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Table 3 Disease characteristics

From: EFFECTS: an expanded access program of everolimus for patients with subependymal giant cell astrocytoma associated with tuberous sclerosis complex

Disease characteristics

Everolimus

N  =  120

Time since TSC diagnosis, median (range), years

9.1 (0.2–30.7)

Time since SEGA diagnosis, median (range), years

3.8 (0–24)

Modified Gomez criteria - major features, n (%)

 Facial angiofibromas or forehead plaques

93 (77.5)

 Non-traumatic ungual or periungual fibroma

18 (15.0)

 Hypomelanotic macules (3 or more)

95 (79.2)

 Shagreen patch (connective tissue nevus)

47 (39.2)

 Multiple retinal nodular hamartomas

23 (19.2)

 Cortical tuber

106 (88.3)

 Subependymal nodule

102 (85.0)

 Subependymal giant cell astrocytoma

120 (100.0)

 Cardiac rhabdomyoma, single or multiple

55 (45.8)

 Lymphangioleiomyomatosis

2 (1.7)

 Renal angiomyolipoma

59 (49.2)

Modified Gomez criteria - minor features, n (%)

 Multiple, randomly distributed pits in dental enamel

6 (5.0)

 Hamartomatous rectal polyps

0 (0.0)

 Bone cysts

2 (1.7)

 Cerebral white matter radial migration lines

16 (13.3)

 Gingival fibromas

6 (5.0)

 Non-renal hamartoma

3 (2.5)

 Retinal achromic patch

4 (3.3)

 'Confetti' skin lesions

15 (12.5)

 Multiple renal cysts

19 (15.8)

  1. SEGA subependymal giant cell astrocytoma, TSC tuberous sclerosis complex