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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 ‐ triple therapy: VX‐152‐tezacaftor‐ivacaftor vs placebo Quality of life: CFQ‐R respiratory domain: absolute change from baseline · adults with cystic fibrosis and with the F508del/MF genotype Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane | no estimate extracted | 1 study · 76 participants | Low |
Summary of findings ‐ triple therapy: VX‐152‐tezacaftor‐ivacaftor vs placebo FEV1 (% predicted): relative change from baseline · adults with cystic fibrosis and with the F508del/MF genotype Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane | no estimate extracted | 1 study · 76 participants | Low |
Summary of findings ‐ triple therapy: VX‐152‐tezacaftor‐ivacaftor vs placebo FEV1 (% predicted): absolute change from baseline · adults with cystic fibrosis and with the F508del/MF genotype Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane | no estimate extracted | 1 study · 76 participants | Low |
Summary of findings ‐ triple therapy: VX‐152‐tezacaftor‐ivacaftor vs placebo Adverse events · adults with cystic fibrosis and with the F508del/MF genotype Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane | no estimate extracted | 1 study · 76 participants | Low |
Summary of findings ‐ triple therapy: VX‐152‐tezacaftor‐ivacaftor vs placebo Time to first pulmonary exacerbation · adults with cystic fibrosis and with the F508del/MF genotype Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane | no estimate extracted | NR | No studies |
Summary of findings ‐ monotherapy: cavosonstat vs placebo Survival · adults and children with cystic fibrosis Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane | no estimate extracted | 1 study · 26 participants | Very low |
Summary of findings ‐ monotherapy: cavosonstat vs placebo Quality of life: total score · adults and children with cystic fibrosis Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene… (2023) · Cochrane | no estimate extracted | NR | No studies |
Intervention NR 50% reduction of tumour sizea · people with TSC Rapamycin and rapalogs for tuberous sclerosis complex (2023) · Cochrane | RR 24.7 [3.5, 173.4] | 2 studies · 162 participants | High |
Intervention NR Frequency of seizure · people with TSC Rapamycin and rapalogs for tuberous sclerosis complex (2023) · Cochrane | no estimate extracted | NR | No studies |
Intervention NR Creatinine level · people with TSC Rapamycin and rapalogs for tuberous sclerosis complex (2023) · Cochrane | no estimate extracted | NR | No studies |
Intervention NR FEV1/FVC ratio · people with TSC Rapamycin and rapalogs for tuberous sclerosis complex (2023) · Cochrane | no estimate extracted | NR | No studies |
Intervention NR Outcomes relevant to participants' well‐being · people with TSC Rapamycin and rapalogs for tuberous sclerosis complex (2023) · Cochrane | no estimate extracted | 1 study · 62 participants | Low |
Intervention NR Adverse events · people with TSC Rapamycin and rapalogs for tuberous sclerosis complex (2023) · Cochrane | RR 1.72 [1.1, 2.67] | 3 studies · 277 participants | Moderate |
Intervention NR Response to skin lesionsb · people with TSC Rapamycin and rapalogs for tuberous sclerosis complex (2023) · Cochrane | RR 5.8 [2.3, 14.5] | 2 studies · 224 participants | High |
Intervention NR Frequency of seizure · people with TSC Rapamycin and rapalogs for tuberous sclerosis complex (2023) · Cochrane | RR 5.3 | 1 study · 366 participants | Moderate |
Intervention NR Number of participants with an increased creatinine level · people with TSC Rapamycin and rapalogs for tuberous sclerosis complex (2023) · Cochrane | RR 0.16 | 1 study · 118 participants | Moderate |
Intervention NR FEV1/FVC ratio · people with TSC Rapamycin and rapalogs for tuberous sclerosis complex (2023) · Cochrane | no estimate extracted | NR | No studies |
Intervention NR Outcomes relevant to participants' well‐being · people with TSC Rapamycin and rapalogs for tuberous sclerosis complex (2023) · Cochrane | no estimate extracted | 1 study · 42 participants | Low |
Intervention NR Adverse events · people with TSC Rapamycin and rapalogs for tuberous sclerosis complex (2023) · Cochrane | RR 1.09 | 5 studies · 680 participants | High |
Intervention NR 50% reduction in tumour size · people with TSC Rapamycin and rapalogs for tuberous sclerosis complex (2023) · Cochrane | no estimate extracted | NR | No studies |
