PMID- 30455224 OWN - NLM STAT- MEDLINE DCOM- 20200527 LR - 20211204 IS - 1708-8267 (Electronic) IS - 1081-5589 (Linking) VI - 67 IP - 3 DP - 2019 Mar TI - Continuous low-dose everolimus shrinkage tuberous sclerosis complex-associated renal angiomyolipoma: a 48-month follow-up study. PG - 686-690 LID - 10.1136/jim-2018-000865 [doi] AB - Tuberous sclerosis complex (TSC) is a rare disease that causes multisystem benign neoplasm, induced by dysregulation of the mammalian target of the rapamycin pathway (mTOR). This study aimed to examine the effects of continuous low-dose everolimus, a potent and selective inhibitor of mTOR, on the treatment of TSC-associated renal angiomyolipoma (AML). Between July 2013 and August 2017, 11 patients with TSC-AML were enrolled for an everolimus therapy protocol. An oral everolimus dose starting at 2.5 mg daily was gradually increased to 5.0 mg daily. All patients were evaluated using MRI or CT scanning at baseline, 12, 24, 36 and 48 months after the start of treatment for measuring changes of renal AML mass volume. Everolimus therapy resulted in significant shrinkage of TSC-AML volume after 48 months follow-up. Serum levels of everolimus were subdivided into group I (<8 ng/mL, n=6) and group II (>8 ng/mL, n=5). The volume reduction rates were 10.6%-65.2% in group I and 42.5%-70.6% in group II. To evaluate the response to treatment, three of six (50%) were responders in group I, and all the patients in group II (5/5, 100%) were responders. The differences in AML volume reduction between the groups were statistically significant at 12 months (p=0.011), 24 months (p=0006), 36 months (p=0.014) and 48 months (p=0.05). These results suggest that continuous low-dose everolimus therapy (2.5-5 mg daily) might be effective in shrinking TSC-AML volume and minimizes adverse effects and subsequent reducing medical costs. CI - (c) American Federation for Medical Research 2019. No commercial re-use. See rights and permissions. Published by BMJ. FAU - Wei, Chang-Ching AU - Wei CC AD - Children's Hospital, China Medical University Hospital, Taichung, Taiwan. AD - School of Medicine, China Medical University, Taichung, Taiwan. FAU - Tsai, Jeng-Daw AU - Tsai JD AD - Division of Nephrology, Department of Pediatrics, MacKay Children's Hospital, Taipei, Taiwan. AD - Department of Medicine, MacKay Medical College, New Taipei City, Taiwan. AD - Department of Pediatrics, Taipei Medical University Hospital, Taipei, Taiwan. AD - Department of Pediatrics, School of Medicine, College of Medicine, Taipei Medical University, Taipei, Taiwan. FAU - Sheu, Ji-Nan AU - Sheu JN AD - School of Medicine, Chung Shan Medical University, Taichung, Taiwan. AD - Department of Paediatrics, Chung Shan Medical University Hospital, Taichung, Taiwan. FAU - Chen, Sung-Lang AU - Chen SL AD - School of Medicine, Chung Shan Medical University, Taichung, Taiwan. AD - Department of Urology, Chung Shan Medical University Hospital, Taichung, Taiwan. FAU - Tsao, Teng-Fu AU - Tsao TF AD - School of Medicine, Chung Shan Medical University, Taichung, Taiwan. AD - Department of Medical Imaging, Chung Shan Medical University Hospital, Taichung, Taiwan. FAU - Yang, Sheng-Hui AU - Yang SH AD - Department of Life Sciences, National Chung Hsing University, Taichung, Taiwan. FAU - Tsai, Jeng-Dau AU - Tsai JD AD - School of Medicine, Chung Shan Medical University, Taichung, Taiwan. AD - Department of Paediatrics, Chung Shan Medical University Hospital, Taichung, Taiwan. LA - eng PT - Journal Article DEP - 20181118 PL - England TA - J Investig Med JT - Journal of investigative medicine : the official publication of the American Federation for Clinical Research JID - 9501229 RN - 0 (Immunosuppressive Agents) RN - 9HW64Q8G6G (Everolimus) RN - EC 2.7.1.1 (MTOR protein, human) RN - EC 2.7.11.1 (TOR Serine-Threonine Kinases) SB - IM MH - Adolescent MH - Adult MH - Angiomyolipoma/diagnostic imaging/*drug therapy MH - Everolimus/*administration & dosage MH - Female MH - Follow-Up Studies MH - Humans MH - Immunosuppressive Agents/*administration & dosage MH - Kidney Neoplasms/diagnostic imaging/*drug therapy MH - Male MH - TOR Serine-Threonine Kinases/*antagonists & inhibitors MH - Time Factors MH - Tuberous Sclerosis/diagnostic imaging/*drug therapy MH - Young Adult OTO - NOTNLM OT - neoplasms OT - rare diseases COIS- Competing interests: None declared. EDAT- 2018/11/21 06:00 MHDA- 2020/05/28 06:00 CRDT- 2018/11/21 06:00 PHST- 2018/10/20 00:00 [accepted] PHST- 2018/11/21 06:00 [pubmed] PHST- 2020/05/28 06:00 [medline] PHST- 2018/11/21 06:00 [entrez] AID - jim-2018-000865 [pii] AID - 10.1136/jim-2018-000865 [doi] PST - ppublish SO - J Investig Med. 2019 Mar;67(3):686-690. doi: 10.1136/jim-2018-000865. Epub 2018 Nov 18.