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Treatment of Genetic Forms of Nephrotic Syndrome

Idiopathic steroid-resistant nephrotic syndrome (SRNS) is most frequently characterized by focal segmental glomerulosclerosis (FSGS) but also other histological lesions, such as diffuse mesangial sclerosis. In the past two decades, a multitude of genetic causes of SRNS have been discovered raising t...

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Autores principales: Kemper, Markus J., Lemke, Anja
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2018
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5879576/
https://www.ncbi.nlm.nih.gov/pubmed/29632851
http://dx.doi.org/10.3389/fped.2018.00072
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author Kemper, Markus J.
Lemke, Anja
author_facet Kemper, Markus J.
Lemke, Anja
author_sort Kemper, Markus J.
collection PubMed
description Idiopathic steroid-resistant nephrotic syndrome (SRNS) is most frequently characterized by focal segmental glomerulosclerosis (FSGS) but also other histological lesions, such as diffuse mesangial sclerosis. In the past two decades, a multitude of genetic causes of SRNS have been discovered raising the question of effective treatment in this cohort. Although no controlled studies are available, this review will discuss treatment options including pharmacologic interventions aiming at the attenuation of proteinuria in genetic causes of SRNS, such as inhibitors of the renin–angiotensin–aldosterone system and indomethacin. Also, the potential impact of other interventions to improve podocyte stability will be addressed. In this respect, the treatment with cyclosporine A (CsA) is of interest, since a podocyte stabilizing effect has been demonstrated in various experimental models. Although clinical response to CsA in children with genetic forms of SRNS is inferior to sporadic SRNS, some recent studies show that partial and even complete response can be achieved even in individual patients inherited forms of nephrotic syndrome. Ideally, improved pharmacologic and molecular approaches to induce partial or even complete remission will be available in the future, thus slowing or even preventing the progression toward end-stage renal disease.
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spelling pubmed-58795762018-04-09 Treatment of Genetic Forms of Nephrotic Syndrome Kemper, Markus J. Lemke, Anja Front Pediatr Pediatrics Idiopathic steroid-resistant nephrotic syndrome (SRNS) is most frequently characterized by focal segmental glomerulosclerosis (FSGS) but also other histological lesions, such as diffuse mesangial sclerosis. In the past two decades, a multitude of genetic causes of SRNS have been discovered raising the question of effective treatment in this cohort. Although no controlled studies are available, this review will discuss treatment options including pharmacologic interventions aiming at the attenuation of proteinuria in genetic causes of SRNS, such as inhibitors of the renin–angiotensin–aldosterone system and indomethacin. Also, the potential impact of other interventions to improve podocyte stability will be addressed. In this respect, the treatment with cyclosporine A (CsA) is of interest, since a podocyte stabilizing effect has been demonstrated in various experimental models. Although clinical response to CsA in children with genetic forms of SRNS is inferior to sporadic SRNS, some recent studies show that partial and even complete response can be achieved even in individual patients inherited forms of nephrotic syndrome. Ideally, improved pharmacologic and molecular approaches to induce partial or even complete remission will be available in the future, thus slowing or even preventing the progression toward end-stage renal disease. Frontiers Media S.A. 2018-03-26 /pmc/articles/PMC5879576/ /pubmed/29632851 http://dx.doi.org/10.3389/fped.2018.00072 Text en Copyright © 2018 Kemper and Lemke. https://creativecommons.org/licenses/by/4.0/ This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
spellingShingle Pediatrics
Kemper, Markus J.
Lemke, Anja
Treatment of Genetic Forms of Nephrotic Syndrome
title Treatment of Genetic Forms of Nephrotic Syndrome
title_full Treatment of Genetic Forms of Nephrotic Syndrome
title_fullStr Treatment of Genetic Forms of Nephrotic Syndrome
title_full_unstemmed Treatment of Genetic Forms of Nephrotic Syndrome
title_short Treatment of Genetic Forms of Nephrotic Syndrome
title_sort treatment of genetic forms of nephrotic syndrome
topic Pediatrics
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5879576/
https://www.ncbi.nlm.nih.gov/pubmed/29632851
http://dx.doi.org/10.3389/fped.2018.00072
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