Mark Tarnopolsky, MD, explains how patient experiences, physical examinations, and quantitative measurements can guide shared treatment decisions in Pompe disease.
Transcript
I think part of that is that treatment decisions are based on what the patient tells us, but also what we can measure.
You know, sometimes patients will underreport or play down some of the issues. So, for example, if I see a patient that has, you know, weaker than a four-minus foot drop, I know they’re probably tripping and stumbling.
And I ask them about that. And, you know, I worry if they might need a brace, for example, for their foot.
So, when I do my examination, it usually leads me to more directed questions about what’s going on. The other issue is I rely on quantitative measurements.
So, in our clinic, we quantitatively measure arm strength, grip strength, knee extension with a very fancy, expensive device that’s very reproducible. And we measure sitting in supine spirometry, as well as the stair climb.
And sometimes we’ll see, you know, that they’re going declining, let’s say, at 2% or 3%. And I’ll see a 7% or 10% decline.
So then that gets me to come back and say, well, “Well, you know, your knee extension is really dropped. Are you still exercising? Did you hurt yourself?” You know, and I use that information to help me from a treatment perspective.
I also can show patients data, for example, if they start a new treatment and their rate of decline is exactly the same. We share that data with them, and they can make a treatment decision or share in that decision.
So we like to use that information to help us understand: Is the patient responding or not responding? And to share that with the patient so that they can share in their treatment decision.
