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Search review questions
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 |
|---|---|---|---|
Prophylaxis therapy vs on‐demand therapy with FEIBA Safety of bypassing agents · adults and children with hemophilia A or B with inhibitors Bypassing agent prophylaxis in people with hemophilia A or B with inhibitors (2017) · Cochrane | no estimate extracted | 2 studies · 67 participants | Low |
High‐dose vs low‐dose rFVIIa Overall bleeding rates: number of bleeds per month · adults and children with hemophilia A or B with inhibitors Bypassing agent prophylaxis in people with hemophilia A or B with inhibitors (2017) · Cochrane | no estimate extracted | 1 study · 22 participants | Moderate |
High‐dose vs low‐dose rFVIIa Annualised bleeding rate · adults and children with hemophilia A or B with inhibitors Bypassing agent prophylaxis in people with hemophilia A or B with inhibitors (2017) · Cochrane | no estimate extracted | 1 study · 24 participants | Ungraded |
High‐dose vs low‐dose rFVIIa Target joint bleeding rate · adults and children with hemophilia A or B with inhibitors Bypassing agent prophylaxis in people with hemophilia A or B with inhibitors (2017) · Cochrane | no estimate extracted | 1 study · 22 participants | Moderate |
Intervention NR Cardiac function · infantile‐onset Pompe disease Enzyme replacement therapy for infantile‐onset Pompe disease (2017) · Cochrane | no estimate extracted | 1 study · 18 participants | Low |
Intervention NR Time to ventilation · infantile‐onset Pompe disease Enzyme replacement therapy for infantile‐onset Pompe disease (2017) · Cochrane | no estimate extracted | 1 study · 18 participants | Low |
Intervention NR Survival · infantile‐onset Pompe disease Enzyme replacement therapy for infantile‐onset Pompe disease (2017) · Cochrane | 61 | 1 study · 18 participants | Low |
Intervention NR Motor development · infantile‐onset Pompe disease Enzyme replacement therapy for infantile‐onset Pompe disease (2017) · Cochrane | no estimate extracted | 1 study · 18 participants | Low |
Intervention NR Quality of life · infantile‐onset Pompe disease Enzyme replacement therapy for infantile‐onset Pompe disease (2017) · Cochrane | no estimate extracted | 1 study · 18 participants | Ungraded |
Intervention NR Infusion‐related events · infantile‐onset Pompe disease Enzyme replacement therapy for infantile‐onset Pompe disease (2017) · Cochrane | RR 0.83 [0.4, 1.76] | 1 study · 18 participants | Low |
Intervention NR Other adverse events · infantile‐onset Pompe disease Enzyme replacement therapy for infantile‐onset Pompe disease (2017) · Cochrane | no estimate extracted | 1 study · 18 participants | Low |
Beta‐2 agonists vs placebo FEV1: change from baseline · adults and children with cystic fibrosis Long‐acting inhaled bronchodilators for cystic fibrosis (2017) · Cochrane | no estimate extracted | 1 study · 16 participants | Very low |
LAMAs vs placebo Other measures of lung function: mean change from baseline in FEV1 AUC0-4h (% predicted) after tiotropium 5.0 µg · adults and children with cystic fibrosis Long‐acting inhaled bronchodilators for cystic fibrosis (2017) · Cochrane | no estimate extracted | 2 studies · 770 participants | Moderate |
LAMAs vs placebo Other measures of lung function: mean change from baseline in trough FEV1 (% predicted) after tiotropium 5.0 µg · adults and children with cystic fibrosis Long‐acting inhaled bronchodilators for cystic fibrosis (2017) · Cochrane | no estimate extracted | 2 studies · 772 participants | Moderate |
Beta‐2 agonists vs placebo Participant‐reported outcomes: quality of life (measured by CFQ‐R) · adults and children with cystic fibrosis Long‐acting inhaled bronchodilators for cystic fibrosis (2017) · Cochrane | no estimate extracted | NR | No studies |
Beta‐2 agonists vs placebo Adverse events after tiotropium 5.0 µg · adults and children with cystic fibrosis Long‐acting inhaled bronchodilators for cystic fibrosis (2017) · Cochrane | no estimate extracted | NR | No studies |
