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%

Avalglucosidase alfa vs alglucosidase alfa

Infusion reactions · adults with LOPD

Avalglucosidase alfa showed no clear difference in infusion reactions compared with alglucosidase alfa (low certainty).

1 finding from 1 review

Enzyme replacement therapy for late‐onset Pompe disease 2023 · Cochrane Headline outcomes only
RR 0.78 [0.42, 1.45]
1 study · 100 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

Avalglucosidase alfa vs alglucosidase alfa

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

Avalglucosidase alfa vs alglucosidase alfa

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

Avalglucosidase alfa vs alglucosidase alfa

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 · 100 participants
Low

Avalglucosidase alfa vs alglucosidase alfa

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

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

1 finding from 1 review

Enzyme replacement therapy for late‐onset Pompe disease 2023 · Cochrane Headline outcomes only
RR 0.92 [0.61, 1.4]
1 study · 100 participants
Low

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

Summary of findings: anti‐pseudomonal antibiotics vs placebo

FEV1 % predicted · adults and children with cystic fibrosis and a positive microbiological isolate of P aeruginosa from a respiratory tract specimen

1 finding from 1 review

Inhaled anti‐pseudomonal antibiotics for long‐term therapy in cystic fibrosis 2022 · Cochrane Headline outcomes only
no estimate extracted
11 studies · 1130 participants
Low

Summary of findings: colistimethate dry powder for inhalation (Colobreathe®) vs tobramycin

Pulmonary exacerbations · adults and children with cystic fibrosis and a positive microbiological isolate of P aeruginosa from a respiratory tract specimen

Summary of findings: colistimethate dry powder for inhalation… showed no clear difference in pulmonary exacerbations: number of pulmonary exacerbations compared with tobramycin (moderate certainty).

1 finding from 1 review

Inhaled anti‐pseudomonal antibiotics for long‐term therapy in cystic fibrosis 2022 · Cochrane Headline outcomes only
RR 1.19 [0.86, 1.64]
1 study · 374 participants
Moderate

Summary of findings: colistimethate dry powder for inhalation (Colobreathe®) vs tobramycin

QoL · adults and children with cystic fibrosis and a positive microbiological isolate of P aeruginosa from a respiratory tract specimen

1 finding from 1 review

Inhaled anti‐pseudomonal antibiotics for long‐term therapy in cystic fibrosis 2022 · Cochrane Headline outcomes only
no estimate extracted
1 study · 374 participants
Low

Summary of findings: colistimethate dry powder for inhalation (Colobreathe®) vs tobramycin

Survival: number of deaths · adults and children with cystic fibrosis and a positive microbiological isolate of P aeruginosa from a respiratory tract specimen

Summary of findings: colistimethate dry powder for inhalation… showed no clear difference in survival: number of deaths compared with tobramycin (low certainty).

1 finding from 1 review

Inhaled anti‐pseudomonal antibiotics for long‐term therapy in cystic fibrosis 2022 · Cochrane Headline outcomes only
RR 0.21 [0.01, 4.32]
1 study · 374 participants
Low

Summary of findings: colistimethate dry powder for inhalation (Colobreathe®) vs tobramycin

Antibiotic resistance: change in mean MIC50 and MIC90 at the end of the trial · adults and children with cystic fibrosis and a positive microbiological isolate of P aeruginosa from a respiratory tract specimen

1 finding from 1 review

Inhaled anti‐pseudomonal antibiotics for long‐term therapy in cystic fibrosis 2022 · Cochrane Headline outcomes only
no estimate extracted
1 study · 374 participants
Low

Summary of findings: colistimethate dry powder for inhalation (Colobreathe®) vs tobramycin

Adverse events: number of treatment related adverse events · adults and children with cystic fibrosis and a positive microbiological isolate of P aeruginosa from a respiratory tract specimen

Summary of findings: colistimethate dry powder for inhalation… may leads to higher adverse events: number of treatment related adverse events compared with tobramycin (low certainty).

1 finding from 1 review

Inhaled anti‐pseudomonal antibiotics for long‐term therapy in cystic fibrosis 2022 · Cochrane Headline outcomes only
RR 1.76 [1.5, 2.08]
1 study · 379 participants
Low

Summary of findings: inhaled TOBI® (IV preparation) vs tobramycin

FEV1 % predicted: absolute change from baseline · adults and children with cystic fibrosis and a positive microbiological isolate of P aeruginosa from a respiratory tract specimen

1 finding from 1 review

Inhaled anti‐pseudomonal antibiotics for long‐term therapy in cystic fibrosis 2022 · Cochrane Headline outcomes only
no estimate extracted
1 study · 32 participants
Very low

Summary of findings: inhaled TOBI® (IV preparation) vs tobramycin

FVC (% predicted) Change from baseline · adults and children with cystic fibrosis and a positive microbiological isolate of P aeruginosa from a respiratory tract specimen

1 finding from 1 review

Inhaled anti‐pseudomonal antibiotics for long‐term therapy in cystic fibrosis 2022 · Cochrane Headline outcomes only
no estimate extracted
1 study · 32 participants
Very low

Summary of findings: inhaled TOBI® (IV preparation) vs tobramycin

Pulmonary exacerbations · adults and children with cystic fibrosis and a positive microbiological isolate of P aeruginosa from a respiratory tract specimen

The review found no eligible studies for this question.

1 finding from 1 review

Inhaled anti‐pseudomonal antibiotics for long‐term therapy in cystic fibrosis 2022 · Cochrane Headline outcomes only
no estimate extracted
Studies not reported
No studies found