Background. Respiratory colonization with Pseudomonas aeruginosa is associated with increased morbidity and mortality in cystic fibrosis (CF) patients. This study aims to assess the clinical characteristics and associated factors of CF infants under two years of age with P. aeruginosa colonization in Türkiye. Method. Of the 1637 patients registered in the Cystic Fibrosis Registry of Türkiye in 2019, 284 patients under two years of age were included in this retrospective cross-sectional study. Patients were classified into two groups: those with P. aeruginosa colonization (Group 1) and those without (Group 2). Cystic fibrosis transmembrane conductance regulator (CFTR) gene functions were categorized according to CFTR mutation functional class. Results. Twenty-three patients (8.1%) were categorized as Group 1 and 262 participants (91.9%) were classified as Group 2. Infants with P. aeruginosa colonization (Group 1) were more likely to have minimal CFTR function compared with those without colonization (87% vs. 39.8%, p = 0.017). In addition, both Staphylococcus aureus colonization (47.8% vs. 7.3%, p < 0.001) and methicillin-resistant S. aureus positivity (17.4% vs. 6.1%, p = 0.042) were observed more commonly in Group 1. There were no statistical differences between the groups in terms of age at diagnosis, gender, mean z-scores of weight and height, newborn screening test positivity, sweat chloride test results, and pancreatic insufficiency (p > 0.05). Univariate logistic regression analysis did not identify significant associated factors for P. aeruginosa colonization. Conclusions. Our findings suggest that minimal CFTR function and S. aureus colonization are associated with P. aeruginosa colonization in CF patients under two years of age. Further studies are needed to investigate associated factors for early P. aeruginosa colonization, eradication treatment effectiveness, and longitudinal outcomes of in CF patients under two years of age.

BACKGROUND:Pulmonary exacerbations (PEs) are associated with a subsequent decline in lung function. We aim to evaluate lung function in cystic fibrosis (CF) patients with frequent PEs in their first 2 years of age using spirometry at age 6. METHODS:This retrospective cohort study included CF patients who were 6 years old from the CF registry of Turkey in 2019. According to the number of PEs, patients were classified: those who had two or fewer PEs in the first 2 years of age were defined as Group 1 and those who had more than two PEs were defined as Group 2. The patients' demographics and clinical characteristics were compared between Group 1 and Group 2. RESULTS:The study included 88 patients who had data on PE from their first 2 years and completed their sixth year by 2019. Fifty-nine patients were included in Group 1 and 29 in Group 2. The mean percent-predictive FEV1 (ppFEV1), percent-predictive FVC (ppFVC) values, and the mean age at first PE were lower in Group 2 than in Group 1 (p = 0.019, p = 0.017, p < 0.001). The patients with chronic Pseudomonas aeruginosa (PA) colonization had lower mean ppFEV1 and ppFVC values than those without (p = 0.001, p = 0.001). Patients with PA in respiratory-sample culture during their first PE had lower ppFEV1 and ppFVC values than those with SA (p = 0.046; 0.018). CONCLUSIONS:This study showed that more frequent PEs in the first 2 years of age and chronic PA colonization were associated with poorer FEV1, FVC, and BMI values in CF patients.

Background. Flexible bronchoscopy (FB) is a critical diagnostic and therapeutic tool in respiratory diseases, enabling airway assessment, sample collection, and therapeutic interventions. Despite international guidelines, practices vary widely across centers. This study aimed to assess current FB practices and to identify variations among pediatric pulmonologists in Türkiye. Methods. A descriptive cross-sectional survey was distributed via email to clinical directors of 19 centers performing FB in March 2023. The survey comprised 80 questions across seven domains: demographics, patient preparation, bronchoscopy procedure, sedation/anesthesia, discharge, bronchoscope cleaning, and respondent comments. Participants were asked to provide accurate and objective data on FB practices at their centers. Results. All 19 centers participated in the survey, achieving a 100% response rate. The median FB experience was 12.5 years (IQR: 1-30), with a median 210 procedures per year per center (min-max: 30-500; IQR: 80-250). The most frequently reported indications were atelectasis (84.2%), bronchiectasis (78.9%), suspected foreign body aspiration (52.6%), and to obtain bronchoalveolar lavage (BAL) in patients with immunodeficiency (52.6%). Nine centers (47.4%) indicated performing advanced bronchoscopic procedures, including endobronchial biopsy (31.6%), bronchoscopic intubations (26.3%), and tracheal/bronchial stent insertion (10.5%). General anesthesia (84.2%) was the predominant sedation method, and propofol (89.5%) the most frequently used agent. Premedication was used in 13 centers (68.4%), with midazolam being the most commonly used agent (76.9%). Twelve centers (63.2%) also used topical lidocaine for local anesthesia. Respondents reported the need for developing interventional procedures and increasing the number of trained bronchoscopy teams. Conclusions. This first comprehensive national survey of pediatric FB practices in Türkiye, encompassing all relevant centers, revealed significant heterogeneity in procedural approaches, particularly regarding sedation protocols, BAL techniques, and interventional capabilities. The findings underscore the importance of developing interventional procedures and enhancing the training of pediatric pulmonologists and interdisciplinary teams practicing pediatric bronchoscopy to improve patient outcomes and procedural consistency.

