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
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
Current version onlySummary of findings for systemic administration of rapalogs
Population: people with TSC
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
50% reduction of tumour sizea | RR 24.7 [3.5, 173.4] | 2 studies · 162 participants | High |
Response to skin lesionsb | RR 5.8 [2.3, 14.5] | 2 studies · 224 participants | High |
Frequency of seizure | RR 5.3 | 1 study · 366 participants | Moderate Downgraded: imprecision, indirectness |
Number of participants with an increased creatinine level | RR 0.16 | 1 study · 118 participants | Moderate Downgraded: imprecision, indirectness |
FEV1/FVC ratio | no estimate extracted | NR | No studies |
Outcomes relevant to participants' well‐being | no estimate extracted | 1 study · 42 participants | Low Downgraded: imprecision, indirectness |
Adverse events | RR 1.09 | 5 studies · 680 participants | High |
Summary of findings for topical administration of rapamycin
Population: people with TSC
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
50% reduction in tumour size | no estimate extracted | NR | No studies |
Response to skin lesion | RR 2.72 [1.76, 4.18] | 2 studies · 187 participants | High |
Frequency of seizure | no estimate extracted | NR | No studies |
Creatinine level | no estimate extracted | NR | No studies |
FEV1/FVC ratio | no estimate extracted | NR | No studies |
Outcomes relevant to participants' well‐being | no estimate extracted | 1 study · 62 participants | Low Downgraded: imprecision, inconsistency, indirectness |
Adverse events | RR 1.72 [1.1, 2.67] | 3 studies · 277 participants | Moderate Downgraded: imprecision, inconsistency, indirectness |
Summary of findings for systemic administration
Population: people with TSC
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
50% reduction of tumour size1 | RR 24.7 | 2 studies · 162 participants | High |
Response to skin lesions2 | RR 5.8 | 2 studies · 224 participants | High |
Frequency of seizure | no estimate extracted | 1 study · 1 participants | Moderate Downgraded: indirectness |
Creatinine level | RR 0.16 | 1 study · 118 participants | High |
Forced expiratory volume in 1 second (FEV1) | no estimate extracted | NR | No studies |
Any adverse events | RR 1.07 | 1 study · 117 participants | High |
Adverse Events leading to dose reduction, interruption or withdrawal | RR 3.14 | 2 studies · 235 participants | High |
Summary of findings for topical administration
Population: people with TSC
| Outcome | Effect | Evidence | Status / GRADE |
|---|---|---|---|
Response to skin lesions ‐ rapamycin (6 months) | RR 1.81 | 1 study · 28 participants | Low Downgraded: risk of bias |