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

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

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Título The prognostic value of the serum ferritin in a southern Brazilian cohort of patients with Gaucher disease
Autor Koppe, Tiago de Bone
Doneda, Divair
Siebert, Marina
Paskulin, Lívia D'Avila
Camargo, Matheus Brunstein
Tirelli, Kristiane Michelin
Vairo, Filippo Pinto e
Daudt, Liane Esteves
Schwartz, Ida Vanessa Doederlein
Abstract 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.
Contido em Genetics and molecular biology. Ribeirão Preto, SP. Vol. 39, no. 1 (Jan./Mar. 2016), p. 30-34
Assunto Biomarcadores
Doença de Gaucher
Ferritinas
[en] Biomarkers
[en] Ferritin
[en] Gaucher disease
[en] Iron metabolism
Origem Nacional
Tipo Artigo de periódico
URI http://hdl.handle.net/10183/147202
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