Current Status of Cystic Fibrosis in Türkiye: Data from the National Registry
Dilber Ademhan Tural1,2, Tuğba Şişmanlar Eyüboğlu3, Güzin Cinel1,2
1Department of Pediatric Pulmonology, Ankara Yıldırım Beyazıt University Faculty of Medicine, Ankara, Türkiye
Insights
The Cystic Fibrosis Registry of Türkiye (CFRT) report shows 2,258 patients with cystic fibrosis (CF) in 2023. Most patients received standard treatments, but only 15.9% used CFTR modulators, indicating a need for broader access.
Area of Science:
- Medical Research
- Pulmonology
- Genetics
Background:
- The Cystic Fibrosis Registry of Türkiye (CFRT) was established in 2017.
- It collects demographic, clinical, genetic, and treatment data for cystic fibrosis (CF) patients.
- This registry is crucial for understanding the CF landscape in Türkiye.
Purpose of the Study:
- To present the 2023 annual data from the CFRT.
- To describe the current status of cystic fibrosis in Türkiye.
- To analyze demographic, clinical, genetic, and treatment data of CF patients.
Main Methods:
- Data extracted from the CFRT's 2023 record.
- Included demographic, clinical, and treatment information.
- Genetic analyses were performed on a majority of patients.
Main Results:
- 2,258 CF patients were recorded in 34 centers in 2023.
- The median age was 9.1 years, with 46.9% female. Median age at diagnosis was 0.3 years.
- F508del was the most common variant (22.1%). Lung function (FEV1, FVC) and BMI z-scores were reported. Pseudomonas aeruginosa colonization was 17.2%.
- 87.1% used inhaled recombinant human DNase, 83.0% used pancreatic enzyme replacement. Only 15.9% used CFTR modulators. 24 deaths occurred, median age 13.3 years.
Conclusions:
- The CFRT report is a valuable resource for CF patient data in Türkiye.
- It highlights current treatment patterns and identifies areas for potential improvement, such as CFTR modulator access.
Objective:
The Cystic Fibrosis Registry of Türkiye (CFRT) was established by the Turkish Pediatric Respiratory Diseases and Cystic Fibrosis Society and has provided detailed information on demographic, clinical, genetic, and treatment-related aspects of cystic fibrosis (CF) patients since 2017. We aimed to describe the current status of CF in Türkiye using CFRT's 2023 annual data.
Material And Methods:
Demographic, clinical, and treatment data were taken from CFRT's 2023 record.
Results:
In 2023, 2,258 patients from 34 centers were recorded. The median age of patients was 9.1 years, and 46.9% were female, with a median age at diagnosis of 0.3 years. Only 14.9% of the patients were older than 18 years. Genetic analyses were completed in 97.3% of patients. The most common variant, F508del, had a total variant frequency of 22.1%. The median percent predicted FEV1 and FVC were 88.0 and 94.0 in those aged 6-17 years 71.0 and 84.0 in those aged ≥18 years, respectively. The median values of body mass index z-scores were -0.5, and -0.5 for patients 2-18 and older than 18 years, respectively. Chronic colonization with Pseudomonas aeruginosa was present in 17.2% of the patients. Most patients used inhaled recombinant human DNase (87.1%) and oral pancreatic enzyme replacement treatment (83.0%). CF transmembrane conductance regulator (CFTR) modulators were used by 15.9% of patients. Over the year, 24 patients died, with a median age at death of 13.3 years.
Conclusion:
The CFRT report provides a valuable resource showing the clinical and laboratory data of patients with CF in the country.
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