Melissa Wright, MD, PhD, explains how functional testing, symptoms, and individual trends can help guide decisions about changing SMA treatment.
How do you know when a patient needs a treatment adjustment?
Transcript
This is an active area of research, as we don’t have a lot of clear data to guide decisions about changing treatment approaches with individual patients.
From a practical standpoint, we do standardized functional tests in the clinic with our physical therapists, and we track those over time. Depending on the age and strength of the patient, we’ll often use tests like the CHOP INTEND, the HFMSE and the six-minute walk test.
The expected trajectory for untreated patients with SMA is progressive decline in scores of all of these tests over time. The expected trajectory for patients with SMA who are treated is stability in these tests over time, or even improvement, especially in younger kids. They’ll often see some improvements in their scores over time.
For reasons we don’t fully understand, some individuals respond better to one treatment than another.
Because of this, most neuromuscular doctors will consider either switching to a different treatment or adding an additional treatment if a patient is showing signs of declining function, because we don’t expect that in treated patients, or if they’re not showing the progression and improvement that we would expect over time.
However, we really don’t have great data to drive these decisions.
And so, it really comes down to monitoring a patient’s individual trends of our objective measures with our physical therapy testing, listening closely to what a patient is telling me about their symptoms and what they’re noticing over time, and then talking with the patient about risks and benefits of trying a different treatment approach and making the decision with them.