INTRODUCTION:Cystic fibrosis (CF) is a complex genetic disorder characterized by significant clinical heterogeneity. We aimed to investigate the genotype-phenotype relationship of nasal polyps (NP) in CF Registry of Turkey (CFRT). METHODS:The study included all children registered in the CFRT in 2023, divided into two groups based on NP presentation. Clinical and demographic data were compared. The Sinus-Nasal Quality of Life (QoL) Survey-5(SN-5) evaluated patients with NP history. RESULTS:A total of 2258 patients were registered, with 115 (5.1%) having NP and 2143 without NP. Age was higher in the NP group (p < 0.001). The first and second sweat tests were higher in the NP group (p < 0.05). Median FEV1% was 89 (31-133) for the NP group and 85 (15-142) for those without NP (p = 0.015). Chronic colonization of MSSA and MRSA, CF-related liver disease, and CF-related diabetes were also more common in NP (p < 0.05). No differences were detected in pulmonary exacerbations (p > 0.05). In the NP group, severe genotype and F508del were more frequent (p < 0.05). Elexacaftor/tezacaftor/ivacaftor use was more common in NP (p = 0.003). A weak negative correlation was found between age and the SN-5 score (p = 0.020, r = -0.231), as well as a weak positive correlation between age and the Visual Analog Scale (VAS) (p = 0.001, r = 0.322). CONCLUSION:Although uncommon, NP is associated with more severe clinical and genetic characteristics in CF. These findings underscore the importance of integrating upper airway evaluation into CF management and highlight the potential for age-related adaptation or improved symptom perception in older patients.

BackgroundAllergic bronchopulmonary aspergillosis (ABPA), is a hypersensitivity reaction in the lung against the antigens of Aspergillus fumigatus. The aim of this study is to determine the incidence of ABPA, and to determine the risk factors leading to the development of ABPA.MethodsThe study was designed as a retrospective cohort study. Using the CF Registry System of Turkey data, the incidence of ABPA was determined and the clinical and demographic characteristics of 44 patients newly diagnosed with ABPA in 2022 and 132 patients with similar mutation weight and age range without ABPA diagnosis in that year were examined and compared. Also risk factors affecting the development of ABPA were determined.ResultsLow pulmonary function test values, having had at least one pulmonary exacerbation in that year, receiving intravenous antibiotic treatment, using inhaled antibiotics, having high number of pulmonary exacerbations, having Pseudomonas aeruginosa colonization, having low body mass index (BMI), having cystic fibrosis related diabetes mellitus were found to be among the risk factors for the development of ABPA.ConclusionsEarly recognition and treatment of ABPA is essential to prevent further damage to the lungs. Patients with frequent pulmonary exacerbations, low BMI and low pulmonary function test values, chronic colonization should definitely be considered for ABPA.

