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.
Background: Cystic fibrosis (CF) is an autosomal recessive disease caused by variants of CFTR gene. Over 2000 variants have been identified, and new drugs called CFTR modulators have been developed to target specific defects in the CFTR protein. However, these drugs are only suitable for patients with certain variants of CFTR , and eligibility rates vary depending on race and geographical region. This study aimed to reveal the detailed genotype and clinical characteristics of people with CF (pwCF) at our center in Turkey, a developing country, who are not eligible for CFTR modulators. Methods: A total of 445 pwCF followed up at Marmara University were reviewed retrospectively. Variants of the patients ineligible to CFTR modulators were classified based on American College of Medical Genetics guidelines, CFTR classification, the change in the encoded protein, and the variant type. Results: The study revealed that 139 (31.2%) patients weren't eligible for CFTR modulators. There were 60 different variants in the 276 alleles, as two were missing. The majority of patients had missense or nonsense variants, and that the most common variant was c.1545_1546del, which can be said unique to this geography. Conclusion: The study highlights the importance of detecting the variants of ineligible patients in detail to guide future approaches for more targeted and effective interventions in CF care. Testing the effectiveness of CFTR modulators for rare or newly occurring variants is crucial to ensure equal access for pwCF to these therapies from different racial backgrounds and ethnic minorities.
4 September 2024 in 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.