Gap finder

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.

Limit to evidence status or GRADE (optional)

2,280 questions in 114 reviews · Cystic Fibrosis and Genetic Disorders

current versions
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.

QuestionEffectEvidenceStatus / GRADE
Summary of findings: Comparison 5 ‐ deferiprone (DFP) plus deferoxamine (DFO) vs DFP
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 extracted4 studies · 369 participantsLow
Summary of findings: Comparison 5 ‐ deferiprone (DFP) plus deferoxamine (DFO) vs DFP
Incidence of SAEs
Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or… (2023) · Cochrane
RR 0.15 [0.01, 2.81]1 study · 213 participantsLow
Summary of findings: Comparison 5 ‐ deferiprone (DFP) plus deferoxamine (DFO) vs DFP
All‐cause mortality
Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or… (2023) · Cochrane
0.77 [0.17, 3.42]2 studies · 237 participantsVery low
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 extractedNRNo 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 extractedNRNo studies
Summary of findings: Comparison 6 ‐ deferiprone (DFP) plus deferoxamine (DFO) vs 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 extracted5 studies · 281 participantsLow
Summary of findings: Comparison 6 ‐ deferiprone (DFP) plus deferoxamine (DFO) vs DFO
Incidence of SAEs
Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or… (2023) · Cochrane
no estimate extracted4 studies · 180 participantsLow
Summary of findings: Comparison 1 ‐ deferiprone (DFP) vs deferoxamine (DFO)
All‐cause mortality
Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or… (2023) · Cochrane
RR 0.47 [0.18, 1.21]3 studies · 376 participantsVery low
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 extractedNRNo 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 extractedNRNo 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 extractedNRNo studies
Summary of findings: Comparison 7 ‐ deferiprone (DFP) plus deferoxamine (DFO) vs DFP plus deferasirox (DFX)
Adherence to iron chelation therapy rates (n, N)
Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or… (2023) · Cochrane
RR 0.84 [0.72, 0.99]1 study · 96 participantsLow
Summary of findings: Comparison 7 ‐ deferiprone (DFP) plus deferoxamine (DFO) vs DFP plus deferasirox (DFX)
Incidence of SAEs
Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or… (2023) · Cochrane
1 [0.06, 16.22]1 study · 96 participantsVery low
Summary of findings: Comparison 7 ‐ deferiprone (DFP) plus deferoxamine (DFO) vs DFP plus deferasirox (DFX)
All‐cause mortality ‐ at 1 year ‐ trial end
Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or… (2023) · Cochrane
RD 01 study · 96 participantsVery low
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 extractedNRNo studies
Summary of findings: Comparison 7 ‐ deferiprone (DFP) plus deferoxamine (DFO) vs DFP plus deferasirox (DFX)
QoL
Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or… (2023) · Cochrane
no estimate extracted1 study · 96 participantsUngraded
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 extractedNRNo 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 extractedNRNo 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 extractedNRNo 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 extractedNRNo 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 extractedNRNo studies
Summary of findings: Comparison 8 ‐ medication management vs standard care
QoL PedsQLTM total score
Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or… (2023) · Cochrane
no estimate extracted1 study · 48 participantsVery low
Summary of findings: Comparison 1 ‐ deferiprone (DFP) vs deferoxamine (DFO)
QoL (
Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or… (2023) · Cochrane
no estimate extracted1 study · 1 participantsVery low
Summary of findings: Comparison 2 ‐ deferasirox (DFX) vs deferiprone (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 extracted3 studies · 452 participantsVery low
Summary of findings: Comparison 2 ‐ deferasirox (DFX) vs deferiprone (DFO)
SAEs Thalassaemia‐related SAEs
Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or… (2023) · Cochrane
RR 0.95 [0.41, 2.17]2 studies · 247 participantsVery low
Summary of findings: Comparison 2 ‐ deferasirox (DFX) vs deferiprone (DFO)
SAEs SCD‐related SAEs
Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or… (2023) · Cochrane
no estimate extracted1 study · 195 participantsVery low
Summary of findings: Comparison 2 ‐ deferasirox (DFX) vs deferiprone (DFO)
All‐cause mortality
Interventions for improving adherence to iron chelation therapy in people with sickle cell disease or… (2023) · Cochrane
0.96 [0.06, 15.42]2 studies · 240 participantsVery low
Hydroxyurea vs placebo
Reduction or prevention of kidney disease progression · people with sickle cell disease
Interventions for chronic kidney disease in people with sickle cell disease (2023) · Cochrane
no estimate extracted1 study · 142 participantsVery low
