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.
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 |
|---|---|---|---|
Alglucosidase alfa plus clenbuterol vs alglucosidase alfa plus placebo QoL · 13 people with LOPD Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane | no estimate extracted | NR | No studies |
Alglucosidase alfa plus clenbuterol vs alglucosidase alfa plus placebo Treatment‐related adverse events · 13 people with LOPD Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane | no estimate extracted | NR | No studies |
Alglucosidase alfa plus albuterol vs alglucosidase alfa plus placebo Change in 6MWT from baseline (metres) · 13 people with LOPD Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane | no estimate extracted | 1 study · 5 participants | Very low |
Alglucosidase alfa plus albuterol vs alglucosidase alfa plus placebo Change in FVC from baseline (% predicted) · 13 people with LOPD Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane | no estimate extracted | 1 study · 5 participants | Very low |
Alglucosidase alfa plus albuterol vs alglucosidase alfa plus placebo Infusion reactions · 13 people with LOPD Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane | no estimate extracted | NR | No studies |
Alglucosidase alfa plus albuterol 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 extracted | NR | No studies |
Alglucosidase alfa plus albuterol 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 extracted | NR | No studies |
Alglucosidase alfa vs placebo Change in FVC (% predicted) · 90 people with LOPD Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane | no estimate extracted | 1 study · 90 participants | Moderate |
Alglucosidase alfa plus albuterol vs alglucosidase alfa plus placebo QoL · 13 people with LOPD Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane | no estimate extracted | NR | No studies |
Alglucosidase alfa plus albuterol vs alglucosidase alfa plus placebo Adverse events · 13 people with LOPD Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane | RR 0.67 [0.38, 1.18] | 1 study · 13 participants | Very low |
VAL‐1221 vs alglucosidase alfa Change from baseline in 6MWT (metres) · adults with LOPD Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane | no estimate extracted | 1 study · 12 participants | Very low |
VAL‐1221 vs alglucosidase alfa Change in FVC (% predicted) · adults with LOPD Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane | no estimate extracted | NR | No studies |
VAL‐1221 vs alglucosidase alfa Infusion reactions · adults with LOPD Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane | OR 2.8 | 1 study · 12 participants | Very low |
VAL‐1221 vs alglucosidase alfa Need for respiratory support · adults with LOPD Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane | no estimate extracted | NR | No studies |
VAL‐1221 vs alglucosidase alfa Use of a walking aid/wheelchair · adults with LOPD Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane | no estimate extracted | NR | No studies |
VAL‐1221 vs alglucosidase alfa QoL · adults with LOPD Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane | no estimate extracted | NR | No studies |
VAL‐1221 vs alglucosidase alfa Treatment‐related adverse events · adults with LOPD Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane | no estimate extracted | NR | No studies |
Cipaglucosidase plus miglustat vs alglucosidase alfa plus placebo 6MWT: mean change from baseline in 6MWT (metres) · adults with LOPD Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane | no estimate extracted | 1 study · 117 participants | Low |
Alglucosidase alfa vs placebo Infusion reactions · 90 people with LOPD Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane | RR 1.21 [0.57, 2.61] | 1 study · 90 participants | Low |
Cipaglucosidase plus miglustat vs alglucosidase alfa plus placebo Change in sitting FVC (% predicted) · adults with LOPD Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane | no estimate extracted | 1 study · 133 participants | Moderate |
Cipaglucosidase plus miglustat vs alglucosidase alfa plus placebo Change in SNIP (% predicted) · adults with LOPD Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane | no estimate extracted | 1 study · 121 participants | Low |
Cipaglucosidase plus miglustat vs alglucosidase alfa plus placebo Infusion reactions · adults with LOPD Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane | RR 0.94 [0.49, 1.8] | 1 study · 123 participants | Low |
Cipaglucosidase plus miglustat vs alglucosidase alfa plus placebo Need for respiratory support · adults with LOPD Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane | no estimate extracted | NR | No studies |
Cipaglucosidase plus miglustat vs alglucosidase alfa plus placebo Use of a walking aid / wheelchair · adults with LOPD Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane | no estimate extracted | NR | No studies |
Cipaglucosidase plus miglustat vs alglucosidase alfa plus placebo QoL: change from baseline to PROMIS score (physical function score and fatigue score) · adults with LOPD Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane | no estimate extracted | 1 study · 121 participants | Low |
Cipaglucosidase plus miglustat vs alglucosidase alfa plus placebo Treatment‐related AEs: total treatment‐emergent adverse events potentially related to treatment · adults with LOPD Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane | RR 0.83 [0.49, 1.4] | 1 study · 123 participants | Low |
Avalglucosidase alfa vs alglucosidase alfa 6MWT: mean change from baseline (metres) · adults with LOPD Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane | no estimate extracted | 1 study · 100 participants | Moderate |
Avalglucosidase alfa vs alglucosidase alfa Change in FVC (% predicted) · adults with LOPD Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane | no estimate extracted | 1 study · 100 participants | Moderate |
Avalglucosidase alfa vs alglucosidase alfa Infusion reactions · adults with LOPD Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane | RR 0.78 [0.42, 1.45] | 1 study · 100 participants | Low |
Alglucosidase alfa vs placebo Need for respiratory support · 90 people with LOPD Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane | no estimate extracted | NR | No studies |
