Objective: To investigate factors influencing the volume response of everolimus and sirolimus in tuberous sclerosis complex (TSC) associated-angiomyolipomas (AML).
Methods: A retrospective analysis of 30 cases of TSC-AML treated by mTOR inhibitors (everolimus 18 cases, and sirolimus 12 cases) between April 2014 and November 2017 at our center was carried out. Epidemiological data, therapeutic response and influence factors were reviewed and analyzed. Age, sex, associated with SEGA and/or LAM or not, plasma rapamycin concentration, AML volume at baseline, and mean CT value of AML in the maximum cross-section at baseline were analyzed as potential influencing factors.
Results: Eighteen patients with 32 lesions in everolimus group and 12 patients with 15 lesions in sirolimus group were included. There was no statistically significant difference of baseline characteristics except for involved side (P = 0.008) between two groups. The mean volume of AML was 1000 ± 1276 cm3 at baseline and 633 ± 1121 cm3 at 6 months after treatment (P < 0.001) in everolimus group, and 1984 ± 2861 cm3 at baseline and 1733 ± 2533 cm3 at 6 months after treatment (P = 0.001) in sirolimus group, respectively. The mean volume reduction of the AML in everolimus and sirolimus groups were 55.56% ± 23.79% and 30.5% ± 22.8% (P = 0.001). Stepwise multiple linear regression analysis revealed that factors influencing the short-term volume response of everolimus and sirolimus for TSC-associated AML were AML volume at baseline (P < 0.001 and 0.038, respectively) and mean CT value at baseline (P < 0.001 and 0.020, respectively). The rates of ≥ 50% volume reduction in high CT value group was much higher than that in low CT value group (90.5% vs. 18.2%, P < 0.001).
Conclusions: Everolimus at 10 mg daily might be more effective than sirolimus at 2 mg daily in treatment of patients with TSC-AML. AML volume and mean CT value at baseline were factors influencing the short-term volume response of everolimus or sirolimus for TSC-AML.
Keywords: Angiomyolipomas; Influence factors; Tuberous sclerosis complex; mTOR inhibitor.