Findings

What do systematic reviews say?

167 questions · 167 findings · 8 reviews · Cystic Fibrosis and Genetic Disorders

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No studies found 80 · 47.9%GRADE not reported 1 · 0.6%Very low 45 · 26.9%Low 34 · 20.4%Moderate 7 · 4.2%High 0 · 0%

Alglucosidase alfa vs placebo

QoL (PCS in SF‐36 (measured on a scale from 0 to 100)) · 90 people with LOPD

1 finding from 1 review

Enzyme replacement therapy for late‐onset Pompe disease 2023 · Cochrane Headline outcomes only
no estimate extracted
1 study · 90 participants
Low

Alglucosidase alfa vs placebo

Treatment‐related AEs: total treatment‐emergent adverse events potentially related to treatment · 90 people with LOPD

Alglucosidase alfa showed no clear difference in treatment‐related AEs compared with placebo (low certainty).

1 finding from 1 review

Enzyme replacement therapy for late‐onset Pompe disease 2023 · Cochrane Headline outcomes only
RR 0.94 [0.64, 1.39]
1 study · 90 participants
Low

Alglucosidase alfa plus clenbuterol vs alglucosidase alfa plus placebo

Change from baseline in 6MWT (metres) · 13 people with LOPD

1 finding from 1 review

Enzyme replacement therapy for late‐onset Pompe disease 2023 · Cochrane Headline outcomes only
no estimate extracted
1 study · 10 participants
Very low

Alglucosidase alfa plus clenbuterol vs alglucosidase alfa plus placebo

FVC (% predicted) Change from baseline · 13 people with LOPD

1 finding from 1 review

Enzyme replacement therapy for late‐onset Pompe disease 2023 · Cochrane Headline outcomes only
no estimate extracted
1 study · 11 participants
Very low

Intervention not reported

12MWT: change from baseline in m walked · children and adults with mucopolysaccharidosis type VI

1 finding from 1 review

Enzyme replacement therapy with galsulfase for mucopolysaccharidosis type VI 2021 · Cochrane Headline outcomes only
no estimate extracted
1 study · 38 participants
Very low

Intervention not reported

3MSC: change in 3MSC rates (stairs/min) from baseline · children and adults with mucopolysaccharidosis type VI

1 finding from 1 review

Enzyme replacement therapy with galsulfase for mucopolysaccharidosis type VI 2021 · Cochrane Headline outcomes only
no estimate extracted
1 study · 38 participants
Very low

Intervention not reported

6MWT: change from baseline in m walked · children and adults with mucopolysaccharidosis type VI

1 finding from 1 review

Enzyme replacement therapy with galsulfase for mucopolysaccharidosis type VI 2021 · Cochrane Headline outcomes only
no estimate extracted
1 study · 38 participants
Very low

Intervention not reported

Respiratory tests: absolute change from baseline in FVC (L) · children and adults with mucopolysaccharidosis type VI

1 finding from 1 review

Enzyme replacement therapy with galsulfase for mucopolysaccharidosis type VI 2021 · Cochrane Headline outcomes only
no estimate extracted
1 study · 38 participants
Very low

Intervention not reported

Cardiac function Â · children and adults with mucopolysaccharidosis type VI

The review found no eligible studies for this question.

1 finding from 1 review

Enzyme replacement therapy with galsulfase for mucopolysaccharidosis type VI 2021 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found

Intervention not reported

GAG level: change in urinary GAG level from baseline ųg/mg creatinine · children and adults with mucopolysaccharidosis type VI

1 finding from 1 review

Enzyme replacement therapy with galsulfase for mucopolysaccharidosis type VI 2021 · Cochrane Headline outcomes only
no estimate extracted
1 study · 38 participants
Low

Intervention not reported

AEs: drug‐related AEs · children and adults with mucopolysaccharidosis type VI

It is very uncertain whether the intervention changes AEs: drug‐related AEs (very low certainty).

1 finding from 1 review

Enzyme replacement therapy with galsulfase for mucopolysaccharidosis type VI 2021 · Cochrane Headline outcomes only
RR 1.93 [0.89, 4.17]
1 study · 39 participants
Very low

Summary of findings: ECM vs NECT plus cimetidine

Change in weight (kg) · adults with cystic fibrosis

It is very uncertain whether summary of findings: ECM changes change in weight (kg) compared with NECT plus cimetidine (very low certainty).

1 finding from 1 review

Pancreatic enzyme replacement therapy for people with cystic fibrosis 2020 · Cochrane Headline outcomes only
MD 0.4
1 study · 12 participants
Very low

Summary of findings: ECM vs ECT

Change in BMI · 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

Summary of findings: ECM vs ECT

Abdominal pain frequency or change in frequency · adults and children with cystic fibrosis

It is very uncertain whether summary of findings: ECM changes frequency of bowel symptoms: abdominal pain (% of days affected) compared with ECT (very low certainty).

1 finding from 1 review

Pancreatic enzyme replacement therapy for people with cystic fibrosis 2020 · Cochrane Headline outcomes only
no estimate extracted
2 studies · 41 participants
Very low

Summary of findings: ECM vs ECT

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

Summary of findings: ECM showed no clear difference in CFA: change in FFE (g/day) compared with ECT (low certainty).

1 finding from 1 review

Pancreatic enzyme replacement therapy for people with cystic fibrosis 2020 · Cochrane Headline outcomes only
no estimate extracted
2 studies · 41 participants
Low

Summary of findings: ECM vs ECT

Adverse events · 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

Summary of findings: ECM vs ECT

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

Summary of findings: ECM vs ECMM

Change in weight (kg) · children with cystic fibrosis and proven pancreatic insufficiency

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

Summary of findings: ECM vs ECMM

Change in height · children with cystic fibrosis and proven pancreatic insufficiency

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

Summary of findings: ECM vs ECMM

Change in BMI · children with cystic fibrosis and proven pancreatic insufficiency

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