Unknown

Dataset Information

0

The effect of everolimus on renal angiomyolipoma in pediatric patients with tuberous sclerosis being treated for subependymal giant cell astrocytoma.


ABSTRACT:

Background

Patients with tuberous sclerosis complex (TSC) often have multiple TSC-associated hamartomas, particularly in the brain and kidney.

Methods

This was a post hoc analysis of pediatric patients being treated for subependymal giant cell astrocytomas (SEGAs) during the phase 3, randomized, double-blind, placebo-controlled EXIST-1 trial. Patients were initially randomly assigned to receive everolimus 4.5 mg/m2/day (target blood trough 5-15 mg/dl) or placebo and could continue in an open-label extension phase. Angiomyolipoma response rates were analyzed in patients aged <18 years with ?1 target angiomyolipoma lesion at baseline. Response was defined as the proportion of patients with a ?50% reduction in the sum volume of target renal angiomyolipomata from baseline, in the absence of new target angiomyolipomata, a >20% increase in kidney volume from nadir, and angiomyolipoma-related bleeding ? grade 2. Tolerability was also assessed.

Results

Overall, this analysis included 33 patients. Renal angiomyolipoma response was achieved by 75.8% of patients (95% confidence interval, 57.7-88.9%), with sustained mean reductions in renal angiomyolipoma volume over nearly 4 years of treatment. In addition, most (?80%) achieved clinically relevant reductions in angiomyolipoma volume (?50%), beginning at week 24 and continuing for the remainder of the study. Everolimus was generally well tolerated in this subgroup, with most adverse events being grade 1 or 2 in severity.

Conclusions

Although everolimus is currently not indicated for this use, this analysis from EXIST-1 demonstrates its long-term efficacy and safety for the treatment of renal angiomyolipoma in pediatric patients undergoing treatment for TSC-associated SEGA.

PROVIDER: S-EPMC5700234 | BioStudies |

REPOSITORIES: biostudies

Similar Datasets

| S-EPMC4924870 | BioStudies
| S-EPMC3389821 | BioStudies
| S-EPMC3520048 | BioStudies
| S-EPMC4852219 | BioStudies
| S-EPMC3797614 | BioStudies
| S-EPMC6128468 | BioStudies
| S-EPMC9290768 | BioStudies
2018-01-01 | S-EPMC5870799 | BioStudies
| S-EPMC3848592 | BioStudies
| S-EPMC6935755 | BioStudies