4.6 Article

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

Journal

JOURNAL OF PEDIATRICS
Volume 187, Issue -, Pages 318-+

Publisher

MOSBY-ELSEVIER
DOI: 10.1016/j.jpeds.2017.05.015

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Funding

  1. Novartis
  2. National Institute of Neurological Disorders and Stroke of the NIH [U01-NS082320, U54-NS092090, P20-NS080199]
  3. Tuberous Sclerosis Alliance
  4. Van Andel Research Institute
  5. Upsher-Smith Pharmaceuticals
  6. German Tuberous Sclerosis Foundation

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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.

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