Cochrane · systematic review · CD011272

Rapamycin and rapalogs for tuberous sclerosis complex

Sasongko, TH, Kademane, K, Chai Soon Hou, S, et al. · 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
14
current version
GRADE rated
9
No studies
5
Included studies
NR
No studies 5 · 35.7%Ungraded 0 · 0%Very low 0 · 0%Low 2 · 14.3%Moderate 3 · 21.4%High 4 · 28.6%

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

Show all 2 versions

Summary of findings for systemic administration of rapalogs

Population: people with TSC
7 questions
OutcomeEffectEvidenceStatus / GRADE
50% reduction of tumour sizea
RR 24.7 [3.5, 173.4]2 studies · 162 participantsHigh
Response to skin lesionsb
RR 5.8 [2.3, 14.5]2 studies · 224 participantsHigh
Frequency of seizure
RR 5.31 study · 366 participantsModerate
Downgraded: imprecision, indirectness
Number of participants with an increased creatinine level
RR 0.161 study · 118 participantsModerate
Downgraded: imprecision, indirectness
FEV1/FVC ratio
no estimate extractedNRNo studies
Outcomes relevant to participants' well‐being
no estimate extracted1 study · 42 participantsLow
Downgraded: imprecision, indirectness
Adverse events
RR 1.095 studies · 680 participantsHigh

Summary of findings for topical administration of rapamycin

Population: people with TSC
7 questions
OutcomeEffectEvidenceStatus / GRADE
50% reduction in tumour size
no estimate extractedNRNo studies
Response to skin lesion
RR 2.72 [1.76, 4.18]2 studies · 187 participantsHigh
Frequency of seizure
no estimate extractedNRNo studies
Creatinine level
no estimate extractedNRNo studies
FEV1/FVC ratio
no estimate extractedNRNo studies
Outcomes relevant to participants' well‐being
no estimate extracted1 study · 62 participantsLow
Downgraded: imprecision, inconsistency, indirectness
Adverse events
RR 1.72 [1.1, 2.67]3 studies · 277 participantsModerate
Downgraded: imprecision, inconsistency, indirectness