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Each row is one question a systematic review asked: who, what, compared with what, and which outcome, with the effect it found and how certain that is. Words match anywhere in the field.
2,280 questions in 114 reviews · Cystic Fibrosis and Genetic Disorders
current versionsNo 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.
| Question | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Summary of findings table ‐ Emicizumab 1.5 mg/kg once weekly vs emicizumab 3.0 mg/kg every 2 weeks Annualized joint Bleeding Rate (AjBR) · people without inhibitors Non‐clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B (2024) · Cochrane | no estimate extracted | 1 study · 71 participants | Moderate |
Summary of findings table ‐ Emicizumab 1.5 mg/kg once weekly vs emicizumab 3.0 mg/kg every 2 weeks Annualized spontaneous Bleeding Rate (AsBR) · people without inhibitors Non‐clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B (2024) · Cochrane | no estimate extracted | 1 study · 71 participants | Moderate |
Summary of findings table ‐ Emicizumab 1.5 mg/kg once weekly vs emicizumab 3.0 mg/kg every 2 weeks Proportion of participants with zero bleeds · people without inhibitors Non‐clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B (2024) · Cochrane | RR 1.25 [0.74, 2.1] | 1 study · 71 participants | Moderate |
Summary of findings table ‐ Emicizumab prophylaxis vs on‐demand therapy Proportion with zero bleeds · people with inhibitors Non‐clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B (2024) · Cochrane | RR 11.31 [1.66, 77.3] | 1 study · 53 participants | Moderate |
Summary of findings table ‐ Emicizumab 1.5 mg/kg once weekly vs emicizumab 3.0 mg/kg every 2 weeks Change in total score, Haem‐A‐QoL Scale from: 0 (better) to 100 · people without inhibitors Non‐clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B (2024) · Cochrane | no estimate extracted | 1 study · 63 participants | Low |
Summary of findings table ‐ Emicizumab 1.5 mg/kg once weekly vs emicizumab 3.0 mg/kg every 2 weeks All adverse events · people without inhibitors Non‐clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B (2024) · Cochrane | RR 1.1 [0.04, 2.97] | 1 study · 71 participants | Moderate |
Summary of findings table ‐ Emicizumab 1.5 mg/kg once weekly vs emicizumab 3.0 mg/kg every 2 weeks Serious adverse events · people without inhibitors Non‐clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B (2024) · Cochrane | RR 0.32 [0.04, 2.97] | 1 study · 71 participants | Moderate |
Summary of findings table ‐ Fitusiran prophylaxis vs on‐demand therapy Annualized Bleeding Rate (ABR) ‐ All treated bleeds · people without inhibitors Non‐clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B (2024) · Cochrane | no estimate extracted | 1 study · 119 participants | Moderate |
Summary of findings table ‐ Fitusiran prophylaxis vs on‐demand therapy Annualized joint Bleeding Rate (AjBR) · people without inhibitors Non‐clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B (2024) · Cochrane | no estimate extracted | 1 study · 119 participants | Moderate |
Summary of findings table ‐ Fitusiran prophylaxis vs on‐demand therapy Annualized spontaneous Bleeding Rate (AsBR) · people without inhibitors Non‐clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B (2024) · Cochrane | no estimate extracted | 1 study · 119 participants | Moderate |
Summary of findings table ‐ Fitusiran prophylaxis vs on‐demand therapy Proportion with zero bleeds · people without inhibitors Non‐clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B (2024) · Cochrane | RR 8.1 [2.04, 32.11] | 1 study · 119 participants | Moderate |
Summary of findings table ‐ Fitusiran prophylaxis vs on‐demand therapy Change in total scores, Haem‐A‐QoL Scale from: 0 (better) to 100 · people without inhibitors Non‐clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B (2024) · Cochrane | no estimate extracted | 1 study · 103 participants | Low |
Summary of findings table ‐ Fitusiran prophylaxis vs on‐demand therapy All adverse events · people without inhibitors Non‐clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B (2024) · Cochrane | RR 1.74 [1.21, 2.5] | 1 study · 119 participants | Moderate |
Summary of findings table ‐ Fitusiran prophylaxis vs on‐demand therapy Adverse events ‐ Serious adverse events · people without inhibitors Non‐clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B (2024) · Cochrane | RR 0.5 [0.15, 1.63] | 1 study · 120 participants | Moderate |
