Tolvaptan in patients with autosomal dominant polycystic kidney disease

New England Journal of Medicine

Volumn 367, Issue 25, 2012, Pages 2407-2418

  Torres, Vicente E ^a   Chapman, Arlene B ^b   Devuyst, Olivier ^c,d   Gansevoort, Ron T ^e   Grantham, Jared J ^f   Higashihara, Eiji ^g   Perrone, Ronald D ^h   Krasa, Holly B ⁱ   Ouyang, John ⁱ   Czerwiec, Frank S ⁱ  

^a MAYO CLINIC   (United States)

^b EMORY UNIVERSITY SCHOOL OF MEDICINE   (United States)

^c CLINIQUES UNIVERSITAIRES SAINT LUC   (Belgium)

^d UNIVERSITY OF ZURICH   (Switzerland)

^e UNIVERSITY MEDICAL CENTER GRONINGEN   (Netherlands)

^f UNIVERSITY OF KANSAS SCHOOL OF MEDICINE   (United States)

^g KYORIN UNIVERSITY   (Japan)

^h TUFTS UNIVERSITY SCHOOL OF MEDICINE   (United States)

ⁱ Otsuka Pharmaceutical Development and Commercialization   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CREATININE; PLACEBO; TOLVAPTAN;

ABNORMAL URINE COMPOSITION; ADULT; ALANINE AMINOTRANSFERASE BLOOD LEVEL; ALBUMINURIA; AQUARESIS; ARTICLE; ASPARTATE AMINOTRANSFERASE BLOOD LEVEL; CONTROLLED STUDY; CREATININE BLOOD LEVEL; CREATININE CLEARANCE; DIARRHEA; DISEASE COURSE; DISEASE SEVERITY; DIZZINESS; DOUBLE BLIND PROCEDURE; DRUG BLOOD LEVEL; DRUG DOSE ESCALATION; DRUG FATALITY; DRUG RESPONSE; DRUG TOLERABILITY; DRUG WITHDRAWAL; FATIGUE; FEMALE; HEADACHE; HUMAN; HYPERTENSION; KIDNEY FAILURE; KIDNEY PAIN; KIDNEY POLYCYSTIC DISEASE; KIDNEY SIZE; MAJOR CLINICAL STUDY; MALE; MULTICENTER STUDY; NOCTURIA; PATIENT COMPLIANCE; PHASE 3 CLINICAL TRIAL; POLLAKISURIA; POLYDIPSIA; POLYURIA; PRIORITY JOURNAL; RANDOMIZED CONTROLLED TRIAL; SIDE EFFECT; THIRST; THORAX PAIN; TOXIC HEPATITIS; XEROSTOMIA;

EID: 84871303897     PISSN: 00284793     EISSN: 15334406     Source Type: Journal    
DOI: 10.1056/NEJMoa1205511     Document Type: Article

References (36)

