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BACKGROUND: Biomarkers of inflammation predictive of cystic fibrosis (CF) disease outcomes would increase the power of clinical trials and contribute to better personalization of clinical assessments. A representative patient cohort would improve searching for believable, generalizable, reproducible and accurate biomarkers. METHODS: We recruited patients from Mountain West CF Consortium (MWCFC) care centers for prospective observational study of sputum biomarkers of inflammation. After informed consent, centers enrolled randomly selected patients with CF who were clinically stable sputum producers, 12 years of age and older, without previous organ transplantation. RESULTS: From December 8, 2014 through January 16, 2016, we enrolled 114 patients (53 male) with CF with continuing data collection. Baseline characteristics included mean age 27 years (SD = 12), 80% predicted forced expiratory volume in 1 s (SD = 23%), 1.0 prior year pulmonary exacerbations (SD = 1.2), home elevation 328 m (SD = 112) above sea level. Compared with other patients in the US CF Foundation Patient Registry (CFFPR) in 2014, MWCFC patients had similar distribution of sex, age, lung function, weight and rates of exacerbations, diabetes, pancreatic insufficiency, CF-related arthropathy and airway infections including methicillin-sensitive or -resistant Staphylococcus aureus, Pseudomonas aeruginosa, Burkholderia cepacia complex, fungal and non-tuberculous Mycobacteria infections. They received CF-specific treatments at similar frequencies. CONCLUSIONS: Randomly-selected, sputum-producing patients within the MWCFC represent sputum-producing patients in the CFFPR. They have similar characteristics, lung function and frequencies of pulmonary exacerbations, microbial infections and use of CF-specific treatments. These findings will plausibly make future interpretations of quantitative measurements of inflammatory biomarkers generalizable to sputum-producing patients in the CFFPR.
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Fibrosis Quística/patología , Staphylococcus aureus Resistente a Meticilina/aislamiento & purificación , Selección de Paciente , Esputo/microbiología , Infecciones Estafilocócicas/patología , Adolescente , Adulto , Fibrosis Quística/microbiología , Fibrosis Quística/terapia , Femenino , Humanos , Pulmón/microbiología , Pulmón/patología , Pulmón/fisiopatología , Masculino , Staphylococcus aureus Resistente a Meticilina/fisiología , Persona de Mediana Edad , Estudios Prospectivos , Infecciones Estafilocócicas/microbiología , Infecciones Estafilocócicas/terapia , Adulto JovenRESUMEN
BACKGROUND: Infantile wheezing is a common problem, but there are no guidelines for the evaluation of infants with recurrent or persistent wheezing that is not relieved or prevented by standard therapies. METHODS: An American Thoracic Society-sanctioned guideline development committee selected clinical questions related to uncertainties or controversies in the diagnostic evaluation of wheezing infants. Members of the committee conducted pragmatic evidence syntheses, which followed the Grading of Recommendations, Assessment, Development, and Evaluation (GRADE) approach. The evidence syntheses were used to inform the formulation and grading of recommendations. RESULTS: The pragmatic evidence syntheses identified few studies that addressed the clinical questions. The studies that were identified constituted very low-quality evidence, consisting almost exclusively of case series with risk of selection bias, indirect patient populations, and imprecise estimates. The committee made conditional recommendations to perform bronchoscopic airway survey, bronchoalveolar lavage, esophageal pH monitoring, and a swallowing study. It also made conditional recommendations against empiric food avoidance, upper gastrointestinal radiography, and gastrointestinal scintigraphy. Finally, the committee recommended additional research about the roles of infant pulmonary function testing and food avoidance or dietary changes, based on allergy testing. CONCLUSIONS: Although infantile wheezing is common, there is a paucity of evidence to guide clinicians in selecting diagnostic tests for recurrent or persistent wheezing. Our committee made several conditional recommendations to guide clinicians; however, additional research that measures clinical outcomes is needed to improve our confidence in the effects of various diagnostic interventions and to allow advice to be provided with greater confidence.