Summary of findings table ‐ Emicizumab prophylaxis vs on‐demand therapy Change from baseline in total score ‐ Haem‐A‐QoL Scale from: 0 (better) to 100 · people with inhibitors Non‐clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B (2024) · Cochrane | no estimate extracted | 1 study · 40 participants | Low |
Summary of findings table ‐ Emicizumab prophylaxis vs on‐demand therapy All adverse events · people with inhibitors Non‐clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B (2024) · Cochrane | RR 1.97 [1.26, 3.1] | 1 study · 52 participants | Moderate |
Summary of findings table ‐ Emicizumab prophylaxis vs on‐demand therapy Serious adverse events · people with inhibitors Non‐clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B (2024) · Cochrane | RR 1.32 [0.48, 3.63] | 1 study · 52 participants | Moderate |
Summary of findings table ‐ Fitusiran prophylaxis vs on‐demand therapy Annualized Bleeding Rate (ABR) ‐ All treated bleeds · people with inhibitors Non‐clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B (2024) · Cochrane | no estimate extracted | 1 study · 57 participants | Moderate |
Summary of findings table ‐ Fitusiran prophylaxis vs on‐demand therapy Annualized spontaneous Bleeding Rate (AsBR) · people with inhibitors Non‐clotting factor therapies for preventing bleeds in people with congenital hemophilia A or B (2024) · Cochrane | no estimate extracted | 1 study · 57 participants | Moderate |
Summary of findings for airway clearance techniques vs no airway clearance techniques Lung function: FEV1 % predicted change from baseline · children and adults with cystic fibrosis Airway clearance techniques compared to no airway clearance techniques for cystic fibrosis (2023) · Cochrane | no estimate extracted | 1 participants | Ungraded |
Summary of findings for airway clearance techniques vs no airway clearance techniques Lung function: LCI · children and adults with cystic fibrosis Airway clearance techniques compared to no airway clearance techniques for cystic fibrosis (2023) · Cochrane | no estimate extracted | 2 studies · 58 participants | Low |
Summary of findings for airway clearance techniques vs no airway clearance techniques Exacerbation rate (number of exacerbations/year) · children and adults with cystic fibrosis Airway clearance techniques compared to no airway clearance techniques for cystic fibrosis (2023) · Cochrane | no estimate extracted | NR | No studies |
Summary of findings for airway clearance techniques vs no airway clearance techniques Mucus transport rate (radio tracer clearance) · children and adults with cystic fibrosis Airway clearance techniques compared to no airway clearance techniques for cystic fibrosis (2023) · Cochrane | no estimate extracted | 6 studies · 55 participants | Very low |
Summary of findings for airway clearance techniques vs no airway clearance techniques Expectorated secretions (sputum wet weight) · children and adults with cystic fibrosis Airway clearance techniques compared to no airway clearance techniques for cystic fibrosis (2023) · Cochrane | no estimate extracted | 5 studies · 47 participants | Very low |
Summary of findings for airway clearance techniques vs no airway clearance techniques Exercise tolerance (MSWT) · children and adults with cystic fibrosis Airway clearance techniques compared to no airway clearance techniques for cystic fibrosis (2023) · Cochrane | no estimate extracted | NR | No studies |
Summary of findings for airway clearance techniques vs no airway clearance techniques Well‐being or QoL · children and adults with cystic fibrosis Airway clearance techniques compared to no airway clearance techniques for cystic fibrosis (2023) · Cochrane | no estimate extracted | NR | No studies |
Hypertonic saline 3% to 7% vs isotonic saline FEV1 (% predicted) change from baseline, short‐term · adults and children with cystic fibrosis (stable lung disease) Nebulised hypertonic saline for cystic fibrosis (2023) · Cochrane | no estimate extracted | 4 studies · 246 participants | Very low |
Hypertonic saline 3% to 7% vs isotonic saline LCI · adults and children with cystic fibrosis (during acute exacerbations of lung disease) Nebulised hypertonic saline for cystic fibrosis (2023) · Cochrane | no estimate extracted | NR | No studies |
Hypertonic saline 3% to 7% vs isotonic saline Mortality · adults and children with cystic fibrosis (during acute exacerbations of lung disease) Nebulised hypertonic saline for cystic fibrosis (2023) · Cochrane | no estimate extracted | 2 studies · 142 participants | Low |
Hypertonic saline 3% to 7% vs isotonic saline Measures of sputum clearance · adults and children with cystic fibrosis (during acute exacerbations of lung disease) Nebulised hypertonic saline for cystic fibrosis (2023) · Cochrane | no estimate extracted | NR | No studies |
