Increasing a dose of medicine can be nerve-wracking

Changes in my son's enzyme replacement therapy spark my worries

Keara Engle avatar

by Keara Engle |

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Note: This column describes the author’s son’s experiences with Nexviazyme and Lumizyme. Not everyone will have the same response to treatment. Consult your doctor before starting or stopping a therapy or changing its dose.

My 6-year-old son, Cayden, has received infusions of enzyme replacement therapy for the entirety of his life. He’s had them since newborn screening led to his diagnosis with infantile-onset Pompe disease. They’re the only current treatment for Pompe.

Even though we’re familiar with the therapy, getting an infusion can still be nerve-wracking. The dose of Nexviazyme (avalglucosidase alfa) Cayden infuses is based on his weight; any time his weight increases, the Nexviazyme dose increases as well.

Fortunately, Cayden usually has no issue with that. He did have a reaction to an enzyme replacement infusion when he was around age 1, but he was on Lumizyme (alglucosidase alfa) at the time. Infusions of that medicine would make him break out in red hives all over.

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This problem continued for a while, and Cayden eventually had to see an allergist to get to the root of the problem. The allergist recommended that we switch him to weekly infusions. This schedule eventually became the standard recommendation for children with infantile-onset Pompe disease who are receiving Lumizyme.

Cayden eventually switched to Nexviazyme in June of 2022. We wanted to get back to a biweekly infusion schedule, and we hoped he’d see better results with this medication.

Cayden hasn’t had a bad reaction to enzyme replacement infusions since his experience with Lumizyme. But I can’t help but worry a bit anytime the infusions change, whether it be the dose, the rate, or the medicines given beforehand.

The most recent change

His dose was increased last week, and his home infusion nurse and I watched him like a hawk. On top of the increased dose, we also got permission to stop one of those prior-to-infusion medicines. Two changes at once? That was definitely worrisome. We do have an EpiPen and anaphylaxis kit at home in case he were to have a negative reaction, but we never want to use them.

Thankfully, Cayden’s infusion last week went as usual. I won’t be as stressed out before the next one because I know he tolerated both changes just fine.

The stress will never subside completely, though. I’ll always be a bit worried about infusions because I remember those negative reactions years ago and fear they could happen again. I guess that’s my motherly instincts, and that’s OK.

Note: Pompe Disease News is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Pompe Disease News or its parent company, BioNews, and are intended to spark discussion about issues pertaining to Pompe disease.


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