Findings

What do systematic reviews say?

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Evidence found and GRADE certainty

16 questions · 16 findings · 9 reviews · Cystic Fibrosis and Genetic Disorders

No studies found 2 · 12.5%GRADE not reported 0 · 0%Very low 1 · 6.3%Low 4 · 25%Moderate 8 · 50%High 1 · 6.3%

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

Intervention not reported

Adverse events ‐ minor Infections Clinical events experienced by children during the 1 year period commencing at entry to the trial · 117 children with homozygous sickle cell (SS) disease

The intervention showed no clear difference in adverse events ‐ minor Infections Clinical events experienced by… (low certainty).

1 finding from 1 review

Cycled inhaled tobramycin vs culture‐based TNS

Adverse effects of antibiotics: proportion of participants with one or more serious adverse event · adults and children with cystic fibrosis and a positive microbiological isolate of P aeruginosa from a respiratory tract specimen

Cycled inhaled tobramycin (TNS) probably leads to lower adverse effects of antibiotics: proportion of participants with one… compared with culture‐based TNS (moderate certainty).

1 finding from 1 review

Summary of findings: tobramycin for inhalation powder vs tobramycin

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

Summary of findings: tobramycin for inhalation powder showed no clear difference in adverse events: number of any adverse event reported compared with tobramycin (moderate certainty).

1 finding from 1 review

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

Summary of findings: aztreonam lysine for inhalation 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: aztreonam lysine for inhalation probably leads to higher adverse events: number of treatment‐related adverse events compared with tobramycin (moderate certainty).

1 finding from 1 review

Summary of findings: continuous cycles alternating aztreonam lysine for inhalation with tobramycin for inhalation solution vs continuous cycles alternating placebo with tobramycin

Adverse events: any adverse event in the comparative phase · adults and children with cystic fibrosis and a positive microbiological isolate of P aeruginosa from a respiratory tract specimen

Summary of findings: continuous cycles alternating aztreonam lysine… showed no clear difference in adverse events: any adverse event in the comparative phase compared with continuous cycles alternating placebo with tobramycin (low certainty).

1 finding from 1 review

Summary of findings: amikacin liposome inhalation suspension (ALIS) vs tobramycin

Number experiencing adverse effects · adults and children with cystic fibrosis and a positive microbiological isolate of P aeruginosa from a respiratory tract specimen

Summary of findings: amikacin liposome inhalation suspension (ALIS) showed no clear difference in adverse events: number of participants experiencing any TEAE compared with tobramycin (moderate certainty).

1 finding from 1 review

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