Findings

What do systematic reviews say?

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Evidence found and GRADE certainty

19 questions · 19 findings · 2 reviews · Cystic Fibrosis and Genetic Disorders · Headline outcomes only

No studies found 5 · 26.3%GRADE not reported 0 · 0%Very low 0 · 0%Low 2 · 10.5%Moderate 8 · 42.1%High 4 · 21.1%

Intervention not reported

50% reduction of tumour sizea · people with TSC

The intervention leads to higher 50% reduction of tumour sizea (high certainty).

1 finding from 1 review

Intervention not reported

Frequency of seizure · people with TSC

The review found no eligible studies for this question.

1 finding from 1 review

Intervention not reported

Creatinine level · people with TSC

The review found no eligible studies for this question.

1 finding from 1 review

Intervention not reported

FEV1/FVC ratio · people with TSC

The review found no eligible studies for this question.

1 finding from 1 review

Intervention not reported

Adverse events · people with TSC

The intervention probably leads to higher adverse events (moderate certainty).

1 finding from 1 review

Intervention not reported

Response to skin lesionsb · people with TSC

The intervention leads to higher response to skin lesionsb (high certainty).

1 finding from 1 review

Intervention not reported

Frequency of seizure · people with TSC

The intervention probably leads to higher frequency of seizure (moderate certainty).

1 finding from 1 review

Intervention not reported

Number of participants with an increased creatinine level · people with TSC

The intervention probably leads to lower number of participants with an increased creatinine level (moderate certainty).

1 finding from 1 review

Intervention not reported

FEV1/FVC ratio · people with TSC

The review found no eligible studies for this question.

1 finding from 1 review

Intervention not reported

Adverse events · people with TSC

The intervention leads to higher adverse events (high certainty).

1 finding from 1 review

Intervention not reported

50% reduction in tumour size · people with TSC

The review found no eligible studies for this question.

1 finding from 1 review

Intervention not reported

Response to skin lesion · people with TSC

The intervention leads to higher response to skin lesion (high certainty).

1 finding from 1 review

Summary of findings ‐ AZLI vs placebo

Participants hospitalised for respiratory exacerbation · adults and children with CF

Summary of findings ‐ AZLI showed no clear difference in participants hospitalised for respiratory exacerbation compared with placebo (moderate certainty).

1 finding from 1 review