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    Comparison of clinical features of cystic fibrosis patients eligible but not on CFTR modulators to ineligible for CFTR modulators
    (Wiley, 2024) Buyuksahin, Halime Nayir; Emiralioglu, Nagehan; Yalcin, Ebru; Sen, Velat; Sen, Hadice Selimoglu; Arslan, Huseyin; Baskan, Azer Kilic
    Introduction: Cystic fibrosis transmembrane conductance regulator (CFTR) modulator drugs target the underlying defect and improve CFTR function. They are a part of standard care in many countries, but not all patients are eligible for these drugs due to age and genotype. Here, we aimed to determine the characteristics of non-eligible patients for CFTR modulators in the CF registry of Turkey (CFRT) to highlight their clinical needs. Methods: This retrospective cohort study included CF patient data from the CFRT in 2021. The decision of eligibility for the CFTR modulator was determined according to the 'Vertex treatment-Finder' on the Vertex (R) website. Demographic and clinical characteristics of patients were compared between eligible (group 1) and ineligible (group 2) groups for CFTR modulators. Results: Among the study population (N = 1527), 873 (57.2%) were in group 1 and 654 (42.8%) were in group 2. There was no statistical difference between groups regarding sex, meconium ileus history, diagnoses via newborn screening, FEV1 z-score, CF-associated complications, organ transplant history, and death. Patients in group 2 had a higher incidence of pancreatic insufficiency (87.7% vs. 83.2%, p = .010), lower median height z-scores (-0.87 vs. -0.55, p < .001), lower median body mass index z-scores (-0.65 vs. -0.50, p < .001), longer days receiving antibiotics due to pulmonary exacerbation (0 [interquartile range, IQR: 0-2] vs. 0 [IQR: 0-7], p = 0.001), and more non-invasive ventilation support (2.6% vs. 0.9%, p = 0.008) than patients in group 1. Conclusion: The ineligible group had worse clinical outcomes than the eligible group. This highlights their need for life-changing drugs to improve clinical outcomes.
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    Flexible bronchoscopy findings in lung amoebiasis: a case report
    (Drunpp-Sarajevo, 2012) Kelekci, Selvi; Sen, Velat; Tuncel, Tuba; Sen, Hadice Selimoglu; Cicek, Muttalip; Erge, Duygu; Gurkan, M. Fuat
    The protozoon Entamoeba histolytica is an agent of human amoebiasis. Amoebiasis is common around the world, especially in tropical and subtropical regions. About 90% of infections are asymptomatic, while the remaining 10% are characterized by dysentery and abscesses observable in the liver or other organs. The liver is the organ most commonly affected by extraintestinal amoebiasis. Pulmonary and invasive amoebiasis is seen in 2-3% of patients, but isolated pulmonary amoebiasis is rarely seen in the pediatric age group. In this study, a 14 year old male patient diagnosed with isolated pulmonary amoebiasis is presented. Diagnosis was based on the detection of trophozoites through direct examination of bronchoalveolar lavage fluid obtained by flexible bronchoscopy, and the presence of amoebic IgG in the blood. Three weeks of metronidazole combined with antimicrobial treatment significantly improved the patient's clinical and radiologic findings. The aim of this report was to present a rare case of childhood pulmonary amoebiasis without liver or other organ involvement and to demonstrate the efficacy of flexible bronchoscopy for diagnosis.