The prognostic value of the serum ferritin in a southern brazilian cohort of patients with Gaucher disease

Detalhes bibliográficos
Autor(a) principal: Koppe, Tiago de Bone
Data de Publicação: 2016
Outros Autores: Moneda, Divair, Siebert, Marina, Paskulin, Lívia D'Avila, Camargo, Matheus Brunstein, Tirelli, Kristiane Michelin, Vairo, Filippo Pinto e, Daudt, Liane Esteves, Schwartz, Ida Vanessa Doederlein
Tipo de documento: Artigo
Idioma: eng
Título da fonte: Repositório Institucional da UFRGS
Texto Completo: http://hdl.handle.net/10183/150137
Resumo: The clinical utility of serum ferritin as a biomarker of disease severity and prognosis in Gaucher disease (GD) is still debated. Here, we aimed to evaluate ferritin and its relation to clinicolaboratory parameters of GD patients seen at the Reference Center for Gaucher Disease of Rio Grande do Sul, Brazil, so as to gather evidence on the utility of ferritin as a biomarker of this condition. A retrospective chart review was performed collecting pre- and posttreatment data from GD patients. Eighteen patients with ferritin levels available before and after treatment were included in the study. Nine of these participants were males, and seventeen had type I GD. All patients were given either enzyme replacement (n = 16) or substrate reduction therapy (n = 2), and ferritin was found to decrease from 756 [318-1441] ng/mL at baseline to 521 [227-626] ng/mL (p = 0.025) after 28.8 months of treatment. Serum ferritin levels did not correlate with measures of disease severity, but showed an association with age at onset of treatment ( = 0.880; n = 18; p < 0.001). In conclusion, although serum ferritin did not correlate with disease severity, after a median 28.8 months of treatment, clinical outcomes had clearly improved, and ferritin levels had decreased.
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spelling Koppe, Tiago de BoneMoneda, DivairSiebert, MarinaPaskulin, Lívia D'AvilaCamargo, Matheus BrunsteinTirelli, Kristiane MichelinVairo, Filippo Pinto eDaudt, Liane EstevesSchwartz, Ida Vanessa Doederlein2016-12-16T02:17:10Z20161415-4757http://hdl.handle.net/10183/150137001005101The clinical utility of serum ferritin as a biomarker of disease severity and prognosis in Gaucher disease (GD) is still debated. Here, we aimed to evaluate ferritin and its relation to clinicolaboratory parameters of GD patients seen at the Reference Center for Gaucher Disease of Rio Grande do Sul, Brazil, so as to gather evidence on the utility of ferritin as a biomarker of this condition. A retrospective chart review was performed collecting pre- and posttreatment data from GD patients. Eighteen patients with ferritin levels available before and after treatment were included in the study. Nine of these participants were males, and seventeen had type I GD. All patients were given either enzyme replacement (n = 16) or substrate reduction therapy (n = 2), and ferritin was found to decrease from 756 [318-1441] ng/mL at baseline to 521 [227-626] ng/mL (p = 0.025) after 28.8 months of treatment. Serum ferritin levels did not correlate with measures of disease severity, but showed an association with age at onset of treatment ( = 0.880; n = 18; p < 0.001). In conclusion, although serum ferritin did not correlate with disease severity, after a median 28.8 months of treatment, clinical outcomes had clearly improved, and ferritin levels had decreased.application/pdfengGenetics and molecular biology. Ribeirão Preto, SP. Vol. 39, no. 1, (2016), p. 30-34FerritinaBiomarcadoresDoença de GaucherMetabolismo : FerroFerritinBiomarkersGaucher diseaseIron metabolismThe prognostic value of the serum ferritin in a southern brazilian cohort of patients with Gaucher diseaseinfo:eu-repo/semantics/articleinfo:eu-repo/semantics/otherinfo:eu-repo/semantics/publishedVersioninfo:eu-repo/semantics/openAccessreponame:Repositório Institucional da UFRGSinstname:Universidade Federal do Rio Grande do Sul (UFRGS)instacron:UFRGSORIGINAL001005101.pdf001005101.pdfTexto completo (inglês)application/pdf462153http://www.lume.ufrgs.br/bitstream/10183/150137/1/001005101.pdfdb0619d85cfdd575b2a74a25592ddef3MD51TEXT001005101.pdf.txt001005101.pdf.txtExtracted Texttext/plain25782http://www.lume.ufrgs.br/bitstream/10183/150137/2/001005101.pdf.txt764435741be623b0938ef4f5eb092113MD52THUMBNAIL001005101.pdf.jpg001005101.pdf.jpgGenerated Thumbnailimage/jpeg1892http://www.lume.ufrgs.br/bitstream/10183/150137/3/001005101.pdf.jpg1bf6751d308e13815166ab8d18ebc950MD5310183/1501372021-08-28 04:40:44.327205oai:www.lume.ufrgs.br:10183/150137Repositório de PublicaçõesPUBhttps://lume.ufrgs.br/oai/requestopendoar:2021-08-28T07:40:44Repositório Institucional da UFRGS - Universidade Federal do Rio Grande do Sul (UFRGS)false
