Table 2

Results of sizes reduction of patients with tuberous sclerosis complex-associated renal angiomyolipoma (TSC-AML) treated with everolimus for 2 years, n=11

PatientsGroupSize (cm3)RR (%)Long-term PR
(months)
Side effect
BeforeAfter
1I84.551.638.9Acne
2I2.31.247.8Oral ulcers/acne
3I2.01.430.0+ (24)Oral ulcers/cellulitis
4I367.7328.910.6Hyperlipidemia
5I79.127.565.2+ (6)Oral ulcer
6II2497.0793.668.2+ (12)Oral ulcers
7II94.835.262.9+ (12)Diarrhea
8II544.5159.970.6+ (24)Oral ulcers
9II2473.71421.942.5+ (60)Oral ulcers, hyperlipidemia/DM/HTN
10I77.245.740.8+ (24)Oral ulcer, hyperlipidemia, amenorrhea, proteinuria
11II66.131.552.3+ (6)Oral ulcer, acne, hyperlipidemia, hyperglycemia amenorrhea, liver function impairment
  • Mean trough level (μg/kg)<8: group I;>8: group II.

  • DM, diabetics mellitus; HTN, hypertension.; PR, partial response; RR, reduction rate.