Koenig 2012.
| Methods | Double‐blind, randomised, placebo‐controlled study. Single centre in Texas, USA. | |
| Participants | 28 participants (mean age at 23 years old, ranging from 13 to > 30 years old, 15 males and 13 females) diagnosed as TSC based on the consensus criteria (Roach 1999) were recruited. 18 participants in the treatment group and 10 participants in the placebo group. Five participants were withdrawn from the study, but it is unclear if they come from the treatment arm or from placebo arm. For a future version of this review, we plan to contact study authors to ascertain if the people with TSC meet the latest diagnostic criteria (Northrup 2013). | |
| Interventions | Participants were were randomised into either: 1. topical (skin application) rapamycin 1 mg per 30 cc (0.003%); or 2. topical (skin application) rapamycin 5 mg per 30 cc (0.015%); or 3. topical (skin application) placebo. Participants were treated for 6 months. |
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| Outcomes | Effect of rapamycin on participant' perception towards their skin appearance following the treatment. The participants were asked if they "got better on the treatment", "got worse on the treatment" or if "the treatment made no difference". Rapamycin concentrations and complete blood counts (including haemoglobin level and platelet count) were measured. Adverse events were reported. | |
| Notes | ‐ | |
| Risk of bias | ||
| Bias | Authors' judgement | Support for judgement |
| Random sequence generation (selection bias) | Unclear risk | Randomisation was done by the manufacturer of the investigational product: "The company randomized the investigational product". However, there was no statement as to how the allocation sequence was generated. |
| Allocation concealment (selection bias) | Unclear risk | Not stated. |
| Blinding of participants and personnel (performance bias) All outcomes | Low risk | Participants and study personnel were blinded: "Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)". |
| Blinding of outcome assessment (detection bias) All outcomes | Low risk | Outcome assessors were blinded. |
| Incomplete outcome data (attrition bias) All outcomes | High risk | Out of 28 participants included into the study, 23 participants completed the intervention. Outcome data were reported only as percentages. While there are dropouts, no reports as to how many dropouts in each treatment arm. |
| Selective reporting (reporting bias) | Low risk | All outcomes that were mentioned in 'Methods' were reported in 'Results' sections of the study report. |
| Other bias | Low risk | ‐ |
CT: computed tomography MRI: magnetic resonance imaging SEGA: subependymal giant cell astrocytomas TSC: tuberous sclerosis complex