VIDEO: Approaching CML treatment in pediatric patients

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Well, pediatrics, this is a really important question. In the United States, we have the benefit of being able to give medications off-label. Because the labeling of the multiple drugs that are available for adults is not there in kids. We don't have enough choices in pediatrics because only one, and now two drugs have been approved. And the second one that's approved besides imatinib is bosutinib (Bosulif, Pfizer). And the part that really irks me is that it was approved in adults 12 years ago, and it just got approved in the last month or so in pediatrics. And kids have an even greater reason to have choices of which therapy to take and to understand the long-term effects. And we don't do that because we don't have those choices. We're using things off-label, and we need a coordinated way to test these faster to make sure we have what we need for kids.

So there's really interesting work looking at pharmacologic differences in children than adults and in different ethnic backgrounds, pharmacogenomic and pharmacogenetic differences. And we're never going to know that in kids. And it's critically important to get a molecular remission for the length of the time that you take tyrosine kinase inhibitors. And in fact, it allows you to get off them sometimes for a holiday. And we'll never know that in children because it's taking us too long to get the drugs thoroughly tested. That's a problem because it's rare. But also it's a problem because we're not coordinated in how we test these in children. So there's a group in Europe that tests, there's a group here that tests. There's a Chinese group that tests. And it takes 12 years to get the label. And even then, there's still more questions about it.

The big problem is we don't have the info we need. It's even hard in adults to know which agent to use first. In kids, it's even more confusing. And also few of them are approved. So at big hospitals, where you have all these things available, you just use it off-label. But there are places where that, and kids, where that isn't possible because they can't afford it. It won't be covered. And that means we're not doing the best we can for children. And it's shameful because we know that tyrosine kinase inhibitors for adults can let you live out your normal lifespan in, for many, many people, that's extraordinary. And kids have an even longer period that they could benefit and we don't have the info we need to be sure we're giving them the best possible chance of that. And furthermore, we don't know all the long-term effects of tyrosine kinase inhibitors on growing bones and bodies and immune system and kidneys and livers and everything else. And that, we're not going to know that, unless we do this in a coordinated fashion. And it's, while it's wonderful that the study got done, it shouldn't have taken 12 years. It just shouldn't have.