Multicenter Phase 2 Trial of Sirolimus for Tuberous Sclerosis: Kidney Angiomyolipomas and Other Tumors Regress and VEGF- D Levels Decrease

@inproceedings{Dabora2011MulticenterP2,
  title={Multicenter Phase 2 Trial of Sirolimus for Tuberous Sclerosis: Kidney Angiomyolipomas and Other Tumors Regress and VEGF- D Levels Decrease},
  author={Sandra L. Dabora and David Neal Franz and Stephen Ashwal and Arthur L Sagalowsky and Francis DiMario and Daniel S. Miles and Drew Cutler and Darcy A Krueger and Raul N. Uppot and Rahmin A Rabenou and Susana Camposano and Jan L Paolini and Fiona Fennessy and Nancy A. Lee and Chelsey L Woodrum and Judith B. Manola and Judy Garber and Elizabeth Anne Thiele},
  booktitle={PloS one},
  year={2011}
}
BACKGROUND Tuberous sclerosis (TSC) related tumors are characterized by constitutively activated mTOR signaling due to mutations in TSC1 or TSC2. METHODS We completed a phase 2 multicenter trial to evaluate the efficacy and tolerability of the mTOR inhibitor, sirolimus, for the treatment of kidney angiomyolipomas. RESULTS 36 adults with TSC or TSC/LAM were enrolled and started on daily sirolimus. The overall response rate was 44.4% (95% confidence intervals [CI] 28 to 61); 16/36 had a… CONTINUE READING
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