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Palmetun Ekbäck et al. Topical rapamycin tuberous sclerosis clinical practice

Table 1: Patients and results
CO 2 Skin Erythema
No. Gender Age, Skin lesions Erythema laser Duration of lesions post post Side Sirolimus
year (pre) (pre) (pre) treatment treatment treatment effects conc
1 M 15 Papules and Medium No 6 months Not improved Not improved None Not done
nodules
2 F 4 Papules Medium Yes 14 months Improved Excellent None Not done
3 M 10 Papules Slight No 6 + 3 months* Improved Improved Dryness Not detected

4 F 2 Papules Medium No 7 months Excellent Excellent None Not done
5 F 13 Papules and Medium Yes 10 months Not improved Improved Dryness Not detected
nodules
6 M 12 Papules and Medium No 8 + 12 months* Not improved Improved Dryness Not detected
nodules
7 M 30 Papules Slight Yes 1.5 + 1 month* Not improved Not Dryness Not done
improved
8 F 43 Papules Medium Yes 9 + 16 months* Excellent Excellent None Not detected
9 F 9 Papules Medium No Drop out - - - Not done
10 M 2 Fibrous cephalic Medium No 9 + 4 months* Not improved Not None Not detected
Plaque improved
11 M 7 Papules Medium No Drop-out - - - Not done
12 M 19 Papules Medium Yes 19 + 7 months* Improved Improved None Not done
13 F 19 Papules Medium Yes 11 + 7 months* Improved Improved None Not done
14 M 18 Papules Medium Yes 5 + 14 months* Improved Improved None Not done
15 M 21 Papules Medium Yes 3 months Not improved Not None Not done
improved
16 F 26 Papules Medium No 33 months Improved Improved None Not done
17 F 29 Papules Slight Yes 5 months † † † Not applicable
18 F 33 Papules Medium Yes 3 months † † † Not applicable
19 F 52 Papules Slight Yes 3 weeks Discontinued Dryness, Not done
itching and
swollen
20 F 9 Papules Medium Yes 6 + 5 months* Improved Improved Itching Not done
21 F 10 Papules Medium Yes 5 + 6 Not improved Improved Stinging Not done
22 F 16 Papules Medium Yes 3 weeks Discontinued Pain, ery- Not done
thema
23 M 32 Papules Severe Yes 1.5 months Not improved Excellent None Not done
*Paused the treatment during summer months; †Received oral rapamycin for internal hamartomas

All patients except three were evaluated during visits The erythema was improved in 12 of 17 patients
where the skin status was compared with the pre- (70%) [Figure 4], of whom 4 had excellent results
treatment photos. Three patients lived far away and [Table 1]. The median age of the patients whose
were evaluated through photos sent by e-mail. Five erythema responded (improved and excellent) was
patients had blood samples taken for rapamycin 12.5 years (2-43 years) and median treatment time
measurement but none had detectable rapamycin levels. was 12.5 months (1.5-33 months). The median age for
the patients whose erythema did not respond was 18
Papules were improved in 9 patients (53%), of whom 2 years (2-30 years) and median treatment time was 4.5
had results classed as excellent [Figures 2 and 3]. The months (2.5-13 months). During the summer pause,
median treatment time for the patients who responded lesions and erythema recurred.
(improved or excellent results) was 16 months (7-
33 months). The patients who did not respond (not Initially, rapamycin was prescribed on a daily basis.
improved results) had a median treatment time of Six of 17 patients (35%) had mild side effects, such
10 months (1.5-13 months). The 3 patients with both as dryness, itching and stinging, but could continue
papules and nodules did not improve. The patient the treatment when using the solution every second
with a fibrous cephalic plaque did not respond to to third day. In practice, the treatment was performed
treatment. The median age of the patients whose skin every second to third day. The reasons for this were
lesions were improved was 13 years (2-43 years). The both side effects and compliance problems. Eleven of
median age of the patients whose skin lesions were these 17 patients had CO 2 laser treatment 3-5 months
not improved was 14 years (2-32 years). [Figures 3, 5, 6 and 7] before rapamycin treatment.

330 Plastic and Aesthetic Research ¦ Volume 3 ¦ October 25, 2016