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Effectiveness and therapeutic aims of enzyme replacement therapy in mucopolysaccharidosis type II (Hunter syndrome)

M.C. García-Jiménez, M. Lafuente-Hidalgo, R. Pérez-Delgado, J. López-Pisón, J.L. Peña-Segura, A. Baldellou-Vázquez [REV NEUROL 2008;47 (Supl. 2):S15-S18] PMID: 19021116 DOI: https://doi.org/10.33588/rn.47S02.2008539

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Volumen 47 | Number S02 | Nº of views of the article 5.224 | Nº of PDF downloads 555 | Article publication date 10/11/2008

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ABSTRACT

INTRODUCTION Mucopolysaccharidosis type II (MPS II), or Hunter syndrome, is a lysosomal disease that is transmitted by recessive inheritance linked to the X chromosome and is caused by an iduronate-2-sulphatase enzyme deficit. The treatment of lysosomal diseases in general and MPS in particular has undergone spectacular changes over the last 25 years. At present, the development of enzyme replacement therapy (ERT) is being used as a new therapeutic option in the treatment of lysosomal diseases. DEVELOPMENT. The development of ERT has offered these patients the first therapeutic benefits. In 2006, the US Food and Drug Administration authorised the use of idursulfase, and in Europe it was approved by the European Medicines Agency in January 2007. The following question arises with respect to treatment with idursulfase today: do we treat Hunter syndrome or not? Despite being a promising alternative, at the present time ERT has a number of limitations that make it necessary to pose this question. Throughout this review we will analyse both the beneficial effects and the current drawbacks of ERT.

CONCLUSIONS To be able to answer this question properly, the following theoretical objectives have to be accomplished: appraisal of the therapeutic effectiveness and clinical relevance of ERT in MPS II, development of suitable biomarkers for monitoring the treatment and setting suitable therapeutic aims that help us to establish the indications of ERT. KeywordsEnzyme replacement therapy Hunter syndrome Idursulfase Mucopolysaccharidosis type II

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Effectiveness and therapeutic aims of enzyme replacement therapy in mucopolysaccharidosis type II (Hunter syndrome)