OBJECTIVES:Cystic fibrosis-related diabetes (CFRD) is the most common comorbidity in patients with cystic fibrosis (CF). This study aimed to determine the prevalence and risk factors of CFRD using data from the CF Registry of Türkiye (CFRT). METHODS:All patients with CF who underwent diabetes screening in 2022 or earlier were included in the study. The clinical and demographic characteristics of patients with CFRD, pre-diabetes (impaired glucose tolerance, impaired fasting glucose, indeterminate glucose tolerance), and those with normal screening results (NSR) were compared. RESULTS:Of the 1,702 patients registered in the data system in 2022, 916 patients from 21 centers who underwent diabetes screening were included in the study. CFRD was diagnosed in 102 patients (11.2 %), there were 56 patients (6.1 %) with pre-diabetes, and NSR were found in 757 patients (82.7 %). The youngest patient diagnosed with diabetes was 6 years old. In patients with CFRD, the median age of patients was found to be higher (p<0.001), body mass index (BMI) was found to be lower (p 0.017); chronic Pseudomonas aeruginosa (P. aeruginosa) colonization (p<0.001), severe mutations (60.8 %, p<0.001), and pancreatic insufficiency (56.1 %, p<0.001) were more frequent; median FEV1% and FVC% values were significantly lower (p<0.001 and p<0.002, respectively) compared to non-diabetic patients. CONCLUSIONS:CFRD is associated with impaired pulmonary function, poor nutritional status, and increased chronic bacterial colonization of the airways. Advanced age, decreased respiratory function, severe genotype, and pancreatic insufficiency are risk factors for CFRD.

Introduction: Advances in neonatal and pediatric intensive care have improved patient survival rates, emphasizing the need for respiratory support in cases of chronic respiratory failure, resulting in the establishment of the Turkish National Pediatric Patients Receiving Home Mechanical Ventilation (HMV) Support Registration System in 2023. This study aims to present the initial findings of the registry. Methods: In this cross-sectional study, epidemiological and demographic data was obtained from the registry and compared between invasive ventilation (IV) and non-invasive ventilation (NIV) groups. Results: The study included 952 patients registered from 23 centers with a median (IQR) age of 6.0 (3.0-12.0) years. IV support was observed in 51.1% of patients (n= 486). There was no difference in sex between IV and NIV groups, but a significant difference in age, nutrition, mobility status and education were observed between the subgroups. The largest patient group had neuromuscular disease, with spinal muscular atrophy being the most common primary diagnosis (15.7%). Most of the caregivers (78.7% in the NIV group and 93.8% in the IV group) received formal training before the initiation of HMV. Conclusion: Turkey's first registry for pediatric HMV patients is introduced. This system helps understand and solve mobility and education issues for these patients in our country. Collaboration with relevant ministries is crucial. In addition, although most of the caregivers have received training before discharge, efforts are still needed to ensure that this rate is 100%. The long-term results will be presented in the future.

Background: We aimed to assess unmet needs of pwCF in the earthquake zone by income level. Methods: Following the February 6, 2023 Earthquake in Turkiye, the shorter version of the 'Your Current Life Situation' (YCLS) survey was adapted for post-earthquake conditions. The adapted YCLS was administered through face-to-face interviews at participants in seven earthquake-affected provinces to determine the insecurity areas and unmet needs in pwCF. Parents completed the survey for those pwCF under 18 years old; those over 18 completed it themselves. Results: Among 255 participants, 91.7% (n=234) had incomes below the poverty threshold and 71.8% (n=183) below the hunger threshold. Post-earthquake, 69% (n=176) lived in overcrowded conditions and 37.6% (n=96) relocated to temporary housing. Under these challenging circumstances, 34.5% (n=88) of pwCF experienced disruptions in routine visits, and 20.8% (n=53) reported disruption in daily CF care routine. Financial and food insecurities were prevalent, with 77.3% (n=197) and 53.3% (n=136) of participants affected, respectively. The potential impact of earthquake-induced relocation on the participants' insecurity status was analyzed. Among those who relocated, financial, transportation, and housing insecurity appeared to be more prevalent (p<0.001). Conclusion: This is the first study to analyze association between income level and unmet needs among pwCF living in earthquake-affected zone. The study revealed significant financial and food insecurity among pwCF in these areas. The findings emphasize the need for disaster-specific emergency action plans to address these vulnerabilities, ensuring access to healthcare and basic needs during crises.

