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Fate of Pediatric Renal Angiomyolipoma During mTOR Inhibitor Treatment in Tuberous Sclerosis Complex

  • Charlotte Q. Wu
    Correspondence
    Address correspondence to: Charlotte Q. Wu M.D., Emory University School of Medicine, Children's Healthcare of Atlanta, Georgia Pediatric Urology, 5730 Glenridge Drive, Suite 200, Atlanta, GA 30328.
    Affiliations
    Division of Pediatric Urology, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, GA
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  • David S. Wolf
    Affiliations
    Division of Pediatric Neurology, Department of Pediatrics, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, GA
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  • Edwin A. Smith
    Affiliations
    Division of Pediatric Urology, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, GA
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Published:February 18, 2020DOI:https://doi.org/10.1016/j.urology.2019.12.041

      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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