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

Interventions for preventing silent cerebral infarcts in people with sickle cell disease

Estcourt, LJ, Kimber, C, et al. · Cochrane Database of Systematic Reviews · 2020

Cystic Fibrosis and Genetic Disorders

Thinnest evidence

Reviews 114

Interventions for preventing silent cerebral infarcts in people with sickle cell disease

Glucocorticoid replacement regimens for treating congenital adrenal hyperplasia

Transcutaneous electrical nerve stimulation (TENS) for pain management in sickle cell disease

Probiotics for people with cystic fibrosis

Physical therapies for postural abnormalities in people with cystic fibrosis

Oral non‐steroidal anti‐inflammatory drug therapy for lung disease in cystic fibrosis

Inhaled corticosteroids for cystic fibrosis

Once‐daily versus multiple‐daily dosing with intravenous aminoglycosides for cystic fibrosis

Positive expiratory pressure physiotherapy for airway clearance in people with cystic fibrosis

Treatment for avascular necrosis of bone in people with sickle cell disease

Statins for children with familial hypercholesterolemia

Antioxidant supplementation for lung disease in cystic fibrosis

Enzyme replacement therapy with laronidase (Aldurazyme®) for treating mucopolysaccharidosis type I

Potentiators (specific therapies for class III and IV mutations) for cystic fibrosis

Splenectomy for people with thalassaemia major or intermedia

Magnesium for treating sickle cell disease

Antifibrinolytic therapy for preventing oral bleeding in patients with haemophilia or Von Willebrand disease undergoing minor oral surgery or dental extractions

Treatments for preventing recurrence of infection with Pseudomonas aeruginosa in people with cystic fibrosis

Interventions for treating neuropathic pain in people with sickle cell disease

Drugs for preventing red blood cell dehydration in people with sickle cell disease

Vitamin A and beta (β)‐carotene supplementation for cystic fibrosis

Standard (head‐down tilt) versus modified (without head‐down tilt) postural drainage in infants and young children with cystic fibrosis

Folate supplementation in people with sickle cell disease

Antifibrinolytic therapy for preventing oral bleeding in people on anticoagulants undergoing minor oral surgery or dental extractions

Ursodeoxycholic acid for cystic fibrosis‐related liver disease