Poliquistosis renal autosómica dominante: ¿quiénes se benefician del tratamiento con tolvaptán? Estado del arte

Ricardo Silvariño; Leticia Peluffo; Eliana Cabrera; Sofía San Román; Cecilia Baccino; Oscar Noboa

doi:10.29193/RMU.38.3.11

Authors

Ricardo Silvariño Universidad de la República, Facultad de Medicina, Centro de Nefrología. Cooperativa de Servicios Médicos, Servicio de Nefrología
Leticia Peluffo Cooperativa de Servicios Médicos, Servicio de Nefrología
Eliana Cabrera Cooperativa de Servicios Médicos, Servicio de Nefrología
Sofía San Román Cooperativa de Servicios Médicos, Servicio de Nefrología
Cecilia Baccino Universidad de la República, Facultad de Medicina, Centro de Nefrología
Oscar Noboa Universidad de la República, Facultad de Medicina, Centro de Nefrología. Cooperativa de Servicios Médicos, Servicio de Nefrología

DOI:

https://doi.org/10.29193/RMU.38.3.11

Keywords:

AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY, CHRONIC KIDNEY DISEASE, TOLVAPTAN

Abstract

Autosomal dominant polycystic kidney disease is the most common hereditary kidney disease. It is characterized by the progressive appearance of renal cysts that usually lead to extreme chronic kidney disease in adulthood. The approval of the use of tolvaptán (V2 vasopressin receptor antagonist) has meant a significant change in the treatment of this disease. In recent years, evidence has proved the benefits of initiating treatment with tolvaptán in patients with a rapidly evolving disease. A descriptive review of the main clinical studies published in 2012-2022 period is carried out and a useful scheme is suggested to select those patients who can benefit from the start of treatment.

