 | What is MPS I? |
| What will happen now? |
| What can you do to help my child? |
| Why don’t my doctors know about this condition? What other doctors will I need to see? |
| What can health care professionals do to help me and my child? |
| What is the chance this will happen again? |
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 GAG. [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. Once therapy stops, levels of GAG may build up again and symptoms may return.
Regular therapy is essential
You could also 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 alpha-L-iduronidase and breaks down that accumulation of GAG. If the therapy is stopped, GAG may once again build up, creating a recurrence of signs and symptoms. [2]
Continuing therapy
It is important to encourage patients to stay on therapy. You may want to emphasize 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.
Expectations of therapy
Patients and their families may have high hopes for quick results with Aldurazyme therapy. Consider offering explanations on how results may vary depending on the types of symptoms experienced by patients. Different organ systems can respond at different rates and can demonstrate a range of improvement, if any at all.
