Findings

What do systematic reviews say?

16 questions · 17 findings · 8 reviews · Cystic Fibrosis and Genetic Disorders

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No studies found 0 · 0%GRADE not reported 0 · 0%Very low 3 · 17.6%Low 8 · 47.1%Moderate 5 · 29.4%High 1 · 5.9%

Oral azithromycin vs placebo

QoL · adults and children with cystic fibrosis

1 finding from 1 review

Macrolide antibiotics (including azithromycin) for cystic fibrosis 2024 · Cochrane Headline outcomes only
no estimate extracted
1 study · 177 participants
Moderate

Bisphosphonates vs placebo or no treatment

Quality of life (change in mean back pain) · adults with β‐thalassaemia and osteoporosis

1 finding from 1 review

Treatment for osteoporosis in people with beta‐thalassaemia 2023 · Cochrane Headline outcomes only
no estimate extracted
1 study · 118 participants
Very low

Inhaled corticosteroids vs placebo

Quality of life: Quality of Well Being score change from baseline · adults and children with cystic fibrosis

1 finding from 1 review

Inhaled corticosteroids for cystic fibrosis 2019 · Cochrane Headline outcomes only
no estimate extracted
1 study · 23 participants
Low

Summary of findings: inhaled antioxidants vs placebo

Quality of life: change in CFQoL score from baseline · adults and children with cystic fibrosis

1 finding from 1 review

Antioxidant supplementation for lung disease in cystic fibrosis 2019 · Cochrane Headline outcomes only
no estimate extracted
1 study · 153 participants
Moderate

Dornase alfa vs placebo or no dornase alfa treatment

Quality of life: CFQ‐R respiratory domain: absolute change from baseline · adults and children with cystic fibrosis

Dornase alfa showed no clear difference in change in quality of life ‐ CFQ‐R respiratory at 1 month compared with placebo or no dornase alfa treatment (low certainty).

2 findings from 1 review

Dornase alfa for cystic fibrosis 2021 · Cochrane Headline outcomes only
MD 0.84
19 participants
Low
Dornase alfa for cystic fibrosis 2021 · Cochrane Headline outcomes only
MD 9.78
19 participants
Low

Summary of findings: oral antioxidants (NAC/GSH) vs placebo

Quality of life: change from baseline in CFQ‐R · adults and children with cystic fibrosis

1 finding from 1 review

Antioxidant supplementation for lung disease in cystic fibrosis 2019 · Cochrane Headline outcomes only
no estimate extracted
1 study · 61 participants
Moderate

Oral plus nebulised antibiotics vs oral antibiotics plus nebulised placebo

Quality of life: CFQ‐R respiratory domain: absolute change from baseline · adults and children with cystic fibrosis and a positive microbiological isolate of MRSA from a respiratory tract specimen

1 finding from 1 review

Interventions for the eradication of meticillin‐resistant Staphylococcus aureus (MRSA) in people with cystic fibrosis 2022 · Cochrane Headline outcomes only
no estimate extracted
1 study · 25 participants
Very low

Summary of findings ‐ monotherapy: cavosonstat vs placebo

Quality of life: CFQ‐R respiratory domain: absolute change from baseline · adults and children with cystic fibrosis

1 finding from 1 review

Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
no estimate extracted
1 study · 24 participants
Very low

Summary of findings ‐ dual therapy: tezacaftor plus ivacaftor vs placebo or ivacaftor alone

Quality of life: CFQ‐R respiratory domain: absolute change from baseline · adults and children with cystic fibrosis

1 finding from 1 review

Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
no estimate extracted
5 studies · 946 participants
Low

Summary of findings ‐ triple therapy: VX‐440‐tezacaftor‐ivacaftor vs placebo

Quality of life: CFQ‐R respiratory domain: absolute change from baseline · adults with cystic fibrosis and with the F508del/MF genotype

1 finding from 1 review

Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
no estimate extracted
1 study · 55 participants
Low

Summary of findings ‐ triple therapy: VX‐152‐tezacaftor‐ivacaftor vs placebo

Quality of life: CFQ‐R respiratory domain: absolute change from baseline · adults with cystic fibrosis and with the F508del/MF genotype

1 finding from 1 review

Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
no estimate extracted
1 study · 76 participants
Low

Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (once daily) vs placebo

Quality of life: CFQ‐R respiratory domain: absolute change from baseline · adults and children with cystic fibrosis

1 finding from 1 review

Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
no estimate extracted
3 studies · 1139 participants
Moderate

Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (twice daily) vs placebo

Quality of life: CFQ‐R respiratory domain: absolute change from baseline · adults and children with cystic fibrosis

1 finding from 1 review

Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
no estimate extracted
1 study · 204 participants
Low

Summary of findings ‐ monotherapy: lumacaftor vs placebo

Quality of life: CFQ‐R respiratory domain: absolute change from baseline · adults and children with cystic fibrosis

1 finding from 1 review

Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
no estimate extracted
1 study · 85 participants
Low

Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (once daily) vs placebo

Quality of life ‐ (EuroQol) EQ‐5D‐3L Index Score (total score): absolute change from baseline · adults and children with cystic fibrosis

1 finding from 1 review

Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
no estimate extracted
2 studies · 1061 participants
Moderate

Ataluren vs placebo

Quality of life: CFQ‐R respiratory domain: absolute change from baseline · adults and children with CF with at least one class I mutation (PTC)

1 finding from 1 review

Ataluren and similar compounds (specific therapies for premature termination codon class I mutations) for cystic… 2023 · Cochrane Headline outcomes only
no estimate extracted
2 studies · 506 participants
High