Findings

What do systematic reviews say?

632 questions · 636 findings · 33 reviews · Cystic Fibrosis and Genetic Disorders

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No studies found 197 · 31%GRADE not reported 13 · 2%Very low 133 · 20.9%Low 159 · 25%Moderate 121 · 19%High 13 · 2%

DFP and DFO vs deferoxamine

Adherence to iron chelation therapy (%, SD)

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
5 studies · 281 participants
Low

DFP and DFO vs DFP and DFX

Adherence to iron chelation therapy ‐ not reported

Summary of findings: Comparison 7 ‐ deferiprone (DFP) plus… may leads to lower adherence to iron chelation therapy rates (n, N) compared with DFP plus deferasirox (DFX) (low certainty).

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
RR 0.84 [0.72, 0.99]
1 study · 96 participants
Low

Deferasirox vs deferiprone (DFO)

Adherence to iron chelation therapy (%, SD)

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
3 studies · 452 participants
Very low

Summary of findings ‐ Hydrotherapy vs no exercise

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

Summary of findings ‐ Hydrotherapy vs no exercise

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

Summary of findings ‐ Hydrotherapy vs no exercise

Pain · 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

Summary of findings ‐ Hydrotherapy vs no exercise

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

Summary of findings ‐ Hydrotherapy vs no exercise

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 · 20 participants
Very low

Summary of findings ‐ Hydrotherapy vs no exercise

Strength: knee flexor and extensor strength · participants with haemophilia

1 finding from 1 review

Exercise for haemophilia 2016 · Cochrane Headline outcomes only
no estimate extracted
1 study · 20 participants
Very low

Summary of findings ‐ monotherapy: lumacaftor vs placebo

Survival · adults and children with cystic fibrosis

1 finding from 1 review

Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
no estimate extracted
2 studies · 147 participants
Low

Summary of findings ‐ monotherapy: lumacaftor vs placebo

Quality of life ‐ total score · adults and children with cystic fibrosis

The review found no eligible studies for this question.

1 finding from 1 review

Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Summary of findings ‐ monotherapy: lumacaftor vs placebo

Quality of life: CFQ‐R respiratory domain: absolute change from baseline · adults and children with cystic fibrosis

1 finding from 1 review

Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
no estimate extracted
1 study · 85 participants
Low

Summary of findings ‐ monotherapy: lumacaftor vs placebo

FEV1 % predicted: relative change from baseline · adults and children with cystic fibrosis

The review found no eligible studies for this question.

1 finding from 1 review

Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Summary of findings ‐ monotherapy: lumacaftor vs placebo

FEV1 % predicted: absolute change from baseline · adults and children with cystic fibrosis

1 finding from 1 review

Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
no estimate extracted
1 study · 61 participants
Moderate

Summary of findings ‐ monotherapy: lumacaftor vs placebo

Adverse events · adults and children with cystic fibrosis

1 finding from 1 review

Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
no estimate extracted
2 studies · 115 participants
Very low

Summary of findings ‐ monotherapy: lumacaftor vs placebo

Time to first pulmonary exacerbation · adults and children with cystic fibrosis

The review found no eligible studies for this question.

1 finding from 1 review

Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Summary of findings ‐ monotherapy: cavosonstat vs placebo

Quality of life: CFQ‐R respiratory domain: absolute change from baseline · adults and children with cystic fibrosis

1 finding from 1 review

Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
no estimate extracted
1 study · 24 participants
Very low

Summary of findings ‐ monotherapy: cavosonstat vs placebo

FEV1 % predicted: relative change from baseline · adults and children with cystic fibrosis

The review found no eligible studies for this question.

1 finding from 1 review

Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Summary of findings ‐ monotherapy: cavosonstat vs placebo

FEV1 % predicted: absolute change from baseline · adults and children with cystic fibrosis

1 finding from 1 review

Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
no estimate extracted
1 study · 26 participants
Very low

Summary of findings ‐ monotherapy: cavosonstat vs placebo

Adverse events: occurring in at least 10% of cavosonstat treated participants · adults and children with cystic fibrosis

1 finding from 1 review

Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants… 2023 · Cochrane Headline outcomes only
no estimate extracted
1 study · 26 participants
Very low