scholarly journals Multicentre feasibility of multiple-breath washout in preschool children with cystic fibrosis and other lung diseases

2020 ◽  
Vol 6 (4) ◽  
pp. 00408-2020
Author(s):  
Mirjam Stahl ◽  
Cornelia Joachim ◽  
Ines Kirsch ◽  
Tatjana Uselmann ◽  
Yin Yu ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Preschool Children ◽  
Lung Diseases ◽  
End Point ◽  
Multiple Breath Washout ◽  
Training Quality ◽  
Central Coordination ◽  
The Mean ◽  
Study Sites ◽  
Central Data

BackgroundMultiple-breath washout (MBW)-derived lung clearance index (LCI) detects early cystic fibrosis (CF) lung disease. LCI was used as an end-point in single- and multicentre settings at highly experienced MBW centres in preschool children. However, multicentre feasibility of MBW in children aged 2–6 years, including centres naïve to this technique, has not been determined systematically.MethodsFollowing central training, 91 standardised nitrogen MBW investigations were performed in 74 awake preschool children (15 controls, 46 with CF, and 13 with other lung diseases), mean age 4.6±0.9 years at investigation, using a commercially available device across five centres in Germany (three experienced, two naïve to the performance in awake preschool children) with central data analysis. Each MBW investigation consisted of several measurements.ResultsOverall success rate of MBW investigations was 82.4% ranging from 70.6% to 94.1% across study sites. The number of measurements per investigation was significantly different between sites ranging from 3.7 to 6.2 (p<0.01), while the mean number of successful measurements per investigation was comparable with 2.1 (range, 1.9 to 2.5; p=0.46). In children with CF, the LCI was increased (median 8.2, range, 6.7–15.5) compared to controls (median 7.3, range 6.5–8.3; p<0.01), and comparable to children with other lung diseases (median 7.9, range, 6.6–13.9; p=0.95).ConclusionThis study demonstrates that multicentre MBW in awake preschool children is feasible, even in centres previously naïve, with central coordination to assure standardised training, quality control and supervision. Our results support the use of LCI as multicentre end-point in clinical trials in awake preschoolers with CF.

Multicenter feasibility of lung clearance index in preschool children with cystic fibrosis and other lung diseases

2020 ◽  
Author(s):  
Mirjam Stahl ◽  
Cornelia Joachim ◽  
Tatjana Uselmann ◽  
Yin Yu ◽  
Ines Kirsch ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Preschool Children ◽  
Lung Diseases ◽  
Lung Clearance ◽  
Lung Clearance Index

The utility of moment ratios and abbreviated endpoints of the multiple breath washout test in preschool children with cystic fibrosis

2020 ◽  
Vol 55 (3) ◽  
pp. 649-653 ◽  
Author(s):  
Michelle Shaw ◽  
Martinus C. Oppelaar ◽  
Renee Jensen ◽  
Sanja Stanojevic ◽  
Stephanie D. Davis ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Preschool Children ◽  
Multiple Breath Washout

scholarly journals Three-center feasibility of lung clearance index in infants and preschool children with cystic fibrosis and other lung diseases

2018 ◽  
Vol 17 (2) ◽  
pp. 249-255 ◽  
Author(s):  
Mirjam Stahl ◽  
Simon Y. Graeber ◽  
Cornelia Joachim ◽  
Sandra Barth ◽  
Isabell Ricklefs ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Preschool Children ◽  
Lung Diseases ◽  
Lung Clearance ◽  
Lung Clearance Index

scholarly journals Inter-test reproducibility of the lung clearance index measured by multiple breath washout

2017 ◽  
Vol 50 (4) ◽  
pp. 1700433 ◽  
Author(s):  
Esther Oude Engberink ◽  
Felix Ratjen ◽  
Stephanie D. Davis ◽  
George Retsch-Bogart ◽  
Reshma Amin ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Preschool Children ◽  
Biological Variability ◽  
Meaningful Change ◽  
Limits Of Agreement ◽  
Intra Class Correlation ◽  
Lung Clearance ◽  
Lung Clearance Index ◽  
Multiple Breath Washout ◽  
Intra Class Correlation Coefficient

The lung clearance index (LCI) has strong intra-test repeatability; however, the inter-test reproducibility of the LCI is poorly defined.The aim of the present study was to define a physiologically meaningful change in LCI in preschool children, which discriminates changes associated with disease progression from biological variability.Repeated LCI measurements from a longitudinal cohort study of children with cystic fibrosis and age-matched controls were collected to define the inter-visit reproducibility of the LCI. Absolute change, the coefficient of variation, Bland–Altman limits of agreement, the coefficient of repeatability, intra-class correlation coefficient, and percentage changes were calculated.LCI measurements (n=505) from 71 healthy and 77 cystic fibrosis participants (aged 2.6–6 years) were analysed. LCI variability was proportional to its magnitude, such that reproducibility defined by absolute changes is biased. A physiologically relevant change for quarterly LCI measurements in health was defined as exceeding ±15%. In clinically stable cystic fibrosis participants, the threshold was higher (±25%); however, for measurements made 24 h apart, the threshold was similar to that observed in health (±17%).A percentage change in LCI greater than ±15% in preschool children can be considered physiologically relevant and greater than the biological variability of the test.

scholarly journals Relevance of Nasal Potential Difference in Diagnosis of Cystic Fibrosis Among Children

Medicina ◽  
2013 ◽  
Vol 49 (4) ◽  
pp. 29
Author(s):  
Arūnas Valiulis ◽  
Iveta Skurvydienė ◽  
Valdonė Misevičienė ◽  
Jūratė Kasnauskienė ◽  
Laimutė Vaidelienė ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Lung Diseases ◽  
Clinical Symptoms ◽  
Potential Difference ◽  
Sweat Test ◽  
Healthy Children ◽  
Test Results ◽  
Obstructive Lung Diseases ◽  
The Mean ◽  
Nasal Potential Difference

