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Recombinant human DNase nebulisation in children with cystic fibrosis: before bedtime or after waking up?
L J van der Giessen1, R Gosselink, W C J Hop
1Department of Paediatric Physiotherapy, Sophia Children's Hospital, Erasmus MC, Rotterdam, The Netherlands. l.vandergiessen@erasmusmc.nl
Insights
Nebulizing recombinant human deoxyribonuclease (rhDNase) before bed is as effective and safe as taking it after waking up for cystic fibrosis patients on maintenance therapy. This finding offers flexibility for rhDNase treatment timing in CF care.
Area of Science:
- Pulmonary Medicine
- Pharmacology
- Genetics
Background:
- Cystic Fibrosis (CF) is a genetic disorder affecting multiple organs, primarily the lungs.
- Recombinant human deoxyribonuclease (rhDNase) is a key therapy for CF, improving lung function.
- Optimizing rhDNase administration timing is crucial for patient adherence and treatment efficacy.
Purpose of the Study:
- To compare the efficacy and safety of nebulizing rhDNase before bedtime versus after waking up.
- To evaluate the impact of rhDNase timing on pulmonary function and nocturnal symptoms in CF patients.
Main Methods:
- A randomized, double-blind, double-dummy, crossover study design.
- Inclusion criteria: CF diagnosis, stable condition, and rhDNase maintenance therapy.
- Primary endpoint: Maximal instantaneous forced flow (MEF(25%)); Secondary endpoints: Nocturnal cough, oxygen saturation.
Main Results:
- No significant difference in MEF(25%) between morning and bedtime rhDNase nebulization.
- No significant differences observed in nocturnal cough frequency or arterial oxygen saturation.
- The study included 24 patients aged 6-19 years on rhDNase maintenance therapy.
Conclusions:
- Nebulizing rhDNase before bedtime is equally effective and safe as administering it after waking up for CF patients.
- Treatment timing flexibility can be offered to patients on maintenance rhDNase therapy.
- This finding supports improved patient convenience without compromising therapeutic outcomes in cystic fibrosis management.
Abstract:
The present study focused on patients with cystic fibrosis (CF), who were on maintenance therapy with recombinant human deoxyribonuclease (rhDNase), with the aim of comparing efficacy and possible side effects of nebulisation of rhDNase when taken before bedtime with efficacy and side effects when taken after waking up. A randomised, double-blind, double-dummy, crossover study group was used. The inclusion criteria were as follows: 1) CF, 2) stable clinical condition and 3) rhDNase maintenance therapy. Patients in group I inhaled rhDNase before bedtime and a placebo after waking up in weeks 1-2. The protocol was reversed during weeks 3-4. Group II patients performed the reverse of this sequence. Patients continued with their daily routine sputum expectoration. The primary end-point was classified as the maximal instantaneous forced flow when 25% of the forced vital capacity remained to be exhaled (MEF(25%)). Pulmonary functions tests were performed on days 0, 7, 14, 21 and 28. At 1, 2, 3 and 4 weeks arterial oxygen saturation and cough frequency were measured during the night. A total of 24 patients completed the study. The mean (range) age of the patients was 13 (6-19) yrs. MEF(25%), taken to be the primary end-point, did not show a significant difference between nebulisation of rhDNase before bedtime compared with when taken after waking up. Nocturnal cough, oxygen saturation, and other secondary end-points were not significantly different between the two study periods. In conclusion, the present study found that it is equally effective and safe to nebulise recombinant human deoxyribonuclease before bedtime compared with when performed after waking up in children with cystic fibrosis, who are on maintenance treatment with recombinant human deoxyribonuclease.
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