Preventive Inhalation of Hypertonic Saline in Infants with Cystic Fibrosis (PRESIS). A Randomized, Double-Blind, Controlled Study

Stahl, Mirjam; Wielpütz, Mark O.; Ricklefs, Isabell; Dopfer, Christian; Barth, Sandra; Schlegtendal, Anne; Graeber, Simon Y.; Sommerburg, Olaf; Diekmann, Gesa; Hüsing, Johannes; Koerner-Rettberg, Cordula; Naehrlich, Lutz; Dittrich, Anna‐Maria; Kopp, Matthias; Mall, Marcus

doi:10.1164/rccm.201807-1203oc

Cited by 107 publications

(93 citation statements)

References 61 publications

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“…Lung disease remains the leading cause of morbidity and mortality in patients with cystic fibrosis (CF) and starts in the first months of life. Therefore, early diagnosis and therapeutic intervention may be most beneficial [1][2][3][4][5][6]. To determine efficacy of new therapies that address early CF lung disease in multicentre studies, sensitive, quantitative noninvasive end-points are needed to detect early lung abnormalities and disease progression in preschool children with CF [7].…”

Section: Introductionmentioning

confidence: 99%

“…The sensitivity of the LCI to detect response to therapy with a spectrum of interventions including inhaled hypertonic saline, inhaled DNase, CF transmembrane conductance regulator (CFTR) modulators and antibiotic therapy for pulmonary exacerbation, has been demonstrated for several paediatric age groups from infancy to school-age, including multicentre studies with performance of MBW either in sedated infants and preschool children or in awake children 3 years and older at centres highly experienced in this technique in the corresponding age group [6,10,[13][14][15][16][17]. In addition, observational studies across two MBW-experienced sites of the Australian Respiratory Early Surveillance Team for Cystic Fibrosis (AREST CF) and an observational study across three MBW-experienced North American CF centres used the LCI as end-point in preschool children [11,18].…”

Section: Introductionmentioning

confidence: 99%

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

et al. 2020

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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.

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Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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

et al. 2020

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“…Die Hoffnung ist, dadurch bereits subklinische Schäden an den Organen zu verhindern und einem Fortschreiten der Erkrankung entgegen zu wirken. In einer randomisierten klinischen Studie wurden neu diagnostizierte Säuglinge mit hypertoner Kochsalzlösung (6 %) im Vergleich zu isotoner (0,9 %) Kochsalzlösung behandelt, was zu einem signifikant besseren Verlauf der Lungenfunktion (LCI) im ersten Lebensjahr führte . Die Lungenfunktion wurde in dieser Studie durch eine neue Methode gemessen, den ▸ Multiple Breath Washout (MBW) mit Bestimmung des ▸ Lung Clearance Index (LCI).…”

Section: Cftr‐modulation Von Der Geburt Bis Zum Lebensendeunclassified

Modulatoren der Kanalfunktion

Düesberg¹,

Bend

Smaczny³

2020

Biologie in unserer Zeit

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Zusammenfassung Diagnose und Therapie der Mukoviszidose sind in den letzten Jahren revolutioniert worden. Mit dem Neugeborenenscreening ist nun eine frühe Diagnose möglich, und die Behandlung mit CFTR‐Modulatoren bekämpft die Erbkrankheit am Basisdefekt. Bei einem frühen Einsatz könnten viele irreversible Schädigungen vermieden und Lebensqualität und Krankheitslast der Patienten deutlich reduziert werden. Laut dem Deutschen Mukoviszidose‐Register hat ein heute geborenes Kind mit Mukoviszidose eine durchschnittliche Lebenserwartung von 53 Jahren [2]. Schon heute gibt es mehr erwachsene Patienten als Kinder mit Mukoviszidose, die vor gar nicht allzu langer Zeit noch als reine Kinderkrankheit galt. Dieser Wandel führt aber auch zu Problemen in der Versorgung, denn die Strukturen im Gesundheitssystem halten nicht Schritt. Viele der erwachsenen Patienten müssen an Kinderkliniken behandelt werden, denn es gibt viel zu wenige Einrichtungen, die auf die Behandlung erwachsener Patienten spezialisiert sind. Hier gibt es bei aller Freude über den Fortschritt noch viele Herausforderungen. Auch der Dokumentation von Langzeitwirkungen der neuen Medikamente kommt eine große Bedeutung zu. Und schließlich dürfen die Patienten (ca. 10 %) nicht vergessen werden, die so seltene Mutationen des CFTR tragen, dass für sie derzeit noch keine der zugelassenen Therapien in Frage kommt. Hier könnte ein personalisierter Ansatz mittels Organoiden greifen, der mit dem HitCF‐Projekt bereits untersucht wird.

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“…Hypertonic saline (HS is widely used in CF, and studies are ongoing to optimize therapeutic recommendations. Feasibility, safety and efficacy of preventive 6% HS compared with 0.9% isotonic saline inhalation was evaluated over a 12‐month period in a double‐blind, controlled trial of 42 infants with CF who were less than 4 months of age at enrollment . Twice daily inhalation of 4 mL of both agents was well tolerated, with no difference in adverse events between the groups.…”

Section: Pulmonary Healthmentioning

confidence: 99%