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: low‐dose vs high‐dose PERT

Frequency of bowel symptoms: stool frequency · adults and children with cystic fibrosis

1 finding from 1 review

Pancreatic enzyme replacement therapy for people with cystic fibrosis 2020 · Cochrane Headline outcomes only
no estimate extracted
1 study · 17 participants
Very low

Summary of findings: low‐dose vs high‐dose PERT

CFA: FFE (g/day) · adults and children with cystic fibrosis

1 finding from 1 review

Pancreatic enzyme replacement therapy for people with cystic fibrosis 2020 · Cochrane Headline outcomes only
no estimate extracted
1 study · 17 participants
Very low

Summary of findings: low‐dose vs high‐dose PERT

Adverse events · adults and children with cystic fibrosis

1 finding from 1 review

Pancreatic enzyme replacement therapy for people with cystic fibrosis 2020 · Cochrane Headline outcomes only
no estimate extracted
2 studies · 146 participants
Very low

Summary of findings: low‐dose vs high‐dose PERT

Pulmonary exacerbations · adults and children with cystic fibrosis

The review found no eligible studies for this question.

1 finding from 1 review

Pancreatic enzyme replacement therapy for people with cystic fibrosis 2020 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Exercise vs no intervention

Adverse events · participants with haemophilia

The review found no eligible studies for this question.

1 finding from 1 review

Exercise for haemophilia 2016 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Exercise vs no intervention

Quality of life · participants with haemophilia

The review found no eligible studies for this question.

1 finding from 1 review

Exercise for haemophilia 2016 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Exercise vs no intervention

Joint health: Modified Colorado Score · participants with haemophilia

1 finding from 1 review

Exercise for haemophilia 2016 · Cochrane Headline outcomes only
no estimate extracted
1 study · 25 participants
Low

Exercise vs no intervention

Reduction in pain intensity · participants with haemophilia

1 finding from 1 review

Exercise for haemophilia 2016 · Cochrane Headline outcomes only
no estimate extracted
1 study · 18 participants
Very low

Exercise vs no intervention

Functional ability · participants with haemophilia

The review found no eligible studies for this question.

1 finding from 1 review

Exercise for haemophilia 2016 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Exercise vs no intervention

Range of Motion: flexion and extension · participants with haemophilia

1 finding from 1 review

Exercise for haemophilia 2016 · Cochrane Headline outcomes only
no estimate extracted
1 study · 18 participants
Very low

Exercise vs no intervention

Strength: bicep strength · participants with haemophilia

1 finding from 1 review

Exercise for haemophilia 2016 · Cochrane Headline outcomes only
no estimate extracted
1 study · 18 participants
Very low

Summary of findings – denosumab vs placebo

BMD Z score (total hip/femoral neck) · adults with β‐thalassaemia and low bone mass

Summary of findings – denosumab showed no clear difference in BMD Z score (total hip/femoral neck) compared with placebo (low certainty).

1 finding from 1 review

Treatment for osteoporosis in people with beta‐thalassaemia 2023 · Cochrane Headline outcomes only
MD 0.05
1 study · 63 participants
Low

Summary of findings – denosumab vs placebo

BMD Z score (lumbar spine) · adults with β‐thalassaemia and low bone mass

Summary of findings – denosumab showed no clear difference in BMD Z score (lumbar spine) compared with placebo (low certainty).

1 finding from 1 review

Treatment for osteoporosis in people with beta‐thalassaemia 2023 · Cochrane Headline outcomes only
no estimate extracted
1 study · 63 participants
Low

Summary of findings – denosumab vs placebo

BMD Z score (wrist) · adults with β‐thalassaemia and low bone mass

Summary of findings – denosumab showed no clear difference in BMD Z score (wrist) compared with placebo (low certainty).

1 finding from 1 review

Treatment for osteoporosis in people with beta‐thalassaemia 2023 · Cochrane Headline outcomes only
no estimate extracted
1 study · 63 participants
Low

Summary of findings – denosumab vs placebo

Incidence of fractures · adults with β‐thalassaemia and low bone mass

The review found no eligible studies for this question.

1 finding from 1 review

Treatment for osteoporosis in people with beta‐thalassaemia 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Summary of findings – denosumab vs placebo

Mobility · adults with β‐thalassaemia and low bone mass

The review found no eligible studies for this question.

1 finding from 1 review

Treatment for osteoporosis in people with beta‐thalassaemia 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Summary of findings – denosumab vs placebo

Quality of life · adults with β‐thalassaemia and low bone mass

The review found no eligible studies for this question.

1 finding from 1 review

Treatment for osteoporosis in people with beta‐thalassaemia 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Summary of findings – denosumab vs placebo

Adverse effects of treatment · adults with β‐thalassaemia and low bone mass

The review found no eligible studies for this question.

1 finding from 1 review

Treatment for osteoporosis in people with beta‐thalassaemia 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Summary of findings ‐ Niprisan® vs placebo

Frequency, severity or duration of painful crises · people with SCD

1 finding from 1 review

Phytomedicines (medicines derived from plants) for sickle cell disease 2020 · Cochrane Headline outcomes only
no estimate extracted
70 participants
Low

Summary of findings ‐ Ciklavit® vs placebo

Frequency of hospital admissions from severe complications of SCD · people with SCD

The review found no eligible studies for this question.

1 finding from 1 review

Phytomedicines (medicines derived from plants) for sickle cell disease 2020 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found