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%

Cipaglucosidase plus miglustat vs alglucosidase alfa plus placebo

Infusion reactions · adults with LOPD

Cipaglucosidase plus miglustat showed no clear difference in infusion reactions compared with alglucosidase alfa plus placebo (low certainty).

1 finding from 1 review

Enzyme replacement therapy for late‐onset Pompe disease 2023 · Cochrane Headline outcomes only
RR 0.94 [0.49, 1.8]
1 study · 123 participants
Low

Cipaglucosidase plus miglustat vs alglucosidase alfa plus placebo

Need for respiratory support · 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

Cipaglucosidase plus miglustat vs alglucosidase alfa plus placebo

Use of a walking aid / wheelchair · 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

Cipaglucosidase plus miglustat vs alglucosidase alfa plus placebo

QoL · adults 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 · 121 participants
Low

Cipaglucosidase plus miglustat vs alglucosidase alfa plus placebo

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

Cipaglucosidase plus miglustat showed no clear difference in treatment‐related AEs: total treatment‐emergent adverse events… compared with alglucosidase alfa plus placebo (low certainty).

1 finding from 1 review

Enzyme replacement therapy for late‐onset Pompe disease 2023 · Cochrane Headline outcomes only
RR 0.83 [0.49, 1.4]
1 study · 123 participants
Low

Alglucosidase alfa vs placebo

Need for respiratory support · 90 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 vs placebo

Use of a walking aid / wheelchair · 90 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 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

Single foetal haemoglobin inducer at any dose or duration vs placebo or usual care

Frequency of transfusion (transfused volume/year) · 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

Transfusion‐free interval (days) · 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

Frequency of transfusion (transfused volume/year) · 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

Transfusion‐free interval (days) · 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

Haemoglobin (g/dL) · people with non‐transfusion‐dependent β‐thalassaemia

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
1 study · 54 participants
Low

Two or more foetal haemoglobin inducers at any dose or duration vs a usual management protocol, placebo or a single foetal haemoglobin inducer

Measures of HbF · 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

Two or more foetal haemoglobin inducers at any dose or duration vs a usual management protocol, placebo or a single foetal haemoglobin inducer

Adverse events · people with non‐transfusion‐dependent β‐thalassaemia

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
2 studies · 104 participants
Very low

Single foetal haemoglobin inducer at any dose or duration vs placebo or usual care

Haemoglobin (g/dL) · people with non‐transfusion‐dependent β‐thalassaemia

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
1 study · 35 participants
Very low