For many with Pompe Disease, Enzyme Replacement Therapy is a part of their routine. For some, it can be weekly, and for others, it can be bi-weekly. This is a life changing drug that replaces some of a deficient enzyme to slow Pompe disease progression. It’s an intravenous infusion that it weight based, and infuses over many hours. Planning for this infusion is complex with several considerations—a big one being where to receive the infusion: home or hospital?
Parents of children with Pompe who receive these infusions shared the pros and cons to consider with both. Let’s start with hospital/clinic infusions:
The pros to consider include leaving the logistics to the facility. They order the drug, store it, prepare it, and infuse it, allowing the patient/family to keep this part off their plate. A second high point is keeping the medical and home environments separate so as to protect the sanctity of home. Others mention enjoying the time in a relaxed environment that forces them to slow down. Many families report forming incredible bonds with the staff and other patients at infusion centers, adding a social aspect to the medical setting.
The cons have their important points as well. Families report being up to 4 hours away from an appropriate infusion facility, lengthening an already tedious infusion timeframe. This can cause infusion days to last from sun up to sun down, which can be especially hard on children with Pompe. Many children can feel bored at the infusion centers because they are confined to one room or area, and switching activities can run repetitive quickly. If not boredom, some children have medical anxiety, making this setting more worrisome than comforting. My own daughter required TWO child life specialists for a year while visiting hospitals and it was exhausting for us both—so this consideration may be dependent on the child
Now let’s consider a few point points for home infusions. Medications are ordered, and shipped to the home where a trained person or hired infusion nurse prepares and administers the infusion within the home environment. There were a lot of pros reported for this, such as feeling the comfort of home, still being productive within the home, and avoidance of long drives to infusion clinics. Children can place their IV pump in a specialized back pack they wear, allowing them to run and play as usual in the home. Parents report that this can keep children from missing too much school as well, as they can continue schoolwork from home in some instances, and for others, the children can attend school while infusing!
The home setting still has its cons. Families report that the logistics of the medication can be an absolute nightmare if any parts of procedure go awry or become delayed. This can cause delays in treatment, lost time, and expensive medical waste. The cost of mishandling these drugs even once is tens of thousands of dollars lost. And while on the subject of cost, a lot of times insurances refuses to cover in-home infusions—taking this option off the table. Another recurrent discussion point was staffing issues and the quality/competency of nurses. While some families grow close to their infusion nurses and even feel like they are family, other nurses cause a lot more stress for patients and families due to incompetency around the specifics of the drug and infusion protocols.
Not all of these affect each patient and family with the same weight, so the decision for infusions really vary in this community. What may be a 10 minute drive for one family, could be a 4 hour drive for another. Some parents want to keep their role as “parent” and not “nurse” too. Others report that their kids feel more at ease that a parent is also their infuser. It’s just another thing about Pompe that isn’t “one size fits all.”
If you’re on the fence about which infusion scenario would best fit your family, find resources that can help bring some discussion points to the table for you to consider. Many can be found through Facebook support and awareness groups with seasoned ERT parents such as Ryker’s Foundation for Pompe Disease, Pompe Warrior Foundation, Grant’s Giants Pompe Awareness Non-Profit, The Eve of Better Days, Evie’s journey with Pompe Disease, Winnie the Warrior, Windsor’s Pompe Journey and several more.
Perspectives of The Pompe World 
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