Beta‐2 agonists vs placebo Pulmonary exacerbations · adults and children with cystic fibrosis Long‐acting inhaled bronchodilators for cystic fibrosis (2017) · Cochrane | no estimate extracted | NR | No studies |
Beta‐2 agonists vs placebo Other measures of lung function · adults and children with cystic fibrosis Long‐acting inhaled bronchodilators for cystic fibrosis (2017) · Cochrane | no estimate extracted | NR | No studies |
LAMAs vs placebo FEV1: mean change from baseline (L) · adults and children with cystic fibrosis Long‐acting inhaled bronchodilators for cystic fibrosis (2017) · Cochrane | no estimate extracted | 1 study · 34 participants | Low |
LAMAs vs placebo Participant‐reported outcomes: quality of life (measured by CFQ‐R) · adults and children with cystic fibrosis Long‐acting inhaled bronchodilators for cystic fibrosis (2017) · Cochrane | no estimate extracted | 2 studies · 974 participants | Low |
LAMAs vs placebo Adverse events after tiotropium 5.0 µg · adults and children with cystic fibrosis Long‐acting inhaled bronchodilators for cystic fibrosis (2017) · Cochrane | RR 0.96 [0.88, 1.04] | 2 studies · 807 participants | High |
LAMAs vs placebo Pulmonary exacerbations: participants reporting ≥1 pulmonary exacerbation during the trial period after tiotropium 5 µg · adults and children with cystic fibrosis Long‐acting inhaled bronchodilators for cystic fibrosis (2017) · Cochrane | RR 0.6 [0.32, 1.12] | 2 studies · 805 participants | Moderate |
Intervention NR Changes in globotriaosylceramide (Gb3) concentration in plasma · Anderson‐Fabry disease Enzyme replacement therapy for Anderson‐Fabry disease (2016) · Cochrane | no estimate extracted | 2 studies · 39 participants | Very low |
Intervention NR Renal function events (ITT population) · Anderson‐Fabry disease Enzyme replacement therapy for Anderson‐Fabry disease (2016) · Cochrane | no estimate extracted | 1 study · 82 participants | Very low |
Intervention NR Symptoms and complications of disease: cardiac events · Anderson‐Fabry disease Enzyme replacement therapy for Anderson‐Fabry disease (2016) · Cochrane | no estimate extracted | 1 study · 82 participants | Very low |
Intervention NR Symptoms and complications of disease: cerebrovascular events (ITT population) · Anderson‐Fabry disease Enzyme replacement therapy for Anderson‐Fabry disease (2016) · Cochrane | no estimate extracted | 1 study · 82 participants | Very low |
Intervention NR Changes in globotriaosylceramide (Gb3) concentration in plasma and tissue · Anderson‐Fabry disease Enzyme replacement therapy for Anderson‐Fabry disease (2016) · Cochrane | no estimate extracted | NR | No studies |
Intervention NR Death · Anderson‐Fabry disease Enzyme replacement therapy for Anderson‐Fabry disease (2016) · Cochrane | no estimate extracted | 1 study · 36 participants | Very low |
Intervention NR Pain Acroparaesthesia and Fabry crises · Anderson‐Fabry disease Enzyme replacement therapy for Anderson‐Fabry disease (2016) · Cochrane | no estimate extracted | NR | No studies |
Intervention NR Renal function · Anderson‐Fabry disease Enzyme replacement therapy for Anderson‐Fabry disease (2016) · Cochrane | no estimate extracted | NR | No studies |
Intervention NR Symptoms and complications of disease: cardiac events · Anderson‐Fabry disease Enzyme replacement therapy for Anderson‐Fabry disease (2016) · Cochrane | no estimate extracted | 1 study · 29 participants | Very low |
Intervention NR Symptoms and complications of disease: cerebrovascular events · Anderson‐Fabry disease Enzyme replacement therapy for Anderson‐Fabry disease (2016) · Cochrane | no estimate extracted | NR | No studies |
Intervention NR Death · Anderson‐Fabry disease Enzyme replacement therapy for Anderson‐Fabry disease (2016) · Cochrane | no estimate extracted | NR | No studies |
Intervention NR Pain The Brief Pain Inventory severity · Anderson‐Fabry disease Enzyme replacement therapy for Anderson‐Fabry disease (2016) · Cochrane | no estimate extracted | 1 study · 26 participants | Low |
Intervention NR Renal function by creatinine clearance · Anderson‐Fabry disease Enzyme replacement therapy for Anderson‐Fabry disease (2016) · Cochrane | no estimate extracted | 1 study · 24 participants | Very low |