Intervention NR Response to skin lesion · people with TSC Rapamycin and rapalogs for tuberous sclerosis complex (2023) · Cochrane | RR 2.72 [1.76, 4.18] | 2 studies · 187 participants | High |
Amlodipine plus standard iron chelation therapy vs standard iron chelation therapy (alone or with placebo) Cardiac T2* value · or population: people with transfusion‐dependent beta thalassaemia Calcium channel blockers for preventing cardiomyopathy due to iron overload in people with… (2023) · Cochrane | no estimate extracted | 4 studies · 191 participants | Low |
Amlodipine plus standard iron chelation therapy vs standard iron chelation therapy (alone or with placebo) MIC · or population: people with transfusion‐dependent beta thalassaemia Calcium channel blockers for preventing cardiomyopathy due to iron overload in people with… (2023) · Cochrane | no estimate extracted | 3 studies · 138 participants | Low |
Amlodipine plus standard iron chelation therapy vs standard iron chelation therapy (alone or with placebo) LVEF (echocardiography) · or population: people with transfusion‐dependent beta thalassaemia Calcium channel blockers for preventing cardiomyopathy due to iron overload in people with… (2023) · Cochrane | no estimate extracted | 3 studies · 136 participants | Low |
Amlodipine plus standard iron chelation therapy vs standard iron chelation therapy (alone or with placebo) Serum ferritin · or population: people with transfusion‐dependent beta thalassaemia Calcium channel blockers for preventing cardiomyopathy due to iron overload in people with… (2023) · Cochrane | no estimate extracted | 4 studies · 187 participants | Low |
Amlodipine plus standard iron chelation therapy vs standard iron chelation therapy (alone or with placebo) Liver T2* value · or population: people with transfusion‐dependent beta thalassaemia Calcium channel blockers for preventing cardiomyopathy due to iron overload in people with… (2023) · Cochrane | no estimate extracted | 3 studies · 127 participants | Low |
Amlodipine plus standard iron chelation therapy vs standard iron chelation therapy (alone or with placebo) LIC · or population: people with transfusion‐dependent beta thalassaemia Calcium channel blockers for preventing cardiomyopathy due to iron overload in people with… (2023) · Cochrane | no estimate extracted | 2 studies · 123 participants | Moderate |
Amlodipine plus standard iron chelation therapy vs standard iron chelation therapy (alone or with placebo) Adverse events · or population: people with transfusion‐dependent beta thalassaemia Calcium channel blockers for preventing cardiomyopathy due to iron overload in people with… (2023) · Cochrane | no estimate extracted | 5 studies · 234 participants | Very low |
Ataluren vs placebo Health‐related quality of life: mean change from baseline in CFQ‐R respiratory domain score Scale: age‐appropriate versions of the CFQ‐R questionnaire · adults and children with CF with at least one class I mutation (PTC) Ataluren and similar compounds (specific therapies for premature termination codon class I mutations) for… (2023) · Cochrane | no estimate extracted | 2 studies · 506 participants | High |
Ataluren vs placebo Respiratory function: FEV1 % predicted (relative change from baseline) · adults and children with CF with at least one class I mutation (PTC) Ataluren and similar compounds (specific therapies for premature termination codon class I mutations) for… (2023) · Cochrane | no estimate extracted | 1 study · 203 participants | High |
Ataluren vs placebo Adverse events relating to treatment · adults and children with CF with at least one class I mutation (PTC) Ataluren and similar compounds (specific therapies for premature termination codon class I mutations) for… (2023) · Cochrane | no estimate extracted | 2 studies · 517 participants | Moderate |
Ataluren vs placebo Pulmonary exacerbations: protocol‐defined Scale: rate · adults and children with CF with at least one class I mutation (PTC) Ataluren and similar compounds (specific therapies for premature termination codon class I mutations) for… (2023) · Cochrane | no estimate extracted | 1 study · 232 participants | High |
