What do systematic reviews say?

58 questions · 59 findings · 22 reviews · Cystic Fibrosis and Genetic Disorders

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No studies found 59 · 100%GRADE not reported 0 · 0%Very low 0 · 0%Low 0 · 0%Moderate 0 · 0%High 0 · 0%

Summary of findings ‐ high‐dose azithromycin vs low‐dose azithromycin

QoL · children with cystic fibrosis

The review found no eligible studies for this question.

1 finding from 1 review

Macrolide antibiotics (including azithromycin) for cystic fibrosis 2024 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

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

Bisphosphonates vs no bisphosphonates

QoL · adults with cystic fibrosis who have 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

CCPT vs AD

QoL · children and adolescents with cystic fibrosis

The review found no eligible studies for this question.

1 finding from 1 review

Conventional chest physiotherapy compared to other airway clearance techniques for cystic fibrosis 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

CCPT vs exercise

QoL · children and young adults with CF

The review found no eligible studies for this question.

1 finding from 1 review

Conventional chest physiotherapy compared to other airway clearance techniques for cystic fibrosis 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Deferasirox vs deferiprone (DFO)

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

Summary of findings: Comparison 3 ‐ deferiprone (DFP) vs deferasirox (DFX)

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

DFX film‐coated tablet vs DFX dispersible tablet

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 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

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

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

VAL‐1221 vs alglucosidase alfa

QoL · adults 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

Digital technology (TIM) vs standard treatment (TBM)

QoL · children aged 5 to 16 years

The review found no eligible studies for this question.

1 finding from 1 review

Digital technology for monitoring adherence to inhaled therapies in people with cystic fibrosis 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Single foetal haemoglobin inducer at any dose or duration vs another 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 another dose or dose regimen of the same 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

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

Statins with cholesterol supplementation vs cholesterol supplementation only

QoL · children and adults with Smith‐Lemli‐Opitz syndrome

The review found no eligible studies for this question.

1 finding from 1 review

Statins for Smith‐Lemli‐Opitz syndrome 2022 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

High‐dose rhGH vs standard‐dose rhGH

QoL · children and young adults with CF

The review found no eligible studies for this question.

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
No studies found