Objective
To evaluate the clinical and radiographic follow-up of renal angiomyolipoma (AML)
in pediatric patients with tuberous sclerosis complex (TSC) on mTOR inhibitors.
Methods
We performed retrospective chart review of children who were diagnosed with TSC between
2000 and 2019 and prescribed everolimus at age ≤18 years. Treatment assessment was
performed in patients who were medically-compliant by serum drug trough levels and
who had at least a baseline and one subsequent renal imaging study.
Results
Nineteen patients were analyzed. Average age of everolimus initiation was 9 years,
and indication was neurologic in 17 (90%). Fourteen patients (73.6%) had AML with
average size of 1.9 (0.4-5) cm. Medication was discontinued due to side effects in
3 (16%) patients. Treatment assessment was analyzed for 15 patients with median medication
exposure 5.1 (0.8-8.5) years. Among 13 with AML, the dominant lesion decreased in
size in 9 (69%) and stayed stable in 4 (31%). Greatest absolute size decrease was
seen for lesions ≥2 cm. No new AML lesions formed during treatment.
Conclusion
Although not currently approved for this indication, everolimus appears to be well-tolerated
with similar efficacy for pediatric AML as in adult AML. Use may be most warranted
in children with AML ≥2 cm.
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Article info
Publication history
Published online: February 18, 2020
Accepted:
December 10,
2019
Received:
October 18,
2019
Footnotes
Financial Disclosures: None.
Identification
Copyright
© 2020 Elsevier Inc. All rights reserved.
