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Letter to the Editor| Volume 51, ISSUE 3, e9-e10, September 2014

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Monitoring of Everolimus Trough Concentrations for Achieving Optimal Tolerance in the Management of Tuberous Sclerosis Complex

      Tuberous sclerosis complex (TSC) is a genetic disorder characterized by the development of a variety of benign tumors along with an increased risk of malignancy. Mechanism of disease is related to the mTOR pathway.
      • Jozwiak J.
      • Jozwiak S.
      • Wlodarski P.
      Possible mechanisms of disease development in tuberous sclerosis.
      In spite of this, individual features have different natural histories, and it is difficult to predict the extent to which a child with newly diagnosed disease is affected.
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      References

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        • Jozwiak S.
        • Wlodarski P.
        Possible mechanisms of disease development in tuberous sclerosis.
        Lancet. 2008 Jan; 9: 73-79
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        Everolimus: in patients with subependymal giant cell astrocytoma associated with tuberous sclerosis complex.
        Pediatr Drugs. 2012; 14: 51-60
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        • et al.
        Everolimus for angiomyolipoma associated with tuberous sclerosis complex or sporadic lymphangioleiomyomatosis (EXIST-2): a multicentre, randomised, double-blind, placebo-controlled trial.
        Lancet. 2013; 9: 817-824
        • Danesi Romano
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        • Ravaud Alain
        Oral and intravenously administered mTOR inhibitors for metastatic renal cell carcinoma: pharmacokinetic considerations and clinical implications.
        Cancer Treat Rev. 2013; 39: 784-792