1. Torres VE, Harris PC, Pirson Y. Autosomal dominant polycystic kidney disease. Lancet 2007;369:1287-301. (Pubitemid 46552051)
2. Grantham JJ. Autosomal dominant polycystic kidney disease. N Engl J Med 2008;359:1477-85.
3. Gattone VH II, Maser RL, Tian C, Rosenberg JM, Branden MG. Developmental expression of urine concentration-associated genes and their altered expression in murine infantile-type polycystic kidney disease. Dev Genet 1999;24:309-18. (Pubitemid 29211795)
4. Gattone VH, Wang X, Harris PC, Torres VE. Inhibition of renal cystic disease development and progression by a vasopressin V2 receptor antagonist. Nat Med 2003;9:1323-6. (Pubitemid 37279858)
5. Torres VE, Wang X, Qian Q, Somlo S, Harris PC, Gattone VH II. Effective treatment of an orthologous model of autosomal dominant polycystic kidney disease. Nat Med 2004;10:363-4. (Pubitemid 38508512)
6. Wang X, Gattone V II, Harris PC, Torres VE. Effectiveness of vasopressin V2 receptor antagonists OPC-31260 and OPC-41061 on polycystic kidney disease development in the PCK rat. J Am Soc Nephrol 2005;16:846-51. (Pubitemid 41710293)
7. Nagao S, Nishii K, Katsuyama M, et al. Increased water intake decreases progression of polycystic kidney disease in the PCK rat. J Am Soc Nephrol 2006;17:2220-7. (Pubitemid 44141912)
8. Wang X, Wu Y, Ward CJ, Harris PC, Torres VE. Vasopressin directly regulates cyst growth in the polycystic kidney disease. J Am Soc Nephrol 2008;19:102-8.
9. Meijer E, Gansevoort RT, de Jong PE, et al. Therapeutic potential of vasopressin V2 receptor antagonist in a mouse model for autosomal dominant polycystic kidney disease: optimal timing and dosing of the drug. Nephrol Dial Transplant 2011;26:2445-53.
10. Yamamura Y, Nakamura S, Itoh S, et al. OPC-41061, a highly potent human vasopressin V2-receptor antagonist: pharmacological profile and aquaretic effect by single and multiple oral dosing in rats. J Pharmacol Exp Ther 1998;287:860-7. (Pubitemid 29131593)
11. Higashihara E, Torres VE, Chapman AB, et al. Tolvaptan in autosomal dominant polycystic kidney disease: three years' experience. Clin J Am Soc Nephrol 2011;6:2499-507.
12. Torres VE, Meijer E, Bae KT, et al. Rationale and design of the TEMPO (Tolvaptan Efficacy and Safety in Management of Autosomal Dominant Polycystic Kidney Disease and its Outcomes) 3/4 study. Am J Kidney Dis 2011;57:692-9.
13. Ravine D, Gibson RN, Walker RG, Sheffield LJ, Kincaid-Smith P, Danks DM. Evaluation of ultrasonographic diagnostic criteria for autosomal dominant polycystic kidney disease 1. Lancet 1994;343:824-7. (Pubitemid 24147512)
14. Grantham JJ, Torres VE, Chapman AB, et al. Volume progression in polycystic kidney disease. N Engl J Med 2006;354:2122-30. (Pubitemid 43736612)
15. Chapman AB, Bost JE, Torres VE, et al. Kidney volume and functional outcomes in autosomal dominant polycystic kidney disease. Clin J Am Soc Nephrol 2012;7:479-86.
16. Cockcroft DW, Gault MH. Prediction of creatinine clearance from serum creatinine. Nephron 1976;16:31-41.
17. Levey AS, Coresh J, Greene T, et al. Using standardized serum creatinine values in the Modification of Diet in Renal Disease study equation for estimating glomerular filtration rate. Ann Intern Med 2006;145:247-54. [Erratum, Ann Intern Med 2008;149:519.] (Pubitemid 46768608)
18. Rule AD, Torres VE, Chapman AB, et al. Comparison of methods for determining renal function decline in early autosomal dominant polycystic kidney disease: the Consortium of Radiologic Imaging Studies of Polycystic Kidney Disease cohort. J Am Soc Nephrol 2006;17:854-62.
19. Horio M, Imai E, Yasuda Y, Watanabe T, Matsuo S. Modification of the CKD epidemiology collaboration (CKD-EPI) equation for Japanese: accuracy and use for population estimates. Am J Kidney Dis 2010;56:32-8.
20. Irazabal MV, Torres VE, Hogan MC, et al. Short-term effects of tolvaptan on renal function and volume in patients with autosomal dominant kidney disease. Kidney Int 2011;80:295-301.
21. Boertien WE, Meijer E, de Jong PE, et al. Short-term effects on efficacy parameters with tolvaptan in subjects with ADPKD at various levels of kidney function. J Am Soc Nephrol 2012;23:243A. abstract.
22. Lefante JJ. The power to detect differences in average rates of change in longitudinal studies. Stat Med 1990;9:437-46. (Pubitemid 20245414)
23. Laird NM, Ware JH. Random-effects model for longitudinal data. Biometrics 1982;38:963-74.
24. Therneau TM, Grambsch PM. Modeling survival data: extending the Cox model. New York: Springer, 2000.
25. - secretion, and in vitro cyst growth of human ADPKD cells stimulated by vasopressin. Am J Physiol Renal Physiol 2011;301:F1005-F1113.
26. Grantham JJ, Chapman AB, Torres VE. Volume progression in autosomal dominant polycystic kidney disease: the major factor determining clinical outcomes. Clin J Am Soc Nephrol 2006;1:148-57.
27. Walz G, Budde K, Mannaa M, et al. Everolimus in patients with autosomal dominant polycystic kidney disease. N Engl J Med 2010;363:830-40. [Errata, N Engl J Med 2010;363:1190, 1977.]
28. Schrier RW, Gross P, Gheorghiade M, et al. Tolvaptan, a selective oral vasopressin V2-receptor antagonist, for hyponatremia. N Engl J Med 2006;355:2099-112. (Pubitemid 44749318)
29. Konstam MA, Gheorghiade M, Burnett JC Jr, et al. Effects of oral tolvaptan in patients hospitalized for worsening heart failure: the EVEREST Outcome Trial. JAMA 2007;297:1319-31. (Pubitemid 46513233)
30. Berl T, Quittnat-Pelletier F, Verbalis JG, et al. Oral tolvaptan is safe and effective in chronic hyponatremia. J Am Soc Nephrol 2010;21:705-12. [Erratum, J Am Soc Nephrol 2010;21:1407.]
31. Matsuzaki M, Hori M, Izumi T, Fukunami M. Efficacy and safety of tolvaptan in heart failure patients with volume overload despite the standard treatment with conventional diuretics: a phase III, randomized, double-blind, placebo-controlled study (QUEST Study). Cardiovasc Drugs Ther 2011;25:Suppl 1:S33-S45.
32. Ruggenenti P, Remuzzi A, Ondei P, et al. Safety and efficacy of long-acting somatostatin treatment in autosomal-dominant polycystic kidney disease. Kidney Int 2005;68:206-16. (Pubitemid 43181378)
33. van Keimpema L, Nevens F, Vanslembrouck R, et al. Lanreotide reduces the volume of polycystic liver: a randomized, double-blind, placebo-controlled trial. Gastroenterology 2009;137:1661-8.
34. Hogan MC, Masyuk TV, Page LJ, et al. Randomized clinical trial of long-acting somatostatin for autosomal dominant polycystic kidney and liver disease. J Am Soc Nephrol 2010;21:1052-61.
35. Serra AL, Poster D, Kistler AD, et al. Sirolimus and kidney growth in autosomal dominant polycystic kidney disease. N Engl J Med 2010;363:820-9.
36. Perico N, Antiga L, Caroli A, et al. Sirolimus therapy to halt the progression of ADPKD. J Am Soc Nephrol 2010;21:1031-40.