Comparison of Stool Microbiome in Children with Cystic Fibrosis Treated with and Without Elexacaftor-Tezacaftor-Ivacaftor-A Pilot Study.

No significant differences in alpha diversity were found between children with CF treated with ETI, children with CF not treated with ETI, and healthy sibling controls for both bacteriome and mycobiome.

• Study was prospective and observational in design
• Children aged 2-17 years with CF were enrolled
• ETI-treated group had received the therapy for at least two months
• Healthy siblings were included as controls
• No significant differences in demographics were observed between the groups

Alpha diversity showed a negative trend with the duration of ETI therapy for both bacteriome and mycobiome in children with CF treated with ETI.

• This trend was observed in the ETI-treated CF group specifically
• The trend was present for both bacterial (bacteriome) and fungal (mycobiome) components of the stool microbiome
• The finding is described as a trend rather than a statistically significant result
• Authors noted this as a preliminary finding requiring confirmation in future studies

Firmicutes and Proteobacteria were the most abundant and core bacterial phyla across all samples regardless of disease status or treatment group.

• These phyla were identified as core members across all samples
• The finding held regardless of CF disease status or ETI treatment
• Healthy sibling controls also shared these dominant phyla
• This suggests these phyla represent a consistent feature of the gut bacteriome in this pediatric population

Ascomycota and Basidiomycota were the most abundant and core fungal phyla across all samples regardless of disease status or treatment group.

• These fungal phyla were identified as core members of the mycobiome across all samples
• The finding held regardless of CF disease status or ETI treatment
• Both CF groups and healthy sibling controls shared these dominant fungal phyla
• This pattern mirrors findings for bacterial phyla in terms of consistency across groups

Prior studies in people with CF demonstrated a positive impact of ivacaftor on the stool microbiome, but studies evaluating the impact of ETI on gut dysbiosis are limited.

• This study was designed to address the gap in knowledge regarding ETI's effect on gut dysbiosis
• ETI combines elexacaftor, tezacaftor, and ivacaftor
• The study is described as a pilot study given the limited prior literature on ETI and gut microbiome
• Authors concluded future studies are needed to confirm or refute their preliminary findings