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Martina A. Steurer Jean Costello Rebecca J. Baer Scott P. Oltman Sky K. Feuer Tania Pacheco-Werner Elizabeth Rogers Marta M. Jankowska Jessica Block Molly McCarthy Matthew S. Pantell Christina Chambers Kelli K. Ryckman Laura L. Jelliffe-Pawlowski 《Paediatric and perinatal epidemiology》2020,34(2):130-138
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Béla Nagy Zsolt Bene Zsolt Fejes Sonya L. Heltshe David Reid Nicola J. Ronan Yvonne McCarthy Daniel Smith Attila Nagy Elizabeth Joseloff György Balla János Kappelmayer Milan Macek Scott C. Bell Barry J. Plant Margarida D. Amaral István Balogh 《Journal of cystic fibrosis》2019,18(2):271-277
Background
We have recently shown that human epididymis protein 4 (HE4) levels correlate with the severity of cystic fibrosis (CF) lung disease. However, there are no data on how HE4 levels alter in patients receiving CFTR modulating therapy.Methods
In this retrospective clinical study, 3 independent CF patient cohorts (US-American: 29, Australian: 12 and Irish: 19 cases) were enrolled carrying at least one Class III CFTR CF-causing mutation (p.Gly551Asp) and being treated with CFTR potentiator ivacaftor. Plasma HE4 was measured by immunoassay before treatment (baseline) and 1–6?months after commencement of ivacaftor, and were correlated with FEV1 (% predicted), sweat chloride, C-reactive protein (CRP) and body mass index (BMI).Results
After 1?month of therapy, HE4 levels were significantly lower than at baseline and remained decreased up to 6?months. A significant inverse correlation between absolute and delta values of HE4 and FEV1 (r?=??0.5376; P?<?.001 and r?=??0.3285; P?<?.001), was retrospectively observed in pooled groups, including an independent association of HE4 with FEV1 by multiple regression analysis (β?=??0.57, P?=?.019). Substantial area under the receiver operating characteristic curve (ROC-AUC) value was determined for HE4 when 7% mean change of FEV1 (0.722 [95% CI 0.581–0.863]; P?=?.029) were used as classifier, especially in the first 2?months of treatment (0.806 [95% CI 0.665–0.947]; P?<?.001).Conclusions
This study shows that plasma HE4 levels inversely correlate with lung function improvement in CF patients receiving ivacaftor. Overall, this potential biomarker may be of value for routine clinical and laboratory follow-up of CFTR modulating therapy. 相似文献34.
Gary E. Stapleton Benjamin W. Eidem Ricardo H. Pignatelli Karina M. Carlson Charles E. Mullins Ronald G. Grifka 《Congenital heart disease》2006,1(3):116-119
Background. A persistent patent ductus arteriosus (PDA) may delay closure of a coexisting atrial septal defect (ASD) due to volume loading and enlargement of the left atrium. The purpose of this study was to investigate the natural history of ASD size in patients with a PDA following transcatheter PDA occlusion. Methods. All patients with an ASD and a PDA who underwent transcatheter PDA occlusion at Texas Children’s Hospital were identified. Patients with ASD diameter <3 mm, or additional cardiac defects were excluded. Eight patients (7 females) with small‐ to moderate‐sized ASDs and a PDA were identified. Patient demographics, echocardiographic data, and cardiac catheterization data were recorded. Data were analyzed by 1‐tailed t‐test. Results. Following PDA occlusion, ASD diameter decreased in 6 of 8 patients by a mean of 3.8 mm (±2.3 mm), including 2 that closed. The median duration of follow‐up was 689 days. One ASD remained unchanged and 1 increased in size. The mean maximum ASD diameter decreased from 6.4 mm (±2.2 mm) to 3.9 mm (±3.4 mm) (P = .03). Two patients underwent subsequent transcatheter ASD occlusion. Conclusion. Following transcatheter PDA occlusion, small‐ to moderate‐sized ASDs have significant probability to decrease in size, and possibly close. In infants and children, we recommend transcatheter PDA occlusion, and serial follow‐up of the size of the ASD. This will allow many small‐ to moderate‐sized ASDs to either close, or become smaller, obviating the need for future intervention. 相似文献
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Functional recovery, the goal of treatment, has long been overlooked in the assessment of effectiveness of pharmacological treatments. However, with the recent shift in paradigm, from syndromal–symptomatic recovery to functional recovery, there appears to be a new interest in the definition and evaluation of functional recovery. Since functional recovery lags symptomatic recovery, sometimes by months or years, the attainment of functional recovery will be determined by both efficacy and long-term compliance. Quetiapine, due to its efficacy in both mania and depression, and effect on cognition may lead to improved functioning in patients with bipolar disorder. 相似文献
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Setting goals to maintain hope. 总被引:3,自引:0,他引:3