Venglustat, Acetato de Lanreotida y Tolvaptan como opciones terapéuticas para el tratamiento de la enfermedad renal poliquística autosómica dominante: revisión narrativa
DOI:
https://doi.org/10.18041/2665-427X/ijeph.2.7246Palabras clave:
enfermedad poliquística renal autosómica dominante, ADPKD, enfermedad poliquística renal, Tolvaptan, antagonistas receptores V2, Venglustat, inhibidor de la glucosilceramida sintasa, acetato de lanreotida, análogo de somatostatinaResumen
Introducción: la enfermedad poliquística renal autosómica (ADPKD), es considerada huérfana hereditaria. Con una prevalencia mundial de 2.7/100,000 hab. Presenta graves complicaciones con desenlaces fatales. Hasta hace poco, el tratamiento se limitó al control de las manifestaciones clínicas; con la aparición del tolvaptan, se empezó a manejar la enfermedad. La introducción terapéutica del inhibidor de glucosilceramida sintetasa (venglustat) y el acetato de lanreotida hacen necesario revisar el estado de estas opciones terapéuticas para estos pacientes.
Métodos: Se realizó una revisión narrativa de la literatura en PubMed, Cochrane Library, OVID, EBSCO y Google Scholar.
Resultados: Se obtuvieron 92 artículos, de los cuales 5 fueron incluidos en el análisis cualitativo. El Ensayo CLINICO TEMPO 3:4, demostró que el tolvaptan redujo la tasa de crecimiento anual en el volumen total de riñón (TKV) del 5.5% al 2.8% y la tasa anual de filtración glomerular estimada (eGFR) disminuyó de −3.70 a −2.72 mL/min/1.73-m2. El estudio THE DIPAK-1 evidenció que el acetato de lanreotida reduce el volumen renal total (4.2% frente al control: 5.6%; diferencia: -1.4% por año; sin diferencias significativas para la incidencia de empeoramiento de la función renal. Los ensayos clínicos con venglustat evidenciaron mejoría preliminar en la TKV y la eGFR, acorde a los resultados de los estudios preclínicos, sin embargo, aún se encuentran en estudio.
Conclusiones: La ADPKD, es una patología con pocas opciones terapéuticas y con recientes tratamientos incorporados, actualmente en investigación. Este artículo establece las bases para un futuro diseño de estudio cuantitativo.
Descargas
Referencias
Solazzo A, Testa F, Giovanella S, Busutti M, Furci L, Carrera P, et al. The prevalence of autosomal dominant polycystic kidney disease (ADPKD): A meta-analysis of European literature and prevalence evaluation in the Italian province of Modena suggest that ADPKD is a rare and underdiagnosed condition. Remuzzi G, editor. PLoS One. 2018; 13(1): e0190430. Doi: 10.1371/journal.pone.0190430.
Iliuta I-A, Kalatharan V, Wang K, Cornec-Le Gall E, Conklin J, Pourafkari M, et al. Polycystic kidney disease without an apparent family history. J Am Soc Nephrol. 2017; 28(9): 2768–76. doi: 10.1681/ASN.2016090938.
Zhou Z, Xu Y, Delcourt C, Shan J, Li Q, Xu J, et al. Is regular screening for intracranial aneurysm necessary in patients with autosomal dominant polycystic kidney disease? a systematic review and meta-analysis. Cerebrovasc Dis. 2017; 44(1–2): 75–82. doi: 10.1159/000476073.
Lee H, Manns B, Taub K, Ghali WA, Dean S, Johnson D, et al. Cost analysis of ongoing care of patients with end-stage renal disease: The impact of dialysis modality and dialysis access. Am J Kidney Dis. 2002; 40(3): 611–22. doi: 10.1053/ajkd.2002.34924.
Brunelli SM, Blanchette CM, Claxton AJ, Roy D, Rossetti S, Gutierrez B. End-stage renal disease in autosomal dominant polycystic kidney disease: A comparison of dialysis-related utilization and costs with other chronic kidney diseases. Clin Outcomes Res. 2015; 7: 65–72. doi: 10.2147/CEOR.S76269.
Ong ACM, Devuyst O, Knebelmann B, Walz G. Autosomal dominant polycystic kidney disease: the changing face of clinical management. Lancet. 2015; 385(9981): 1993–2002. Doi: 10.1016/S0140-6736(15)60907-2
McEwan P, Bennett Wilton H, Ong ACM, Ørskov B, Sandford R, Scolari F, et al. A model to predict disease progression in patients with autosomal dominant polycystic kidney disease (ADPKD): The ADPKD outcomes model. BMC Nephrol. 2018;19(1): 1–12. doi: 10.1186/s12882-017-0804-2.
