Predictor
|
Measure
|
N
|
Category
|
Mean
|
± SD
|
95% CI
|
p-value*
|
---|
Sociodemographic aspects
|
Sex
|
VAS
|
64
|
Male
|
67.9
|
19.7
|
63.0–72.9
|
0.768
|
54
|
Female
|
66.7
|
21.2
|
60.9–72.5
|
Index (TTO)
|
64
|
Male
|
0.796
|
0.267
|
0.730–0.863
|
0.320
|
54
|
Female
|
0.843
|
0.257
|
0.773–0.914
|
Age
|
VAS
|
54
|
18–28 y
|
72.0
|
20.4
|
66.4–77.6
|
0.020
|
62
|
29–61 y
|
68.2
|
19.6
|
58.3–68.2
|
Index (TTO)
|
54
|
18–28 y
|
0.834
|
0.238
|
0.769–0.898
|
0.309
|
62
|
29–61 y
|
0.800
|
0.286
|
0.727–0.873
|
Employment
|
VAS
|
49
|
Yes
|
70.8
|
18.2
|
65.6–76.0
|
0.017
|
33
|
No
|
58.7
|
21.7
|
51.0–66.4
|
Index (TTO)
|
49
|
Yes
|
0.920
|
0.115
|
0.887–0.953
|
< 0.001†
|
33
|
No
|
0.661
|
0.341
|
0.540–0.782
|
Grade of disability
|
VAS
|
36
|
0–80
|
72.0
|
15.9
|
66.6–77.4
|
0.001†
|
56
|
90–100
|
58.6
|
20.3
|
53.1–64.0
|
Index (TTO)
|
36
|
0–80
|
0.916
|
0.101
|
0.882–0.950
|
< 0.001†
|
56
|
90–100
|
0.681
|
0.318
|
0.596–0.766
|
Clinical aspects
|
Mutation type
|
VAS
|
24
|
TSC1
|
72.8
|
18.5
|
65.0–80.6
|
0.198
|
30
|
TSC2
|
65.0
|
24.2
|
56.0–74.1
|
Index (TTO)
|
24
|
TSC1
|
0.803
|
0.288
|
0.682–0.924
|
0.662
|
30
|
TSC2
|
0.793
|
0.296
|
0.682–0.904
|
Active epilepsy
|
VAS
|
50
|
Yes
|
60.0
|
20.1
|
54.3–65.7
|
0.001†
|
71
|
Sz freedom > 12 m
|
72.3
|
18.9
|
68.2–77.4
|
Index (TTO)
|
50
|
Yes
|
0.729
|
0.305
|
0.642–0.816
|
0.001†
|
71
|
Sz freedom > 12 m
|
0.883
|
0.205
|
0.834–0.939
|
Structural brain
|
VAS
|
83
|
Yes
|
65.4
|
20.3
|
61.0–69.9
|
0.116
|
35
|
No
|
72.0
|
20.0
|
65.1–78.8
|
Index (TTO)
|
83
|
Yes
|
0.793
|
0.281
|
0.732–0.855
|
0.123
|
35
|
No
|
0.876
|
0.206
|
0.805–0.947
|
SEGA
|
VAS
|
50
|
Yes
|
62.2
|
20.5
|
56.3–68.0
|
0.017
|
68
|
No
|
71.2
|
19.5
|
66.5–75.9
|
Index (TTO)
|
50
|
Yes
|
0.802
|
0.268
|
0.725–0.877
|
0.597
|
68
|
No
|
0.830
|
0.260
|
0.767–0.893
|
Neuropsychiatric
|
VAS
|
60
|
Yes
|
59.1
|
21.4
|
53.5–64.7
|
< 0.001†
|
61
|
No
|
75.4
|
15.7
|
71.3–75.4
|
Index (TTO)
|
60
|
Yes
|
0.693
|
0.314
|
0.610–0.776
|
< 0.001†
|
61
|
No
|
0.938
|
0.107
|
0.911–0.966
|
AML
|
VAS
|
69
|
Yes
|
65.9
|
21.2
|
60.8–71.0
|
0.317
|
49
|
No
|
69.4
|
19.2
|
63.9–74.9
|
