usually not seriously ill and have a relatively good quality of life (
Respiratory symptoms predominate in the clinical picture in patients with cystic fibrosis and determine the prognosis in more than 90% of patients. The first respiratory symptoms of cystic fibrosis in 60% of the children manifest themselves by the age of 6 months, by the end of the first year – in 80%, and by the second year – in 91% of the patients. In 2% of the children, the disease can occur after 2 years of age (Kondratieva et al. 2013).
Respiratory symptoms and manifestations most commonly include the upper respiratory tract – chronic rhinitis / rhinorrhea (> 90% at > 8 months), nasal polyposis – 10–32%, sinusitis and involvement of the lower respiratory tract, with the early symptom being chronic cough (excruciating), whooping cough, nocturnal), wet rales, obstructive pulmonary disease (wheezing, tachydispnea) with bronchial hyperreactivity in 50% of cases and a positive bronchodilator test in some patients. Late respiratory symptoms include respiratory failure, exacerbation of respiratory distress, hypercapnia, pulmonary hypertension, cor pulmonale (Kapranov et al. 2011).
Children appearance with cystic fibrosis is characteristic – retardation in physical development, dystrophic changes of skin and hair, enlarged, often deformed chest, large abdomen (sometimes the occurrence of umbilical hernia), thin limbs with deformed extremities of the fingers with “drum sticks” and nails like “clock glass”. With severe disease, the processes in the lungs are constantly progressing, exacerbations occur more often, phlegm is purulent, with a rotten odor. Bronchial obstructive syndrome is severe and is associated with a narrowed lumen of the bronchi as a result of inflammatory changes in the bronchial wall and the accumulation of viscous secretions. In the evolution of the disease, the symptoms of hypoxia gradually increase – shortness of breath, cyanosis, tachycardia, the cor pulmonale clinic, respiratory and heart failure develops (
The pulmonary function is an important measure of disease severity and prognosis in cystic fibrosis patients. Spirometry was performed including measurement of FVC (forced life capacity), FEV1 (forced expiratory volume for 1 sec), FEF25-27 (forced expiratory flow between 25% and 75% of vital capacity) (Debray et al. 2006). FEV1 is the most proven clinical means of lethal prognosis and is the primary means of measuring the outcome in many clinical trials (
Treatment of cystic fibrosis patients is a difficult, not yet fully resolved, task. It is strictly individualized, pharmacological and non-pharmacological, requiring a complex therapeutic approach (
The purpose of therapy is to control lung infections and improve nutritional status, early adequate behavior and delay definitive changes (
A small number of pathogens, initially S. aureus and H. influenzae, are found in the lungs of patients with CF, followed by chronic colonization with P. aeruginosa. Antibiotic courses are used for treatment. Even oral courses (but lasting 2–4 weeks) are sometimes sufficient. For more severe infections, intravenous administration is required (
Sputum microbiological examination in patients with cystic fibrosis should be performed at least once every 3 months.
One of the important components of treatment is kinesitherapy. Depending on their health status, patients with cystic fibrosis may perform locomotor activity in hospital under the guidance of paramedics (respiratory rehabilitation), at home (adapted physical activity), at school or in sports (Sugny et al. 2018). Its main purpose is to clear the bronchial tree from the viscous secretion. The tasks of kinesitherapy are to relax the respiratory muscles and the diaphragm, reduce the frequency of breathing and control the exhalation, facilitate the removal of the bronchial secretion and its evacuation by increasing the volume of bronchial oscillations and the speed of air flow, improving exercise mobility. in coughing and expectoration and training in compensatory diaphragmatic breathing (
Kinesitherapy is performed against the background of inhalation treatment (
As the disease progresses, complications and conditions are observed that require immediate intervention. These include atelectasis, pneumothorax, pulmonary hemorrhage. In asymptomatic, limited pneumothorax detected by chance, the patient is actively observed for 24 hours (
If clinical symptoms do not appear and the changes do not progress radiologically, regular follow-up behavior may be initiated. In all other cases, drainage, possibly surgery (
Cystic fibrosis is a complex disease that requires a holistic treatment approach. In recent years, medicine has progressed significantly with CF with a dramatic improvement in life expectancy (
The establishment of specialized centers with trained and experienced professionals is essential in order to provide optimal patient care. These include frequent clinical evaluations, follow-up of complications, and early interventions for the treatment of patients with cystic fibrosis (