Figure 1.
Photographs taken from a TSC and a BHD patient show angiofibromas and fibrofolliculomas, respectively. Notice the resemblance between the two types of lesion.
Figure 2.
* The patient who was lost to follow-up at 3 months was excluded from the analysis of the 3 month results. ** For the patients who were lost to follow-up at 6 months, the 3-month data were carried forward in the analysis of the 6-month results.
Table 1.
Baseline characteristics of included patients.
Figure 3.
Photographic recording of cosmetic status during rapamycin treatment.
Close up of standardised facial photographs taken at baseline, 3 months and 6 months after starting treatment with topical rapamycin or placebo per facial half. Photos of a representative patient are shown, revealing no visible improvement or worsening of the facial fibrofolliculomas.
Figure 4.
Changes in cosmetic status of fibrofolliculomas after treatment with rapamycin scored by doctors and patients.
BHD patients with facial fibrofolliculomas were treated with topical rapamycin and placebo for six months. The degree of improvement compared to baseline was scored on a 7-point Likert scale by both doctors (A) and patients (B). Doctors and patients reported no change in the majority of cases. Patients reported improvement more frequently for the rapamycin compared to the placebo treated facial side.
Figure 5.
Changes in the number of fibrofolliculomas after treatment with rapamycin.
BHD patients with facial fibrofolliculomas were treated with topical rapamycin and placebo for six months. The number of fibrofolliculomas in a predefined area of the face was determined and compared to baseline. The reduction in the number of fibrofolliculomas was stronger upon placebo treatment.
Table 2.
Between-treatment comparison of mean change in fibrofolliculoma size compared to baseline (in mm) using the T-test for paired samples.
Table 3.
The inter-observer variation of scores used to measure the effect of rapamycin using the intraclass correlation coefficient (ICC).
Figure 6.
Local side effects of topical rapamycin treatment.
Side effects of both topical rapamycin and placebo treatment were registered at each clinical assessment. Burning, erythema, dryness and itching were the most commonly reported local side effects. All side effects other than dryness and itching were reported more frequently for the rapamycin treated facial half.