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 ‐ monotherapy: cavosonstat 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

Amlodipine plus standard iron chelation therapy vs standard chelation therapy with or without placebo

Cardiac T2* value · or population: people with transfusion‐dependent beta thalassaemia

1 finding from 1 review

Calcium channel blockers for preventing cardiomyopathy due to iron overload in people with transfusion‐dependent beta… 2023 · Cochrane Headline outcomes only
no estimate extracted
4 studies · 191 participants
Low

Amlodipine plus standard iron chelation therapy vs standard chelation therapy with or without placebo

MIC · or population: people with transfusion‐dependent beta thalassaemia

1 finding from 1 review

Calcium channel blockers for preventing cardiomyopathy due to iron overload in people with transfusion‐dependent beta… 2023 · Cochrane Headline outcomes only
no estimate extracted
3 studies · 138 participants
Low

Amlodipine plus standard iron chelation therapy vs standard chelation therapy with or without placebo

LVEF (echocardiography) · or population: people with transfusion‐dependent beta thalassaemia

1 finding from 1 review

Calcium channel blockers for preventing cardiomyopathy due to iron overload in people with transfusion‐dependent beta… 2023 · Cochrane Headline outcomes only
no estimate extracted
3 studies · 136 participants
Low

Amlodipine plus standard iron chelation therapy vs standard chelation therapy with or without placebo

Ferritin (µg/L) · or population: people with transfusion‐dependent beta thalassaemia

1 finding from 1 review

Calcium channel blockers for preventing cardiomyopathy due to iron overload in people with transfusion‐dependent beta… 2023 · Cochrane Headline outcomes only
no estimate extracted
4 studies · 187 participants
Low

Amlodipine plus standard iron chelation therapy vs standard chelation therapy with or without placebo

Liver T2* value · or population: people with transfusion‐dependent beta thalassaemia

1 finding from 1 review

Calcium channel blockers for preventing cardiomyopathy due to iron overload in people with transfusion‐dependent beta… 2023 · Cochrane Headline outcomes only
no estimate extracted
3 studies · 127 participants
Low

Amlodipine plus standard iron chelation therapy vs standard chelation therapy with or without placebo

LIC · or population: people with transfusion‐dependent beta thalassaemia

1 finding from 1 review

Calcium channel blockers for preventing cardiomyopathy due to iron overload in people with transfusion‐dependent beta… 2023 · Cochrane Headline outcomes only
no estimate extracted
2 studies · 123 participants
Moderate

Amlodipine plus standard iron chelation therapy vs standard chelation therapy with or without placebo

Adverse events · or population: people with transfusion‐dependent beta thalassaemia

1 finding from 1 review

Calcium channel blockers for preventing cardiomyopathy due to iron overload in people with transfusion‐dependent beta… 2023 · Cochrane Headline outcomes only
no estimate extracted
5 studies · 234 participants
Very low

Ataluren vs placebo

Quality of life: CFQ‐R respiratory domain: absolute change from baseline · adults and children with CF with at least one class I mutation (PTC)

1 finding from 1 review

Ataluren and similar compounds (specific therapies for premature termination codon class I mutations) for cystic… 2023 · Cochrane Headline outcomes only
no estimate extracted
2 studies · 506 participants
High

Ataluren vs placebo

FEV1 % predicted: relative change from baseline · adults and children with CF with at least one class I mutation (PTC)

1 finding from 1 review

Ataluren and similar compounds (specific therapies for premature termination codon class I mutations) for cystic… 2023 · Cochrane Headline outcomes only
no estimate extracted
1 study · 203 participants
High

Ataluren vs placebo

Adverse events relating to treatment Scale: mild, moderate, severe and total · adults and children with CF with at least one class I mutation (PTC)

1 finding from 1 review

Ataluren and similar compounds (specific therapies for premature termination codon class I mutations) for cystic… 2023 · Cochrane Headline outcomes only
no estimate extracted
2 studies · 517 participants
Moderate

Ataluren vs placebo

Pulmonary exacerbations · adults and children with CF with at least one class I mutation (PTC)

1 finding from 1 review

Ataluren and similar compounds (specific therapies for premature termination codon class I mutations) for cystic… 2023 · Cochrane Headline outcomes only
no estimate extracted
1 study · 232 participants
High

Ataluren vs placebo

Nutrition and growth Change in: a) weight (kg) b) BMI (kg/m2) c) height (m) · adults and children with CF with at least one class I mutation (PTC)

1 finding from 1 review

Ataluren and similar compounds (specific therapies for premature termination codon class I mutations) for cystic… 2023 · Cochrane Headline outcomes only
no estimate extracted
2 studies · 506 participants
High

Ataluren vs placebo

Sweat chloride level ‐ change from baseline Scale: mmol/L · adults and children with CF with at least one class I mutation (PTC)

1 finding from 1 review

Ataluren and similar compounds (specific therapies for premature termination codon class I mutations) for cystic… 2023 · Cochrane Headline outcomes only
no estimate extracted
1 study · 194 participants
High

Ataluren vs placebo

Mortality rate · adults and children with CF with at least one class I mutation (PTC)

1 finding from 1 review

Ataluren and similar compounds (specific therapies for premature termination codon class I mutations) for cystic… 2023 · Cochrane Headline outcomes only
no estimate extracted
2 studies · 518 participants
High

Hydroxyurea vs placebo

Reduction or prevention of kidney disease progression · people with sickle cell disease

1 finding from 1 review

Interventions for chronic kidney disease in people with sickle cell disease 2023 · Cochrane Headline outcomes only
no estimate extracted
1 study · 142 participants
Very low

Hydroxyurea vs placebo

Serious adverse events · people with sickle cell disease

Hydroxyurea showed no clear difference in serious adverse events compared with placebo (low certainty).

1 finding from 1 review

Interventions for chronic kidney disease in people with sickle cell disease 2023 · Cochrane Headline outcomes only
RR 0.39 [0.13, 1.16]
1 study · 193 participants
Low

Hydroxyurea vs placebo

Quality of life · people with sickle cell disease

The review found no eligible studies for this question.

1 finding from 1 review

Interventions for chronic kidney disease in people with sickle cell disease 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Angiotensin‐converting enzyme inhibitors vs placebo

Reduction or prevention of kidney disease progression · people with sickle cell disease

1 finding from 1 review

Interventions for chronic kidney disease in people with sickle cell disease 2023 · Cochrane Headline outcomes only
no estimate extracted
1 study · 22 participants
Very low

Angiotensin‐converting enzyme inhibitors vs placebo

Serious adverse events · people with sickle cell disease

The review found no eligible studies for this question.

1 finding from 1 review

Interventions for chronic kidney disease in people with sickle cell disease 2023 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found