Avalglucosidase alfa vs alglucosidase alfa Need for respiratory support · adults with LOPD Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane | no estimate extracted | NR | No studies |
Avalglucosidase alfa vs alglucosidase alfa Use of a walking aid / wheelchair · adults with LOPD Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane | no estimate extracted | NR | No studies |
Avalglucosidase alfa vs alglucosidase alfa QoL: change from baseline in EQ‐5D‐5L score · adults with LOPD Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane | no estimate extracted | 1 study · 100 participants | Low |
Avalglucosidase alfa vs alglucosidase alfa Treatment‐related AEs: total treatment‐emergent adverse events potentially related to treatment · adults with LOPD Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane | RR 0.92 [0.61, 1.4] | 1 study · 100 participants | Low |
Alglucosidase alfa vs placebo Use of a walking aid/wheelchair · 90 people with LOPD Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane | no estimate extracted | NR | No studies |
Alglucosidase alfa vs placebo QoL (PCS in SF‐36 (measured on a scale from 0 to 100)) · 90 people with LOPD Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane | no estimate extracted | 1 study · 90 participants | Low |
Alglucosidase alfa vs placebo Treatment‐related AEs · 90 people with LOPD Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane | RR 0.94 [0.64, 1.39] | 1 study · 90 participants | Low |
Alglucosidase alfa plus clenbuterol vs alglucosidase alfa plus placebo Change in 6MWT from baseline (metres) · 13 people with LOPD Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane | no estimate extracted | 1 study · 10 participants | Very low |
Alglucosidase alfa plus clenbuterol vs alglucosidase alfa plus placebo Change in FVC from baseline (% predicted) · 13 people with LOPD Enzyme replacement therapy for late‐onset Pompe disease (2023) · Cochrane | no estimate extracted | 1 study · 11 participants | Very low |
Motivational interventions to improve adherence to airway clearance treatment for people with CF ‐ specifically‐composed music vs no music Adherence to treatment Morisky‐Green questionnaire (number of participants showing full adherence) · people with CF Interventions for improving adherence to airway clearance treatment and exercise in people with cystic… (2023) · Cochrane | RR 1.56 [1.01, 2.4] | 1 study · 30 participants | Very low |
Motivational interventions to improve adherence to airway clearance treatment for people with CF ‐ self‐selected music vs no music Frequency of non‐hospitalised respiratory infection · people with CF Interventions for improving adherence to airway clearance treatment and exercise in people with cystic… (2023) · Cochrane | no estimate extracted | NR | No studies |
Motivational interventions to improve adherence to airway clearance treatment for people with CF ‐ self‐selected music vs no music FEV1 % predicted (change from baseline) · people with CF Interventions for improving adherence to airway clearance treatment and exercise in people with cystic… (2023) · Cochrane | no estimate extracted | NR | No studies |
Motivational interventions to improve adherence to airway clearance treatment for people with CF ‐ self‐selected music vs no music FVC % predicted (change from baseline) · people with CF Interventions for improving adherence to airway clearance treatment and exercise in people with cystic… (2023) · Cochrane | no estimate extracted | NR | No studies |
Motivational interventions to improve adherence to airway clearance treatment for people with CF ‐ self‐selected music vs no music QoL (change from baseline) · people with CF Interventions for improving adherence to airway clearance treatment and exercise in people with cystic… (2023) · Cochrane | no estimate extracted | NR | No studies |
Motivational interventions to improve adherence to airway clearance treatment for people with CF ‐ self‐selected music vs no music Exercise capacity (change from baseline) · people with CF Interventions for improving adherence to airway clearance treatment and exercise in people with cystic… (2023) · Cochrane | no estimate extracted | NR | No studies |
Motivational interventions to improve adherence to airway clearance treatment for people with CF ‐ specifically‐composed music vs self‐selected music Adherence to treatment Morisky‐Green questionnaire (number of participants showing full adherence) · people with CF Interventions for improving adherence to airway clearance treatment and exercise in people with cystic… (2023) · Cochrane | RR 1.52 [0.97, 2.38] | 1 study · 28 participants | Very low |
Motivational interventions to improve adherence to airway clearance treatment for people with CF ‐ specifically‐composed music vs self‐selected music Frequency of hospitalisation for respiratory infection · people with CF Interventions for improving adherence to airway clearance treatment and exercise in people with cystic… (2023) · Cochrane | RR 0.29 [0.03, 2.45] | 1 study · 28 participants | Very low |
Motivational interventions to improve adherence to airway clearance treatment for people with CF ‐ specifically‐composed music vs self‐selected music Frequency of non‐hospitalised respiratory infection · people with CF Interventions for improving adherence to airway clearance treatment and exercise in people with cystic… (2023) · Cochrane | no estimate extracted | NR | No studies |
Motivational interventions to improve adherence to airway clearance treatment for people with CF ‐ specifically‐composed music vs self‐selected music FEV1 % predicted (change from baseline) · people with CF Interventions for improving adherence to airway clearance treatment and exercise in people with cystic… (2023) · Cochrane | no estimate extracted | NR | No studies |
Motivational interventions to improve adherence to airway clearance treatment for people with CF ‐ specifically‐composed music vs self‐selected music FVC % predicted (change from baseline) · people with CF Interventions for improving adherence to airway clearance treatment and exercise in people with cystic… (2023) · Cochrane | no estimate extracted | NR | No studies |