Artículos relacionadosArtículos relacionadosArtículos relacionados
Artículos afines de siicsalud publicados en los últimos 4 meses
EVALUACIÓN Y EL TRATAMIENTO DE LA ENFERMEDAD RENAL CRÓNICA
Kidney International 105(4S):205-245
Difundido en siicsalud: 23 oct 2024
TOLERABILIDAD A LARGO PLAZO DE SIROLIMUS EN EL TRASPLANTE DE PULMÓN
Transplantation Proceedings 55(10):2470-2477
Difundido en siicsalud: 5 nov 2024

USO DE SIROLIMUS EN PACIENTES PEDIATRICOS CON POLIQUISTOSIS RENAL AUTOSOMICA DOMINANTE

(especial para SIIC © Derechos reservados)
La poliquistosis renal autosómica dominante se caracteriza por la aparición de quistes renales. Durante 24 meses se asignaron al azar 12 pacientes a un grupo de tratamiento con sirolimus.        
Autor:
José Miguel Liern
Columnista Experto de SIIC

Institución:
Hospital General De Niños "dr. Ricardo Gutiérrez", Buenos Aires, Argentina


Artículos publicados por José Miguel Liern
Coautores
Federico Mora* Graciela Vallejo* Paola Codianni** 
Médico, Hospital General De Niños "dr. Ricardo Gutiérrez", Buenos Aires, Argentina, Buenos Aires, Argentina*
Médica, Hospital General De Niños "dr. Ricardo Gutiérrez", Buenos Aires, Argentina, Buenos Aires, Argentina**
Aprobación
15 de Septiembre, 2014
Primera edición
27 de Octubre, 2014
Segunda edición, ampliada y corregida
7 de Junio, 2021

Resumen
Introducción: La poliquistosis renal autosómica dominante (PQRAD) se caracteriza por la aparición de quistes renales. Objetivos: Evaluar el crecimiento del volumen renal y de los quistes en los pacientes tratados con sirolimus, en comparación con aquellos que reciben el tratamiento habitual para la PQRAD; evaluar la aparición de efectos adversos relacionados con el uso de sirolimus; evaluar los cambios en la presión arterial, la proteinuria y el filtrado glomerular (FG) estimado. Materiales y métodos: Durante 24 meses se asignaron al azar 12 pacientes con PQRAD a un grupo de tratamiento con sirolimus (6 pacientes recibieron sirolimus 2 a 3 mg/m2/día, con un máximo de 5 mg/día) o a un grupo control (bajo tratamiento habitual). Resultados: De los 12 pacientes, los 6 pertenecientes al grupo de tratamiento con sirolimus tuvieron, al final del estudio, un aumento del volumen renal total y del volumen quístico del 13% y el 32%, respectivamente. En el grupo control, los 6 pacientes restantes tuvieron aumentos del 11% y el 23%, respectivamente. El FG fue normal para ambos grupos. La proteinuria para los grupos de sirolimus y control fue inicialmente 7.3 mg/m²/h y 6 mg/m2/h, respectivamente. Al finalizar el estudio era normal para ambos grupos. Tres pacientes tenían hipertensión arterial, pero a los 24 meses la presión arterial fue normal. Los efectos adversos observados fueron: anemia, diarrea y úlceras bucales. Conclusiones: El sirolimus no disminuyó el volumen renal ni el quístico. No hubo un aumento significativo en la proteinuria o una disminución en el FG. La media de la presión arterial se mantuvo normal.

Palabras clave
sirolimus, enfermedad renal poliquistica, enfermedad renal cronica, volumen renal, resonancia magnetica nuclear


Artículo completo

(castellano)
Extensión:  +/-6.65 páginas impresas en papel A4
Exclusivo para suscriptores/assinantes

Abstract
Background: Autosomal Dominant Polycystic Kidney Disease (ADPKD) is characterized by the development of kidney cysts. Objectives: To assess the growth of kidney and cystic volume in patients treated with sirolimus compared with patients receiving the usual treatment for ADPKD; assess the occurrence of adverse effects associated with the use of sirolimus; evaluate changes in blood pressure, proteinuria and estimated glomerular filtration rate (eGFR). Materials and methods: For a 24-month period, 12 patients with ADPKD were randomly allocated to a sirolimus group (6 patients, received sirolimus, 2-3 mg/m2/day, max. 5 mg/day) and the other 6 patients were allocated to a control group (receiving conventional treatment). Results: Out of the 12 patients, the 6 patients in the sirolimus group had a total increase in kidney and cystic volume of 13% and 32%, respectively, by the end of the study. In the control group, the other 6 patients had increases of 11% and 23%, respectively. eGFR was normal in both groups. Baseline proteinuria was 7.3 mg/m²/hour and 6 mg/m2/hour in the sirolimus and control groups, respectively. By the end of the study, it was normal in both groups. Three patients had high blood pressure at baseline, but it was normalized at 24 months. Adverse effects were: anemia, diarrhea, and mouth sores. Conclusions: Sirolimus did not reduce kidney and cystic volume. No significant increase in proteinuria or decrease in eGFR were observed. Mean blood pressure remained normal.