Committee for Orphan Medicinal Products. Public summary of opinion on orphan designation Lanreotide acetate for the treatment of autosomal dominant polycystic kidney disease. EMA/COMP/432098/2015, European Medicines Agency; 2015. Disponible en: https://www.ema.europa.eu/en/documents/orphan-designation/eu/3/15/1514-public-summary-opinion-orphan-designation-lanreotide-acetate-treatment-autosomal-dominant_en.pdf.
European Medicines Agency. Public summary of opinion on orphan designation Venglustat for the treatment of autosomal dominant polycystic kidney disease. EMA/840293/2018, European Medicines Agency; 2019. Disponible en: https://www.ema.europa.eu/en/documents/orphan-review/eu/3/18/2122-public-summary-opinion-orphan-designation-venglustat-treatment-autosomal-dominant-polycystic_en.pdf.
Peterschmitt MJ, Crawford NPS, Gaemers SJM, Ji AJ, Sharma J, Pham TT. Pharmacokinetics, pharmacodynamics, safety, and tolerability of oral venglustat in healthy volunteers. Clin Pharmacol Drug Dev. 2021;10(1): 86–98. doi: 10.1002/cpdd.865.
Meijer E, Visser FW, Van Aerts RMM, Blijdorp CJ, Casteleijn NF, D’Agnolo HMA, et al. Effect of lanreotide on kidney function in patients with autosomal dominant polycystic kidney disease the DIPAK 1 randomized clinical trial. JAMA. 2018; 320(19): 2010–9. doi: 10.1001/jama.2018.15870.
Chebib FT, Perrone RD, Chapman AB, Dahl NK, Harris PC, 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
Bae KT, Czerwiec FS, Gansevoort RT, Ouyang JJ, Torres VE, Chapman AB, 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(5): 692–9. Available from: doi: 10.1053/j.ajkd.2010.11.029
Casteleijn NF, Blais JD, Chapman AB, Czerwiec FS, 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. Available from: doi: 10.1053/j.ajkd.2016.08.028.
Wyatt CM, Le Meur Y. REPRISE: tolvaptan in advanced polycystic kidney disease. Kidney Int. 2018; 93(2): 292–5. Available from: doi: 10.1016/j.kint.2017.12.002
Messchendorp AL, Spithoven EM, Casteleijn NF, Dam WA, Van Den Born J, Tonnis WF, et al. Association of plasma somatostatin with disease severity and progression in patients with autosomal dominant polycystic kidney disease. BMC Nephrology. 2018; 19: 368. Doi: 10.1186/s12882-018-1176-y.
Genzyme. Multicenter, randomized, double-blind, placebo-controlled two stage study to characterize the efficacy, safety, tolerability and pharmacokinetics of GZ/SAR402671 in patients at risk of rapidly progressive autosomal dominant polycystic kidney disease (ADPKD). ClinicalTrials.gov. ClinicalTrials.gov Identifier: NCT03523728; 2018. Available from: https://clinicaltrials.gov/ct2/show/NCT03523728.
Natoli TA, Smith LA, Rogers KA, Wang B, Komarnitsky S, Budman Y, et al. Inhibition of glucosylceramide accumulation results in effective blockade of polycystic kidney disease in mouse models. Nat Med. 2010; 16(7): 788–92. doi: 10.1038/nm.2171.
Myint TM, Rangan GK, Webster AC. Treatments to slow progression of autosomal dominant polycystic kidney disease: Systematic review and meta-analysis of randomized trials. Nephrology. 2014; 19(4): 217–26. doi: 10.1111/nep.12211.
Publicado
Versiones
- 2023-07-18 (3)
- 2021-04-12 (2)
- 2020-12-30 (1)
Número
Sección
Licencia
Derechos de autor 2020 Interdisciplinary Journal of Epidemiology and Public Health
Esta obra está bajo una licencia internacional Creative Commons Atribución-NoComercial-SinDerivadas 4.0.
-
Attribution — You must give appropriate credit, provide a link to the license, and indicate if changes were made. You may do so in any reasonable manner, but not in any way that suggests the licensor endorses you or your use.
-
NonCommercial — You may not use the material for commercial purposes.
-
NoDerivatives — If you remix, transform, or build upon the material, you may not distribute the modified material.
- No additional restrictions — You may not apply legal terms or technological measures that legally restrict others from doing anything the license permits.
