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