Mark Tarnopolsky, MD, discusses what patient-reported data helps doctors the most in Pompe care decisions.
Transcript
There’s a number of patient-reported outcome symptoms where people can fill out various scales. Problem is, there’s so many of them out there.
We tend not to rely on them, but we very carefully ask patients, you know, “How are you getting around the house? Have you had any falls? What can you manage? What are called your activities of daily living?”
So with every patient we ask them, “Can you feed yourself? Can you do your hair? Can you get in and out of a bathtub? Can you get on and off of a toilet? Do you have any assistive devices? Are you couch surfing or looking for supports?”
If so, we need to talk about things like a cane or a walker. So those sort of really, patient relevant activities of daily living are the things that we ask. Because really, that’s that’s what is impacted in the patients and we need to get their perspective on how things are doing.
Sometimes a 10% drop in their knee extension strength in a quantitative device. They don’t really feel it. But when they, actually tell us that they, you know, can no longer get out of a chair, that’s a patient-reported outcome. That’s very important.
But we have to carefully go through a checklist of activities of daily living. And, you know, we all know what those are. Cooking, cleaning, eating. Mouth care, bathing, toileting. Those are really the main activities of daily living that we always go through that checklist to get the patient’s perspective on how they’re doing.
