Findings

What do systematic reviews say?

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

Quality of life ‐ total score · adults and children with cystic fibrosis

The review found no eligible studies for this question.

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
Studies not reported
No studies found

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

FEV1 % predicted: relative change from baseline · adults and children with cystic fibrosis

The review found no eligible studies for this question.

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
Studies not reported
No studies found

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

FEV1 % predicted: 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 ‐ dual therapy: lumacaftor plus ivacaftor (twice daily) vs placebo

Adverse events · 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 ‐ dual therapy: lumacaftor plus ivacaftor (twice daily) vs placebo

Time to first pulmonary exacerbation · adults and children with cystic fibrosis

The review found no eligible studies for this question.

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
Studies not reported
No studies found

Summary of findings: colistimethate dry powder for inhalation (Colobreathe®) vs tobramycin

Pulmonary exacerbations · adults and children with cystic fibrosis and a positive microbiological isolate of P aeruginosa from a respiratory tract specimen

Summary of findings: colistimethate dry powder for inhalation… showed no clear difference in pulmonary exacerbations: number of pulmonary exacerbations compared with tobramycin (moderate certainty).

1 finding from 1 review

Inhaled anti‐pseudomonal antibiotics for long‐term therapy in cystic fibrosis 2022 · Cochrane Headline outcomes only
RR 1.19 [0.86, 1.64]
1 study · 374 participants
Moderate

Summary of findings: colistimethate dry powder for inhalation (Colobreathe®) vs tobramycin

QoL · adults and children with cystic fibrosis and a positive microbiological isolate of P aeruginosa from a respiratory tract specimen

1 finding from 1 review

Inhaled anti‐pseudomonal antibiotics for long‐term therapy in cystic fibrosis 2022 · Cochrane Headline outcomes only
no estimate extracted
1 study · 374 participants
Low

Summary of findings: colistimethate dry powder for inhalation (Colobreathe®) vs tobramycin

Survival: number of deaths · adults and children with cystic fibrosis and a positive microbiological isolate of P aeruginosa from a respiratory tract specimen

Summary of findings: colistimethate dry powder for inhalation… showed no clear difference in survival: number of deaths compared with tobramycin (low certainty).

1 finding from 1 review

Inhaled anti‐pseudomonal antibiotics for long‐term therapy in cystic fibrosis 2022 · Cochrane Headline outcomes only
RR 0.21 [0.01, 4.32]
1 study · 374 participants
Low

Summary of findings: colistimethate dry powder for inhalation (Colobreathe®) vs tobramycin

Antibiotic resistance: change in mean MIC50 and MIC90 at the end of the trial · adults and children with cystic fibrosis and a positive microbiological isolate of P aeruginosa from a respiratory tract specimen

1 finding from 1 review

Inhaled anti‐pseudomonal antibiotics for long‐term therapy in cystic fibrosis 2022 · Cochrane Headline outcomes only
no estimate extracted
1 study · 374 participants
Low

Summary of findings: colistimethate dry powder for inhalation (Colobreathe®) vs tobramycin

Adverse events: number of treatment related adverse events · adults and children with cystic fibrosis and a positive microbiological isolate of P aeruginosa from a respiratory tract specimen

Summary of findings: colistimethate dry powder for inhalation… may leads to higher adverse events: number of treatment related adverse events compared with tobramycin (low certainty).

1 finding from 1 review

Inhaled anti‐pseudomonal antibiotics for long‐term therapy in cystic fibrosis 2022 · Cochrane Headline outcomes only
RR 1.76 [1.5, 2.08]
1 study · 379 participants
Low

Summary of findings: colistimethate dry powder for inhalation (Colobreathe®) vs tobramycin

FEV1 (% predicted): mean change from baseline · adults and children with cystic fibrosis and a positive microbiological isolate of P aeruginosa from a respiratory tract specimen

1 finding from 1 review

Inhaled anti‐pseudomonal antibiotics for long‐term therapy in cystic fibrosis 2022 · Cochrane Headline outcomes only
no estimate extracted
1 study · 374 participants
Low

Summary of findings: colistimethate dry powder for inhalation (Colobreathe®) vs tobramycin

FVC (% predicted) Change from baseline · adults and children with cystic fibrosis and a positive microbiological isolate of P aeruginosa from a respiratory tract specimen

1 finding from 1 review

Inhaled anti‐pseudomonal antibiotics for long‐term therapy in cystic fibrosis 2022 · Cochrane Headline outcomes only
no estimate extracted
1 study · 374 participants
Low

DFP and DFO vs DFP

Adherence to iron chelation therapy (%, SD)

1 finding from 1 review

Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or thalassaemia 2023 · Cochrane Headline outcomes only
no estimate extracted
4 studies · 369 participants
Low

DFP and DFO vs DFP

SAEs

Summary of findings: Comparison 5 ‐ deferiprone (DFP) plus… showed no clear difference in incidence of SAEs compared with DFP (low certainty).

1 finding from 1 review

Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or thalassaemia 2023 · Cochrane Headline outcomes only
RR 0.15 [0.01, 2.81]
1 study · 213 participants
Low

DFP and DFO vs DFP

All‐cause mortality

1 finding from 1 review

Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or thalassaemia 2023 · Cochrane Headline outcomes only
0.77 [0.17, 3.42]
2 studies · 237 participants
Very low

DFP and DFO vs DFP

Sustained adherence

The review found no eligible studies for this question.

1 finding from 1 review

Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or thalassaemia 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

DFP and DFO vs DFP

QoL

The review found no eligible studies for this question.

1 finding from 1 review

Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or thalassaemia 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

DFP and DFO vs deferoxamine

Adherence to iron chelation therapy (%, SD)

1 finding from 1 review

Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or thalassaemia 2023 · Cochrane Headline outcomes only
no estimate extracted
5 studies · 281 participants
Low

DFP and DFO vs deferoxamine

SAEs

1 finding from 1 review

Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or thalassaemia 2023 · Cochrane Headline outcomes only
no estimate extracted
4 studies · 180 participants
Low