Intervention NR Symptoms and complications of disease:cardiac events · Anderson‐Fabry disease Enzyme replacement therapy for Anderson‐Fabry disease (2016) · Cochrane | no estimate extracted | NR | No studies |
Intervention NR Symptoms and complications of disease: cerebrovascular events · Anderson‐Fabry disease Enzyme replacement therapy for Anderson‐Fabry disease (2016) · Cochrane | no estimate extracted | NR | No studies |
Intervention NR Changes in globotriaosylceramide (Gb3) concentration in plasma · Anderson‐Fabry disease Enzyme replacement therapy for Anderson‐Fabry disease (2016) · Cochrane | no estimate extracted | 1 study · 58 participants | Very low |
Intervention NR Death · Anderson‐Fabry disease Enzyme replacement therapy for Anderson‐Fabry disease (2016) · Cochrane | no estimate extracted | 1 study · 82 participants | Very low |
Intervention NR Pain · Anderson‐Fabry disease Enzyme replacement therapy for Anderson‐Fabry disease (2016) · Cochrane | no estimate extracted | NR | No studies |
Intervention NR SCD knowledge:children Pre‐ and post‐knowledge assessment Duration of · patients with sickle cell disease Interventions for patients and caregivers to improve knowledge of sickle cell disease and recognition of its… (2016) · Cochrane | no estimate extracted | 4 studies · 160 participants | Moderate |
Intervention NR Healthcare utilization clinic visits, emergency room visits and hospital admissions Duration of · patients with sickle cell disease Interventions for patients and caregivers to improve knowledge of sickle cell disease and recognition of its… (2016) · Cochrane | no estimate extracted | 2 studies · 176 participants | Moderate |
Intervention NR Positive coping Coping attempts and coping strategies Duration of · patients with sickle cell disease Interventions for patients and caregivers to improve knowledge of sickle cell disease and recognition of its… (2016) · Cochrane | no estimate extracted | 5 studies · 208 participants | Low |
Intervention NR Depression standardised depression scores · patients with sickle cell disease Interventions for patients and caregivers to improve knowledge of sickle cell disease and recognition of its… (2016) · Cochrane | no estimate extracted | 3 studies · 135 participants | Moderate |
Intervention NR Patient‐reported signs and symptoms/ self‐management · patients with sickle cell disease Interventions for patients and caregivers to improve knowledge of sickle cell disease and recognition of its… (2016) · Cochrane | no estimate extracted | NR | No studies |
Intervention NR SCD knowledge: caregiver Pre‐ and post‐knowledge assessment Duration of · caregivers of patients with sickle cell disease Interventions for patients and caregivers to improve knowledge of sickle cell disease and recognition of its… (2016) · Cochrane | no estimate extracted | 1 study · 39 participants | Low |
Intervention NR Family relationship Pre‐intervention and post‐intervention assessment Home visits and training session for family members Patient‐caregiver relationships assessed pre‐intervention and immediately post‐intervention. · caregivers of patients with sickle cell disease Interventions for patients and caregivers to improve knowledge of sickle cell disease and recognition of its… (2016) · Cochrane | no estimate extracted | 2 studies · 76 participants | Moderate |
Intervention NR Caregiver‐reported signs and symptoms Lead to self management · caregivers of patients with sickle cell disease Interventions for patients and caregivers to improve knowledge of sickle cell disease and recognition of its… (2016) · Cochrane | no estimate extracted | NR | No studies |
Summary of findings ‐ Exercise vs no intervention Adverse events · participants with haemophilia Exercise for haemophilia (2016) · Cochrane | no estimate extracted | NR | No studies |
Summary of findings ‐ Exercise A vs Exercise B Balance: Biodex stability parameters · participants with haemophilia Exercise for haemophilia (2016) · Cochrane | no estimate extracted | 1 study · 30 participants | Very low |