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Improvement in Renal Cystic Disease of Tuberous Sclerosis Complex After Treatment with Mammalian Target of Rapamycin Inhibitor.

Abstract
Renal cysts occur in approximately 50% of patients with tuberous sclerosis complex, but their clinical significance and response to treatment are unknown. Abdominal imaging of 15 patients with tuberous sclerosis complex-associated renal cystic disease who had received mammalian target of rapamycin inhibitor therapy for other tuberous sclerosis complex-related indications was evaluated. Reductions in cyst number, sum diameter, and volume were observed.
AuthorsBrian J Siroky, Alexander J Towbin, Andrew T Trout, Hannah Schäfer, Anna R Thamann, Karen D Agricola, Cynthia Tudor, Jamie Capal, Bradley P Dixon, Darcy A Krueger, David N Franz
JournalThe Journal of pediatrics (J Pediatr) Vol. 187 Pg. 318-322.e2 (08 2017) ISSN: 1097-6833 [Electronic] United States
PMID28600153 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't, Research Support, N.I.H., Extramural)
CopyrightCopyright © 2017 Elsevier Inc. All rights reserved.
Chemical References
  • Immunosuppressive Agents
  • Sirolimus
Topics
  • Adolescent
  • Child
  • Child, Preschool
  • Female
  • Humans
  • Immunosuppressive Agents (therapeutic use)
  • Infant
  • Kidney (pathology)
  • Kidney Diseases, Cystic (drug therapy, etiology)
  • Magnetic Resonance Imaging
  • Male
  • Sirolimus (therapeutic use)
  • Treatment Outcome
  • Tuberous Sclerosis (complications, drug therapy)
  • Young Adult

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