Findings

What do systematic reviews say?

34 questions · 34 findings · 14 reviews · Cystic Fibrosis and Genetic Disorders

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No studies found 16 · 47.1%GRADE not reported 0 · 0%Very low 4 · 11.8%Low 8 · 23.5%Moderate 6 · 17.6%High 0 · 0%

Bisphosphonates vs placebo

QoL · children with cystic fibrosis who have not had a lung transplant

The review found no eligible studies for this question.

1 finding from 1 review

Bisphosphonates for osteoporosis in people with cystic fibrosis 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Alglucosidase alfa plus clenbuterol vs alglucosidase alfa plus placebo

QoL · 13 people with LOPD

The review found no eligible studies for this question.

1 finding from 1 review

Enzyme replacement therapy for late‐onset Pompe disease 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Alglucosidase alfa plus albuterol vs alglucosidase alfa plus placebo

QoL · 13 people with LOPD

The review found no eligible studies for this question.

1 finding from 1 review

Enzyme replacement therapy for late‐onset Pompe disease 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Two or more foetal haemoglobin inducers at any dose or duration vs a usual management protocol, placebo or a single foetal haemoglobin inducer

QoL · people with non‐transfusion‐dependent β‐thalassaemia

The review found no eligible studies for this question.

1 finding from 1 review

Foetal haemoglobin inducers for reducing blood transfusion in non‐transfusion‐dependent beta‐thalassaemias 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Single foetal haemoglobin inducer at any dose or duration vs placebo or usual care

QoL · people with non‐transfusion‐dependent β‐thalassaemia

The review found no eligible studies for this question.

1 finding from 1 review

Foetal haemoglobin inducers for reducing blood transfusion in non‐transfusion‐dependent beta‐thalassaemias 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

High‐dose rhGH vs placebo

QoL · children and young adults with CF

1 finding from 1 review

Recombinant growth hormone therapy for cystic fibrosis in children and young adults 2021 · Cochrane Headline outcomes only
no estimate extracted
1 study · 41 participants
Very low

Standard rhGH vs placebo

QoL · children and young adults with CF

1 finding from 1 review

Recombinant growth hormone therapy for cystic fibrosis in children and young adults 2021 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
Very low

Summary of findings: garlic supplementation vs placebo

QoL · children aged over eight and adults with cystic fibrosis and chronic pulmonary infection with P aeruginosa

The review found no eligible studies for this question.

1 finding from 1 review

Antibiotic adjuvant therapy for pulmonary infection in cystic fibrosis 2020 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Summary of findings: nitric oxide vs placebo

QoL · children (12 or over) and young adults with cystic fibrosis

The review found no eligible studies for this question.

1 finding from 1 review

Antibiotic adjuvant therapy for pulmonary infection in cystic fibrosis 2020 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Zinc vs placebo

QoL · children with cystic fibrosis

The review found no eligible studies for this question.

1 finding from 1 review

Antibiotic adjuvant therapy for pulmonary infection in cystic fibrosis 2020 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Summary of findings: β‐carotene vs placebo

QoL · adults and children with cystic fibrosis

The review found no eligible studies for this question.

1 finding from 1 review

Antibiotic adjuvant therapy for pulmonary infection in cystic fibrosis 2020 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Pregabalin vs placebo

QoL · adults and children with sickle cell disease experiencing neuropathic pain

1 finding from 1 review

Interventions for treating neuropathic pain in people with sickle cell disease 2019 · Cochrane Headline outcomes only
no estimate extracted
1 study · 16 participants
Very low

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 · 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

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

Summary of findings: monoclonal antibody KB001‐A vs placebo

QoL · children (12 or over) and adults with cystic fibrosis and lung infection

1 finding from 1 review

Antibiotic adjuvant therapy for pulmonary infection in cystic fibrosis 2020 · Cochrane Headline outcomes only
no estimate extracted
1 study · 27 participants
Low

Summary of findings ‐ ivacaftor vs placebo

QoL (CFQ‐R) Respiratory domain · 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 · 222 participants
Moderate

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

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

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