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1.
IntroductionIdiopathic pulmonary fibrosis (IPF) is progressive and irreversible. Some discrepancies about IPF staging exists, especially in mild phases. Forced vital capacity (FVC) higher than 80% has been considered early or mild IPF even for the design of clinical trials.MethodsSpanish multicentre, observational, retrospective study of IPF patients diagnosed between 2012 and 2016, based on the ATS/ERS criteria, which presented FVC greater or equal 80% at diagnosis. Clinical and demographic characteristics, lung function, radiological pattern, treatment, and follow-up were analyzed.Results225 IPF patients were included, 72.9% were men. The mean age was 69.5 years. The predominant high-resolution computed tomography (HRCT) pattern was consistent usual interstitial pneumonia (UIP) (51.6%). 84.7% of patients presented respiratory symptoms (exertional dyspnea and/or cough) and 33.33% showed oxygen desaturation below 90% in the 6 min walking test (6MWT). Anti-fibrotic treatment was initiated at diagnosis in 55.11% of patients. Median FVC was 89.6% (IQR 17) and 58.7% of patients had a decrease of diffusion lung capacity for carbon monoxide (DLCO) below 60% of theoretical value; most of them presented functional progression (61.4%) and higher mortality at 3 years (20.45%). A statistically significant correlation with the 3-years mortality was observed between DLCO <60% and consistent UIP radiological pattern.ConclusionsPatients with preserved FVC but presenting UIP radiological pattern and moderate–severe DLCO decrease at diagnosis associate an increased risk of progression, death or lung transplantation. Therefore, in these cases, preserved FVC would not be representative of early or mild IPF.  相似文献   
2.
目的评估肝脏瞬时弹性成像、天冬氨酸转氨酶与血小板比值指数(APRI)及基于4因子的肝纤维化指数(FIB-4)对儿童非酒精性脂肪性肝病(NAFLD)肝纤维化的诊断价值。方法选取湖南省儿童医院2015年8月至2020年10月已行肝穿刺病理活检的非酒精性脂肪性肝病100例进行回顾性研究,收集肝脏病理组织和临床资料。采用受试者操作特征曲线(ROC曲线)分析肝脏硬度(LSM)值、APRI及FIB-4诊断儿童NAFLD所致不同肝脏纤维化的诊断价值。结果LSM值、APRI、FIB-4诊断肝纤维化(S≥1)的ROC曲线下面积(AUC)分别为0.701[95%可信区间(CI):0.579~0.822,P=0.011]、0.606(95%CI:0.436~0.775,P=0.182)、0.568(95%CI:0.397~0.740,P=0.387),最佳临界值分别为6.65 kPa、21.20、0.18;LSM值、APRI、FIB-4诊断显著肝纤维化(S≥2)的AUC分别为0.660(95%CI:0.552~0.768,P=0.006)、0.578(95%CI:0.464~0.691,P=0.182)、0.541(95%CI:0.427~0.655,P=0.482),最佳临界值分别为7.35 kPa、24.78、0.22;LSM值、APRI、FIB-4诊断进展期肝纤维化(S≥3)的AUC分别为0.639(95%CI:0.446~0.832,P=0.134)、0.613(95%CI:0.447~0.779,P=0.223)、0.587(95%CI:0.411~0.764,P=0.346),最佳临界值分别为8.55 kPa、26.66、0.27。结论瞬时弹性成像技术对儿童NAFLD肝纤维化有较好的诊断价值,优于APRI和FIB-4。  相似文献   
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4.
This paper is the first in a series providing updated guidance on the definition, evaluation and management of people with a Cystic Fibrosis Transmembrane conductance Regulator (CFTR)-Related Disorder (CFTR-RD). The need for this update relates to more precise characterisation of CFTR gene variants and improved assessment of CFTR protein dysfunction. The exercise is co-ordinated by the European CF Society Standards of Care Committee and Diagnostic Network Working Group and involves stakeholder engagement. This first paper was produced by a core group using an extensive literature review and papers graded for their quality. Subsequent wider stakeholder agreement was achieved.The definition of a CFTR-RD remains “a clinical condition with evidence of CFTR protein dysfunction that does not fulfil the diagnostic criteria for CF”. Clearer guidance on CFTR dysfunction and relevant CFTR variants will be provided. Thresholds for clinical presentations are presented and the paradigm that pathobiological processes may be evident in more than one organ is agreed. In this paper we reflect on the early patient journey, highlighting that CF specialists as well as other relevant specialists should be involved in the care of people with a CFTR-RD.  相似文献   
5.
Hepatitis C virus(HCV)is a significant cause of hepatocellular carcinoma(HCC).The direct-acting antivirals marked a new era of HCV therapy and are associated with greater than 95%cure rate.Successful treatment of chronic hepatitis C greatly reduces the risk of HCC.A proportion of patients,especially those with pre-existing cirrhosis,remain at risk for HCC despite sustained virologic response(SVR).Diabetes mellitus,hepatic steatosis,alcohol consumption and lack of fibrosis regression are associated with risks of HCC after HCV cure.Noninvasive modalities such as aspartate aminotransferase to platelet ratio index and fibrosis-4 index and transient elastography have been used to monitor hepatic fibrosis.More recently,various fibrosis scores have been combined with clinical parameters and other novel biomarkers to predict risks of HCC for patients who achieved SVR.These models still need to be validated and standardized prior to applying to routine clinical care.  相似文献   
6.
<正>纤维化是一种组织受到破坏后的修复反应,主要由于炎症因子过度刺激引发实质细胞坏死、细胞外基质(extracellular matrix,ECM)分泌过剩,大量沉积于细胞间质,形成胶原蛋白排列紊乱的病理表现,严重者导致器官结构紊乱和功能障碍,甚至发生器官衰竭[1]。据报道,纤维化是一种不可逆的病理过程[2],目前临床上对纤维化疾病以干预和对症治疗为主[3],尚无特定的治疗药物。肿瘤坏死因子α(tumor necrosis factor-α,TNF-α)和白细胞介素1β(interleukin-1β,IL-1β)[4-5]已被确定为多种纤维化疾病治疗的关键靶点。那么,阻断炎症因子的信号途径是否可控制纤维化的发生、发展?G蛋白偶联受体30(G-protein-coupled receptor 30,GPR30)是一种7次跨膜雌激素受体蛋白,  相似文献   
7.
8.