dc.title.pt_BR.fl_str_mv The prognostic value of the serum ferritin in a southern brazilian cohort of patients with Gaucher disease
title The prognostic value of the serum ferritin in a southern brazilian cohort of patients with Gaucher disease
spellingShingle The prognostic value of the serum ferritin in a southern brazilian cohort of patients with Gaucher disease
Koppe, Tiago de Bone
Ferritina
Biomarcadores
Doença de Gaucher
Metabolismo : Ferro
Ferritin
Biomarkers
Gaucher disease
Iron metabolism
title_short The prognostic value of the serum ferritin in a southern brazilian cohort of patients with Gaucher disease
title_full The prognostic value of the serum ferritin in a southern brazilian cohort of patients with Gaucher disease
title_fullStr The prognostic value of the serum ferritin in a southern brazilian cohort of patients with Gaucher disease
title_full_unstemmed The prognostic value of the serum ferritin in a southern brazilian cohort of patients with Gaucher disease
title_sort The prognostic value of the serum ferritin in a southern brazilian cohort of patients with Gaucher disease
author Koppe, Tiago de Bone
author_facet Koppe, Tiago de Bone
Moneda, Divair
Siebert, Marina
Paskulin, Lívia D'Avila
Camargo, Matheus Brunstein
Tirelli, Kristiane Michelin
Vairo, Filippo Pinto e
Daudt, Liane Esteves
Schwartz, Ida Vanessa Doederlein
author_role author
author2 Moneda, Divair
Siebert, Marina
Paskulin, Lívia D'Avila
Camargo, Matheus Brunstein
Tirelli, Kristiane Michelin
Vairo, Filippo Pinto e
Daudt, Liane Esteves
Schwartz, Ida Vanessa Doederlein
author2_role author
author
author
author
author
author
author
author
dc.contributor.author.fl_str_mv Koppe, Tiago de Bone
Moneda, Divair
Siebert, Marina
Paskulin, Lívia D'Avila
Camargo, Matheus Brunstein
Tirelli, Kristiane Michelin
Vairo, Filippo Pinto e
Daudt, Liane Esteves
Schwartz, Ida Vanessa Doederlein
dc.subject.por.fl_str_mv Ferritina
Biomarcadores
Doença de Gaucher
Metabolismo : Ferro
topic Ferritina
Biomarcadores
Doença de Gaucher
Metabolismo : Ferro
Ferritin
Biomarkers
Gaucher disease
Iron metabolism
dc.subject.eng.fl_str_mv Ferritin
Biomarkers
Gaucher disease
Iron metabolism
description The clinical utility of serum ferritin as a biomarker of disease severity and prognosis in Gaucher disease (GD) is still debated. Here, we aimed to evaluate ferritin and its relation to clinicolaboratory parameters of GD patients seen at the Reference Center for Gaucher Disease of Rio Grande do Sul, Brazil, so as to gather evidence on the utility of ferritin as a biomarker of this condition. A retrospective chart review was performed collecting pre- and posttreatment data from GD patients. Eighteen patients with ferritin levels available before and after treatment were included in the study. Nine of these participants were males, and seventeen had type I GD. All patients were given either enzyme replacement (n = 16) or substrate reduction therapy (n = 2), and ferritin was found to decrease from 756 [318-1441] ng/mL at baseline to 521 [227-626] ng/mL (p = 0.025) after 28.8 months of treatment. Serum ferritin levels did not correlate with measures of disease severity, but showed an association with age at onset of treatment ( = 0.880; n = 18; p < 0.001). In conclusion, although serum ferritin did not correlate with disease severity, after a median 28.8 months of treatment, clinical outcomes had clearly improved, and ferritin levels had decreased.
publishDate 2016
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dc.relation.ispartof.pt_BR.fl_str_mv Genetics and molecular biology. Ribeirão Preto, SP. Vol. 39, no. 1, (2016), p. 30-34
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