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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.
856 questions in 87 reviews · Cystic Fibrosis and Genetic Disorders
current versionsNo studies 856 · 100%Ungraded 0 · 0%Very low 0 · 0%Low 0 · 0%Moderate 0 · 0%High 0 · 0%
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 ‐ monotherapy: lumacaftor 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 |
Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (twice daily) 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 |
Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (twice daily) vs placebo FEV1 % predicted: relative change from baseline · 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 |
Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor (twice daily) vs placebo Time to first pulmonary exacerbation · 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 |
Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor 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 |
Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor vs placebo Quality of life: respiratory domain · 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 |
Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor vs placebo FEV1 % predicted: relative change from baseline · 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 |
Summary of findings ‐ dual therapy: lumacaftor plus ivacaftor vs placebo Time to first pulmonary exacerbation · 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 |
Summary of findings ‐ dual therapy: tezacaftor plus ivacaftor vs placebo or ivacaftor alone 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 |
Summary of findings ‐ monotherapy: lumacaftor vs placebo FEV1 % predicted: relative change from baseline · 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 |
Summary of findings ‐ triple therapy: VX‐659‐tezacaftor‐ivacaftor/deutivacaftor vs control Quality of life: total score · adults with cystic fibrosis and either F508del/MF or F508del/F508del 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 ‐ triple therapy: VX‐659‐tezacaftor‐ivacaftor/deutivacaftor vs control Time to first pulmonary exacerbation · adults with cystic fibrosis and either F508del/MF or F508del/F508del 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 ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor/deutivacaftor vs control Quality of life: total score · adults with cystic fibrosis and either F508del/MF or F508del/F508del 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 ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor/deutivacaftor vs control Time to first pulmonary exacerbation · adults with cystic fibrosis and either F508del/MF or F508del/F508del 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 ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor vs control (ivacaftor alone or tezacaftor plus ivacaftor) Quality of life: total score · adults and children aged 12 years and over with cystic fibrosis and either F508del/gating variant or F508del/residual… 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 ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor vs control (ivacaftor alone or tezacaftor plus ivacaftor) Quality of life: CFQ‐R respiratory domain: absolute change from baseline · adults and children aged 12 years and over with cystic fibrosis and either F508del/gating variant or F508del/residual… 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 ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor vs control (ivacaftor alone or tezacaftor plus ivacaftor) FEV1 (% predicted): relative change from baseline · adults and children aged 12 years and over with cystic fibrosis and either F508del/gating variant or F508del/residual… 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 ‐ triple therapy: elexacaftor‐tezacaftor‐ivacaftor vs control (ivacaftor alone or tezacaftor plus ivacaftor) Time to first pulmonary exacerbation · adults and children aged 12 years and over with cystic fibrosis and either F508del/gating variant or F508del/residual… 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 ‐ triple therapy: VX‐440‐tezacaftor‐ivacaftor vs placebo Quality of life: total score · 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: lumacaftor vs placebo Time to first pulmonary exacerbation · 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 |
Summary of findings ‐ triple therapy: VX‐440‐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 ‐ triple therapy: VX‐152‐tezacaftor‐ivacaftor vs placebo Quality of life: total score · 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 ‐ 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 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 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 FEV1/FVC ratio · people with TSC Rapamycin and rapalogs for tuberous sclerosis complex (2023) · Cochrane | no estimate extracted | NR | No studies |
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 |
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) 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 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 5 ‐ deferiprone (DFP) plus deferoxamine (DFO) vs DFP 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 5 ‐ deferiprone (DFP) plus deferoxamine (DFO) vs DFP 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 6 ‐ deferiprone (DFP) plus deferoxamine (DFO) vs DFO All‐cause mortality 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 6 ‐ deferiprone (DFP) plus deferoxamine (DFO) vs 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 6 ‐ deferiprone (DFP) plus deferoxamine (DFO) vs 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 7 ‐ deferiprone (DFP) plus deferoxamine (DFO) vs DFP plus 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 8 ‐ medication management vs standard care Adherence to iron chelation 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 8 ‐ medication management vs standard care SAEs 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 1 ‐ deferiprone (DFP) vs deferoxamine (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 8 ‐ medication management vs standard care Mortality 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 8 ‐ medication management vs standard care 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 |
Angiotensin‐converting enzyme inhibitors vs vitamin C Serious adverse events · children with sickle cell anaemia and microalbuminuria Interventions for chronic kidney disease in people with sickle cell disease (2023) · Cochrane | no estimate extracted | NR | No studies |
Angiotensin‐converting enzyme inhibitors vs vitamin C Mortality due to any cause · children with sickle cell anaemia and microalbuminuria Interventions for chronic kidney disease in people with sickle cell disease (2023) · Cochrane | no estimate extracted | NR | No studies |
Angiotensin‐converting enzyme inhibitors vs vitamin C Quality of life · children with sickle cell anaemia and microalbuminuria Interventions for chronic kidney disease in people with sickle cell disease (2023) · Cochrane | no estimate extracted | NR | No studies |
Hydroxyurea vs placebo Quality of life · people with sickle cell disease Interventions for chronic kidney disease in people with sickle cell disease (2023) · Cochrane | no estimate extracted | NR | No studies |
Angiotensin‐converting enzyme inhibitors vs placebo Serious adverse events · people with sickle cell disease Interventions for chronic kidney disease in people with sickle cell disease (2023) · Cochrane | no estimate extracted | NR | No studies |