Naslov
Enzyme levels in leukocytes of two patients on rh-alpha-L-iduronidase therapy

Autori
Barišić, Ingeborg ; Huzjak , Nevenka ; Tokić, Višnja ; Fumić, Ksenija ; Mrsić, Mirando ;

Vrsta, podvrsta i kategorija rada
Sažeci sa skupova, sažetak, znanstveni

Izvornik
Abstracts. 8th International Symposium on Mucopolysaccharide and Related Diseases. New therapeutic strategies - what can we hopefully expect? / - Mainz, 2004

Skup
8th International Symposium on Mucopolysaccharide and Related Diseases. New therapeutic strategies - what can we hopefully expect?

Mjesto i datum
Mainz, Njemačka, 10.06.2004. - 13.06.2004

Vrsta sudjelovanja
Poster

Vrsta recenzije
Nije recenziran

Ključne riječi
enzyme replacement therapy; MPS I; enzyme levels; rh-alpha-L-iduronidase

Sažetak
The Hurler syndrome is a lysosomal storage disorder with an extremely severe clinical course due to the complete or almost complete deficiency of the active alpha-L-iduronidase enzyme that participates in the stepwise degradation of dermatan and heparan sulphate. The rh alpha-L-iduronidase is administrated weekly in order to compensate the deficiency of enzymes included in the degradation of glycosaminoglicans. There is still a need for further understanding of the enzyme pharmacokinetics in humans. The data are presented on one-year alpha-L-iduronidase level surveillance in two patients with muccopolysaccharidosis type I (Mb. Hurler). While the enzyme level in the first patient was sufficient during the whole monitored period, allowing prolongation of the period between doses to be considered, the other patient showed no adequate biochemical response to the therapy. Regular surveillance of the alpha-L-iduronidase level in blood is necessary to allow early recognition of possible poor response to the usual dosage, to identify the possible adverse factors influencing the enzyme level and to make timely therapeutic intervention possible.

Izvorni jezik
Engleski

Znanstvena područja
Javno zdravstvo i zdravstvena zaštita

POVEZANOST RADA