SMA: Patient Adherence to Therapy

John Brandsema, MD: I do think another area that is interesting to consider here is compliance and adherence to therapy. With nusinersen, we are very sure people are getting their maintenance doses because we are controlling that when we give the lumbar puncture. We know if somebody is little late, we can start asking them, “When you are coming in? We need to get you scheduled.” Everyone is nervous about people not taking risdiplam because of the daily need for this steady state. There is also concern about how long you can have no access to this before your motor neurons start to become unwell—whether it is because the medication did not get delivered to you properly, you are unwell and cannot swallow and are vomiting for a period of time, or you just have decided you are too cool for your medication and you are 14. Whatever the reason may be, I think there is no forgiveness here. If someone with epilepsy does not take their medication and they have a seizure, you can say, “You really need to take your medicine.” Here, if you lose motor neurons, we cannot get those back once you are not taking your medication. I have been trying to think a little about other areas where we have a lot of experience with this, like in the cases of transplant and other things. How do you ensure that you have a good therapeutic alliance with your patient, that you know for sure they are getting access, and that they are appropriately maintaining their therapy?

Julie Parsons, MD: Going back to one of your other points, John, with the other drugs, which I am really interested in, we have talked about the intrathecal medication to target the motor neurons without a lot of systemic involvement. With risdiplam, you are choosing to give a medication that you know is systemic. Is that an advantage or disadvantage, or do we even know at this point?

John Brandsema, MD: That is such an interesting thing to discuss; we could spend the whole rest of the day on this because in other tissues besides motor neurons, the manifestations of this disease are not very obvious, unless patients have a very severe early onset. We know they can have cardiac disease, and dysautonomia occurs in patients with early onset. There are some liver and immune system things that have been established in the literature, but we do not tend to see things in our monitoring of these more stable, especially higher functioning, patients that are really important to recognize in terms of other systemic involvement.

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Transcript Edited for Clarity