Inhaled dry powder mannitol in children with cystic fibrosis: A randomised efficacy and safety trial

Boeck, K. De; Haarman, Eric G.; Hull, James H.; Lands, L. C.; Moeller, Alexander; Munck, À.; Riethmüller, Joachim; Tiddens, Harm A.W.M.; Volpi, Sonia; Leadbetter, Joanna; Charlton, Brett; Malfroot, Anne

doi:10.1016/j.jcf.2017.02.003

Cited by 31 publications

(20 citation statements)

References 25 publications

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“…DPI mannitol made significant improvements in spirometry indices and exacerbation frequency in children with CF aged 6-17 years. 26 The improvement in FEV1 with DPI mannitol was found as effective as dornase alfa. 12 Contrary to our findings, Minasian et al 12 reported that the combination of DPI and dornase alfa had no significant effect on FEV1.…”

Section: Discussionmentioning

confidence: 91%

Comparison of inhaled mannitol/dornase alfa combination and daily dornase alfa alone in children with cystic fibrosis

Tural

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Emiralioğlu

et al. 2021

Pediatric Pulmonology

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Objectives: Inhaled recombinant human deoxyribonuclease (dornase alfa) and osmotic agents such as inhaled mannitol are used for improving the clearance of secretions of cystic fibrosis (CF) patients. We aimed to evaluate the long-term clinical effects of adding dry powder inhaled (DPI) mannitol in subjects with CF who are taking daily dornase alfa. Method:We conducted a retrospective case-control study on subjects with CF. The effect of DPI mannitol was assessed by comparing DPI mannitol and dornase alfa combination with daily dornase alfa alone in children with CF during a 12-month period. The primary outcome measures of the study were absolute changes in percent predicted forced expiratory volume in 1 s (FEV1) and FEV1 z-scores and the secondary outcomes included other spirometry indices, body mass index, frequency of pulmonary exacerbations, SPO 2 , and sputum microbiology.Result: Of a total of 28 patients who committed to use DPI mannitol treatments during the study period, five had a positive challenge with DPI mannitol and two were aged over 18 years. Therefore, the mannitol treatment group consisted of 21 patients. However, the effect of DPI mannitol was analyzed using 15 patients in the mannitol treatment group who received DPI mannitol for at least 12 months, and 18 patients who only used dornase alfa constituted the control group. The median absolute change in FEV1 between baseline and the third month; and baseline and the 12th month were significantly higher in the mannitol treatment group (p = 0.038, p = 0.004, respectively). When the groups are compared with respect to absolute zscore changes, all spirometry indices, except FVC at the end of 3 months, showed statistically significant improvements in the mannitol treatment group. Some secondary outcomes like pulmonary exacerbation frequency during the study year and median absolute body mass index z-score changes from baseline to the end of the study showed no significant differences between the groups (p = 0.735, p = 0.161, respectively). No colonization changes were observed in the treatment group.Conclusions: This study showed that in those patients who tolerated long-term (12 months) treatment with DPI mannitol and dornase alfa made greater improvements

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Section: Discussionmentioning

confidence: 91%

Comparison of inhaled mannitol/dornase alfa combination and daily dornase alfa alone in children with cystic fibrosis

Tural

Yalçın

Emiralioğlu

et al. 2021

Pediatric Pulmonology

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“…In a subsequent study of children with CF aged 6 to 17 years, inhaled mannitol was well‐tolerated and associated with improvement in lung function and a decreased incidence of pulmonary exacerbations, irrespective of disease severity …”

Section: Mucus Hydrators: Hypertonic Saline and Mannitolmentioning

confidence: 99%

Aerosolized agents for airway clearance in cystic fibrosis

Southern

Clancy

Ranganathan

2019

Pediatric Pulmonology

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The outlook for people with cystic fibrosis (CF) has improved considerably as a result of conventional therapies including aerosolized agents for airway clearance. These will continue to play a significant role in maintaining well‐being and improving survival, even as newer agents emerge that correct the underlying CF defect. In this review, we explore the evidence supporting the use of dornase alfa, hypertonic saline, and mannitol in improving mucus clearance in patients with CF from different age groups with differing disease severity. We also discuss the clinical use of these agents in the context of available international guidelines as well as practical considerations in the clinic, highlighting the importance of a multidisciplinary approach and shared decision‐making. Unanswered questions regarding the optimal use of these agents are highlighted.

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“…In children with CF, inhaled mannitol was associated with significant improvements in lung function and sputum weight, irrespective of rhDNase use, age, or disease severity. Inhaled mannitol was well tolerated and was associated with a reduced incidence of PEx [ 35 ].…”

Section: Obstruction Treatmentmentioning

confidence: 99%

Treatment of Pulmonary Disease of Cystic Fibrosis: A Comprehensive Review

et al. 2021

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Cystic fibrosis (CF) is a genetic disease that causes absence or dysfunction of a protein named transmembrane conductance regulatory protein (CFTR) that works as an anion channel. As a result, the secretions of the organs where CFTR is expressed are very viscous, so their functionality is altered. The main cause of morbidity is due to the involvement of the respiratory system as a result of recurrent respiratory infections by different pathogens. In recent decades, survival has been increasing, rising by around age 50. This is due to the monitoring of patients in multidisciplinary units, early diagnosis with neonatal screening, and advances in treatments. In this chapter, we will approach the different therapies used in CF for the treatment of symptoms, obstruction, inflammation, and infection. Moreover, we will discuss specific and personalized treatments to correct the defective gene and repair the altered protein CFTR. The obstacle for personalized CF treatment is to predict the drug response of patients due to genetic complexity and heterogeneity of uncommon mutations.

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Inhaled dry powder mannitol in children with cystic fibrosis: A randomised efficacy and safety trial

Cited by 31 publications

References 25 publications

Comparison of inhaled mannitol/dornase alfa combination and daily dornase alfa alone in children with cystic fibrosis

Comparison of inhaled mannitol/dornase alfa combination and daily dornase alfa alone in children with cystic fibrosis

Aerosolized agents for airway clearance in cystic fibrosis

Treatment of Pulmonary Disease of Cystic Fibrosis: A Comprehensive Review

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