Importance of compliance

Your patients might be concerned that their condition will prevent them from continuing in all of their normal, day-to-day activities. It can be useful to reassure your patients (and their families) that their feelings are not unusual.

It may be difficult to explain the role of Aldurazyme^® (laronidase) to patients. When discussing Aldurazyme^® with your patients, you may want to cover the following discussion points:

MPS I is a lifelong disease [1] Regular replacement of the alpha-L-iduronidase enzyme with Aldurazyme® helps prevent the buildup of glycosaminoglycans (GAG’s).[2]

You might explain to patients that, in order to continue to benefit from the treatment, patients may need to receive intravenous infusions for the rest of their lives, even though they may feel better after a number of infusions. Once therapy stops, levels of GAG may build up again and in theory, symptoms may return.

It could also be useful to explain in patient terms that people with MPS I are deficient in the enzyme alpha-L-iduronidase, which is responsible for breaking down the accumulation of GAG[1]. When administered to patients, Aldurazyme® enzyme replacement therapy acts like ‘natural’ alpha-L-iduronidase and breaks down that accumulation of GAG[2]. If the therapy is stopped, GAG may once again build up, leading to disease progression and, based on the mechanism of action of Aldurazyme®, potential recurring of signs and symptoms.

It is important to encourage patients to stay on therapy. You may want to emphasise that MPS I is a lifelong disorder, and that Aldurazyme® is not a cure. It is important to understand your patient’s expectations, and help them maintain therapeutic compliance in order to continue to benefit.

Patients and their families may have high hopes for quick results with Aldurazyme® therapy. Consider offering explanations on how results may vary from patient to patient and depend largely on the severity and type of symptoms at the beginning of treatment.

References:

1. Neufeld EF, Muenzer J. The mucopolysaccharidoses. In: Scriver CR, Beaudet AL, Sly WS, Valle D, Childs B, Kinzler KW, et al, editors. The Metabolic and Molecular Bases of Inherited Disease. Vol. III. 8th ed. New York: McGraw-Hill; 2001. p. 3421-8.

2. Aldurazyme® [Summary of Product Characteristics]. Naarden, The Netherlands: Genzyme Europe B.V.; 2003.