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Ruidos Respiratorios/diagnóstico , Humanos , Lactante , Recién Nacido , Recurrencia , Pruebas de Función Respiratoria , Sociedades , Estados UnidosRESUMEN
Airway inflammation underlies cystic fibrosis (CF) pulmonary exacerbations. In a prospective multicenter study of randomly selected, clinically stable adolescents and adults, we assessed relationships between 24 inflammation-associated molecules and the future occurrence of CF pulmonary exacerbation using proportional hazards models. We explored relationships for potential confounding or mediation by clinical factors and assessed sensitivities to treatments including CF transmembrane regulator (CFTR) protein synthesis modulators. Results from 114 participants, including seven on ivacaftor or lumacaftor-ivacaftor, representative of the US CF population during the study period, identified 10 biomarkers associated with future exacerbations mediated by percent predicted forced expiratory volume in 1 s. The findings were not sensitive to anti-inflammatory, antibiotic, and CFTR modulator treatments. The analyses suggest that combination treatments addressing RAGE-axis inflammation, protease-mediated injury, and oxidative stress might prevent pulmonary exacerbations. Our work may apply to other airway inflammatory diseases such as bronchiectasis and the acute respiratory distress syndrome.
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BACKGROUND: Low health literacy has been identified as an independent predictor of poor asthma control. The Institute of Medicine considers the role of information technology (IT) as critical in providing "safe, effective, patient centered, timely, efficient, and equitable" care with the potential to reduce health disparities in underserved populations. The aim of this study was to design and evaluate an interactive computer-based questionnaire to assess asthma symptoms in children of parents with limited health literacy and/or limited English proficiency. METHODS: Volunteer caregivers attending a mobile asthma clinic were randomly assigned to complete the electronic or the paper-and-pencil version of an asthma screening questionnaire (ASQ) in their language of choice (English or Spanish). In the electronic version, a tablet computer was used to present the ASQ questions as video clips and to collect information through the touchscreen. Participants also completed a demographic questionnaire, a brief health literacy questionnaire, and a system usability and satisfaction questionnaire. Reliability of the paper and electronic self-assessments was evaluated by comparing each participant's answers to information they provided during a nurse-guided structured interview (gold standard). RESULTS: A total of 48 parents participated in the study, 26 completed the electronic ASQ and 21 the paper-and-pencil form. Thirty-five percent of the children had well-controlled asthma (n = 17). Most participants were Spanish speaking (67%) Hispanic (n = 44) mothers (n = 43) with a median age of 32 years. More than half had ≤8 years of education (n = 25) and earned <$20,000 per year (n = 27). The median health literacy score was 32 (range 0-36). The correlation between health literacy scores and years of education was significant (ρ = .47, p < .01). Concordance between the electronic ASQ and the nurse interview was significantly higher than concordance between the paper ASQ and the nurse interview (68% versus 54%; p < .01). All parents who completed the electronic questionnaire reported being satisfied; 96% felt comfortable using it, and found it simple to use. CONCLUSIONS: By facilitating the assessment of asthma symptoms at manageable cost, interactive information technology tools may help reduce barriers to access due to inadequate levels of English proficiency and health literacy.
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Asma/terapia , Alfabetización en Salud/métodos , Disparidades en Atención de Salud , Educación del Paciente como Asunto/métodos , Adulto , Asma/prevención & control , Preescolar , Femenino , Humanos , Entrevistas como Asunto , Persona de Mediana Edad , Padres , Proyectos Piloto , Análisis de Regresión , Factores Socioeconómicos , Encuestas y Cuestionarios , Adulto JovenRESUMEN
OBJECTIVE: Cystic fibrosis (CF)-related diabetes (CFRD) is associated with increased morbidity and mortality. Improved detection and management may improve outcomes; however, actual practice falls short of published guidelines. We studied efforts to improve CFRD screening and management in the Mountain West CF Consortium (MWCFC). RESEARCH DESIGN AND METHODS: This is a prospective observational cohort study evaluating quality improvement by accredited CF centers in Arizona, Colorado, New Mexico, and Utah performed between 2002 and 2008. After Institutional Review Board (IRB) approval, centers evaluated adherence with CF Foundation guidelines for CFRD. Each center developed and implemented quality improvement plans to improve both screening and management. Centers were reassessed 1â year later. RESULTS: Initially, each CF center had low adherence with screening recommendations (26.5% of eligible patients) that did not improve during the study. However, patients with confirmed CFRD markedly increased (141 (12% of MWCFC patients) to 224 (17%), p<0.001), and with improved adherence to management guidelines, patients with CFRD had increased weight (56.8-58.9â kg, p<0.001), body mass index (21.1-21.4, p=0.003), and weight-for-age z-score (-1.42 to -0.84, p<0.001). Quality improvement methods were specific to the practice settings of each center but shared the common goal of adhering to CFRD care guidelines. 1â year after implementation, no center significantly differed from any other in level of adherence to guidelines. CONCLUSIONS: Improving adherence with CFRD care guidelines requires substantial effort and may be incompletely successful, particularly for CFRD screening, but the effort may significantly improve patient monitoring and clinically relevant outcomes such as weight.