Introduction

New massive sequencing techniques make it possible to determine the composition of airway microbiota in patients with cystic fibrosis (CF). However, the relationship between the composition of lung microbiome and the clinical status of paediatric patients is still not fully understood.

Material and methods

A cross-sectional observational study was conducted on induced sputum samples from children with CF and known mutation in the CFTR gene. The bacterial sequences of the 16SrRNA gene were analyzed and their association with various clinical variables studied.

Results

Analysis of the 13 samples obtained showed a core microbiome made up of Staphylococcus spp., Streptococcus spp., Rothia spp., Gemella spp. and Granulicatella spp., with a small number of Pseudomonas spp. The cluster of patients with less biodiversity were found to exhibit a greater number of sequences of Staphylococcus spp., mainly Staphylococcus aureus (p 0.009) and a greater degree of lung damage.

Conclusion

An airway microbiome with greater biodiversity may be an indicator of less pronounced disease progression, in which case new therapeutic interventions that prevent reduction in non-pathogenic species of the airway microbiota should be studied.  相似文献   
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10.
BackgroundPulmonary exacerbations (PEx) in cystic fibrosis (CF) patients reduce quality of life. Lung function, measured by the percent predicted forced expiratory volume in 1 s (ppFEV1), is widely used to evaluate PEx treatments. We analyzed the correspondence of ppFEV1 with 8 patient-reported symptom-based questions from the Cystic Fibrosis Respiratory Symptom Diary–Chronic Respiratory Infection Symptom Score (CFRSD-CRISS).MethodsData were derived from the observational Standardized Treatment of Pulmonary Exacerbations (STOP) study. CF patients who had CFRSD-CRISS and ppFEV1 measurements on ≥2 timepoints were included: 1) day of initial PEx, 2) 7 days later, and/or 3) end of PEx. We calculated age-stratified Spearman correlation coefficients and 95% confidence intervals (95% CIs) between the change in ppFEV1 and change in CFRSD-CRISS items from index to day 7 and from index to the end of PEx treatment.ResultsLung function and symptom scores improved by the end of treatment; however, correlations between ppFEV1 and the specific CFRSD-CRISS measures were mostly weak to moderate. An exception was that among patients <18, we observed moderately strong correlations between changes in ppFEV1 and cough severity (r = ?0.58 (95% CI: ?0.80, ?0.21)), mucus quantity (r = ?0.51 (?0.77, ?0.11)), and wheezing (r = ?0.53 (?0.78, ?0.14)) from index until end of treatment.ConclusionsAs novel treatments are developed for PEx, it is important to ensure that improvement is measured meaningfully. The generally weak associations between patient-reported symptoms and ppFEV1 that we found suggest that these measures capture different aspects of the disease and both metrics are important when evaluating new treatments.  相似文献   
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