RT Book, Section
A1 Irazabal, Maria V.
A1 Torres, Vicente E.
A2 Lerma, Edgar V.
A2 Rosner, Mitchell H.
A2 Perazella, Mark A.
SR Print(0)
ID 1149115998
T1 Cystic Diseases of the Kidney
T2 CURRENT Diagnosis & Treatment: Nephrology & Hypertension, 2e
YR 2017
FD 2017
PB McGraw-Hill Education
PP New York, NY
SN 9781259861055
LK accessmedicine.mhmedical.com/content.aspx?aid=1149115998
RD 2024/04/20
AB Renal  cystic  disease  includes  a  myriad  of  conditions  characterized  by  the  presence  of  multiple  hereditary  or  acquired  renal  cysts,  which  differ  in  their  clinical  presentation,  prognosis,  and  management.  Renal  cysts  are  composed  of  smooth-walled,  enclosed  fluid-filled  circular  structures  that  form  in  the  kidney  by  focal  out  pouching  of  renal  tubules.  Although  the  mechanism  underlying  cyst  formation  remain  to  be  fully  elucidated,  defects  in  the  primary  ciliary  sensing  mechanisms,  intracellular  calcium  regulation,  and  cellular  cyclic  AMP  (cAMP)  accumulation  have  been  shown  to  contribute  to  cyst  formation  in  autosomal  dominant  and  autosomal  recessive  polycystic  kidney  diseases  (ADPKD  and  ARPKD),  which  represent  a  significant  cause  of  morbidity  and  mortality  in  children  and  young  adults.  Currently,  there  are  no  FDA  approved  treatments  for  ADPKD,  and  the  existing  standard  of  care  includes  risk  modification,  management  of  complications,  and  renal  replacement  therapy  with  dialysis  or  organ  transplantation.  Several  drugs  designed  to  slow  or  arrest  the  progression  of  ADPKD  have  shown  promise  in  preclinical  models  and  clinical  trials,  including  vasopressin  receptor  antagonists  and  somatostatin  analogs.  The  vasopressin-2  receptor  antagonist  Tolvaptan  has  been  the  most  successful  drug  therapy  to  reduce  the  rate  of  increase  in  total  kidney  volume  (TKV)  and  ameliorate  renal  functional  decline  in  patients  with  ADPKD.  Further  elucidation  of  the  mechanisms  implicated  in  cyst  formation  and  development  would  provide  impetus  for  correcting  the  underlying  abnormalities  in  cystic  pathways.