Objective. The aim of this study was to estimate the significance of nasal potential difference (NPD) in the diagnosis of cystic fibrosis (CF) in children with clinical symptoms suggestive of the disease, positive sweat test results, and/or genetically confirmed diagnosis. Material and Methods. NPD measurements according to the modifications by Alton were performed in 50 children with clinical CF symptoms supported by positive sweat test results, 50 children with other obstructive lung diseases, and 50 healthy children. A subgroup of 17 children with the diagnosis confirmed by 2 identified mutations in the CF transmembrane regulatory gene was analyzed individually. Results. The mean NPD value recorded in 50 children with clinical symptoms of CF supported by positive sweat test results and/or genetic analysis was –28.0 mV [SD, 10.2]. The mean NPD value in the subgroup of children with 2 identified mutations in the CF gene (n=17) was more negative than in the subgroup of children with unrecognized mutations (n=33) (–37.1 mV [SD, 7.0] vs. –23.4 mV [SD, 8.3], P<0.001). The mean NPD value in patients with other obstructive lung diseases and healthy children was significantly more positive than in the group of CF children with positive sweat test results and/or identified mutations (–18.1 mV [SD, 3.6] and –15.5 mV [SD, 4.3] vs. –28.0 mV [SD, 10.2], P<0.001). The NPD cut point value for the genetically confirmed diagnosis of CF was –35.0 mV (sensitivity, 93.9%; specificity, 88.2%), while in general, the NPD prognostic value was –24.0 mV (sensitivity, 58.0%; specificity, 98.0%) Conclusions. The NPD measurement is a valuable tool for the diagnosis of CF in children, but further studies are necessary to establish NPD values related to the CF genotype and to reduce the intrasubject variability of this test.

Multiple-Breath Washout as a Marker of Lung Disease in Preschool Children with Cystic Fibrosis

2005 ◽  
Vol 171 (3) ◽  
pp. 249-256 ◽  
Author(s):  
Paul Aurora ◽  
Andrew Bush ◽  
Per Gustafsson ◽  
Cara Oliver ◽  
Colin Wallis ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Preschool Children ◽  
Lung Disease ◽  
Multiple Breath Washout

Heart rate as a marker of relapse during withdrawal of heart failure therapy in patients with recovered dilated cardiomyopathy: an analysis from TRED-HF

2020 ◽  
Vol 41 (Supplement_2) ◽  
Author(s):  
B Halliday ◽  
A Vazir ◽  
R Owen ◽  
J Gregson ◽  
R Wassall ◽  
...  
Keyword(s):  
Heart Rate ◽  
Cardiac Function ◽  
Dilated Cardiomyopathy ◽  
Funding Source ◽  
End Point ◽  
Therapy Withdrawal ◽  
The Mean ◽  
Withdrawal Of Therapy ◽  
Continue Therapy

Abstract Introduction In TRED-HF, 40% of patients with recovered dilated cardiomyopathy (DCM) relapsed in the short-term during phased withdrawal of drug therapy. Non-invasive markers of relapse may be used to monitor patients who wish a trial of therapy withdrawal and provide insights into the pathophysiological drivers of relapse. Purpose To investigate the relationship between changes in heart rate (HR) and relapse amongst patients with recovered DCM undergoing therapy withdrawal in TRED-HF. Methods Patients with recovered DCM were randomised to phased withdrawal of therapy or to continue therapy for 6 months. After 6 months of continued therapy, those in the control arm underwent withdrawal of therapy in a single arm crossover phase. HR was measured at each study visit. Mean HR and 95% confidence intervals (CI) were calculated at baseline, 45 days after baseline, 45 days prior to the end of the study or relapse and at the end of the study or relapse. Patients were stratified by treatment arm and the occurrence of the primary relapse end-point. Heart rate at follow-up was compared amongst patients who had therapy withdrawn and relapsed versus those who had therapy withdrawn and did not. ANCOVA was used to adjust for differences in HR at baseline between the two groups. Results Of 51 patients randomised, 26 were assigned to continue therapy and 25 to withdraw therapy. In the randomised and cross-over phases, 20 patients met the primary relapse end-point; one patient withdrew from the study and one patient completed follow-up in the control arm but did not enter the cross-over phase. Mean HR (standard deviation) at baseline and follow-up for (i) patients in the control arm was 69.9 (9.8) & 65.9 (9.1) respectively; (ii) for those who had therapy withdrawn and did not relapse was 64.6 (10.7) & 74.7 (10.4) respectively; and (iii) for those who had therapy withdrawn and relapsed was 68.3 (11.3) & 86.1 (11.8) respectively [all beats per minute]. The mean change in HR between the penultimate visit and the final visit for those who had therapy withdrawn and did not relapse was −2.4 (9.7) compared to 3.1 (15.5) for those who relapsed. After adjusting for differences in HR at baseline, the mean difference in HR measured at follow-up between patients who underwent therapy withdrawal and did, and did not relapse was 10.4bpm (95% CI 4.0–16.8; p=0.002) (Figure 1 & Table 1). Conclusion(s) A larger increase in HR may be a simple and effective marker of relapse for patients with recovered DCM who have insisted on a trial of therapy withdrawal. Whether HR control is crucial to the maintenance of remission amongst patients with improved cardiac function, or is simply a marker of deteriorating cardiac function, warrants further investigation. Funding Acknowledgement Type of funding source: Public grant(s) – National budget only. Main funding source(s): British Heart Foundation

Sign in / Sign up

Export Citation Format

Share Document