Everolimus safety and efficacy for renal angiomyolipomas associated with tuberous sclerosis complex: a Spanish expanded access trial

Table 1 Baseline patient characteristics (N = 19)

Patient characteristics	Value
Age (years), median (IQR)	38.0 (29.0–43.0)
Sex, n (%)
Female	13 (68.4)
Male	6 (31.6)
Race, n (%)
White	19 (100)
Main comorbidities, n (%)^a
Hypertension	3 (15.8)
Asthma	2 (10.5)
Hypercholesterolaemia	2 (10.5)
Major features for TSC diagnosis, n (%)^a
Renal AML	19 (100)
Facial angiofibromas/forehead plaque	15 (79.0)
Cortical tuber	10 (52.6)
Hypomelanotic macules (≥3)	7 (36.8)
Subependymal nodule	7 (36.8)
Non-traumatic ungual/periungual fibroma	4 (21.1)
Single/multiple cardiac rhabdomyoma	4 (21.1)
Lymphangiomyomatosis	3 (15.8)
Connective tissue nevus	2 (10.5)
Minor features for TSC diagnosis, n (%)^a
Multiple renal cysts	11 (57.9)
Multiple randomly distributed pits in dental enamel	3 (15.8)
Bone cysts	3 (15.8)
Non-renal hamartoma	2 (10.5)
“Confetti” skin lesions	1 (5.26)
Sum of volumes of target renal AML lesions (cm³), median (IQR)	260.0 (127.8–322.2)
Volume of right kidney (cm³), median (IQR)	329.4 (193.0–979.7)^b
Volume of left kidney (cm³), median (IQR)	299.0 (184.2–404.7)^c

Abbreviations: AML angiomyolipoma, IQL interquartile range, SD standard deviation, TSC tuberous sclerosis complex
^aMultiple response variable
^bOne patient had previously undergone nephrectomy of right kidney and two had technical problems for renal volume assessment; N = 16
^cThree patients had previously undergone nephrectomy of left kidney and one had technical problems for renal volume assessment; N = 15

ISSN: 1750-1172