Findings

What do systematic reviews say?

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Evidence found and GRADE certainty

64 questions · 65 findings · 17 reviews · Cystic Fibrosis and Genetic Disorders

No studies found 13 · 20%GRADE not reported 0 · 0%Very low 8 · 12.3%Low 19 · 29.2%Moderate 22 · 33.8%High 3 · 4.6%

Antifibrinolytic therapy (tranexamic acid or epsilon aminocaproic acid) vs Placebo, no intervention or usual care (with or without placebo)

Side effects or other adverse events · People with haemophilia (A or B) undergoing minor oral surgery or dental extractions

Antifibrinolytic therapy (tranexamic acid or epsilon aminocaproic… showed no clear difference in side effects or other adverse events compared with placebo, no intervention or usual care (with or without placebo) (low certainty).

1 finding from 1 review

Antifibrinolytic therapy - locally-applied tranexamic acid (TXA) mouthwash/solution (SoF eligibility: TXA or EACA; all included trials used topical TXA) vs Placebo mouthwash

Adverse effects requiring withdrawal/discontinuation of study medication · People on continuous VKA treatment undergoing minor oral surgery or dental extractions (eligibility included DOACs and all ages; all four…

Antifibrinolytic therapy - locally-applied tranexamic acid (TXA)… showed no clear difference in side effects or other adverse events requiring withdrawal compared with placebo (placebo solution/mouthwash) (moderate certainty).

1 finding from 1 review

Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor vs control (ivacaftor alone or tezacaftor plus ivacaftor)

Adverse events ‐ treatment emergent · adults and children aged 12 years and over with cystic fibrosis and either F508del/gating variant or F508del/residual function genotype

Summary of findings ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor showed no clear difference in adverse events: number of participants with treatment‐emergent… compared with control (ivacaftor alone or tezacaftor plus ivacaftor) (moderate certainty).

1 finding from 1 review

Summary of findings: Single vs combination intravenous anti‐pseudomonal antibiotic therapy

Adverse events during treatment · adults and children with cystic fibrosis

The review found no eligible studies for this question.

2 findings from 1 review

Summary of findings table ‐ Emicuzimab 1.5 mg/kg weekly prophylaxis vs on‐demand therapy

All adverse events · people without inhibitors

Summary of findings table ‐ Emicuzimab 1.5 mg/kg weekly prophylaxis probably leads to higher all adverse events compared with on‐demand therapy (moderate certainty).

1 finding from 1 review