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Evidence found and GRADE certainty

873 questions · 883 findings · 38 reviews · Cystic Fibrosis and Genetic Disorders

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No studies found 319 · 36.1%GRADE not reported 18 · 2%Very low 198 · 22.4%Low 186 · 21.1%Moderate 147 · 16.6%High 15 · 1.7%

Summary of findings: amikacin liposome inhalation suspension (ALIS) vs tobramycin

Number experiencing adverse effects · adults and children with cystic fibrosis and a positive microbiological isolate of P aeruginosa from a respiratory tract specimen

Summary of findings: amikacin liposome inhalation suspension (ALIS) showed no clear difference in adverse events: number of participants experiencing any TEAE compared with tobramycin (moderate certainty).

1 finding from 1 review

Summary of findings table ‐ Emicuzimab 3.0 mg/kg bi‐weekly prophylaxis vs on‐demand therapy

Proportion with zero bleeds · people without inhibitors

Summary of findings table ‐ Emicuzimab 3.0 mg/kg bi‐weekly prophylaxis showed no clear difference in proportion of participants with zero bleeds compared with on‐demand therapy (low certainty).

1 finding from 1 review

Summary of findings table ‐ Emicuzimab 3.0 mg/kg bi‐weekly prophylaxis vs on‐demand therapy

All adverse events · people without inhibitors

Summary of findings table ‐ Emicuzimab 3.0 mg/kg bi‐weekly prophylaxis probably leads to higher all adverse events compared with on‐demand therapy (moderate certainty).

1 finding from 1 review

Summary of findings table ‐ Emicuzimab 3.0 mg/kg bi‐weekly prophylaxis vs on‐demand therapy

Serious adverse events · people without inhibitors

Summary of findings table ‐ Emicuzimab 3.0 mg/kg bi‐weekly prophylaxis showed no clear difference in serious adverse events compared with on‐demand therapy (moderate certainty).

1 finding from 1 review

Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor vs control (ivacaftor alone or tezacaftor plus ivacaftor)

Survival · adults and children aged 12 years and over with cystic fibrosis and either F508del/gating variant or F508del/residual function genotype
1 finding from 1 review

Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor vs control (ivacaftor alone or tezacaftor plus ivacaftor)

Quality of life: total score · adults and children aged 12 years and over with cystic fibrosis and either F508del/gating variant or F508del/residual function genotype

The review found no eligible studies for this question.

1 finding from 1 review

Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor vs control (ivacaftor alone or tezacaftor plus ivacaftor)

Quality of life: CFQ‐R respiratory domain: absolute change from baseline · adults and children aged 12 years and over with cystic fibrosis and either F508del/gating variant or F508del/residual function genotype

The review found no eligible studies for this question.

1 finding from 1 review

Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor vs control (ivacaftor alone or tezacaftor plus ivacaftor)

FEV1 % predicted: relative change from baseline · adults and children aged 12 years and over with cystic fibrosis and either F508del/gating variant or F508del/residual function genotype

The review found no eligible studies for this question.

1 finding from 1 review

Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor vs control (ivacaftor alone or tezacaftor plus ivacaftor)

FEV1 % predicted: absolute change from baseline · adults and children aged 12 years and over with cystic fibrosis and either F508del/gating variant or F508del/residual function genotype
1 finding from 1 review

Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor vs control (ivacaftor alone or tezacaftor plus ivacaftor)

Adverse events ‐ treatment emergent · adults and children aged 12 years and over with cystic fibrosis and either F508del/gating variant or F508del/residual function genotype

Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor showed no clear difference in adverse events: number of participants with treatment‐emergent… compared with control (ivacaftor alone or tezacaftor plus ivacaftor) (moderate certainty).

1 finding from 1 review

Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor vs control (ivacaftor alone or tezacaftor plus ivacaftor)

Time to first pulmonary exacerbation · adults and children aged 12 years and over with cystic fibrosis and either F508del/gating variant or F508del/residual function genotype

The review found no eligible studies for this question.

1 finding from 1 review