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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® 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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OBJECTIVE: It is known that tuberculosis is frequently seen among refugees. Hatay province is one of the cities that substantially expose to migration of refugees after Syrian civil war. In this study, it was aimed to compare frequency of new pulmonary tuberculosis (PTB) cases and treatment success/cure rates between Turkish and Syrian patients. FINDINGS: The study included 211 patients with PTB (178 Turkish and 33 Syrian patients) registered to Hatay Tuberculosis Outpatient Clinic between 2010 and 2013. On the basis of years, number of PTB patients registered was 53 (Turkish/Syrian: 52/1) in 2010, 44 (44/0) in 2011, 41 (39/2) in 2012, and 73 (43/30) in 2013. There were no significant differences between Turkish and Syrian patients regarding age groups, gender, marital status, contact history, smear result, and drug sensitivity assays when treatment success was considered (p>0.05). Directly observed therapy (DOT) rate was higher in patients who achieved successful treatment (97.6% vs. 2.4%; p<0.001). Number of patients successfully treated was smaller among Syrian patients (63.6% vs. 88.8%; p<0.001). Leaving the treatment and/or transfer rates were higher among Syrian patients (30.3% vs. 3.9%; p<0.001). During the study period, drug-resistant tuberculosis was detected in one Syrian and 3 Turkish patients. CONCLUSIONS: Although PTB frequency has increased in Hatay province within prior 4 years, treatment success among local population is still within limits established by World Health Organization (WHO). However, the treatment goal could not be achieved when considered together with refugees. To improve treatment success in refugees, implementation of a new national tuberculosis is needed control program in this population.