References

1) Ars E, Bernis C, Fraga G, Furlano M, Martínez V, Martins J, et al. Documento de consenso de poliquistosis renal autosómica dominantedel grupo de trabajo de enfermedades hereditarias de la Sociedad Española de Nefrología: revisión 2020. Nefrología 2020; 42(4), 367-89. doi: 10.1016/j.nefro.2021.05.009.
2) Suwabe T, Shukoor S, Chamberlain A, Killian J, King B, Edwards M, et al. Epidemiology of autosomal dominant polycystic kidney disease in Olmsted County. Clin J Am Soc Nephrol 2020; 15(1):69-79. doi: 10.2215/CJN.05900519.
3) Neumann H, Jilg C, Bacher J, Nabulsi Z, Malinoc A, Hummel B, et al. Epidemiology of autosomal-dominant polycystic kidney disease: an in-depth clinical study for south-western Germany. Nephrol Dial Transplant 2013; 28(6):1472-87. doi: 10.1093/ndt/gfs551.
4) Martínez V, Comas J, Arcos E, Díaz J, Muray S, Cabezuelo J, et al. Renal replacement therapy in ADPKD patients: a 25-year survey based on the Catalan registry. BMC Nephrol 2013; 14:186. doi: 10.1186/1471-2369-14-186.
5) González-Bedat M, Luzardo L, Oroza V, Ceretta M, Silvariño R, et al. Registro Uruguayo de DIálisis. Informe 2019. Montevideo: SUN, 2019. Disponible en https://www.nefrologia.hc.edu.uy/images/INFORME_ANUAL_RUD_2019._ACTIVIDADES_INCIDENCIA_Y_PREVALENCIA_2021.pdf. (Consulta: 20/5/22).
6) Müller R, Messchendorp A, Birn H, Capasso G, Cornec-Le Gall E, Devuyst O, et al. An update on the use of tolvaptan for autosomal dominant polycystic kidney disease: consensus statement on behalf of the ERA Working Group on Inherited Kidney Disorders, the European Rare Kidney Disease Reference Network and Polycystic Kidney Disease International. Nephrol Dial Transplant 2022; 37(5):825-39. doi: 10.1093/ndt/gfab312.
7) Torres V, Harris P. Strategies targeting cAMP signaling in the treatment of polycystic kidney disease. J Am Soc Nephrol 2014; 25(1):18-32. doi: 10.1681/ASN.2013040398.
8) Chebib F, Perrone R, Chapman A, Dahl N, Harris P, Mrug M, et al. A practical guide for treatment of rapidly progressive ADPKD with Tolvaptan. J Am Soc Nephrol 2018; 29(10):2458-70. doi: 10.1681/ASN.2018060590.
9) Chapman, A. B., Bost, J. E., Torres, V. E., Guay-Woodford, L., Bae, K. T., Landsittel, D., ... & Grantham, J. J. (2012). Kidney volume and functional outcomes in autosomal dominant polycystic kidney disease. Clinical Journal of the American Society of Nephrology, 7(3), 479-486.
10) Torres V, Chapman A, Devuyst O, Gansevoort R, Grantham J, Higashihara E, et al. Tolvaptan in patients with autosomal dominant polycystic kidney disease. N Engl J Med 2012; 367(25):2407-18. doi: 10.1056/NEJMoa1205511.
11) Torres V, Higashihara E, Devuyst O, Chapman A, Gansevoort R, Grantham J, et al. Effect of Tolvaptan in autosomal dominant polycystic kidney disease by CKD Stage: results from the TEMPO 3:4 Trial. Clin J Am Soc Nephrol 2016; 11(5):803-11. doi: 10.2215/CJN.06300615.
12) Torres V, Chapman A, Devuyst O, Gansevoort R, Perrone R, Dandurand A, et al. Multicenter, open-label, extension trial to evaluate the long-term efficacy and safety of early versus delayed treatment with tolvaptan in autosomal dominant polycystic kidney disease: the TEMPO 4:4 Trial. Nephrol Dial Transplant 2018; 33(3):477-89. doi: 10.1093/ndt/gfx043.
13) Torres V, Chapman A, Devuyst O, Gansevoort R, Perrone R, Koch G, et al. Tolvaptan in later-stage autosomal dominant polycystic kidney disease. N Engl J Med 2017; 377(20):1930-42. doi: 10.1056/NEJMoa1710030.
14) Edwards M, Chebib F, Irazabal M, Ofstie T, Bungum L, Metzger A, et al. Long-term administration of tolvaptan in autosomal dominant polycystic kidney disease. Clin J Am Soc Nephrol 2018; 13(8):1153-61. doi: 10.2215/CJN.01520218.
15) Higashihara E, Torres V, Chapman A, Grantham J, Bae K, Watnick T, et al. Tolvaptan in autosomal dominant polycystic kidney disease: three years’ experience. Clin J Am Soc Nephrol 2011; 6(10):2499-507. doi: 10.2215/CJN.03530411.
16) Grantham J, Torres V, Chapman A, Guay-Woodford L, Bae K, King BJr, et al. Volume progression in polycystic kidney disease. N Engl J Med 2006; 354(20):2122-30. doi: 10.1056/NEJMoa054341.
17) Torres V, Abebe K, Chapman A, Schrier R, Braun W, Steinman T, et al. Angiotensin blockade in late autosomal dominant polycystic kidney disease. N Engl J Med 2014; 371(24):2267-76. doi: 10.1056/NEJMoa1402686.
18) Torres V, Chapman A, Devuyst O, Gansevoort R, Perrone R, Lee J, et al. Multicenter study of long-term safety of Tolvaptan in later-stage autosomal dominant polycystic kidney disease. Clin J Am Soc Nephrol 2020; 16(1):48-58. doi: 10.2215/CJN.10250620.
19) Casteleijn N, Blais J, Chapman A, Czerwiec F, Devuyst O, Higashihara E, et al. Tolvaptan and kidney pain in patients with autosomal dominant polycystic kidney disease: secondary analysis from a randomized controlled trial. Am J Kidney Dis 2017; 69(2):210-9. doi: 10.1053/j.ajkd.2016.08.028.
20) Chebib F, Torres V. Assessing risk of rapid progression in autosomal dominant polycystic kidney disease and special considerations for disease-modifying therapy. Am J Kidney Dis 2021; 78(2):282-92. doi: 10.1053/j.ajkd.2020.12.020.
21) Uchiyama K, Kitayama C, Yanai A, Ishibashi Y. The effect of trichlormethiazide in autosomal dominant polycystic kidney disease patients receiving tolvaptan: a randomized crossover controlled trial. Sci Rep 2021; 11(1):17666. doi: 10.1038/s41598-021-97113-w.
22) Jamadar A, Suma S, Mathew S, Fields T, Wallace D, Calvet J, et al. The tyrosine-kinase inhibitor Nintedanib ameliorates autosomal-dominant polycystic kidney disease. Cell Death Dis 2021; 12(10):947. doi: 10.1038/s41419-021-04248-9.
23) Blazer-Yost B, Bacallao R, Erickson B, LaPradd M, Edwards M, Sheth N, et al. A randomized phase 1b cross-over study of the safety of low-dose pioglitazone for treatment of autosomal dominant polycystic kidney disease. Clin Kidney J 2021; 14(7):1738-46. doi: 10.1093/ckj/sfaa232.
24) Pastor-Soler N, Li H, Pham J, Rivera D, Ho P, Mancino V, et al. Metformin improves relevant disease parameters in an autosomal dominant polycystic kidney disease mouse model. Am J Physiol Renal Physiol 2022; 322(1):F27-F41. doi: 10.1152/ajprenal.00298.2021.
25) Nguyen Q, Hoang T, Ryu H, Oh K, Kim J. Synergistic antiproliferative effects of all-trans retinoic acid and paclitaxel on autosomal dominant polycystic kidney disease epithelial cells. Biomed Res Int 2021; 2021:1242916. doi: 10.1155/2021/1242916.
26) Carullo N, Zicarelli M, Casarella A, Nicotera R, Castagna A, Urso A, et al. Retarding progression of chronic kidney disease in autosomal dominant polycystic kidney disease with metformin and other therapies: an update of new insights. Int J Gen Med 2021; 14:5993-6000. doi: 10.2147/IJGM.S305491.
27) Casarella A, Nicotera R, Zicarelli M, Urso A, Presta P, Deodato F, et al. Autosomic dominant polycystic kidney disease and metformin: Old knowledge and new insights on retarding progression of chronic kidney disease. Med Res Rev 2022; 42(1):629-40. doi: 10.1002/med.21850.