Findings

What do systematic reviews say?

697 questions · 708 findings · 47 reviews · Cystic Fibrosis and Genetic Disorders

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No studies found 263 · 37.1%GRADE not reported 12 · 1.7%Very low 146 · 20.6%Low 168 · 23.7%Moderate 98 · 13.8%High 21 · 3%

Summary of findings ‐ ivacaftor vs placebo

Time to first pulmonary exacerbation · adults and children with cystic fibrosis and with at least one G551D CFTR mutation

Summary of findings ‐ ivacaftor probably leads to lower time to first pulmonary exacerbation compared with placebo (moderate certainty).

1 finding from 1 review

Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis 2019 · Cochrane Headline outcomes only
HR 0.46 [0.29, 0.73]
1 study · 161 participants
Moderate

Summary of findings ‐ ivacaftor vs placebo

Survival · adults and children with cystic fibrosis and with at least one R117H CFTR mutation

1 finding from 1 review

Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis 2019 · Cochrane Headline outcomes only
no estimate extracted
1 study · 69 participants
GRADE not reported

Summary of findings ‐ ivacaftor vs placebo

QoL · adults and children with cystic fibrosis and with at least one R117H CFTR mutation

The review found no eligible studies for this question.

1 finding from 1 review

Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis 2019 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Summary of findings ‐ ivacaftor vs placebo

QoL (CFQ‐R) Respiratory domain · adults and children with cystic fibrosis and with at least one R117H CFTR mutation

1 finding from 1 review

Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis 2019 · Cochrane Headline outcomes only
no estimate extracted
1 study · 69 participants
Moderate

Summary of findings ‐ ivacaftor vs placebo

FEV1 % predicted: relative change from baseline · adults and children with cystic fibrosis and with at least one R117H CFTR mutation

1 finding from 1 review

Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis 2019 · Cochrane Headline outcomes only
no estimate extracted
1 study · 69 participants
Moderate

Summary of findings ‐ ivacaftor vs placebo

FEV1 L and % predicted Absolute change from baseline · adults and children with cystic fibrosis and with at least one R117H CFTR mutation

1 finding from 1 review

Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis 2019 · Cochrane Headline outcomes only
no estimate extracted
1 study · 69 participants
Moderate

Summary of findings ‐ ivacaftor vs placebo

QoL · adults and children with cystic fibrosis and with the F508del CFTR mutation

The review found no eligible studies for this question.

1 finding from 1 review

Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis 2019 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Summary of findings ‐ ivacaftor vs placebo

Adverse events · adults and children with cystic fibrosis and with at least one R117H CFTR mutation

1 finding from 1 review

Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis 2019 · Cochrane Headline outcomes only
no estimate extracted
1 study · 69 participants
Low

Summary of findings ‐ ivacaftor vs placebo

Time to first pulmonary exacerbation · adults and children with cystic fibrosis and with at least one R117H CFTR mutation

Summary of findings ‐ ivacaftor showed no clear difference in time to first pulmonary exacerbation compared with placebo (moderate certainty).

1 finding from 1 review

Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis 2019 · Cochrane Headline outcomes only
HR 0.93 [0.42, 2.08]
1 study · 69 participants
Moderate

Summary of findings ‐ ivacaftor vs placebo

QoL (CFQ‐R) Respiratory domain · adults and children with cystic fibrosis and with the F508del CFTR mutation

1 finding from 1 review

Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis 2019 · Cochrane Headline outcomes only
no estimate extracted
1 study · 140 participants
Moderate

Summary of findings ‐ ivacaftor vs placebo

FEV1 % predicted: relative change from baseline · adults and children with cystic fibrosis and with the F508del CFTR mutation

1 finding from 1 review

Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis 2019 · Cochrane Headline outcomes only
no estimate extracted
1 study · 140 participants
Moderate

Summary of findings ‐ ivacaftor vs placebo

FEV1 % predicted: absolute change from baseline · adults and children with cystic fibrosis and with the F508del CFTR mutation

1 finding from 1 review

Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis 2019 · Cochrane Headline outcomes only
no estimate extracted
1 study · 140 participants
Moderate

Summary of findings ‐ ivacaftor vs placebo

Adverse events · adults and children with cystic fibrosis and with the F508del CFTR mutation

1 finding from 1 review

Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis 2019 · Cochrane Headline outcomes only
no estimate extracted
1 study · 140 participants
Low

Summary of findings ‐ ivacaftor vs placebo

Time to first pulmonary exacerbation · adults and children with cystic fibrosis and with the F508del CFTR mutation

The review found no eligible studies for this question.

1 finding from 1 review

Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis 2019 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Summary of findings ‐ ivacaftor vs placebo

Survival · adults and children with cystic fibrosis and with at least one G551D CFTR mutation

1 finding from 1 review

Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis 2019 · Cochrane Headline outcomes only
no estimate extracted
3 studies · 238 participants
GRADE not reported

Summary of findings ‐ ivacaftor vs placebo

QoL · adults and children with cystic fibrosis and with at least one G551D CFTR mutation

The review found no eligible studies for this question.

1 finding from 1 review

Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis 2019 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Intravenous magnesium sulphate vs placebo

Change in frequency of vaso‐occlusive painful crises (as measured by pain diary days with significant pain) · people with sickle cell disease

1 finding from 1 review

Magnesium for treating sickle cell disease 2019 · Cochrane Headline outcomes only
no estimate extracted
1 study · 104 participants
Moderate

Oral magnesium pidolate vs placebo or no intervention

Length of hospital stay · people with sickle cell disease

The review found no eligible studies for this question.

1 finding from 1 review

Magnesium for treating sickle cell disease 2019 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Oral magnesium pidolate vs placebo or no intervention

Change in dense red cells in sickle cell disease · people with sickle cell disease

The review found no eligible studies for this question.

1 finding from 1 review

Magnesium for treating sickle cell disease 2019 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Oral magnesium pidolate vs placebo or no intervention

Effects of K‐Cl cotransport red cell K+ and Mg2+ content (RBC magnesium) mM/kg Hb · people with sickle cell disease

1 finding from 1 review

Magnesium for treating sickle cell disease 2019 · Cochrane Headline outcomes only
no estimate extracted
1 study · 24 participants
Moderate