Renal Function Outcomes in Tuberous Sclerosis Complex Patients Receiving Everolimus for Renal Angiomyolipoma

Everolimus has been proved effective in treating renal angiomyolipoma (RAML) associated with tuberous sclerosis complex (TSC). However, its impact on renal function remains uncertain.

We analyzed changes in the estimated glomerular filtration rate (eGFR) in 54 TSC-RAML patients undergoing 1-yr everolimus treatment, based on the data from two previously registered clinical trials.

After 1 yr of everolimus treatment, no significant change in eGFR was observed in the overall cohort of 54 patients (102 [81-121] vs 103 [79-122] ml/min/1.73 m², p = 0.8 for baseline vs post-treatment eGFR). Notably, patients with a tumor volume reduction (TVR) of ≥500 ml showed a significant improvement in eGFR (101 [95, 119] vs 115 [99, 122] ml/min/1.73 m², p = 0.009), as did those with a tumor shrinkage rate (TSR) of ≥30% (99 [80, 117] vs 104 [76, 119] ml/min/1.73 m², p = 0.02). Patients who experienced an eGFR improvement exhibited a significantly greater TVR (423 [86, 1035] vs 104 [28, 340] ml, p = 0.005) and higher TSR (40% ± 20% vs 26% ± 21%, p = 0.015) compared with those without improvement. Correlation analyses revealed a positive association between eGFR change and both TVR (r = 0.392, p = 0.003) and TSR (r = 0.430, p = 0.0012). In the linear mixed model, both TVR (β = 0.0046, p = 0.011) and TSR (β = 15.0, p < 0.001) were independently and significantly associated with eGFR change.

Everolimus demonstrates a favorable renal safety profile in TSC-RAML patients and improves renal function in patients with substantial tumor reduction, supporting its use as a therapeutic option for renal function preservation.

In this study, we evaluated kidney function in patients with renal angiomyolipoma associated with tuberous sclerosis complex (TSC-RAML), who received everolimus treatment. We found that kidney function was generally stable and even improved in patients with marked tumor reduction. These results suggest that everolimus may help preserve or enhance kidney health in selected patients with TSC-RAML.