Clinical evaluation of biomarkers in Gaucher disease.
Acta Paediatr Suppl
; 94(447): 47-50; discussion 37-8, 2005 Mar.
Article
en En
| MEDLINE
| ID: mdl-15895712
ABSTRACT
UNLABELLED Novel or candidate biomarkers require thorough evaluation to establish their utility in a clinical setting. This paper describes an evaluation of several established enzyme markers of Gaucher disease and a newly-described chemokine, pulmonary and activation-regulated chemokine (PARC). The ability of the biomarkers to rank patients with Gaucher disease in order of disease severity and organ bulk, and to reflect changes in key clinical parameters in response to enzyme replacement therapy were evaluated. PARC concentrations were found to be reliably correlated with visceral disease and with key clinical responses to enzyme replacement in an unbiased manner. Unlike chitotriosidase and serum angiotensin-converting enzyme activity, genetic variation in serum PARC did not appear to influence its utility as a biomarker. CONCLUSION:
For each new candidate biomarker of lysosomal storage diseases, a similar clinical evaluation will be required, though the approach will need to be modified according to the clinical features and natural history of each disorder.
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Colección:
01-internacional
Base de datos:
MEDLINE
Asunto principal:
Fosfatasa Ácida
/
Quimiocinas CC
/
Enfermedad de Gaucher
/
Hexosaminidasas
Límite:
Humans
Idioma:
En
Revista:
Acta Paediatr Suppl
Año:
2005
Tipo del documento:
Article
País de afiliación:
Reino Unido