Background: Advances in neonatal and pediatric intensive care have improved patient survival rates, emphasizing the need for respiratory support in cases of chronic respiratory failure, resulting in the establishment of the Turkish National Pediatric Patients Receiving Home Mechanical Ventilation (HMV) Support Registration System in 2023. This study aims to present the initial findings of the registry. Methods: In this cross-sectional study, epidemiological and demographic data were obtained from the registry and compared between invasive ventilation (IV) and non-IV (NIV) groups. Results: The study included 952 patients registered from 23 centers with a median (interquartile range) age of 6.0 (3.0-12.0) years. IV support was observed in 51.1% of patients (n = 486). There was no difference in sex between IV and NIV groups, but a significant difference in age, nutrition, mobility status, and education were observed between the subgroups. The largest patient group had neuromuscular disease, with spinal muscular atrophy being the most common primary diagnosis (15.7%). Most of the caregivers (78.7% in the NIV group and 93.8% in the IV group) received formal training before the initiation of HMV. Conclusion: Turkey's first registry for pediatric HMV patients is introduced. This system helps understand and solve mobility and education issues for these patients in our country. Collaboration with relevant ministries is crucial. In addition, although most of the caregivers have received training before discharge, efforts are still needed to ensure that this rate is 100%. The long-term results will be presented in the future.

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.

BACKGROUND:Due to the amazing developments in modulatory treatments, genetic analysis of cystic fibrosis (CF) patients has become even more important. More than 2000 disease-causing variants of the cystic fibrosis transmembrane conductance regulator (CFTR) gene have been found, and their ethnic and geographical distributions vary. We aimed to present the first genetic data from the Southeastern Anatolia region of Turkey and evaluate patients' clinical and genetic characteristics and identify modulatory drugs covering a wider range of mutations by detecting and reporting new mutations. METHODS:Our study included 337 CF patients from three CF reference centers in the Southeastern Anatolia region. RESULTS:Ninety-one distinct mutations and four significant deletions were identified by analyzing CFTR mutations. The most prevalent mutation in our research was F508del (8.92%); the second most prevalent mutation was 2183AA->G, and the third most prevalent mutation was R347P. Additionally, a novel mutation (V1160X) was identified in two siblings. Only 33.5% of our patients qualified for CFTR modulator medication therapy. CONCLUSION:This study elucidates the diverse nature of CFTR mutations in the Turkish population. The heterogenous genetic pool of the Southeastern Anatolia region is more similar to Mesopotamia than to other regions of our country, and receives immigration from the East. Detection and reporting of novel mutations and CFTR mutations that occur at very low frequencies from different populations living in various geographical areas are essential for identifying modulatory medicines that cover a broader range of mutations and also help genetic diagnosis of CF in newborn screening.

BACKGROUND:Complications such as parapneumonic effusion (PPE) and necrotizing pneumonia (NP) can be noted in 3% of patients with community-acquired pneumonia and may cause functional lung damage. OBJECTIVE:We aimed to investigate the short- and long-term effects of PPE and NP on lung function and the impact of treatment modalities and radiological sequelae on results. MATERIAL AND METHODS:This multicenter retrospective study includes children aged 0-18 years hospitalized for PPE and NP after community-acquired pneumonia. Demographic, clinical, radiological, and spirometry findings were collected during diagnosis and follow-up. RESULTS:Of 123 children (62 female, median age 57 [interquartile range 71.5] months), 78 were diagnosed with NP and 45 with PPE. According to the defined periods, spirometric evaluation was performed in the first 3 months in 23 patients, between the 3rd and 6th months in 27 patients, and between the 6th and 9th months in 37 patients. At 3 months post-discharge, abnormal spirometry (18.18% restrictive, 36.36% combined spirometry) was observed with a rate of 54.54% in NP, and with a rate of 25.0% (8.33% restrictive, 16.66% combined spirometry) in PPE. At 6-9 months, normal spirometry was observed with a rate of 87% in both groups. FVC% values increased over time in both the NP and PPE groups; however, statistically significant improvement was observed only in the PPE group. In this group, FVC% was significantly higher in the patients who received antibiotics with chest tube and/or fibrinolytic therapy (p = 0.022). Furthermore, those without radiological sequelae had significantly higher FVC% values compared to those with sequelae (p = 0.023) in the PPE group. CONCLUSION:Radiological sequelae and restrictive spirometric patterns were initially more common in NP compared to PPE. However, spirometry indicated significant improvement in both groups by the end of the 9-month follow-up period.

