Inhaled Dry Powder Mannitol Treatment in Pediatric Patients with Cystic Fibrosis: Evaluation of Clinical Data in a Real-World Setting

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dc.contributor.authorDuman, Ipek
dc.contributor.authorUenal, Gokcen
dc.contributor.authorYilmaz, Asli Imran
dc.contributor.authorGueney, Ahmet Yasin
dc.contributor.authorDurduran, Yasemin
dc.contributor.authorPekcan, Sevgi
dc.date.accessioned2024-02-23T14:20:58Z
dc.date.available2024-02-23T14:20:58Z
dc.date.issued2022
dc.departmentNEÜen_US
dc.description.abstractBackground: Cystic fibrosis (CF) is a genetic disorder, in which defective clearance of airway secretions leads to progressive lung function loss. Inhaled mannitol is used to increase sputum and mucociliary clearance. There are little data from real-world studies on the effectiveness of mannitol in children. Our objective was to evaluate the spirometry and clinical results of mannitol in pediatric patients.Methods: We retrospectively reviewed the records of 30 children and adolescents with CF receiving inhaled mannitol who were already on recombinant human deoxyribonuclease (rhDNase) treatment. The change in forced expiratory volume in 1 second (FEV1) from baseline at 2-4 months was the primary outcome. Secondary measures were other spirometry results, body mass index (BMI), hospital admissions, sputum characteristics, and positive bacterial colonization.Results: Compared to baseline, we found significant improvement in percent predicted FEV1 at 2-4 months of treatment; 84.50 (58.00-99.00) vs. 96.00 (66.00-106.00) (P = 0.0007). The absolute change in FEV1 was +11.5% at 2-4 months, +6.5% at 5-7 months, and +4% at 8-12 months. Also, significant improvements in other spirometry results were observed. Adolescents had significantly lower FEV1 results, but the improvement in their lung function was sustained for a more extended period than children. Mannitol provided easier sputum removal, increased sputum volume, significant decline in hospitalizations, and significantly fewer patients with positive sputum cultures. A significant increase in BMI at 8-12 months was observed. Cough was the most frequent adverse effect.Conclusion: In a real-world setting, our results demonstrated that adding mannitol to rhDNase therapy is tolerable in pediatric patients with CF and may provide improved spirometry and clinical outcomes. In addition, our results showed that mannitol provided recovery in overall lung function at 2-4 months, which was sustained up to 12 months together with improved BMI, easier sputum removal, and a decline in bacterial colonization and hospital admissions. However, cough was the most frequent side effect.en_US
dc.identifier.doi10.1089/ped.2021.0127
dc.identifier.endpage26en_US
dc.identifier.issn2151-321X
dc.identifier.issn2151-3228
dc.identifier.issue1en_US
dc.identifier.pmid35285672en_US
dc.identifier.scopus2-s2.0-85127565772en_US
dc.identifier.scopusqualityQ3en_US
dc.identifier.startpage19en_US
dc.identifier.urihttps://doi.org/10.1089/ped.2021.0127
dc.identifier.urihttps://hdl.handle.net/20.500.12452/13389
dc.identifier.volume35en_US
dc.identifier.wosWOS:000772094200001en_US
dc.identifier.wosqualityQ4en_US
dc.indekslendigikaynakWeb of Scienceen_US
dc.indekslendigikaynakScopusen_US
dc.indekslendigikaynakPubMeden_US
dc.language.isoenen_US
dc.publisherMary Ann Liebert, Incen_US
dc.relation.ispartofPediatric Allergy Immunology And Pulmonologyen_US
dc.relation.publicationcategoryMakale - Uluslararası Hakemli Dergi - Kurum Öğretim Elemanıen_US
dc.rightsinfo:eu-repo/semantics/closedAccessen_US
dc.subjectAdolescentsen_US
dc.subjectChildrenen_US
dc.subjectCystic Fibrosisen_US
dc.subjectMannitolen_US
dc.subjectPulmonary Functionen_US
dc.subjectReal-World Dataen_US
dc.titleInhaled Dry Powder Mannitol Treatment in Pediatric Patients with Cystic Fibrosis: Evaluation of Clinical Data in a Real-World Settingen_US
dc.typeArticleen_US

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