α-L-iduronidase: clinical benefits in mucopolysaccharidosis I

Inpharma 1272 - 27 Jan 2001 α-L-iduronidase: clinical benefits in mucopolysaccharidosis I α-L-iduronidase * reduces lysosomal storage and ameliorates some disease manifestations in patients with mucopolysaccharidosis I, report researchers from the US. In this study, 10 such patients (aged 5–22 years) received IV α-L-iduronidase 125 000 U/kg once weekly for 52 weeks. ** At 52 weeks, the mean reduction from baseline in liver volume (–25%) was significant; the size of the liver was normal for age and bodyweight in 8 patients by 26 weeks. The mean decrease from baseline in spleen size (–20%) was also significant. Rapid reductions in urinary glycosaminoglycans excretion were seen after 3–4 weeks’ treatment; the mean reduction at 52 weeks (–63%) was significant. Significant mean reductions in the degree of restriction of shoulder flexion and elbow extension were also seen. In the 7 patients who had apnoea at baseline, all had reductions in the number of episodes of apnoea and hypopnoea per night during treatment. In addition, all patients reported improvements in endurance and fewer limitations on the ability to perform the activities of daily life. In the 6 prepubertal patients, significant increases from baseline were seen in the rates of growth in height (from 2.8 to 5.17 cm/year; normal rate 5 cm/year) and bodyweight (from 1.66 to 3.83 kg/year; normal rate 3 kg/year). * BioMarin; phase III ** This study was supported in part by BioMarin Pharmaceutical. Kakkis ED, et al. Enzyme-replacement therapy in mucopolysaccharidosis I. New England Journal of Medicine 344: 182-188, 18 Jan 2001 800852381 1 Inpharma 27 Jan 2001 No. 1272 1173-8324/10/1272-0001/$14.95 Adis © 2010 Springer International Publishing AG. All rights reserved

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Transcript of α-L-iduronidase: clinical benefits in mucopolysaccharidosis I

Inpharma 1272 - 27 Jan 2001

α-L-iduronidase: clinical benefits inmucopolysaccharidosis I

α-L-iduronidase* reduces lysosomal storage andameliorates some disease manifestations in patientswith mucopolysaccharidosis I, report researchers fromthe US.

In this study, 10 such patients (aged 5–22 years)received IV α-L-iduronidase 125 000 U/kg once weeklyfor 52 weeks.**

At 52 weeks, the mean reduction from baseline inliver volume (–25%) was significant; the size of the liverwas normal for age and bodyweight in 8 patients by 26weeks. The mean decrease from baseline in spleen size(–20%) was also significant. Rapid reductions in urinaryglycosaminoglycans excretion were seen after 3–4weeks’ treatment; the mean reduction at 52 weeks(–63%) was significant.

Significant mean reductions in the degree ofrestriction of shoulder flexion and elbow extension werealso seen. In the 7 patients who had apnoea at baseline,all had reductions in the number of episodes of apnoeaand hypopnoea per night during treatment. In addition,all patients reported improvements in endurance andfewer limitations on the ability to perform the activitiesof daily life.

In the 6 prepubertal patients, significant increasesfrom baseline were seen in the rates of growth in height(from 2.8 to 5.17 cm/year; normal rate 5 cm/year) andbodyweight (from 1.66 to 3.83 kg/year; normal rate 3kg/year).* BioMarin; phase III** This study was supported in part by BioMarin Pharmaceutical.

Kakkis ED, et al. Enzyme-replacement therapy in mucopolysaccharidosis I. NewEngland Journal of Medicine 344: 182-188, 18 Jan 2001 800852381