Poliquistosis renal autosómica dominante: ¿quiénes se benefician del tratamiento con tolvaptán?
Estado del arte
Palabras clave:
RIÑÓN POLIQUÍSTICO AUTOSÓMICO DOMINANTE, ENFERMEDAD RENAL CRÓNICA, TOLVAPTÁN
Resumen
La poliquistosis renal autosómica dominante es la enfermedad renal hereditaria más frecuente. Se caracteriza por la progresiva aparición de quistes renales que suelen conducir a la enfermedad renal crónica extrema en la edad adulta. La aprobación del uso de tolvaptán (antagonista del receptor V2 de la vasopresina) ha marcado un cambio significativo en el tratamiento de esta enfermedad. En los últimos años apareció evidencia que demuestra el beneficio en iniciar tratamiento con tolvaptán en pacientes que presentan una enfermedad con rápida evolución. Se realiza una revisión descriptiva de los principales estudios clínicos publicados en el periodo 2012-2022 y se sugiere un esquema de utilidad para seleccionar aquellos pacientes que pueden beneficierse del inicio de tratamiento.
Citas
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.
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.
Publicado
2022-11-08
Cómo citar
1.
Silvariño R, Peluffo L, Cabrera E, San Román S, Baccino C, Noboa O. Poliquistosis renal autosómica dominante: ¿quiénes se benefician del tratamiento con tolvaptán?. Rev. Méd. Urug. [Internet]. 8 de noviembre de 2022 [citado 20 de abril de 2024];38(3):e38312. Disponible en: http://www2.rmu.org.uy/ojsrmu311/index.php/rmu/article/view/906
Sección
Trabajos de Revisión o Actualización y Puestas al día
