PMID- 20663789
OWN - NLM
STAT- MEDLINE
DCOM- 20110201
LR  - 20211203
IS  - 1460-2385 (Electronic)
IS  - 0931-0509 (Linking)
VI  - 25
IP  - 11
DP  - 2010 Nov
TI  - Low-dose rapamycin reduces kidney volume angiomyolipomas and prevents the loss of 
      renal function in a patient with tuberous sclerosis complex.
PG  - 3787-91
LID - 10.1093/ndt/gfq456 [doi]
AB  - Tuberous sclerosis complex (TSC) is caused by constitutively activated mammalian 
      target of rapamycin (mTOR) resulting in non-malignant tumours of several organs 
      including renal angiomyolipomas (AMLs). AMLs may originate renal failure, 
      hypertension and spontaneous life-threatening bleeding. Recent reports suggest a 
      possible beneficial role of the mTOR inhibitor rapamycin for TSC. However, safety 
      and efficiency of rapamycin in TSC patients as an anti-proliferative agent are 
      still undefined. A 40-year-old man with sporadic TSC and a history of spontaneous 
      bleeding from his left kidney AMLs received low-dose rapamycin for 12 months, and 
      this was associated with a reduction in bilateral kidney AML volume, 
      stabilization and even improvement of renal function. There was also a reduction 
      of facial angiofibromas, improvement of blood pressure control and absence of AML 
      bleeding over this time period. Brain lesion images remained stable, and no 
      significant rapamycin-associated side effects were noted. To the best of our 
      knowledge, this is the first report of a case of reduction in renal AML volume 
      together with preservation of renal function in a patient with TSC receiving 
      low-dose rapamycin. These data suggest that it could be the result of the 
      anti-angiogenic, anti-fibrotic and anti-proliferative effects of rapamycin.
FAU - Peces, Ramon
AU  - Peces R
AD  - Servicio de Nefrologia, Hospital Universitario La Paz, Madrid, Spain. 
      cpeces@varnetmail.com
FAU - Peces, Carlos
AU  - Peces C
FAU - Cuesta-Lopez, Emilio
AU  - Cuesta-Lopez E
FAU - Perez-Duenas, Virginia
AU  - Perez-Duenas V
FAU - Vega-Cabrera, Cristina
AU  - Vega-Cabrera C
FAU - Azorin, Sebastian
AU  - Azorin S
FAU - Selgas, Rafael
AU  - Selgas R
LA  - eng
PT  - Case Reports
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
DEP - 20100727
PL  - England
TA  - Nephrol Dial Transplant
JT  - Nephrology, dialysis, transplantation : official publication of the European 
      Dialysis and Transplant Association - European Renal Association
JID - 8706402
RN  - EC 2.7.11.1 (TOR Serine-Threonine Kinases)
RN  - W36ZG6FT64 (Sirolimus)
SB  - IM
MH  - Adult
MH  - Angiomyolipoma/*drug therapy
MH  - Humans
MH  - Kidney/pathology/physiopathology
MH  - Kidney Neoplasms/*drug therapy
MH  - Magnetic Resonance Imaging
MH  - Male
MH  - Sirolimus/*therapeutic use
MH  - TOR Serine-Threonine Kinases/*antagonists & inhibitors
MH  - Tuberous Sclerosis/*drug therapy/pathology/physiopathology
EDAT- 2010/07/29 06:00
MHDA- 2011/02/02 06:00
CRDT- 2010/07/29 06:00
PHST- 2010/07/29 06:00 [entrez]
PHST- 2010/07/29 06:00 [pubmed]
PHST- 2011/02/02 06:00 [medline]
AID - gfq456 [pii]
AID - 10.1093/ndt/gfq456 [doi]
PST - ppublish
SO  - Nephrol Dial Transplant. 2010 Nov;25(11):3787-91. doi: 10.1093/ndt/gfq456. Epub 
      2010 Jul 27.