Comparison of clinical features of cystic fibrosis patients eligible but not on CFTR modulators to ineligible for CFTR modulators.

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.

Ertapenem plus meropenem combination treatment in carbapenem-resistant Klebsiella pneumoniae bacteremia: an analysis of 53 cases.

Herein, we aimed to describe the outcomes of patients with blood stream infections due to carbapenem-resistant Klebsiella pneumoniae (CR-Kp) who received ertapenem plus meropenem combination treatment (EMCT). A total of 53 patients with culture proven CR-Kp bacteremia treated with ertapenem + meropenem were included. The patients with secondary bacteremia due to urinary tract infection exhibited a significantly lower 1-month mortality (OMM), particularly in those with microbiological eradication and those with end-of-treatment success. Salvage EMCT resulted in 49% 1-month survival.

Clinical characteristics of patients requiring lung transplantation referral in national cystic fibrosis registry data.

BACKGROUND: We aimed to determine the number of cystic fibrosis (CF) patients recorded in the Cystic Fibrosis Registry of Türkiye (CFRT) who were in need of lung transplantation (LT) referral and examine clinical differences between patients who were LT candidates due to rapid forced expiratory volume in one second (FEV1) decline and LT candidates without rapid FEV1 decline in the last year to identify a preventable cause in patients with such rapid FEV1 decline. METHODS: All CF patients recorded in the CFRT in 2018 were evaluated in terms of LT. Patients were divided into those with FEV1 below 50% and in need of LT due to a decrease of 20% or more in the previous year (Group 1) and those who did not have FEV1 decline of more than 20% in the previous year but had other indications for LT (Group 2). Demographic and clinical features were compared between the two groups. RESULTS: Of 1488 patients registered in CFRT, 58 had a need for LT. Twenty patients were included in Group 1 and others in Group 2. Our findings did not reveal any significant variations in treatment, chronic infection status, or complications between the two groups. The average weight z-score was significantly higher in Group 1. Positive correlations were detected between weight z-score and FEV1 in 2017 in Group 1 and between FEV1 values in 2017 and 2018 in Group 2. CONCLUSIONS: There appears to be a relationship between the nutritional status and weight z-scores of CF patients and pulmonary function, which may indirectly affect the need for lung transplantation referral.

Mortality predictors on the day of healthcare-associated Acinetobacter baumanni bacteremia in intensive care unit.

INTRODUCTION: Mortality of healthcare-associated Acinetobacter baumannii bacteremia can be 50-60% in intensive care units (ICUs). We aimed to determine the risk factors for 28-day mortality in patients with sepsis due to A. baumannii bacteremia during their ICU follow-up. METHODOLOGY: Demographic characteristics, disease severity scores on admission and bacteremia day (BD), resistance status, invasive interventions, and laboratory values showing the infection and severity of the BD, were compared between groups with and without mortality as a retrospective cohort study in the ICU of a tertiary hospital. RESULTS: Of a total of 2411 patients, there were 192 cases of bacteremia. After applying the exclusion criteria, 39 patients were recruited for the study, 25 of whom died (mortality rate 64.1%). Higher age, Simplified Acute Physiology Score II (SAPS II) on admission and high Sequential Organ Failure Assessment Score (SOFA), Red Blood Cell Distribution Width (RDW) (p < 0.001), and C-Reactive Protein (CRP) (p = 0.002) on the BD and invasive intervention in follow-up were associated with mortality. When CRP and RDW were both positive, sensitivity was 72%, specificity was 100%, negative predictive value was 33%, and positive predictive value was 100% for the 28-day mortality after BD. Based on multivariate analysis, CRP and RDW values on the BD were independent risk factors for mortality. CONCLUSIONS: It is critical to monitor SOFA, RDW, and CRP values in older ICU patients with SAPS II scores and who undergo invasive intervention in follow-up. Increases in these parameters may indicate bacteremia with high mortality due to A. baumannii.

Clinical Predictors of Response to Tocilizumab: A Retrospective Multicenter Study.

OBJECTIVE: A substantial number of patients with coronavirus disease-2019 (COVID-19) demonstrate severe infection. Cytokine storm is an underlying condition that worsens clinical outcomes. As an interleukin-6 receptor antagonist, tocilizumab is a promising treatment option for COVID-19. This study aimed to evaluate the clinical predictors of mortality for critically ill COVID-19 patients receiving tocilizumab therapy. MATERIAL AND METHODS: The retrospective cohort study was conducted in 4 centers' both wards and intensive care units between March 20 and May 20, 2020. Demographic, clinical, and laboratory data were consecutively drawn from medical records. The primary endpoint was in-hospital mortality. RESULTS: In this study, 39 patients (28.2% female) were included, and the mortality rate was 25.6% (n = 10). There was statistically significant difference between survivor and non-survivor groups regarding age (53.0 (46.5-65.0) vs. 75.0 (68.25-81.25), respectively,P = .001), CALL score (8.0 (7.0-10.0) vs. 12.0 (9.75-13.0), P = .001), GRAM score (119.5 (99.5-142.0) vs. 155.0 (129.8-226.0), P = .004), and white blood cell count (k/mL) (5.6 (3.8-8.6) vs. 8.0 (7.6-9.3), P = .003). The patients who were on invasive mechanical ventilation at the time of tocilizumab administration had a higher mortality rate (100% vs. 25.9%, P < .001). Besides, arterial partial pressure of oxygen/ fraction of inspiratory oxygen (PaO2/FiO2) ratio on day 7, but not on days 0, 1, and 3 of tocilizumab therapy, was associated with mortal- ity. C-reactive protein (mg/dL) tended to be lower in the survivor group; however, it was not statistically significant (68.4 (32.7-157.5) vs. 113.5 (77.7-219.0), P = .058). CONCLUSION: This study demonstrated that advanced age, increased leukocyte count, higher CALL and GRAM scores, and the need for invasive mechanical ventilation revealed a worse prognosis after tocilizumab treatment.