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 extractedNRNo 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 extractedNRNo 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 extractedNRNo studies
Hydroxyurea vs placebo
Serious adverse events · people with sickle cell disease
Interventions for chronic kidney disease in people with sickle cell disease (2023) · Cochrane
RR 0.39 [0.13, 1.16]1 study · 193 participantsLow
Hydroxyurea vs placebo
All‐cause mortality · people with sickle cell disease
Interventions for chronic kidney disease in people with sickle cell disease (2023) · Cochrane
no estimate extracted1 study · 193 participantsLow
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 extractedNRNo studies
Angiotensin‐converting enzyme inhibitors vs placebo
Reduction or prevention of kidney disease progression · people with sickle cell disease
Interventions for chronic kidney disease in people with sickle cell disease (2023) · Cochrane
no estimate extracted1 study · 22 participantsVery low
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 extractedNRNo studies
Angiotensin‐converting enzyme inhibitors vs placebo
All‐cause mortality · people with sickle cell disease
Interventions for chronic kidney disease in people with sickle cell disease (2023) · Cochrane
no estimate extractedNRNo studies
Angiotensin‐converting enzyme inhibitors 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 extractedNRNo studies
Angiotensin‐converting enzyme inhibitors vs vitamin C
Reduction or prevention of kidney disease progression · children with sickle cell anaemia and microalbuminuria
Interventions for chronic kidney disease in people with sickle cell disease (2023) · Cochrane
no estimate extracted1 study · 170 participantsVery low
Full‐mouth ultrasonic scaling plus photodynamic therapy vs full‐mouth ultrasonic scaling alone
Change in class of malocclusion · people with thalassaemia
Treatment of dental and orthodontic complications in thalassaemia (2023) · Cochrane
no estimate extractedNRNo studies
Full‐mouth ultrasonic scaling plus photodynamic therapy vs full‐mouth ultrasonic scaling alone
Resolution or reduction in severity of any other dental complication: reduction in DMFT index · people with thalassaemia
Treatment of dental and orthodontic complications in thalassaemia (2023) · Cochrane
no estimate extractedNRNo studies
Full‐mouth ultrasonic scaling plus photodynamic therapy vs full‐mouth ultrasonic scaling alone
Resolution or reduction in severity of any other dental complication: reduction in severity of gingivitis (measured by BoP) · people with thalassaemia
Treatment of dental and orthodontic complications in thalassaemia (2023) · Cochrane
no estimate extracted1 study · 29 participantsVery low
Full‐mouth ultrasonic scaling plus photodynamic therapy vs full‐mouth ultrasonic scaling alone
Resolution or reduction in severity of any other dental complication: reduction in severity of periodontitis (measured by reduction in clinical attachment loss) · people with thalassaemia
Treatment of dental and orthodontic complications in thalassaemia (2023) · Cochrane
no estimate extractedNRNo studies
Full‐mouth ultrasonic scaling plus photodynamic therapy vs full‐mouth ultrasonic scaling alone
Resolution or reduction in severity of any other dental complication: oral infections (measured by reduction in or resolution of lesions such as periapical cysts, abscesses, or space infections) · people with thalassaemia
Treatment of dental and orthodontic complications in thalassaemia (2023) · Cochrane
no estimate extractedNRNo studies
Full‐mouth ultrasonic scaling plus photodynamic therapy vs full‐mouth ultrasonic scaling alone
Resolution or reduction in severity of any other dental complication: systemic infections (measured by number of episodes after treatment for dental complications under antibiotic prophylaxis) · people with thalassaemia
Treatment of dental and orthodontic complications in thalassaemia (2023) · Cochrane
no estimate extractedNRNo studies
Full‐mouth ultrasonic scaling plus photodynamic therapy vs full‐mouth ultrasonic scaling alone
Any other adverse effects related to treatment · people with thalassaemia
Treatment of dental and orthodontic complications in thalassaemia (2023) · Cochrane
no estimate extractedNRNo studies
Alglucosidase alfa vs placebo
Change from baseline in 6MWT (metres) · 90 people with LOPD
Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane
no estimate extracted1 study · 90 participantsModerate
Alglucosidase alfa plus clenbuterol vs alglucosidase alfa plus placebo
Infusion reactions · 13 people with LOPD
Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane
no estimate extractedNRNo studies
Alglucosidase alfa plus clenbuterol vs alglucosidase alfa plus placebo
Need for respiratory support · 13 people with LOPD
Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane
no estimate extractedNRNo studies
Alglucosidase alfa plus clenbuterol vs alglucosidase alfa plus placebo
Use of a walking aid / wheelchair · 13 people with LOPD
Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane
no estimate extractedNRNo studies