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INTRODUCTION: Cystic fibrosis (CF) is a disease caused by mutations in the gene located on chromosome 7 that encodes the CF transmembrane conductance regulator protein. Several trials have demonstrated the efficacy and safety of the ELE/TEZ/IVA combination in patients who have at least one F508del mutation. The main objective of the study was to evaluate the safety at 3 and 6 months of treatment with ELE/TEZ/IVA in adult patients with CF. METHODS: This is a real-life, prospective, single-center, cross-sectional study that included adult patients from the CF multidisciplinary unit. The demographic and clinical characteristics of all patients were recorded. During the time of the study, 3 visits were carried out (baseline, at 3 and at 6 months). Side effects were recorded during the follow-up time. RESULTS: 3 months after the start of treatment, a statistically significant improvement was observed. of lung function, BMI, pulmonary exacerbations and energy level, as well as in all the categories of the CFQ-R questionnaire except in the digestive domain. This improvement was maintained, but not increased at 6 months in all variables, except BMI, where differences were observed between 3 and 6 months of treatment. CONCLUSIONS: In the cohort studied, treatment with ELE/TEZ/IVA has a good safety profile. and produces an early improvement in lung function, BMI, quality of life and the "energy level" of adult patients with CF, which is maintained at 6 months of treatment.
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Fibrose Cística , Humanos , Adulto , Fibrose Cística/tratamento farmacológico , Fibrose Cística/genética , Estudos Transversais , Estudos Prospectivos , Qualidade de Vida , MutaçãoRESUMO
INTRODUCTION: Bronchiectasis is typically treated with inhaled antibiotics in clinical practice. However, there is a striking lack of standardised procedures for the preparation of noncommercial solutions. We used biochemical parameters to analyse the safety and tolerability of inhaled antibiotics in patients with bronchiectasis, and determined potential associations between the inhaled antibiotics used and adherence to the medications and quality of life. METHODS: We conducted a literature review, biochemical testing, and a pilot study of patients admitted to our hospital with noncystic fibrosis bronchiectasis. The MEDLINE database was searched for studies involving inhaled antibiotics to treat bronchiectasis. We analysed the pH, osmolality, and sodium and chloride ion concentrations of the antibiotics used. The pilot study included patients receiving inhaled antibiotic treatment. Demographic data, adherence, and quality of life were recorded and assessed. We determined potential associations between the study variables. RESULTS: The literature review identified 429 articles: 106 included precise instructions for diluting antibiotics, and 18 reported data on the biochemical parameters analysed. Laboratory results showed that some antibiotic dilutions were outside the range of tolerability, especially those involving dry powders for intravenous infusion, which must be diluted for their inhalation. Adherence was good in more than 80% of the patients, and higher in men and older patients. Men reported better quality of life. No associations were found between the antibiotics used and the other variables. CONCLUSION: Regarding the biochemical parameters analysed, there is a lack of information on the tolerability and biochemical safety of noncommercial dilutions of inhaled antibiotics used to treat bronchiectasis.
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Antibacterianos , Bronquiectasia , Administração por Inalação , Bronquiectasia/tratamento farmacológico , Humanos , Masculino , Projetos Piloto , Qualidade de VidaRESUMO
BACKGROUND: The survival of women with cystic fibrosis (CF) is lower than that of men by approximately 5 years. While various factors have been put forward to account for this discrepancy, no specific reasons have been established. Our hypothesis was that anatomical-structural involvement is more pronounced in women with CF than in men and that this is reflected in thoracic HRCT findings. MATERIAL AND METHODS: We performed a prospective multicentre study, in which adult patients were consecutively included over 18 months. Chest HRCT was performed, and findings were scored by 2 thoracic radiologists using the modified Bhalla system. We also studied respiratory function, applied the CFQR 14+ questionnaire, and collected clinical variables. RESULTS: Of the 360 patients followed up in the participating units, 160 were eventually included. Mean age was 28 years, and 47.5% were women. The mean±SD global score on the modified Bhalla score was 13.7±3.8 in women and 15.2±3.8 in men (p=0.024). The highest scores were observed for sacculations, bronchial generations, and air trapping in women. Women had lower BMI, %FEV1, %FVC, and %DLCO. Similarly, the results for the respiratory domain in CFQR 14+ were worse in women, who also had more annual exacerbations. CONCLUSIONS: This is the first study to provide evidence of the implication of sex differences in HRCT findings in patients with CF. Women with CF present a more severe form of the disease that results in more frequent exacerbations, poorer functional and nutritional outcomes, deterioration of quality of life, and greater structural damage.
