Mahasin Al Shakirchi, Kimmo Sorjonen, Lena Hjelte, Lena Klingspor, Peter Bergman, Petrea Ericson, Marcus Svedberg, Ulrika Lindberg, Christine Hansen, Isabelle de Monestrol
{"title":"Impact of lumacaftor/ivacaftor on the bacterial and fungal respiratory pathogens in cystic fibrosis: a prospective multicenter cohort study in Sweden.","authors":"Mahasin Al Shakirchi, Kimmo Sorjonen, Lena Hjelte, Lena Klingspor, Peter Bergman, Petrea Ericson, Marcus Svedberg, Ulrika Lindberg, Christine Hansen, Isabelle de Monestrol","doi":"10.1177/17534666241254090","DOIUrl":null,"url":null,"abstract":"<p><strong>Background: </strong>A significant decline in pulmonary exacerbation rates has been reported in CF patients homozygous for F508del treated with lumacaftor/ivacaftor. However, it is still unclear whether this reduction reflects a diminished microbiological burden.</p><p><strong>Objectives: </strong>The aim of this study was to determine the impact of lumacaftor/ivacaftor on the bacterial and fungal burden.</p><p><strong>Design: </strong>The study is a prospective multicenter cohort study including 132 CF patients homozygous for F508del treated with lumacaftor/ivacaftor.</p><p><strong>Methods: </strong>Clinical parameters as well as bacterial and fungal outcomes 1 year after initiation of lumacaftor/ivacaftor were compared to data from 2 years prior to initiation of the treatment. Changes in the slope of the outcomes before and after the onset of treatment were assessed.</p><p><strong>Results: </strong>Lung function measured as ppFEV1 (<i>p</i> < 0.001), body mass index (BMI) in adults (<i>p</i> < 0.001), and BMI <i>z</i>-score in children (<i>p</i> = 0.007) were improved after initiation of lumacaftor/ivacaftor. In addition, the slope of the prevalence of <i>Streptococcus pneumoniae</i> (<i>p</i> = 0.007) and <i>Stenotrophomonas maltophilia</i> (<i>p</i> < 0.001) shifted from positive to negative, that is, became less prevalent, 1 year after treatment, while the slope for <i>Candida albicans</i> (<i>p</i> = 0.009), <i>Penicillium</i> spp (<i>p</i> = 0.026), and <i>Scedosporium apiospermum</i> (<i>p</i> < 0.001) shifted from negative to positive.</p><p><strong>Conclusion: </strong>The current study showed a significant improvement in clinical parameters and a reduction of some of CF respiratory microorganisms 1 year after starting with lumacaftor/ivacaftor. However, no significant changes were observed for <i>Pseudomonas aeruginosa, Staphylococcus aureus</i>, or <i>Aspergillus fumigatus</i>, key pathogens in the CF context.</p>","PeriodicalId":3,"journal":{"name":"ACS Applied Electronic Materials","volume":null,"pages":null},"PeriodicalIF":4.3000,"publicationDate":"2024-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11119492/pdf/","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"ACS Applied Electronic Materials","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1177/17534666241254090","RegionNum":3,"RegionCategory":"材料科学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"","PubModel":"","JCR":"Q1","JCRName":"ENGINEERING, ELECTRICAL & ELECTRONIC","Score":null,"Total":0}
引用次数: 0
Abstract
Background: A significant decline in pulmonary exacerbation rates has been reported in CF patients homozygous for F508del treated with lumacaftor/ivacaftor. However, it is still unclear whether this reduction reflects a diminished microbiological burden.
Objectives: The aim of this study was to determine the impact of lumacaftor/ivacaftor on the bacterial and fungal burden.
Design: The study is a prospective multicenter cohort study including 132 CF patients homozygous for F508del treated with lumacaftor/ivacaftor.
Methods: Clinical parameters as well as bacterial and fungal outcomes 1 year after initiation of lumacaftor/ivacaftor were compared to data from 2 years prior to initiation of the treatment. Changes in the slope of the outcomes before and after the onset of treatment were assessed.
Results: Lung function measured as ppFEV1 (p < 0.001), body mass index (BMI) in adults (p < 0.001), and BMI z-score in children (p = 0.007) were improved after initiation of lumacaftor/ivacaftor. In addition, the slope of the prevalence of Streptococcus pneumoniae (p = 0.007) and Stenotrophomonas maltophilia (p < 0.001) shifted from positive to negative, that is, became less prevalent, 1 year after treatment, while the slope for Candida albicans (p = 0.009), Penicillium spp (p = 0.026), and Scedosporium apiospermum (p < 0.001) shifted from negative to positive.
Conclusion: The current study showed a significant improvement in clinical parameters and a reduction of some of CF respiratory microorganisms 1 year after starting with lumacaftor/ivacaftor. However, no significant changes were observed for Pseudomonas aeruginosa, Staphylococcus aureus, or Aspergillus fumigatus, key pathogens in the CF context.