Dr Sarah Tasian Highlights Challenges With Improving Outcomes in Pediatric AML

One of the challenges with treating children with acute myeloid leukemia is that many of the novel drugs are not available in children. Current treatment with chemotherapy really requires balancing increasing doses with the short-term and long-term toxicities, said Sarah Tasian, MD, attending physician in the Division of Oncology at Children’s Hospital of Philadelphia.

Transcript

What challenges have there been that have resulted in little improvement in outcomes for children with AML?

I think one of the biggest problems is that there really have not been new drugs for children with AML really in the past 40 years. In the last year, 2 years, there been a lot of new drugs that have been very exciting in terms of small molecule inhibitors or new immunotherapies, but they've largely been restricted to the adult population. And, you know, virtually none of these drugs are actually available to children. So, I think that's been one of the major challenges.

I think the second other major challenges, we have intensified chemotherapy to its maximum limit in our patients, and it has improved outcomes in the past several decades, but we really are to maximal intensity right now, where it's just simply isn't possible to gain any more benefit from you know, higher doses of these drugs. And these drugs are associated with really many short-term and long-term toxicities, both in terms of infection, low blood counts, and a lot of long-term toxicity in terms of children's heart function.

When treating children who have decades left in their lives, how do you balance between treating the cancer but managing long-term toxicities?

I think that's one of the things that's really challenging, as we currently cure about two-thirds of our children now, but some of it comes at very high costs. We currently transplant about a third of our patients in first remission. And that's obviously associated with short growth, sometimes endocrine dysfunction, infertility, which when you have 75 years ahead of you is a major deal.

We also have problems, I think, in particular with cardiotoxicity. We know that giving high doses of anthracycline to our young children is essential for curing them. But when it comes at the expense of you know, reduced cardiac output or even needing a heart transplant later, that seems like too expensive a cost for these therapies.