Ataluren vs placebo Nutrition and growth: change in weight (kg), BMI (kg/m2) or height (m) · adults and children with CF with at least one class I mutation (PTC) Ataluren and similar compounds (specific therapies for premature termination codon class I mutations) for… (2023) · Cochrane | no estimate extracted | 2 studies · 506 participants | High |
Ataluren vs placebo Sweat chloride level ‐ change from baseline Scale: mmol/L · adults and children with CF with at least one class I mutation (PTC) Ataluren and similar compounds (specific therapies for premature termination codon class I mutations) for… (2023) · Cochrane | no estimate extracted | 1 study · 194 participants | High |
Ataluren vs placebo Mortality rate · adults and children with CF with at least one class I mutation (PTC) Ataluren and similar compounds (specific therapies for premature termination codon class I mutations) for… (2023) · Cochrane | no estimate extracted | 2 studies · 518 participants | High |
Summary of findings: Comparison 1 ‐ deferiprone (DFP) vs deferoxamine (DFO) Adherence to iron chelation therapy (%, SD) Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or… (2023) · Cochrane | no estimate extracted | 7 studies · 612 participants | Very low |
Summary of findings: Comparison 2 ‐ deferasirox (DFX) vs deferiprone (DFO) Sustained adherence Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or… (2023) · Cochrane | no estimate extracted | NR | No studies |
Summary of findings: Comparison 2 ‐ deferasirox (DFX) vs deferiprone (DFO) QoL Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or… (2023) · Cochrane | no estimate extracted | NR | No studies |
Summary of findings: Comparison 3 ‐ deferiprone (DFP) vs deferasirox (DFX) Adherence to iron chelation (%, SD) Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or… (2023) · Cochrane | no estimate extracted | 1 study · 390 participants | Low |
Summary of findings: Comparison 3 ‐ deferiprone (DFP) vs deferasirox (DFX) SAE (chelation‐related) (n/N) Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or… (2023) · Cochrane | 1.54 [0.44, 5.39] | 1 study · 390 participants | Very low |
Summary of findings: Comparison 3 ‐ deferiprone (DFP) vs deferasirox (DFX) Total SAEs Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or… (2023) · Cochrane | RR 0.95 [0.46, 1.96] | 1 study · 390 participants | Very low |
Summary of findings: Comparison 3 ‐ deferiprone (DFP) vs deferasirox (DFX) All‐cause mortality (n/N) Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or… (2023) · Cochrane | RD 0 | 1 study · 390 participants | Low |
Summary of findings: Comparison 3 ‐ deferiprone (DFP) vs deferasirox (DFX) Sustained adherence Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or… (2023) · Cochrane | no estimate extracted | NR | No studies |
Summary of findings: Comparison 3 ‐ deferiprone (DFP) vs deferasirox (DFX) QoL Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or… (2023) · Cochrane | no estimate extracted | NR | No studies |
Summary of findings: Comparison 4 ‐ deferasirox (DFX) film‐coated tablets vs DFX dispersible tablets Adherence to iron chelation therapy (%, SD) Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or… (2023) · Cochrane | no estimate extracted | 1 study · 91 participants | Very low |
Summary of findings: Comparison 4 ‐ deferasirox (DFX) film‐coated tablets vs DFX dispersible tablets Sustained adherence to iron chelation therapy (%, SD) Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or… (2023) · Cochrane | no estimate extracted | 1 study · 54 participants | Very low |
Summary of findings: Comparison 1 ‐ deferiprone (DFP) vs deferoxamine (DFO) Total reported SAEs (from therapy, disease, non‐adherence) Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or… (2023) · Cochrane | RR 1.43 [0.83, 2.46] | 1 study · 228 participants | Very low |
Summary of findings: Comparison 4 ‐ deferasirox (DFX) film‐coated tablets vs DFX dispersible tablets Incidence of SAEs Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or… (2023) · Cochrane | RR 1.22 [0.62, 2.37] | 1 study · 173 participants | Very low |
Summary of findings: Comparison 4 ‐ deferasirox (DFX) film‐coated tablets vs DFX dispersible tablets All‐cause mortality Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or… (2023) · Cochrane | 7.3 [0.14, 368.15] | 1 study · 173 participants | Very low |
Summary of findings: Comparison 4 ‐ deferasirox (DFX) film‐coated tablets vs DFX dispersible tablets QoL Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or… (2023) · Cochrane | no estimate extracted | NR | No studies |