OBJECTIVES:There is limited research on thyroid function in pediatric patients with cystic fibrosis (pwCF). This study aimed to determine the frequency of thyroid dysfunction in children and adolescents with CF and to evaluate iodine deficiency and selenium status in pwCF. METHODS:Sixty-two CF patients and 62 control subjects were evaluated. The anthropometric measurements, nutritional status, FEV1(Forced-expiratory-volume in 1 s) percentage, thyroid function tests (TSH, FT4, FT3), urinary iodine and selenium levels, hospitalization status in the last six months, antibiotic usage, and colonization status with staphylococcus or pseudomonas were assessed for the cases. RESULTS:The mean age of the patient group was 10.84 ± 4.04 years. All CF patients were receiving multivitamin supplementation. Malnutrition was present in 50 % of patients, bacterial colonization in 29 %, FEV1 decrease in 38.5 %, subclinical hypothyroidism (SH) in 12.9 %, iodine deficiency in 87 % and exocrine pancreatic insufficiency in 100 %. T3 levels were found to be higher in pwCF. No significant difference was found between malnutrition and FEV1 and urinary iodine and selenium levels. Compared to the control group, pwCF had lower urinary iodine levels. CONCLUSIONS:To the best of our knowledge, our study is one of the few in the literature to investigate urinary selenium levels alongside iodine in PwCF. Further research is needed to clarify and interpret elevated urinary selenium levels in this context. It was shown that iodine deficiency and the rate of SH were relatively high in pwCF. However, it was still thought that correcting iodine deficiency in these patients could improve thyroid dysfunction associated with CF.

INTRODUCTION:Newborn screening (NBS) for cystic fibrosis (CF) facilitates early diagnosis and has been shown to significantly improve long-term clinical outcomes. In this study, we aimed to evaluate the 7-year results of the immunoreactive trypsinogen (IRT)/IRT NBS of Turkey. METHODS:The study included all CF patients who were born after NBS implementation, and who were enrolled in the CF Registry of Turkey (CFRT) in 2022. Patients were divided into three groups according to NBS results: Group 1 with positive NBS, Group 2 with negative NBS, and Group 3 with no screening or unknown screening results. All clinical and demographic data were compared between the three groups. RESULTS:A total of 853 patients were included in the study, 668 (78.3%) patients were in Group 1, 90 (10.5%) in Group 2, and 95 (11.2%) in Group 3. The age at diagnosis was 0.17 (0.08-0.33) years in Group 1, 0.50 (0.25-1.0) in Group 2, and 0.33 (0.17-0.75) in Group 3 (p<0.001). The first and second sweat test results and frequency of pancreatic insufficiency were lowest in Group 2 (p<0.05). Median FEV1 (%) was 88 (77-103) in Group 1, 90 (71.5-104) in Group 2, 89.5 (81.75-97.5) in Group 3 (p>0.05). 49% of the patients had a severe genotype and it was detected most frequently in Group 1 (p=0.021). CONCLUSIONS:Patients with pancreatic sufficiency may be missed by IRT/IRT NBS and lower and negative sweat test results may contribute to delays in CF diagnosis. Approximately 22% of patients are not diagnosed through this screening method.