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Fibrose Cística , Adulto , Fibrose Cística/diagnóstico por imagem , Feminino , Volume Expiratório Forçado , Humanos , Masculino , Estudos Prospectivos , Qualidade de Vida , Caracteres Sexuais , Tomografia Computadorizada por Raios XRESUMO
OBJECTIVES: The objective of this study was to analyze the predictive value of the modified Bhalla score in high-resolution computed tomography (HRCT) for assessment of pulmonary exacerbations (PEx) in cystic fibrosis (CF) patients. We also describe the relationship between this score and pulmonary function test results. METHODS: We performed a multicenter and prospective study where adult patients with CF were included consecutively over 18 months. All patients underwent HRCT with acquisition in inspiration and expiration. The results were analyzed by an expert radiologist who assigned a modified Bhalla score value. Lung function was also assessed, and clinical variables were collected. Follow-up lasted approximately 1 year, and PEx were registered. RESULTS: The study population comprised 160 subjects selected from 360 CF patients monitored in the participating CF units. The mean age was 28 years, 47.5% were women, and mean forced expiratory volume in 1 s (FEV1) was 67.5%. The mean global modified Bhalla score was 14.5 ± 0.31 points. Pulmonary function test (PFT) results and the modified Bhalla score correlated well, mainly forced vital capacity (FVC) and FEV1. We constructed a statistical model based on the overall Bhalla score to predict the number of PEx. CONCLUSIONS: The overall modified Bhalla score can predict future PEx in CF patients. This useful tool can help to prevent PEx in higher risk patients. KEY POINTS: ⢠Pulmonary function test results and the modified Bhalla score correlated well with FVC and FEV1. ⢠The total modified Bhalla score can predict the number of exacerbations in adult CF patients. ⢠Our findings highlight the need to establish a unified protocol for chest HRCT during the follow-up of adult patients with CF in order to anticipate possible complications and determine their impact on pulmonary function.
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Fibrose Cística , Adulto , Fibrose Cística/complicações , Fibrose Cística/diagnóstico por imagem , Feminino , Volume Expiratório Forçado , Humanos , Pulmão/diagnóstico por imagem , Masculino , Estudos Prospectivos , Capacidade VitalRESUMO
BACKGROUND: Health-related quality of life (QoL) is one of the most important end points in bronchiectasis (BE). However, the majority of health-related QoL questionnaires are time-consuming or not validated in BE. The COPD Assessment Test (CAT) is an easy-to-use questionnaire. The objective of this study was to perform a complete validation of the CAT in BE. METHODS: This was an observational, multicenter, prospective study in patients with BE. Psychometric properties of the CAT were measured: internal consistency (Cronbach α), repeatability (test-retest; intraclass correlation coefficient), discriminant validity (correlation with severity scores), convergent validity (correlation with some validated QoL questionnaire and other clinical variables of interest), longitudinal validity (measuring before and after each exacerbation during follow-up to determine the sensitivity to change and responsiveness), predictive validity to future exacerbations, and finally minimum clinically important difference. RESULTS: Ninety-six patients were included and followed up for 1 year. Their mean age was 62.2 (15.6) years (79.2% women). The CAT showed excellent internal consistency (α, 0.95) and repeatability (intraclass correlation coefficient, 0.95). The validity of the CAT was excellent in all the measures (almost all with a Pearson coefficient > 0.40) except for the correlations with severity scores (Pearson coefficient between 0.22 and 0.26). Sensitivity to change before and after exacerbations was set at between 5.4 and 5.8 points. A CAT value ≥ 10 points showed prognostic value for patients with more than one exacerbation, and finally the minimum clinically important difference was set at 3 points. CONCLUSIONS: The CAT presented excellent psychometric properties and is a questionnaire that is easy to use and interpret in patients with BE.
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Bronquiectasia/diagnóstico , Psicometria , Doença Pulmonar Obstrutiva Crônica/diagnóstico , Qualidade de Vida , Bronquiectasia/epidemiologia , Bronquiectasia/fisiopatologia , Bronquiectasia/psicologia , Feminino , Humanos , Masculino , Pessoa de Meia-Idade , Psicometria/métodos , Psicometria/normas , Doença Pulmonar Obstrutiva Crônica/fisiopatologia , Doença Pulmonar Obstrutiva Crônica/psicologia , Reprodutibilidade dos Testes , Índice de Gravidade de Doença , Espanha/epidemiologia , Inquéritos e Questionários , Avaliação de Sintomas/métodosRESUMO
The most common infections caused by nontuberculous mycobacteria (NTM) are lung infections. The microorganisms causing these infections most frequently are Mycobacterium avium complex, Mycobacterium kansasii and Mycobacterium abscessus complex. Their incidence has increased in the last three decades. After identifying an NTM in the respiratory tract, clinical and radiological aspects must be considered to determine if isolations are clinically relevant. Predisposing conditions that could contribute to infection must also be investigated. Pulmonary disease due to NTM is presented in three clinical forms: a) pneumonitis due to hypersensitivity; b) fibrocavitary form; and c) nodular-bronchiectasic. The diagnosis of respiratory disease due to NTM does not make it obligatory to immediately initiate treatment. Before initiating the latter, other factors must be considered, such as age, comorbidities, life expectancy, due to the prolonged nature of treatments, with potential side effects and, in many cases, only a slight response to the treatment.