PMID- 25258166
OWN - NLM
STAT- MEDLINE
DCOM- 20150407
LR  - 20211203
IS  - 1471-2490 (Electronic)
IS  - 1471-2490 (Linking)
VI  - 14
DP  - 2014 Sep 25
TI  - Percutaneous cryoablation for tuberous sclerosis-associated renal angiomyolipoma 
      with neoadjuvant mTOR inhibition.
PG  - 77
LID - 10.1186/1471-2490-14-77 [doi]
AB  - BACKGROUND: Renal angiomyolipomas (AMLs) are frequent in tuberous sclerosis and 
      are responsible for a significant proportion of the morbidity in adulthood, 
      mainly from bleeding complications, which are correlated to the size of the AMLs. 
      We describe the case of a 19-year-old female with multiple bilateral renal 
      angiomyolipomas. CASE PRESENTATION: The renal AMLs measured up to 6 cm in size. 
      She was first treated with a low dose of the mammalian target of rapamycin (mTOR) 
      inhibitor sirolimus (up to 3 mg/day over a 12-month period) and following 
      significant AML size reduction, percutaneous cryoablation was performed. No 
      side-effects of either treatment were reported. At 12 months post-cryoablation, 
      no recurrence of the AML was noted. CONCLUSION: This is the first report of this 
      treatment strategy and the case study reveals that combining a low dose of an 
      mTOR inhibitor with percutaneous cryoablation to treat small tumors mitigates the 
      side-effects while providing a good clinical outcome. This therapeutic approach 
      is a novel tool for the clinician involved in the management of patients with 
      tuberous sclerosis.
FAU - Krummel, Thierry
AU  - Krummel T
AD  - Department of Nephrology, University Hospital, Strasbourg, France. 
      thierry.krummel@chru-strasbourg.fr.
FAU - Garnon, Julien
AU  - Garnon J
FAU - Lang, Herve
AU  - Lang H
FAU - Gangi, Afshin
AU  - Gangi A
FAU - Hannedouche, Thierry
AU  - Hannedouche T
LA  - eng
PT  - Journal Article
DEP - 20140925
PL  - England
TA  - BMC Urol
JT  - BMC urology
JID - 100968571
RN  - EC 2.7.11.1 (TOR Serine-Threonine Kinases)
RN  - W36ZG6FT64 (Sirolimus)
SB  - IM
MH  - Adult
MH  - Angiomyolipoma/complications/pathology/*therapy
MH  - Cryosurgery/*methods
MH  - Female
MH  - Humans
MH  - Kidney Neoplasms/complications/pathology/*therapy
MH  - Neoadjuvant Therapy
MH  - Sirolimus/*therapeutic use
MH  - TOR Serine-Threonine Kinases/*antagonists & inhibitors
MH  - Treatment Outcome
MH  - Tuberous Sclerosis/*complications
MH  - Young Adult
PMC - PMC4177726
EDAT- 2014/09/27 06:00
MHDA- 2015/04/08 06:00
PMCR- 2014/09/25
CRDT- 2014/09/27 06:00
PHST- 2014/02/03 00:00 [received]
PHST- 2014/09/22 00:00 [accepted]
PHST- 2014/09/27 06:00 [entrez]
PHST- 2014/09/27 06:00 [pubmed]
PHST- 2015/04/08 06:00 [medline]
PHST- 2014/09/25 00:00 [pmc-release]
AID - 1471-2490-14-77 [pii]
AID - 10.1186/1471-2490-14-77 [doi]
PST - epublish
SO  - BMC Urol. 2014 Sep 25;14:77. doi: 10.1186/1471-2490-14-77.