Donaldson 2020.
Study characteristics | ||
Methods | RCT. Design: parallel, double‐blind. Location: single‐centre study, USA. Duration: 4 weeks. |
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Participants | Children aged ≥ 5 to <18 years with confirmed CF and stable lung disease (FEV1 > 60%). Age, mean (SD): HS 11.9 (3.3) years; IS 9.8 (3.6) years; overall 11.1 (3.5) years. Sex, number (%) female: HS 8/14 (57%); IS 4/9 (44%); overall 12/23 (52%). Disease status Post‐BD FVC % predicted, mean (SD): HS 101.4 (12.3); IS 100.0 (10.9); overall 100.8 (11.5). Post‐BD FEV1 % predicted, mean (SD): HS 97.0 (12.6); IS 93.2 (13.4); overall 95.5 (12.8). Post‐BD FEF25-75 % predicted, mean (SD): HS 91.1 (24.9); IS 86.2 (37.8); overall 89.7 (27.9). Taking inhaled antibiotics, number (%): HS 2 (14%); IS 1 (11%); overall 3 (13%). On chronic HS, number (%): HS 1 (7%); IS 3 (33%); overall 4 (17%). Exclusion criteria Individuals with unstable lung disease, who did not tolerate HS, or who were unable or unwilling to withhold prescribed HS for at least 2 weeks before the screening visit were excluded. 24 screened, 23 randomised, 20 completed treatment and follow‐up. |
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Interventions |
Intervention: inhaled HS (6% NaCl, 4 mL) three times a day for 28 days. Control: inhaled IS (0.12% NaCl, 4 mL) three times a day for 28 days. Both treatments were given via an eFlow nebuliser. |
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Outcomes |
Primary outcome Change in average whole lung MCC measured over 90 min (AveClr90) between baseline and 4 weeks of treatment. Secondary outcomes Change in FEV1 % predicted, CFQ‐R respiratory symptom domain score (Quittner 2009). |
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Notes | A sample size calculation was carried out to provide 80% power at a significance level of 0.05 to detect a treatment difference of 6.6% (absolute change in MCC). | |
Risk of bias | ||
Bias | Authors' judgement | Support for judgement |
Random sequence generation (selection bias) | Low risk | The randomisation schedule was generated and maintained by the investigational drug service. |
Allocation concealment (selection bias) | Low risk | The randomisation schedule was generated and maintained by the investigational drug service. |
Blinding of participants and personnel (performance bias) All outcomes | Unclear risk | Participants, care provider and investigator were all blinded to treatment allocation. Taste masking was not done, therefore it is unclear whether the participants were truly blinded. |
Blinding of outcome assessment (detection bias) All outcomes | Low risk | Outcome assessors were also blinded to treatment allocation. |
Incomplete outcome data (attrition bias) All outcomes | Low risk | 24 participants were recruited but there was one screen failure. 23 participants were randomised and 20 participants completed the study. Sample size calculation was carried out. |
Selective reporting (reporting bias) | Low risk | All outcomes listed in the methods and in the trial registration document are reported in the results. |
Other bias | Low risk | No other risk of bias identified. |