EBMT 2026 | Patient selection for gene therapy in SCD: insights into real-world decision-making

Before now, there was a study criterion for patients. So the study criterion is that you had to have had vaso-occlusive events, about four of them in a 24-month period. And they were very specific. You can’t have had strokes. You couldn’t have had more. They were excluded from the trial. Now, the problem is that that has translated, but that’s not real life. We have patients that have a pain crisis and we know that the older these patients get the worse they become. So you have a 12-year-old who’s qualified to go into a study who has had one pain crisis in a one-year period and you’re asking me to deny them that when I know that by the time they turn 16, they may have had multiple organ affectations versus what the study is saying. And so there is no clear guideline. And I think in my personal opinion, I think this has to be personalized individual by individual. But like I said, right now, I think some, and I think we’ll talk about that later on, but the most important thing is that we have the criteria, but I don’t necessarily think that that works for everybody that is coming in for gene therapy. 

And I think it’s a very difficult one because the resources are limited. If we have unlimited resources, I think it’s easy for me to say it should be individualized based on shared decision making. But at the same time, we know that that resource is not available for everybody. And so I think there should be a pairing of the two. There should be a mirroring of the two. I’ll give you a perfect example. I have a 15-year-old that has never had a pain crisis before in his life. And he comes to me and says, I want to get gene therapy. And at the same time, I have this 19-year-old that has had multiple pain crises. And I know my resources are limited. I think I’ll be offering it to the 19-year-old compared to that 15-year-old. But that doesn’t necessarily mean that I’m saying no to the 15-year-old, I’m just saying maybe not now. But what makes that right and what makes that wrong is a little bit tough. 

So the point I’m trying to make is that I do not believe in the strict timeline, the strict eligibility criteria. But at the same time, as much as I believe it should be a shared decision-making, I have to have at the back of my mind what are the resources available for such institutions. If they have the resources to offer it to everybody, go for it, but if they don’t necessarily have that, I think there should be some thought behind every decision that has been made and who is being offered gene therapy.

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