Index (TTO)
|
70
|
Yes
|
0.805
|
0.276
|
0.739–0.872
|
0.979
|
51
|
No
|
0.835
|
0.244
|
0.765–0.906
|
Lymphangioleio-myomatosis
|
VAS
|
12
|
Yes
|
67.5
|
12.7
|
59.4–75.6
|
0.844
|
106
|
No
|
67.4
|
21.1
|
63.3–71.4
|
Index (TTO)
|
12
|
Yes
|
0.819
|
0.320
|
0.615–1.023
|
0.839
|
106
|
No
|
0.818
|
0.257
|
0.768–0.867
|
Skin
|
VAS
|
114
|
Yes
|
67.1
|
20.5
|
63.3–70.9
|
0.469
|
4
|
No
|
75.0
|
13.5
|
63.3–96.6
|
Index (TTO)
|
116
|
Yes
|
0.815
|
0.267
|
0.767–0.865
|
0.504
|
5
|
No
|
0.890
|
0.087
|
0.753–1.028
|
Number of affected organs
|
VAS
|
30
|
1–3
|
80.9
|
12.3
|
76.3–85.5
|
ref.
|
< 0.001†
|
72
|
4–6
|
64.5
|
20.3
|
59.7–69.3
|
0.005
|
16
|
7–8
|
55.1
|
20.3
|
44.2–65.9
|
0.048
|
Index (TTO)
|
30
|
1–3
|
0.931
|
0.159
|
0.872–0.990
|
ref.
|
0.001†
|
72
|
4–6
|
0.805
|
0.264
|
0.743–0.867
|
0.037
|
16
|
7–8
|
0.662
|
0.330
|
0.487–0.838
|
0.009
|
Therapeutic aspects
|
Anti-seizure medication polytherapya
|
VAS
|
53
|
Yes
|
62.3
|
20.2
|
61.7–74.9
|
0.223
|
37
|
No
|
68.3
|
19.8
|
57.3–68.4
|
Index (TTO)
|
53
|
Yes
|
0.759
|
0.282
|
0.682–0.837
|
0.081
|
40
|
No
|
0.851
|
0.250
|
0.767–0.934
|
Everolimus
|
VAS
|
50
|
Yes
|
61.6
|
20.3
|
55.8–67.4
|
0.007†
|
68
|
No
|
71.6
|
19.4
|
66.9–76.3
|
Index (TTO)
|
50
|
Yes
|
0.772
|
0.286
|
0.691–0.854
|
0.141
|
68
|
No
|
0.851
|
0.241
|
0.793–0.910
|
LAEP
|
VAS
|
34
|
< 35
|
62.6
|
19.7
|
58.3–66.9
|
< 0.001†
|
83
|
≥35
|
78.4
|
17.5
|
72.3–84.5
|
Index (TTO)
|
34
|
< 35
|
0.757
|
0.287
|
0.695–0.820
|
< 0.001†
|
83
|
≥35
|
0.960
|
0.093
|
0.928–0.993
|
- AML angiomyolipoma, CI confidence interval, EQ-5D EuroQoL-5 dimensions, TTO time-trade-off method, VAS visual analogue scale, LAEP Liverpool Adverse Events Profile, m months, QOLIE-31 Quality of Life in Epilepsy Inventory-31 items, ref. reference category, SD standard deviation, SEGA subependymal giant cell astrocytoma, sz seizure, TSC tuberous sclerosis complex, y years
- aIncludes only individuals with TSC-associated epilepsy/seizures
- *Comparisons corrected for multiple testing using the Benjamini–Hochberg false-discovery rate method, † and bold type denotes a q-value of < 0.05 (false discovery rate)