PMID- 26403211 OWN - NLM STAT- MEDLINE DCOM- 20161227 LR - 20211203 IS - 1468-3083 (Electronic) IS - 0926-9959 (Linking) VI - 30 IP - 4 DP - 2016 Apr TI - Systemic effects of treatment with mTOR inhibitors in tuberous sclerosis complex: a comprehensive review. PG - 586-94 LID - 10.1111/jdv.13356 [doi] AB - Tuberous sclerosis complex (TSC) is a genetic multisystem disorder associated with constitutive overactivation of the mammalian target of rapamycin (mTOR) pathway and characterized by development of benign tumours in various organs. mTOR inhibitors have proven to be effective in the targeted therapy of certain TSC-associated pathologies such as subependymal giant cell astrocytomas (SEGAs) and renal angiomyolipomas (AMLs). Accumulating experimental and clinical data suggest that mTOR inhibitors might have a systemic, disease-modifying influence on affected individuals. This systematic review provides an analysis of available clinical data concerning systemic effect of mTOR inhibitors and the influence of mTOR inhibition on different manifestations of TSC in individual patients. CI - (c) 2015 European Academy of Dermatology and Venereology. FAU - Sadowski, K AU - Sadowski K AD - Department of Neurology and Epileptology, The Children's Memorial Health Institute, Warsaw, Poland. FAU - Kotulska, K AU - Kotulska K AD - Department of Neurology and Epileptology, The Children's Memorial Health Institute, Warsaw, Poland. FAU - Schwartz, R A AU - Schwartz RA AD - Dermatology, Pediatrics, Medicine, and Preventive Medicine and Community Health, Rutgers New Jersey Medical School, Rutgers University, Newark, NJ, USA. AD - Rutgers University School of Public Affairs and Administration, Newark, NJ, USA. FAU - Jozwiak, S AU - Jozwiak S AD - Department of Neurology and Epileptology, The Children's Memorial Health Institute, Warsaw, Poland. AD - Department of Child Neurology, Warsaw Medical University, Warsaw, Poland. LA - eng PT - Journal Article PT - Research Support, Non-U.S. Gov't PT - Review PT - Systematic Review DEP - 20150925 PL - England TA - J Eur Acad Dermatol Venereol JT - Journal of the European Academy of Dermatology and Venereology : JEADV JID - 9216037 RN - EC 2.7.1.1 (MTOR protein, human) RN - EC 2.7.11.1 (TOR Serine-Threonine Kinases) SB - IM MH - Humans MH - TOR Serine-Threonine Kinases/*antagonists & inhibitors MH - Tuberous Sclerosis/*drug therapy EDAT- 2015/09/26 06:00 MHDA- 2016/12/28 06:00 CRDT- 2015/09/26 06:00 PHST- 2015/03/10 00:00 [received] PHST- 2015/08/06 00:00 [accepted] PHST- 2015/09/26 06:00 [entrez] PHST- 2015/09/26 06:00 [pubmed] PHST- 2016/12/28 06:00 [medline] AID - 10.1111/jdv.13356 [doi] PST - ppublish SO - J Eur Acad Dermatol Venereol. 2016 Apr;30(4):586-94. doi: 10.1111/jdv.13356. Epub 2015 Sep 25.