Cochrane Review Group

Cystic Fibrosis and Genetic Disorders

114 systematic reviews in Cystic Fibrosis and Genetic Disorders ask 2,280 questions. 37.5% have no studies to answer them, 24.1% rest on very low certainty evidence and 13.8% reach moderate or high certainty.

Reviews

114

Questions

2,280

current versions

No studies

37.5%

Moderate or high

13.8%

No studies 856 · 37.5%Ungraded 58 · 2.5%Very low 550 · 24.1%Low 502 · 22%Moderate 271 · 11.9%High 43 · 1.9%

Ungraded means an effect was reported without a GRADE rating; it is not a gap. No studies means the review found nothing to answer the question. The six categories are a display order, not a score.

Search questions in Cystic Fibrosis and Genetic Disorders

Thinnest evidence

All no studies and very low questions

Question	Effect	Evidence	Status / GRADE
Summary of findings ‐ high‐dose azithromycin vs low‐dose azithromycin Adverse events · children with cystic fibrosis Macrolide antibiotics (including azithromycin) for cystic fibrosis (2024) · Cochrane	no estimate extracted	NR	No studies
Summary of findings ‐ high‐dose azithromycin vs low‐dose azithromycin Hospitalisations · children with cystic fibrosis Macrolide antibiotics (including azithromycin) for cystic fibrosis (2024) · Cochrane	no estimate extracted	NR	No studies
Summary of findings ‐ high‐dose azithromycin vs low‐dose azithromycin New acquisition of CF pathogens: PA · children with cystic fibrosis Macrolide antibiotics (including azithromycin) for cystic fibrosis (2024) · Cochrane	no estimate extracted	NR	No studies
Summary of findings ‐ high‐dose azithromycin vs low‐dose azithromycin Need for additional antibiotics · children with cystic fibrosis Macrolide antibiotics (including azithromycin) for cystic fibrosis (2024) · Cochrane	no estimate extracted	NR	No studies
Summary of findings ‐ high‐dose azithromycin vs low‐dose azithromycin QoL · children with cystic fibrosis Macrolide antibiotics (including azithromycin) for cystic fibrosis (2024) · Cochrane	no estimate extracted	NR	No studies
Summary of findings ‐ nebulised azithromycin vs oral azithromycin FEV1 % predicted: mean change from baseline · children with cystic fibrosis Macrolide antibiotics (including azithromycin) for cystic fibrosis (2024) · Cochrane	no estimate extracted	1 study · 45 participants	Very low
Summary of findings ‐ nebulised azithromycin vs oral azithromycin Pulmonary exacerbations: time to next exacerbation · children with cystic fibrosis Macrolide antibiotics (including azithromycin) for cystic fibrosis (2024) · Cochrane	no estimate extracted	NR	No studies
Summary of findings ‐ nebulised azithromycin vs oral azithromycin Adverse events: number of treatment‐related adverse events · children with cystic fibrosis Macrolide antibiotics (including azithromycin) for cystic fibrosis (2024) · Cochrane	no estimate extracted	1 study · 45 participants	Very low
Summary of findings ‐ nebulised azithromycin vs oral azithromycin Hospitalisations · children with cystic fibrosis Macrolide antibiotics (including azithromycin) for cystic fibrosis (2024) · Cochrane	no estimate extracted	NR	No studies
Summary of findings ‐ nebulised azithromycin vs oral azithromycin New acquisition of CF pathogens: PA (mean change in PA count (log10 PA CFU/g sputum)) · children with cystic fibrosis Macrolide antibiotics (including azithromycin) for cystic fibrosis (2024) · Cochrane	no estimate extracted	1 study · 45 participants	Very low
Summary of findings ‐ nebulised azithromycin vs oral azithromycin Need for additional antibiotics · children with cystic fibrosis Macrolide antibiotics (including azithromycin) for cystic fibrosis (2024) · Cochrane	no estimate extracted	NR	No studies
Summary of findings ‐ nebulised azithromycin vs oral azithromycin QoL: change in PEDsQL‐4 scale · children with cystic fibrosis Macrolide antibiotics (including azithromycin) for cystic fibrosis (2024) · Cochrane	no estimate extracted	1 study · 45 participants	Very low

Reviews 114

Macrolide antibiotics (including azithromycin) for cystic fibrosis

Southern, KW, Solis-Moya, A, et al. · Cochrane Database of Systematic Reviews · 2024

Cystic Fibrosis and Genetic Disorders

Thinnest evidence

Reviews 114

Macrolide antibiotics (including azithromycin) for cystic fibrosis

Bronchoscopy‐guided antimicrobial therapy for cystic fibrosis

Antioxidant supplementation for sickle cell disease

Non‐clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B

Airway clearance techniques compared to no airway clearance techniques for cystic fibrosis

Nebulised hypertonic saline for cystic fibrosis

Bisphosphonates for osteoporosis in people with cystic fibrosis

Conventional chest physiotherapy compared to other airway clearance techniques for cystic fibrosis

Antibiotic strategies for eradicating Pseudomonas aeruginosa in people with cystic fibrosis

Nebuliser systems for drug delivery in cystic fibrosis

Active cycle of breathing technique for cystic fibrosis

Treatment for osteoporosis in people with beta‐thalassaemia

Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del)

Rapamycin and rapalogs for tuberous sclerosis complex

Calcium channel blockers for preventing cardiomyopathy due to iron overload in people with transfusion‐dependent beta thalassaemia

Ataluren and similar compounds (specific therapies for premature termination codon class I mutations) for cystic fibrosis

Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or thalassaemia

Interventions for chronic kidney disease in people with sickle cell disease

Treatment of dental and orthodontic complications in thalassaemia

Enzyme replacement therapy for late‐onset Pompe disease

Interventions for improving adherence to airway clearance treatment and exercise in people with cystic fibrosis

Digital technology for monitoring adherence to inhaled therapies in people with cystic fibrosis

Psychological interventions for improving adherence to inhaled therapies in people with cystic fibrosis

Foetal haemoglobin inducers for reducing blood transfusion in non‐transfusion‐dependent beta‐thalassaemias

Surgery for nasal polyposis in cystic fibrosis