INTRODUCTION: Elexacaftor/tezacaftor/ivacaftor (ETI) was used through the early access programme in Spain from December 2019 in cystic fibrosis (CF) patients with homozygous or heterozygous F508del mutation with advanced lung disease. METHODOLOGY: Multicentre, ambispective, observational, study in which 114 patients in follow-up in 16 national CF units were recruited. Clinical data, functional tests, nutritional parameters, quality of life questionnaires, microbiological isolates, number of exacerbations, antibiotic treatments and side effects were collected. The study also compared patients with homozygous and heterozygous F508del mutations. RESULTS: Of the 114 patients, 85 (74.6%) were heterozygous for F508del mutation, and the mean age was 32.2±9.96 years. After 30 months of treatment, lung function measured by FEV1% showed improvement from 37.5 to 48.6 (p<0.001), BMI increased from 20.5 to 22.3 (p<0.001), and all isolated microorganisms decreased significantly. The total number of exacerbations was also significantly reduced from 3.9 (±2.9) to 0.9 (±1.1) (p<0.001). All items in the CFQ-R questionnaire showed improvement, except for the digestive domain. Oxygen therapy use decreased by 40%, and only 20% of patients referred for lung transplantation remained on the active transplant list. ETI was well-tolerated, with only 4 patients discontinuing treatment due to hypertransaminemia. CONCLUSIONS: ETI decreases the number of exacerbations, increases lung function and nutritional parameters, decrease in all isolated microorganisms, for 30 months of treatment. There is an improvement in the CFQ-R questionnaire score except for the digestive item. It is a safe and well-tolerated drug.
Cystic Fibrosis , Adult , Humans , Young Adult , Aminophenols/therapeutic use , Aminophenols/adverse effects , Cystic Fibrosis/drug therapy , Cystic Fibrosis/genetics , Cystic Fibrosis Transmembrane Conductance Regulator/adverse effects , Cystic Fibrosis Transmembrane Conductance Regulator/genetics , Cystic Fibrosis Transmembrane Conductance Regulator/therapeutic use , Mutation , Quality of Life
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Humans , Young Adult , Adult , Middle Aged , Aged , Aged, 80 and over , Cystic Fibrosis/drug therapy , Cystic Fibrosis/diagnosis , Treatment Outcome , Spain , Quality of Life
Introduction: Occurrence of malignant pleural effusion (PE) is known to be associated with a poor prognosis, but the mortality of patients with non-malignant effusions has not been sufficiently studied. Our objective was to describe the clinical course and explore risk factors associated with all-cause mortality at 1, 5 and 10 years in patients who develop a PE. Methods: Retrospective observational study of patients undergoing diagnostic thoracentesis during the decade 2008-2017 in a pulmonology service. Demographic, biochemical, pathological and evolutionary variables were evaluated. The etiology of the effusions was determined using standardized criteria. Results: Pleural fluid samples from 358 patients with a mean age of 68.9 years (SD 15.1 years), 69.2% males, were analyzed. Malignant (29.4%), parapneumonic (19.8%) and secondary to heart failure (18.9%) effusions predominated. Patients with malignant and heart failure related PE had 1-year mortality rates of 60.0% and 30.8%, respectively, and 85% and 64.7% at 5 years. Male gender (hazard ratio [HR] 1.46; 95% CI: 1.03-2.07), positive cytology for malignancy (HR 1.66; 95% CI: 1.03-2.68) and effusion recurrence (HR 1.61; 95% CI: 1.17-2.21) were associated with a worse prognosis and 5-year mortality. Conclusions: Patients undergoing thoracentesis for effusion have a high short and long-term mortality. In our series of hospitalized patients with PE, the factors associated with higher mortality at 1 and 5 years were age, male sex, recurrence of PE, and coexistence of malignancy.