Key words
sirolimus, autosomal dominant polycystic disease, chronic renal failure, renal volume, nuclear magnetic resonance


Clasificación en siicsalud
Artículos originales > Expertos de Iberoamérica >
página   www.siicsalud.com/des/expertocompleto.php/

Especialidades
Principal: Nefrología y Medio Interno, Pediatría
Relacionadas: Diagnóstico por Imágenes, Medicina Nuclear



Comprar este artículo
Extensión: 6.65 páginas impresas en papel A4

file05.gif (1491 bytes) Artículos seleccionados para su compra



Enviar correspondencia a:
José Miguel Liern, 1435, Gallo 1330, Ciudad de Buenos Aire, Argentina
Bibliografía del artículo
1. Harris PC, Torres VE, et al. Polycystic kidney disease. Annu Rev Med 60:321-337, 2009.
2. Boyer O, Niaudet P, et al. Prognosis of autosomal dominant polycystic kidney disease diagnosed in utero or at birth. Pediatr Nephrol 22:380-388, 2007.
3. Avner E, Sweeney W. Diagnosis and management of childhood polycystic kidney disease Pediatr Nephrol 26:675-692, 2011.
4. García-González MA, Menezes LF, Piontek KB, et al. Genetic interaction studies link autosomal dominant and recessive polycystic kidney disease in a common pathway. Hum Mol Genet 16:1940-1950, 2007.
5. Kim I, Li C, Liang D, Chen XZ, et al. Polycystin-2 expression is regulated by a PC2-binding domain in the intracellular portion of fibrocystin. J Biol Chem 283:31559-31566, 2008.
6. Yoder BK. Role of primary cilia in the pathogenesis of polycystic kidney disease. J Am Soc Nephrol 18:1381-1388, 2007.
7. Waters AM, Beales PL. Ciliopathies: an expanding disease spectrum. Pediatr Nephrol 26:1039-1056, 2011.
8. De Almeida EA, de Oliveira EI, et al. Ambulatory blood pressure measurement in young normotensive patients with autosomal dominant polycystic kidney disease. Rev Port Cardiol 26:235-243, 2007.
9. Ecder T, Chapman AB, Brosnahan GM, et al. Effect of antihypertensive therapy on renal function and urinary albumin excretion in hypertensive patients with autosomal dominant polycystic kidney disease. Am J Kidney Dis 35:427-432, 2000.
10. Bergmann C, Zerres K. Early manifestations of polycystic kidney disease. Lancet 369:2157, 2007.
11. Zafar I, Ravichandran K, Belibi FA, et al. Sirolimus attenuates disease progression in an orthologous mouse model of human autosomal dominant polycystic kidney disease. Kidney Int 78(8):754-761, 2010.
12. Wahl PR, Serra AL, Le Hir M, et al. Inhibition of mTOR with sirolimus slows disease progression in Han:SPRD rats with autosomal dominant polycystic kidney disease (ADPKD). Nephrol Dial Transplant 21(3):598-604, 2006.
13. Tao Y, Kim J, Schrier RW, Edelstein CL. Rapamycin markedly slows disease progression in a rat model of polycystic kidney disease. J Am Soc Nephrol 16(1):46-51, 2005.
14. Ravine D, Gibson RN, Walker RG, et al. Evaluation of ultrasonographic diagnostic criteria for autosomal dominant polycystic kidney disease. Lancet 343: 824-827, 1994.
15. Schwartz GJ, Munoz A, Schneider MF, et al. New equations to estimate GFR in children with CKD. J Am Soc Nephrol 20:629-637, 2009.
16. Dell KM. The spectrum of polycystic kidney disease in children.
Adv Chronic Kidney Dis 18(5):339-347, 2011.
17. Chapman AB. Autosomal dominant polycystic kidney disease: time for a change? J Am Soc Nephrol 18(5):1399-1407, 2007.
18. Kim I, Fu Y, Hui K, Moeckel G, Mai W, et al. Fibrocystin/polyductin modulates renal tubular formation by regulating polycystin-2 expression and function. J Am Soc Nephrol 19:455-468, 2008.
19. Fliegauf M, Benzing T, Omran H. When cilia go bad: cilia defects and ciliopathies. Nat Rev Mol Cell Biol 8:880-893, 2007.
20. Grantham JJ, Cook LT, Wetzel LH, et al. Evidence of extraordinary growth in the progressive enlargement of renal cysts. Clin J Am Soc Nephrol 5:889-896, 2010.
21. Pei Y. Practical genetics for autosomal dominant polycystic kidney disease. Nephron Clin Pract 118(1):c19-30, 2011.
22. Liebau M, Serra A. Looking at the (w) hole: magnet resonance imaging in polycystic kidney disease. Pediatr Nephrol 28:1771-1783, 2013.
23. Chapman AB, Wei W. Imaging approaches to patients with polycystic kidney disease. Semin Nephrol 31:237-244, 2011.
24. Avni FE, Garel L, Cassart M, et al. Perinatal assessment of hereditary cystic renal diseases: the contribution of sonography. Pediatr Radiol 36:405-414, 2006.
