evidence/gaps
Cochrane · systematic review · CD012040

Ataluren and similar compounds (specific therapies for premature termination codon class I mutations) for cystic fibrosis

Aslam, AA, Sinha, IP, Southern, KW · Cochrane Database of Systematic Reviews · 2023 · Cochrane Library

Cystic Fibrosis and Genetic Disorders2 versionsAMSTAR 2: not assessed
SoF tableOnly the outcomes this review put in its Summary of Findings table are listed (the Cochrane Handbook caps them at about seven per comparison). Its other analyses are not shown here.
Questions
7
current version
GRADE rated
7
No studies
0
Included studies
NR
No studies 0 · 0%Ungraded 0 · 0%Very low 0 · 0%Low 0 · 0%Moderate 1 · 14.3%High 6 · 85.7%

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.

Questions and results

Current version only

Ataluren versus placebo for people with cystic fibrosis with class I mutations (PTCs)

Population: adults and children with CF with at least one class I mutation (PTC)
CD012040.PUB3 (current) · 7 questions
OutcomeEffectEvidenceStatus / GRADE
Health‐related quality of life: mean change from baseline in CFQ‐R respiratory domain score Scale: age‐appropriate versions of the CFQ‐R questionnaire
Ataluren vs placebo
no estimate extracted2 studies · 506 participantsHigh
Respiratory function: FEV1 % predicted (relative change from baseline)
Ataluren vs placebo
no estimate extracted1 study · 203 participantsHigh
Adverse events relating to treatment
Ataluren vs placebo
no estimate extracted2 studies · 517 participantsModerate
Downgraded: imprecision
Pulmonary exacerbations: protocol‐defined Scale: rate
Ataluren vs placebo
no estimate extracted1 study · 232 participantsHigh
Nutrition and growth: change in weight (kg), BMI (kg/m2) or height (m)
Ataluren vs placebo
no estimate extracted2 studies · 506 participantsHigh
Sweat chloride level ‐ change from baseline Scale: mmol/L
Ataluren vs placebo
no estimate extracted1 study · 194 participantsHigh
Mortality rate
Ataluren vs placebo
no estimate extracted2 studies · 518 participantsHigh

Comparison

Population: adults and children with CF with at least one class I mutation (PTC)
CD012040.PUB2 · 8 questions
OutcomeEffectEvidenceStatus / GRADE
Health Related Quality of Life Scale: age appropriate versions of CFQ‐R questionnaire
no estimate extracted1 study · 232 participantsModerate
Downgraded: imprecision
Respiratory function: % predicted FEV₁ (relative change from baseline)
MD 2.971 study · 232 participantsHigh
Adverse events relating to treatment Scale: mild, moderate, severe Episodes of oropharyngeal pain
RR 0.28 [0.1, 0.83]1 study · 238 participantsModerate
Downgraded: imprecision
Adverse events relating to treatment Scale: mild, moderate, severe Episodes of "acute kidney injury"
RR 17.7 [2.4, 130.47]1 study · 238 participantsModerate
Downgraded: imprecision
Pulmonary exacerbations ‐ protocol‐defined (modified Fuchs' criteria) Scale: rate
no estimate extracted1 study · 232 participantsModerate
Downgraded: imprecision
Nutrition and growth Change in: a) weight (kg) b) BMI (kg/m2) c) height (m)
no estimate extracted1 study · 232 participantsModerate
Downgraded: imprecision
Sweat chloride level ‐ change from baseline Scale: mmol/L
no estimate extracted1 study · 194 participantsModerate
Downgraded: imprecision
Mortality rate
no estimate extracted1 study · 232 participantsModerate
Downgraded: imprecision