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

Siroky, Brian J; Towbin, Alexander J; Trout, Andrew T; Schäfer, Hannah; Thamann, Anna R; Agricola, Karen D; Tudor, Cynthia; Capal, Jamie et al. · J Pediatr · 2017

case_series · Level IV

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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.

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