Abstract
Background: Inhaled hypertonic saline (HS) is an attractive agent for chronic maintenance therapy in infants and toddlers with cystic fibrosis (CF) because it improves defective mucociliary clearance. Prior to undertaking a clinical trial of HS efficacy in young children with CF, tolerability, adherence, and safety must be established. Methods: Three-center, open label evaluation of the short-term tolerability, adherence, and safety of 7% HS administered twice daily for 14 days in children with CF 12-30 months of age. The primary objective was to evaluate the proportion of participants unable to tolerate single and repeated doses of 7% HS according to protocol-defined criteria. Participants inhaled a test dose of HS at the enrollment visit; test dose intolerance was defined as fulfillment of at least one of 4 criteria. Participants who tolerated the test dose inhaled 7% HS twice daily for 14 ± 2 days. Results: Twenty children were enrolled. One was withdrawn due to maternal concern over fussiness with application of the facemask for the test dose. Of the 19 participants administered the test dose, 1 was withdrawn due to test dose intolerance (5%, 95% confidence interval 0, 26%). Eighteen participants completed the study; 1was intolerant (95% CI 0, 27%) at the final visit due to newwheezes on examin association with an upper respiratory infection and otitis media. Home symptom diaries demonstrated cough as the main symptom in the hour following inhalation, which decreased in frequency over the study period. Adherence as assessed by daily home diary and returned study drug ampoules was high. Participants reported receiving both treatments on a median of 100% of days; a median of 25 ampoules were used during a median of 13 days. Conclusions: 7% HS appears well tolerated for up to 14 days in infants and toddlers with CF, with high adherence. These results provide encouraging short-term tolerability and adherence data for future trials assessing the safety and efficacy of 7% HS in young children with CF.
| Original language | English (US) |
|---|---|
| Pages (from-to) | 666-671 |
| Number of pages | 6 |
| Journal | Pediatric Pulmonology |
| Volume | 46 |
| Issue number | 7 |
| DOIs | |
| State | Published - Jul 2011 |
| Externally published | Yes |
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Keywords
- Cystic fibrosis
- Hypertonic saline
- Infants
- Safety
ASJC Scopus subject areas
- Pediatrics, Perinatology, and Child Health
- Pulmonary and Respiratory Medicine
Cite this
Inhaled hypertonic saline in infants and toddlers with cystic fibrosis : Short-term tolerability, adherence, and safety. / Rosenfeld, Margaret; Davis, Stephanie; Brumback, Lyndia; Daniel, Stephen; Rowbotham, Ron; Johnson, Robin; McNamara, Sharon; Jensen, Renee; Barlow, Carol; Ratjen, Felix.
In: Pediatric Pulmonology, Vol. 46, No. 7, 07.2011, p. 666-671.Research output: Contribution to journal › Article
}
TY - JOUR
T1 - Inhaled hypertonic saline in infants and toddlers with cystic fibrosis
T2 - Short-term tolerability, adherence, and safety
AU - Rosenfeld, Margaret
AU - Davis, Stephanie
AU - Brumback, Lyndia
AU - Daniel, Stephen
AU - Rowbotham, Ron
AU - Johnson, Robin
AU - McNamara, Sharon
AU - Jensen, Renee
AU - Barlow, Carol
AU - Ratjen, Felix
PY - 2011/7
Y1 - 2011/7
N2 - Background: Inhaled hypertonic saline (HS) is an attractive agent for chronic maintenance therapy in infants and toddlers with cystic fibrosis (CF) because it improves defective mucociliary clearance. Prior to undertaking a clinical trial of HS efficacy in young children with CF, tolerability, adherence, and safety must be established. Methods: Three-center, open label evaluation of the short-term tolerability, adherence, and safety of 7% HS administered twice daily for 14 days in children with CF 12-30 months of age. The primary objective was to evaluate the proportion of participants unable to tolerate single and repeated doses of 7% HS according to protocol-defined criteria. Participants inhaled a test dose of HS at the enrollment visit; test dose intolerance was defined as fulfillment of at least one of 4 criteria. Participants who tolerated the test dose inhaled 7% HS twice daily for 14 ± 2 days. Results: Twenty children were enrolled. One was withdrawn due to maternal concern over fussiness with application of the facemask for the test dose. Of the 19 participants administered the test dose, 1 was withdrawn due to test dose intolerance (5%, 95% confidence interval 0, 26%). Eighteen participants completed the study; 1was intolerant (95% CI 0, 27%) at the final visit due to newwheezes on examin association with an upper respiratory infection and otitis media. Home symptom diaries demonstrated cough as the main symptom in the hour following inhalation, which decreased in frequency over the study period. Adherence as assessed by daily home diary and returned study drug ampoules was high. Participants reported receiving both treatments on a median of 100% of days; a median of 25 ampoules were used during a median of 13 days. Conclusions: 7% HS appears well tolerated for up to 14 days in infants and toddlers with CF, with high adherence. These results provide encouraging short-term tolerability and adherence data for future trials assessing the safety and efficacy of 7% HS in young children with CF.
AB - Background: Inhaled hypertonic saline (HS) is an attractive agent for chronic maintenance therapy in infants and toddlers with cystic fibrosis (CF) because it improves defective mucociliary clearance. Prior to undertaking a clinical trial of HS efficacy in young children with CF, tolerability, adherence, and safety must be established. Methods: Three-center, open label evaluation of the short-term tolerability, adherence, and safety of 7% HS administered twice daily for 14 days in children with CF 12-30 months of age. The primary objective was to evaluate the proportion of participants unable to tolerate single and repeated doses of 7% HS according to protocol-defined criteria. Participants inhaled a test dose of HS at the enrollment visit; test dose intolerance was defined as fulfillment of at least one of 4 criteria. Participants who tolerated the test dose inhaled 7% HS twice daily for 14 ± 2 days. Results: Twenty children were enrolled. One was withdrawn due to maternal concern over fussiness with application of the facemask for the test dose. Of the 19 participants administered the test dose, 1 was withdrawn due to test dose intolerance (5%, 95% confidence interval 0, 26%). Eighteen participants completed the study; 1was intolerant (95% CI 0, 27%) at the final visit due to newwheezes on examin association with an upper respiratory infection and otitis media. Home symptom diaries demonstrated cough as the main symptom in the hour following inhalation, which decreased in frequency over the study period. Adherence as assessed by daily home diary and returned study drug ampoules was high. Participants reported receiving both treatments on a median of 100% of days; a median of 25 ampoules were used during a median of 13 days. Conclusions: 7% HS appears well tolerated for up to 14 days in infants and toddlers with CF, with high adherence. These results provide encouraging short-term tolerability and adherence data for future trials assessing the safety and efficacy of 7% HS in young children with CF.
KW - Cystic fibrosis
KW - Hypertonic saline
KW - Infants
KW - Safety
UR - http://www.scopus.com/inward/record.url?scp=79959828264&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=79959828264&partnerID=8YFLogxK
U2 - 10.1002/ppul.21425
DO - 10.1002/ppul.21425
M3 - Article
VL - 46
SP - 666
EP - 671
JO - Pediatric Pulmonology
JF - Pediatric Pulmonology
SN - 8755-6863
IS - 7
ER -