Skip to main content

Table 1 Enzyme replacement therapy (ERT) regimens for mucopolysaccharidoses (MPS)

From: Enzyme replacement therapy: efficacy and limitations

 

MPS I

MPS II

MPS IVA

MPS VI

Enzyme deficiency

α-l Iduronidase (IDUA)

Iduronate-2-sulphatase (IDS)

N-acetylgalactosamine-6-sulphatase (GALNS)

N-acetylgalactosamine-4-sulphatase (arylsulphatase B; ARSB)

Glycosaminoglycan accumulation

Dermatan sulphate and heparan sulphate

Dermatan sulphate and heparan sulphate

Keratan sulphate and chondroitin-6-sulphate

Dermatan sulphate

Drug

Laronidase (Aldurazyme®; Genzyme Europe B.V., Gooimeer 10, NL-1411 DD Naarden, The Netherlands), available since 2003

Recombinant human idursulphase (Elaprase®; Shire Human Genetic Therapies, Inc., Cambridge, MA, USA), available since 2006

Elosulphase alpha (Vimizim™; Bio Marin Pharmaceutical, Inc., Novato, CA, USA), available since 2014

Galsulphase (Naglazyme®; Bio Marin Pharmaceutical, Inc., Novato, CA, USA), available since 2005

Dosage

0.58 mg/kg body weight administered once every week as an intravenous infusion. The initial infusion rate of 10 μg/kg/h may be increased every 15 min, if tolerated, to a maximum of 200 μg/kg/h. The total volume of the administration should be delivered in approximately 3–4 h

0.5 mg/kg body weight administered once a week as intravenous infusions over 3 h. The duration of infusion can be shortened gradually to 1 h if there are no infusion-associated reactions (IARs)

2 mg/kg body weight administered once a week. The total volume of the infusion should be delivered over approximately 4 h

1 mg/kg body weight administered once a week as an intravenous infusion over 4 h

Official suggested premedication

With initial administration of Aldurazyme or upon re-administration following interruption of treatment due to previous IARs, pre-treatment with antihistamines and/or antipyretics approximately 60 min prior to the start of the infusion is recommended

Antihistamines and/or corticosteroids can be considered for those patients who have experienced previous IARs during the infusions

Patients should receive antihistamines with or without antipyretics 30 to 60 min prior to start of infusion

Antihistamines with or without antipyretics approximately 30–60 min prior to the start of infusion

Home treatment

Available

Available

Not available

Not available