Findings

What do systematic reviews say?

7 questions · 7 findings · 3 reviews · Cystic Fibrosis and Genetic Disorders

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No studies found 0 · 0%GRADE not reported 0 · 0%Very low 2 · 28.6%Low 3 · 42.9%Moderate 2 · 28.6%High 0 · 0%

Summary of findings: cycled vs culture‐based antibiotic therapy

Adverse events: severe adverse events (total) · children (aged 1 to 12 years) with CF and a newly isolated PA infection

Summary of findings: cycled showed no clear difference in adverse events: severe adverse events (total) compared with culture‐based antibiotic therapy (moderate certainty).

1 finding from 1 review

Treatments for preventing recurrence of infection with Pseudomonas aeruginosa in people with cystic fibrosis 2019 · Cochrane Headline outcomes only
RR 0.65 [0.39, 1.11]
1 study · 253 participants
Moderate

Summary of findings ‐ Inhaled mannitol vs control (non‐respirable mannitol)

Adverse events relating to treatment Scale: mild, moderate, severe and total · adults, children and young people with CF

1 finding from 1 review

Inhaled mannitol for cystic fibrosis 2020 · Cochrane Headline outcomes only
no estimate extracted
123 participants
Low

Summary of findings ‐ Inhaled mannitol vs dornase alfa

Adverse events relating to treatment Scale: mild, moderate, severe and total · children and young adults with CF

1 finding from 1 review

Inhaled mannitol for cystic fibrosis 2020 · Cochrane Headline outcomes only
no estimate extracted
231 participants
Very low

Summary of findings ‐ Inhaled mannitol plus dornase alfa vs dornase alfa

Adverse events relating to treatment Scale: mild, moderate, severe and total · children and young adults with CF

1 finding from 1 review

Inhaled mannitol for cystic fibrosis 2020 · Cochrane Headline outcomes only
no estimate extracted
231 participants
Very low

Summary of findings ‐ 400 mg inhaled mannitol vs 50 mg inhaled mannitol

Adverse events relating to treatment Scale: mild, moderate, severe and total · adults, children and young people with CF

1 finding from 1 review

Inhaled mannitol for cystic fibrosis 2020 · Cochrane Headline outcomes only
no estimate extracted
2 studies · 600 participants
Moderate

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

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