BACKGROUND:Modulators have revolutionized cystic fibrosis (CF) management, but their effects on respiratory pathogens remain unclear. This study evaluated changes in lower respiratory tract pathogen detection after modulator therapy in children with CF, registered in the Cystic Fibrosis Registry of Turkey. METHOD:This retrospective, multicenter cohort study included children receiving modulator therapy between 2020 and 2022. Chronic respiratory tract colonization rates before and after therapy were compared, along with inhaler treatments, oral steroid, azithromycin use, pulmonary function tests, and hospitalizations for pulmonary exacerbations. The cohort was stratified by age, modulator type, and lung disease severity. Changes in microbiologic data over a 1-year period were also analyzed for children not receiving modulator therapy. RESULTS:A total of 101 children (mean age 9.95 ± 4.44 years) were included. Following modulator therapy, respiratory cultures of 57 (56.4%) were negative. Among 32 children with chronic Pseudomonas aeruginosa (P. aeruginosa) colonization, 14 (44%) showed negative respiratory cultures after receiving modulator therapy (p = 0.039). Conversion to culture-negative status was significant for methicillin-sensitive Staphylococcus aureus (MSSA) (p = 0.022) and methicillin-resistant Staphylococcus aureus (MRSA) (p = 0.034), with ETI therapy yielding the highest conversion rates. Inhaled antibiotic use for chronic respiratory pathogens decreased significantly (p = 0.039), and spirometry parameters improved (p < 0.001). Among 1232 children not receiving modulators, 180 (14.6%) had negative respiratory cultures when examined at 1-year interval. In the same group, intermittent/chronic P. aeruginosa colonization was negative in 58 cases, while 85 developed new growth, following modulator therapy, with positivity rates rising from 16.3% to 18.5% (p = 0.030). No significant changes in other pathogen detection were observed. CONCLUSION:Modulators, particularly ETI, reduced respiratory pathogen detection and improved lung function in children with CF.

Introduction: asthma is the most common chronic disease among children. The cases of asthma are increasing dramatically. Families play an important role in childhood asthma management. Children with chronic diseases and their mothers show stress and anxiety incidences, which are supported by publications. The main purpose of this study was to understand parental stress and associated coping behaviors of mothers of children with asthma. To the best of our knowledge, this is the first study conducted in this field. Materials and method: our study was conducted prospectively, and we included 75 asthmatic children and their mothers as study group. In the control group, 60 healthy children and their mothers were included. Data was collected by the researchers using the Clinical Interview form, the Beck Anxiety Scale and Coping with Stress Scale of Style. Results: a total of 135 patients (75 patients, 60 control groups) were included in the study. There was no significant difference between the groups in terms of the age of the participants and the age of the mothers. When compared in terms of asthma diagnosis during pregnancy, the mothers in the study group had a statistically significant higher history of asthma during pregnancy (p<0.05), but the mothers were similar in terms of smoking. When the mean anxiety scores of the mothers were compared with the duration of asthma of the child in the study group, it was observed that the mean anxiety scores of the mothers increased as the duration of asthma increased, but there was no statistically significant difference, whereas when the anxiety scores of the mothers were compared between the groups, it was observed that the level of severe anxiety was statistically significantly higher in the study group.In the mothers of children with asthma, optimistic attitudes and self-confident approach, compared to healthy children of mothers who had low scores and were found to be statistically significantly different. Conclusions: this study revealed that asthmatic children's mothers'stress and anxiety levels increased. It has been determined that during the follow-ups and treatments of children with asthma, giving support to families will help and improve both the patients' treatment process and the dialog between doctors and patients' relatives.

Introduction:Cystic fibrosis (CF) transmembrane conductance regulator protein (CFTR) modulators have significantly improved health outcomes in patients with cystic fibrosis (pwCF). However, in Turkey, access is limited due to lack of insurance coverage, and treatment is only granted in 3-month periods via court rulings. This study aimed to compare clinical outcomes between patients receiving continuous vs. intermittent modulator therapy. Methods:In this retrospective multicenter study, data from 229 CF patients across 14 centers in Turkey who received highly effective modulator therapy (HEMT) for at least six months were analyzed. Patients were grouped based on whether they received treatment continuously (Group 1) or with interruptions (Group 2). Changes in percent predicted forced expiratory volume in one second (ppFEV₁) and body mass index (BMI) were evaluated at baseline, 3 months, and 6 months. For Group 2, ppFEV₁ was also assessed during interruption periods. Results:Of the 229 patients, 38.4% received continuous treatment while 61.5% experienced treatment interruptions. Both groups showed significant improvements in ppFEV₁ over six months (p < 0.001). However, Group 2 experienced a significant decline during interruption periods (p < 0.001), followed by recovery upon reinitiation. BMI also increased significantly in both groups (p < 0.05). Patients with baseline ppFEV₁ < 70% showed greater improvement compared to those with milder disease. Conclusion:Short-term clinical outcomes in ppFEV₁ and BMI were similar between continuous and intermittent treatment. However, treatment interruptions may reduce cumulative benefits, potentially impacting long-term outcomes. Ensuring uninterrupted access to HEMT is essential, especially in low- and middle-income countries.