BETA - What are the treatment options for childhood AML, and how is the best treatment course determined?

NGS, or next generation sequencing, is a type of molecular test where you're looking in a very detailed zoomed in manner at the genome or the genetic material that makes up your leukemia to look for mutations. And this has been looked at in research labs to learn more about the genetics of leukemia, but it's also now being examined to look for very, very tiny amounts of leukemia in the body. After we finish treating someone with chemotherapy, we look to see if there's any leukemia left in the body, and that's looking for what's called MRD, or minimal residual disease. Ideally, we don't want to find any. Over time, our tools to look for minimal residual disease have gotten better and better. It used to be a few decades ago that what you would do is just look under the microscope and see, do I see any leukemia cells? Of course, the human eye is only so good at detecting a single leukemia cell, for example. The next phase of detecting minimal residual disease was to do flow cytometry, which is a laboratory test where they look at various markers on the cell surface. So the sensitivity of a computer, a machine, the flow cytometer, to pick up tiny, tiny amounts of leukemia cells is much better than the human eye. So that became the standard for looking for minimal residual disease. The current wave of technology development is going even further than flow cytometry, and that's the next generation sequencing. So using what we know about the genetics of the leukemia, we can look for even smaller and smaller amounts of leukemia left in the body, which may help us determine the trajectory of someone's treatment. Does this person require more chemotherapy? How deep is their remission? That sort of thing. Yes. Most hospitals that are treating oncology patients will do their own flow cytometry testing, although there are centralized laboratories that people also send flow cytometry testing to. So yes, MSK has their own flow cytometry lab, which will look at whatever type of cell you're trying to detect, whether it's a myeloma cell or a leukemia cell, they can detect the various markers on the cell surface using the flow cytometer. When it comes to children with cancer, the good news is there aren't that many children that get cancer. It's not a childhood disease for the most part. And so although that's a good thing, what that means is that there are fewer places and fewer people that take care of children who have cancer, and that goes for all types of pediatric cancer, including AML. So if your child is being diagnosed with AML, then they need to be at a place that treats children who have AML. Generally, you're looking for a pediatric hospital because that's the type of place that will have an oncology department for children. Depending on where you live in the country, that may be very close to you or may be very far from you. In less populous areas, there may be a long distance between you and the next pediatric oncology center. If you live in a place like New York or Boston or a very populous place where there are multiple pediatric hospitals, then it's a slightly different situation. So many parents who have children with AML do travel far distances to receive care for their children because it's not the kind of thing that can be done at your local community hospital generally. One of the major turning points in the treatment of pediatric leukemia over the last 50 years is the development of central nervous system prophylaxis. When doctors first started figuring out how to treat children with leukemia, and again, that would be primarily ALL, not AML, but when they started figuring out how to treat those children, they were able to get children into remission to get treatment. But then very, very commonly, there would be relapses within the brain and the spinal cord. That's called the central nervous system. So what they realized is that we need to treat the central nervous system with chemotherapy up front to prevent a relapse in the brain. The reason for that is that the body is very smart and has protected the central nervous system with what's called the blood-brain barrier. That means that toxins and other bad things can be transmitted to the brain. And so the blood-brain barrier also protects the brain from some of our chemotherapy agents. So we give the chemotherapy agents into the blood. We treat all the leukemia that's in the bones and the blood, but the leukemia cells that are in the brain go untreated. And so for that reason, we provide what's called prophylaxis, which is preventative chemotherapy to the brain for children with leukemia. So that's what we do. In pediatrics, we encourage all children who are able to, who are eligible to be enrolled on a trial to be enrolled on a trial. We realized a long time ago that since pediatric cancer was so rare that the only way to learn more about how to treat these children better was to do as many well-organized trials as possible. So we encourage all children who are able to, who are eligible to be enrolled on a trial to be enrolled on a clinical trial. We encourage that because you will be adding to the general knowledge about how to treat these patients and improve the future chances of future patients. That being said, not everyone is at a place or being treated at a time when they are not able to be treated at a time when they are not able to be treated at a time when they are not able to be treated at a time when they are not able to be treated at a time But not everyone is at a place or being treated at a time where they are eligible for a trial, which is okay. But we do encourage it when possible. If a child is being treated at a hospital where there is a trial available that the child is eligible for, I would encourage parents to enroll their child on that trial because there will only be trials offered that are considered safe and worth the risk. And the idea of any trial that would be offered to a patient upfront would only be attempting to improve upon the standard of care. That being said, the standard of care treatment for pediatric AML is very good. The survival rates are much better than they used to be. And so if a trial is not available, then that child will be offered standard of care therapy and have nearly 100% chance of getting into remission and nearly a 70% chance of staying in remission. Generally, radiation is not used in AML. We do sometimes use radiation in ALL for very specific circumstances, including transplant, but it's not usually used in pediatric AML.