A four week trial of hypertonic saline in children with mild cystic fibrosis lung disease: Effect on mucociliary clearance and clinical outcomes
| Autor: | T. Danielle Samulski, Jihong Wu, William D. Bennett, Agathe Ceppe, Scott H. Donaldson, Stephanie D. Davis, Aaron T. Trimble, Kirby L. Zeman, Caroline LaFave |
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| Rok vydání: | 2020 |
| Předmět: |
Male
0301 basic medicine Pulmonary and Respiratory Medicine Spirometry medicine.medical_specialty Cystic Fibrosis Mucociliary clearance Population Placebo Cystic fibrosis Gastroenterology Article 03 medical and health sciences 0302 clinical medicine Double-Blind Method Statistical significance Internal medicine Administration Inhalation medicine Humans Child education Saline Solution Hypertonic education.field_of_study medicine.diagnostic_test business.industry Nebulizers and Vaporizers medicine.disease Hypertonic saline Clinical trial 030104 developmental biology 030228 respiratory system Mucociliary Clearance Pediatrics Perinatology and Child Health Female business |
| Zdroj: | J Cyst Fibros |
| ISSN: | 1569-1993 |
| DOI: | 10.1016/j.jcf.2020.07.009 |
| Popis: | Background Hypertonic saline (HS) is commonly prescribed for children with cystic fibrosis (CF) despite the absence of strong data indicating clinical efficacy in a population with mild lung disease. We hypothesized that HS treatment would result in a sustained improvement in mucociliary clearance (MCC) in children with CF who had minimal lung disease, thus providing evidence for a biologically relevant effect that also may be associated with clinical improvements. Methods We performed a randomized, placebo controlled, double blind study of 6% versus 0.12% sodium chloride, delivered three-times daily with an eFlow nebulizer for 4 weeks. MCC was measured using gamma scintigraphy at baseline, 2-hours after the first study treatment, and ~12-hours after the final dose (at day 28). Spirometry, respiratory symptoms (CFQ-R), and safety were also assessed. Results Study treatments were generally well tolerated and safe. HS (6% sodium chloride) resulted in a significant, sustained improvement from baseline in whole lung clearance after 4 weeks of therapy (p = 0.014), despite absence of a prolonged single-dose effect after the initial dose. This sustained change (12 hrs after prior dose) was significantly greater when compared to placebo (0.12% sodium chloride) treatment (p = 0.016). Improvements in spirometry with HS did not reach statistical significance but correlated with MCC changes. Conclusions The observed sustained improvement in MCC with HS suggests that this treatment may yield health benefits, even in relatively mildly affected children with CF. Highlighting this physiologic finding is important due to the lack of meaningful, validated endpoints in this population. |
| Databáze: | OpenAIRE |
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