title: Cost-effectiveness of tolvaptan in autosomal dominant polycystic kidney disease.
abstract: Chinese translation
  
  
  BACKGROUND
  In the TEMPO (Tolvaptan Efficacy and Safety in Management of Autosomal Dominant Polycystic Kidney Disease and Its Outcomes) trial, tolvaptan significantly reduced expansion of kidney volume and loss of kidney function.
  
  
  OBJECTIVE
  To determine how the benefits of tolvaptan seen in TEMPO may relate to longer-term health outcomes, such as progression to end-stage renal disease (ESRD) and death, and cost-effectiveness.
  
  
  METHODS
  A decision-analytic model.
  
  
  METHODS
  Published literature from 1993 to 2012.
  
  
  METHODS
  Persons with early autosomal dominant polycystic kidney disease.
  
  
  METHODS
  Lifetime.
  
  
  CONCLUSIONS
  Societal.
  
  
  METHODS
  Patients received tolvaptan therapy until death, development of ESRD, or liver complications or no tolvaptan therapy.
  
  
  METHODS
  Median age at ESRD onset, life expectancy, discounted quality-adjusted life-years and lifetime costs (in 2010 U.S. dollars), and incremental cost-effectiveness ratios.
  
  
  RESULTS
  Tolvaptan prolonged the median age at ESRD onset by 6.5 years and increased life expectancy by 2.6 years. At $5760 per month, tolvaptan cost $744 100 per quality-adjusted life-year gained compared with standard care.
  
  
  RESULTS
  For patients with autosomal dominant polycystic kidney disease that progressed more slowly, the cost per quality-adjusted life-year gained was even greater for tolvaptan.
  
  
  CONCLUSIONS
  Although TEMPO followed patients for 3 years, the main analysis assumed that clinical benefits persisted over patients' lifetimes.
  
  
  CONCLUSIONS
  Assuming that the benefits of tolvaptan persist in the longer term, the drug may slow progression to ESRD and reduce mortality rates. However, barring an approximately 95% reduction in price, cost-effectiveness does not compare favorably with many other commonly accepted medical interventions.
  
  
  BACKGROUND
  National Institutes of Health and Agency for Healthcare Research and Quality.
authors.forename: Kevin F
authors.initials: KF
authors.lastname: Erickson
authors.forename: Glenn M
authors.initials: GM
authors.lastname: Chertow
authors.forename: Jeremy D
authors.initials: JD
authors.lastname: Goldhaber-Fiebert
mesh_terms: D000328:Adult; D017668:Age of Onset; D000368:Aged; D000369:Aged, 80 and over; D001552:Benzazepines / Q000009:adverse effects / Q000191:economics* / Q000627:therapeutic use*; D003362:Cost-Benefit Analysis; D018450:Disease Progression; D005260:Female; D005919:Glomerular Filtration Rate / Q000187:drug effects; D006801:Humans; D007676:Kidney Failure, Chronic / Q000209:etiology; D008017:Life Expectancy; D008297:Male; D008390:Markov Chains; D008875:Middle Aged; D016891:Polycystic Kidney, Autosomal Dominant / Q000150:complications / Q000188:drug therapy* / Q000503:physiopathology; D019057:Quality-Adjusted Life Years; D000077602:Tolvaptan
chemical_list: D001552:Benzazepines; D000077602:Tolvaptan
references.citation: Natl Vital Stat Rep. 2010 Jun 28;58(21):1-40
references.pmid: 21043319
references.citation: Kidney Int. 2000 Sep;58(3):925-43
references.pmid: 10972657
references.citation: Med Decis Making. 1993 Apr-Jun;13(2):89-102
references.pmid: 8483408
references.citation: Am J Kidney Dis. 2002 Jun;39(6):1127-34
references.pmid: 12046022
references.citation: N Engl J Med. 1996 Apr 11;334(15):939-45
references.pmid: 8596594
references.citation: N Engl J Med. 1994 Mar 31;330(13):877-84
references.pmid: 8114857
references.citation: N Engl J Med. 2012 Dec 20;367(25):2407-18
references.pmid: 23121377
references.citation: N Engl J Med. 2012 Dec 27;367(26):2541-2
references.pmid: 23121375
references.citation: N Engl J Med. 2004 Sep 23;351(13):1296-305
references.pmid: 15385656
references.citation: Clin Exp Nephrol. 2011 Aug;15(4):539-45
references.pmid: 21431900
references.citation: Kidney Int. 2005 Dec;68(6):2801-8
references.pmid: 16316356
references.citation: Kidney Int. 2003 Feb;63(2):678-85
references.pmid: 12631134
references.citation: JAMA. 1996 Oct 16;276(15):1253-8
references.pmid: 8849754
references.citation: Clin J Am Soc Nephrol. 2009 Mar;4(3):560-6
references.pmid: 19261830
references.citation: Health Aff (Millwood). 2004 Jul-Aug;23(4):176-83
references.pmid: 15318578
references.citation: Neurology. 1996 Mar;46(3):861-9
references.pmid: 8618713
references.citation: Am J Kidney Dis. 2001 Oct;38(4):777-84
references.pmid: 11576881