Kidney & blood pressure research · 2025
Fabry Disease Nephropathy: Compendium of "in vitro" and "in vivo" Renal Effects of Globotriaosylsphingosine.
СтатьяОбзорНазвание и аннотация: Перевод в очереди
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Jaurretche Sebastián Pedro Antonio, Del Rosal María Victoria, Brajkovic María Luana, Isaac Juan Ignacio, Venera Graciela, Pérez Germán
Аннотация
UNLABELLED: <p>Background: Renal damage in Fabry disease (FD) is a consequence of pathological and progressive glycosphingolipids accumulation, which occurs in different magnitudes among FD phenotypes, but is a constant renal tissue phenomenon in all patients with renal involvement. A significant correlation between plasma Lyso-Gb3 (Gb3 analog) and disease severity has been described but, there is lack of information from clinical studies regarding the correlation between Lyso-Gb3 and renal events in humans and, the few results have certain discrepancies. In clinical practice, it is necessary to have disease-specific biomarkers, non-invasive and useful in diagnosis, prognosis and therapeutic response. Evidence relating to the prognostic value of lyso-GL3 is mixed.
SUMMARY: We reviewed the evidence on renal effect of plasma and/or urinary Lyso-Gb3 in FD patients and the usefulness as a biomarker of diagnostic and therapeutic response. Additionally, the in vitro renal effects of Lyso-Gb3 were reviewed. The literature search was conducted in PubMed, Embase, Scopus, Cochrane, and Google academic. Search terms were (Fabry disease + [Lyso-Gb3 or Lyso-GL3] + ["renal" or "kidney"]).
INCLUSION CRITERIA: (i) "in vitro" studies in which a direct effect of Lyso-Gb3 on kidney tissue has been studied, both in animals and/or humans; (ii) retrospective, cross-sectional, or prospective "in vivo" studies in which a correlation between Lyso-Gb3 and renal clinical events or renal function biomarkers has been studied.
KEY MESSAGES: Ten studies presented evidence of "in vitro" renal damage due to Lyso-Gb3; 4 studies presented correlation between plasma Lyso-Gb3 and renal events in FD patients; and 3 studies reported correlation between urinary Lyso-Gb3 and renal events in FD patients.
CONCLUSION: The small number of publications and the methodological heterogeneity do not allow a statistical analysis of them. Regarding the "in vivo" and "in vitro" renal effects of Lyso-Gb3 in FD: There is evidence of harmful effects on renal cells in vitro due to Lyso-Gb3 but this evidence is not sufficient to use Lyso-Gb3 (neither plasma nor urinary) as a biomarker for monitoring renal damage in FD patients. Plasma Lyso-Gb3 is a useful biomarker in (i) FD diagnosis and (ii) stratification of phenotype and severity of FD. In FD "classic" patients receiving enzyme replacement therapy (ERT), it is useful to determine plasma Lyso-Gb3 periodically because an inconsistent reduction during ERT may indicate the formation of neutralizing anti-agalsidase antibodies. </p>.
SUMMARY: We reviewed the evidence on renal effect of plasma and/or urinary Lyso-Gb3 in FD patients and the usefulness as a biomarker of diagnostic and therapeutic response. Additionally, the in vitro renal effects of Lyso-Gb3 were reviewed. The literature search was conducted in PubMed, Embase, Scopus, Cochrane, and Google academic. Search terms were (Fabry disease + [Lyso-Gb3 or Lyso-GL3] + ["renal" or "kidney"]).
INCLUSION CRITERIA: (i) "in vitro" studies in which a direct effect of Lyso-Gb3 on kidney tissue has been studied, both in animals and/or humans; (ii) retrospective, cross-sectional, or prospective "in vivo" studies in which a correlation between Lyso-Gb3 and renal clinical events or renal function biomarkers has been studied.
KEY MESSAGES: Ten studies presented evidence of "in vitro" renal damage due to Lyso-Gb3; 4 studies presented correlation between plasma Lyso-Gb3 and renal events in FD patients; and 3 studies reported correlation between urinary Lyso-Gb3 and renal events in FD patients.
CONCLUSION: The small number of publications and the methodological heterogeneity do not allow a statistical analysis of them. Regarding the "in vivo" and "in vitro" renal effects of Lyso-Gb3 in FD: There is evidence of harmful effects on renal cells in vitro due to Lyso-Gb3 but this evidence is not sufficient to use Lyso-Gb3 (neither plasma nor urinary) as a biomarker for monitoring renal damage in FD patients. Plasma Lyso-Gb3 is a useful biomarker in (i) FD diagnosis and (ii) stratification of phenotype and severity of FD. In FD "classic" patients receiving enzyme replacement therapy (ERT), it is useful to determine plasma Lyso-Gb3 periodically because an inconsistent reduction during ERT may indicate the formation of neutralizing anti-agalsidase antibodies. </p>.