Hypertonic saline 3% to 7% vs isotonic saline Pulmonary exacerbations · adults and children with cystic fibrosis (during acute exacerbations of lung disease) Nebulised hypertonic saline for cystic fibrosis (2023) · Cochrane | HR 0.86 [0.57, 1.3] | 1 study · 132 participants | Low |
Hypertonic saline 3% to 7% vs isotonic saline Adverse events · adults and children with cystic fibrosis (during acute exacerbations of lung disease) Nebulised hypertonic saline for cystic fibrosis (2023) · Cochrane | no estimate extracted | 2 studies · 142 participants | Very low |
Hypertonic saline vs rhDNase with FEV1 (% predicted) change from baseline, short‐term · adults and children with cystic fibrosis Nebulised hypertonic saline for cystic fibrosis (2023) · Cochrane | no estimate extracted | 1 study · 14 participants | Very low |
Hypertonic saline vs rhDNase with FEV1 (% predicted) change from baseline, long‐term · adults and children with cystic fibrosis Nebulised hypertonic saline for cystic fibrosis (2023) · Cochrane | no estimate extracted | 1 study · 47 participants | Very low |
Hypertonic saline vs rhDNase with LCI · adults and children with cystic fibrosis Nebulised hypertonic saline for cystic fibrosis (2023) · Cochrane | no estimate extracted | NR | No studies |
Hypertonic saline vs rhDNase with Mortality · adults and children with cystic fibrosis Nebulised hypertonic saline for cystic fibrosis (2023) · Cochrane | no estimate extracted | NR | No studies |
Hypertonic saline vs rhDNase with Measures of sputum clearance · adults and children with cystic fibrosis Nebulised hypertonic saline for cystic fibrosis (2023) · Cochrane | no estimate extracted | NR | No studies |
Hypertonic saline 3% to 7% vs isotonic saline FEV1 (% predicted) change from baseline, long‐term · adults and children with cystic fibrosis (stable lung disease) Nebulised hypertonic saline for cystic fibrosis (2023) · Cochrane | no estimate extracted | 1 study · 134 participants | Low |
Hypertonic saline vs rhDNase with Pulmonary exacerbations · adults and children with cystic fibrosis Nebulised hypertonic saline for cystic fibrosis (2023) · Cochrane | no estimate extracted | 1 study · 47 participants | Very low |
Hypertonic saline vs rhDNase with Adverse events · adults and children with cystic fibrosis Nebulised hypertonic saline for cystic fibrosis (2023) · Cochrane | no estimate extracted | 1 study · 47 participants | Very low |
Hypertonic saline vs amiloride FEV1: change from baseline, short‐term · adults and children with cystic fibrosis Nebulised hypertonic saline for cystic fibrosis (2023) · Cochrane | no estimate extracted | NR | No studies |
Hypertonic saline vs amiloride FEV1: change from baseline, long‐term · adults and children with cystic fibrosis Nebulised hypertonic saline for cystic fibrosis (2023) · Cochrane | no estimate extracted | NR | No studies |
Hypertonic saline vs amiloride LCI · adults and children with cystic fibrosis Nebulised hypertonic saline for cystic fibrosis (2023) · Cochrane | no estimate extracted | NR | No studies |
Hypertonic saline vs amiloride Mortality · adults and children with cystic fibrosis Nebulised hypertonic saline for cystic fibrosis (2023) · Cochrane | no estimate extracted | NR | No studies |
Hypertonic saline vs amiloride Measures of sputum clearance · adults and children with cystic fibrosis Nebulised hypertonic saline for cystic fibrosis (2023) · Cochrane | no estimate extracted | 1 study · 12 participants | Very low |
Hypertonic saline vs amiloride Pulmonary exacerbations · adults and children with cystic fibrosis Nebulised hypertonic saline for cystic fibrosis (2023) · Cochrane | no estimate extracted | NR | No studies |
Hypertonic saline vs amiloride Adverse events · adults and children with cystic fibrosis Nebulised hypertonic saline for cystic fibrosis (2023) · Cochrane | no estimate extracted | NR | No studies |
Hypertonic saline vs sodium‐2‐mercaptoethane sulphonate (Mistabron®) FEV1: short‐term · adults and children with cystic fibrosis Nebulised hypertonic saline for cystic fibrosis (2023) · Cochrane | no estimate extracted | NR | No studies |
Hypertonic saline 3% to 7% vs isotonic saline LCI · adults and children with cystic fibrosis (stable lung disease) Nebulised hypertonic saline for cystic fibrosis (2023) · Cochrane | no estimate extracted | 1 study · 10 participants | Very low |
Hypertonic saline vs sodium‐2‐mercaptoethane sulphonate (Mistabron®) FEV1: long‐term · adults and children with cystic fibrosis Nebulised hypertonic saline for cystic fibrosis (2023) · Cochrane | no estimate extracted | NR | No studies |