25. Avni FE, Guissard G, Hall M, Janssen F, DeMaertelaer V, Rypens F. Hereditary polycystic kidney diseases in children: changing sonographic patterns through childhood. Pediatr Radiol 32:169-174, 2002.
26. Chakraborty S, McHugh K. Cystic diseases of the kidney in children. Imaging 17:69-75, 2005.
27. Cadnapaphornchai MA, Masoumi A, Strain JD, et al. Magnetic resonance imaging of kidney and cyst volume in children with ADPKD. Clin J Am Soc Nephrol 6:369-376, 2010.
28. Torres VE, King BF, Chapman AB, Brummer ME, et al. Magnetic resonance measurements of renal blood flow and disease progression in autosomal dominant polycystic kidney disease. Clin J Am Soc Nephrol 2:112-120, 2007.
29. Arlene b. Chapman, Lisa m. Guay-Woodford. Renal structure in early autosomal-dominant polycystic kidney disease (ADPKD): The Consortium for Radiologic Imaging Studies of Polycystic Kidney Disease (CRISP) cohort. Kidney International 64:1035-1045, 2003.
30. Serra A, Kistler A, Poster D, et al. Clinical proof-of-concept trial to assess the therapeutic effect of sirolimus in patients with autosomal dominant polycystic kidney disease: SUISSE ADPKD study. BMC Nephrology 8:13, 2007.
31. Walz G, Budde K, Mannaa M, Nurnberger J,Wanner C, et al. Everolimus in patients with autosomal dominant polycystic kidney disease. N Engl J Med 363:830-840, 2010.
32. Liern M, Diéguez S, De Reyes V. Antiproteinuric effect of use additive of enalapril and losartan in normotensive pediatric patients with proteinuria. Rev Esp Nefrol 24(6):553-558, 2004.
33. De Almeida EA, de Oliveira EI, Lopes JA, et al. Ambulatory blood pressure measurement in young normotensive patients with autosomal dominant polycystic kidney disease. Rev Port Cardiol 26:235-243, 2007.
34. Ozkok A, Akpinar TS, Tufan F, Kanitez NA, et al. Clinical characteristics and predictors of progression of chronic kidney disease in autosomal dominant polycystic kidney disease: a single center experience. Clin Exp Nephrol 17(3):345-351, 2013.
35. Novalic Z, van der Wal AM, Leonhard WN, Koehl G, Breuning MH, et al. Dose-dependent effects of sirolimus on mTOR signaling and polycystic kidney disease. J Am Soc Nephrol 23:842-853, 2012.
36. Canaud G, Knebelmann B, Harris PC, Vrtovsnik F, et al. Therapeutic mTOR inhibition in autosomal dominant polycystic kidney disease: what is the appropriate serum level? Am J Transplant 10:1701-1706, 2010.
37. Shillingford JM, Piontek KB, Germino GG, Weimbs T. Rapamycin ameliorates PKD resulting from conditional inactivation of Pkd1. J Am Soc Nephrol 21:489-497, 2010.
38. Zafar I, Belibi FA, He Z, et al. Long-term rapamycin therapy in the Han:SPRD rat model of polycystic kidney disease (PKD) Nephrol Dial Transplant 24:2349-2353, 2009.
39. Thaunat O, Beaumont C, Chatenoud L, Lechaton S, et al. Anemia after Late Introduction of Sirolimus May Correlate with Biochemical Evidence of a Chronic Inflammatory State. Transplantation 80(9):1212-1219, 2005.
40. Van Biesen W, Vanholder R, Veys N, Verbeke F, et al. Efficacy of erythropoietin administration in the treatment of anemia immediately after renal transplantation. Transplantation 79(3):367-368, 2005.
41. Oppenheimer F, Alonso A, Arias M, Campistol JM, et al. Manejo de sirolimus en la práctica clínica. Nefrologia 26(Suppl 2):64-93, 2006.
42. Perico N, Remuzzi A, Remuzzi G. Sirolimus therapy to halt the progression of ADPKD.(Sirena study) clinical Research Center for Rare Diseases, J Am Soc Nephrol 21(6):1031-1040, 2010.
43. Fick-Brosnahan GM, Belz MM, McFann KK, Johnson AM, Schrier RW. Relationship between renal volume growth and renal function in autosomal dominant polycystic kidney disease: a longitudinal study. Am J Kidney Dis 39:1127-1134, 2002.
44. Grantham JJ, Torres VE, Chapman AB, Guay-Woodford LM, et al. Volume progression in polycystic kidney disease. N Engl J Med 354(20):2122-2130, 2006.
45. Van Gelder T, Ter Meulen CG, Hene R, et al. Oral ulcers in kidney transplant recipients treated with sirolimus and mycophenolate mofetil. Transplantation 75(6):788-791, 2003.

 
 
 
 
 
 
 
 
 
 
 
 
Está expresamente prohibida la redistribución y la redifusión de todo o parte de los contenidos de la Sociedad Iberoamericana de Información Científica (SIIC) S.A. sin previo y expreso consentimiento de SIIC.
ua31618
Inicio/Home

Copyright siicsalud © 1997-2024 ISSN siicsalud: 1667-9008