Şeyda Karabulut

Clinical Characteristics and Treatment Outcomes After First Achromobacter Isolation in Children with Cystic Fibrosis (Pre–CFTR Modulator Era) - Thoracic Research and Practice

Objective: This study aimed to evaluate 5‐year outcomes of a structured quality improvement (QI) program implemented as part of the standard of care at our CF center, focusing on changes in body mass index (BMI) and forced expiratory volume in 1 s (FEV₁) in pwCF followed at our institution. Methods: The medical records of pwCF registered at our CF center between January 1, 2018, and December 31, 2022, were retrospectively reviewed. Demographic and clinical data of pwCF submitted by our center to the European Cystic Fibrosis Society Patient Registry (ECFSPR) were used for this analysis. Structured QI strategies were initiated in 2018 after collaboration with the University of Michigan CF Center and focused on nutrition, respiratory care, and treatment adherence. Time‐dependent changes in FEV1 percent predicted (FEV1pp) were evaluated using a linear mixed‐effects regression model. Results: All 440 pwCF followed at our center during the study period were included in the analysis. Over 5 years, BMI z‐scores improved significantly in children ( p < 0.001), while adult BMI remained stable ( p = 0.10). No significant linear change in mean FEV₁pp was observed in adjusted longitudinal models ( p > 0.05), however, descriptive analyses showed an increase in median FEV₁pp among adults. Higher BMI was associated with better lung function ( p < 0.001) and lower IV antibiotic burden in correlation analyses (children: p < 0.001; adults: p = 0.045). The estimated mean survival age of the analysis cohort was 46.1 years. Conclusion: To our knowledge, this is the first report of a cohort of Turkish pwCF summarizing 5‐year longitudinal registry data with survival estimates. These findings suggest that sustained, center‐wide QI efforts may help maintain or improve clinical outcomes, particularly in settings with limited access to CFTR modulators.

Background: Cystic fibrosis (CF) is a life‐threatening disease that requires extensive knowledge for effective management. This study aimed to assess the knowledge levels of parents of children diagnosed with CF through newborn screening (NBS), evaluate their experiences during the diagnostic process, and determine the impact of education on their CF knowledge. Methods: This quasi‐experimental study involved 47 parents of children aged 0–30 months diagnosed with CF through NBS at four CF centers in Istanbul, Turkey. Parents completed a questionnaire assessing their CF knowledge before and after receiving face‐to‐face education, informational brochures, and an online webinar. The questionnaire covered general CF features, lung health, sexual function and infertility, and gastrointestinal issues. Results: The study revealed significant deficiencies in parental knowledge about CF and the NBS process. Only 25.5% of parents were informed about NBS prenatally, and 51.1% received information about CF when NBS results were positive. After educational intervention, correct response rates significantly increased for general characteristics ( p = 0.003), sexual health ( p < 0.001), lung health ( p = 0.007), and overall knowledge ( p < 0.001). Parents of children older than 12 months showed more pronounced improvement in knowledge across various sections compared to parents of younger children. Conclusion: The study highlights the need for a more robust educational framework to equip parents with comprehensive knowledge about CF. Improved communication strategies about NBS processes and repeated educational interventions are necessary to address knowledge gaps and enhance the quality of life for CF patients and their families.

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.

Objective: Sleep-disordered breathing (SDB) is a prevalent concern in individuals with neuromuscular diseases (NMD), significantly impacting respiratory function and sleep quality. This study aimed to retrospectively evaluate the demographic, clinical, and baseline polysomnographic data of children with NMD to investigate the guiding effect of the Pediatric Sleep Questionnaire (PSQ) and the modified Epworth Sleepiness Scale (ESS-CHAD) in detecting SDB. Method: A retrospective analysis was conducted on children aged 2-18 years with NMD who underwent polysomnography (PSG) between January 2012 and January 2024. The study assessed various clinical parameters, including age, gender, BMI, underlying disease, PSG results, the PSQ and ESS-CHAD scores, and treatment methods. Statistical analyses were conducted to compare those with and without obstructive sleep apnea syndrome (OSAS). Results: Of the 174 patients included in the study, 90 patients (51.7%) had normal PSG, 56 patients (32.2%) had mild (OSAS), 12 patients (6.9%) had moderate OSAS, and 16 patients (9.2%) had severe OSAS. PSQ and ESS-CHAD were not significantly different between patients with and without OSAS (p>0.05). The most common treatment initiated was noninvasive ventilation (NIV), recommended for 23% of patients. Conclusion: PSG is the gold standard for diagnosing SDB in children with NMD. While PSQ and ESS-CHAD may be useful for screening in the general pediatric population, they are inadequate in identifying OSAS in children with NMD. Early diagnosis and treatment of SDB are crucial for improving outcomes in this patient group.

Background: Recent interest in health and wellness highlights the impact of the social determinants of health (SDH). The WHO defines SDH as "the conditions in which people are born, grow, live, work, and age" and "the basic enabling factors of these conditions.” Our study focused on identifying the challenges patients with Cystic Fibrosis (pwCF) face after a severe earthquake beyond medical care. Methods: Using the 'Your Current Life Situation' survey, we assessed SDH among pwCFs in The CF Center in the earthquake zone, with parents responding for under the age of 18 years and adult pwCFs self-responding. Results: A total of 224 pwCF were included in our study from Diyarbakır, Şanlıurfa, Mersin, Gaziantep, Adana, Hatay and Malatya cities. 54% were male (n=120), mean age was 8.1 (SD ± 5.3) years. Of the patients, 92% (n=207) were aged <18 years. Median values of the BMI z-score (IQR) was -0.7(−1.7;0.3) Of the patients, 74% (n=166) had an income below the hunger threshold and 20% (n=44) had an income below the poverty threshold. When the SDH six domain was examined, it was observed that the highest level of insecurity was in the social domain (88%, n=98). This was followed by food insecurity (86%) and transportation insecurity (62%). Conclusion: The challenges and issues associated with CF family experiences living in the earthquake zone will be systematically identified and appropriate resources will be created to address them.

Aim: In our study, we aimed to determine the changes in diagnosis, treatment and follow-up of our patients who were followed in our Cystic Fibrosis (CF) Centre between 2018 and 2022. Method: The records of patients registered to our CF centre between January 1, 2018, and December 31, 2022 were retrospectively examined. Continuous variables are presented as mean (±SD) and median (IQR). Statistical analysis was performed using the general linear model with IBM Statistical Package for the Social Sciences (SPSS) Version 28. Results: We retrospectively reviewed the records of 484 patients registered at our centre. A total of 440 patient records were included in the study. The median age (IQR) at diagnosis was 4 (2-13) months. Yearly comparisons of median age, FEV₁% predicted values, and BMI z-score mean values from 2018 to 2022 revealed no statistically significant difference in FEV₁% predicted values (p=0.74). However, there was a statistically significant increase in mean BMI z-score (p1% pp values was observed only in the group using modulators.

Sleep apnea,central apnea,children