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USO DE SIROLIMUS EN PACIENTES PEDIATRICOS CON POLIQUISTOSIS RENAL AUTOSOMICA DOMINANTE
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USO DE SIROLIMUS EN PACIENTES PEDIATRICOS CON POLIQUISTOSIS RENAL AUTOSOMICA DOMINANTE

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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
20 de Noviembre, 2015

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

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Extensión:  +/-6.65 páginas impresas en papel A4
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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



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