Insights Into Pediatric Oncology on Childhood Cancer Day: Advances, Challenges, and Survivorship

Burton Eliot Appel, MD

International Childhood Cancer Day serves as a worldwide initiative to raise awareness of the hardships encountered by patients with childhood cancer, with another focus on emphasizing the need for equal access to comprehensive treatment and care for all affected individuals.

“We’re always thinking ahead to survivorship, which is a very big topic in pediatric oncology,” Burton Eliot Appel, MD, stated. “We want to increase the number of cancer survivors, but we want those survivors to have a good quality of life, or as good as we can make it. The balance between curing the greatest number of children while decreasing late effects of the therapy is the key to pediatric oncology.”

In an interview with OncLive^®, Appel highlighted the most common types of childhood cancers and the difficulties that oncologists may face when treating these patients as well as the importance of factoring in emotional and psychological support for patients and their families.

Appel is a pediatric hematologist/oncologist at the Hackensack University Medical Center and Jersey Shore University Medical Center, in Hackensack and Neptune, New Jersey, respectively.

OncLive: What are some of the most common types of childhood cancers you encounter in your practice? How do their treatment approaches differ?

Appel: We see a few different categories of cancer in children and young adults. The biggest category is leukemia and lymphoma, which we think of as blood or hematologic cancers, and their treatment is primarily chemotherapy.

Sometimes patients benefit from radiation, but we try to limit or avoid the use of radiation, particularly in our youngest children. Another big category of pediatric cancer is brain tumors or tumors of the central nervous system. There the treatment approaches are very different; surgery is very important if it’s safe and feasible to do so. Radiation is also important but carries with it a lot of potential adverse effects for the youngest patients. Additionally, chemotherapy does have a role, but not as much as surgery and radiation.

Everything else is things like bone tumors, kidney tumors, and tumors of the muscle and soft tissue. There, any number of combinations of surgery, radiation, and chemotherapy can be used.

What are some key challenges in diagnosing and treating patients with childhood cancers, and how have advancements helped address these challenges?

Our overall goal in pediatric oncology is to cure the greatest number of patients. You might think, ‘Well, that’s obvious; wouldn’t any oncologist say that?’ However, it’s different from if you treat a 75 or 80-year-old, and you are thinking about giving the patient 5 more years of life with good quality. That’s not what we’re aiming for with a child or young adult, as we want to cure as many patients as possible. We also want to do that in a way that minimizes long-term effects of their therapy.

In terms of other challenges, sometimes we find delays in diagnosis and that can certainly happen to adults too. However, many adults are more tuned in to the possibility of cancer and will seek medical attention or benefit from screening. Many adults will have colon cancer screenings or breast cancer screenings. However, in kids, there is no such thing because we never expect a child to develop cancer. Therefore, sometimes we’ll see delays in diagnosis. Even if a parent brings their child to the primary care doctor, emergency room, or urgent care they don’t think that cancer is even a possibility. It may take a while for a child to be diagnosed so that’s another challenge.

How do you approach the possibility of relapse in pediatric patients?

Another challenge we think about is relapse. What we tend to do in our goal of trying to cure as many children as possible is to treat very aggressively or intensively at the first diagnosis. What that means is a child who relapses may have already been exposed to the most effective therapies for that disease. If a child relapses after that, [we ask], what do we do with them, and that’s a real challenge.

There are also things like quality of life, and we take that very seriously during the child’s therapy, and once they’re [cured] hopefully. For some of the diseases that we manage, particularly leukemia, the duration of therapy is quite long at 2 and a half years. If you can imagine a 3-year-old diagnosed with leukemia, they are going to be treated for 2 and a half years; that’s almost their entire lifetime.

We want the child to develop to achieve the normal milestones that their peers and siblings would. Trying to do that, while still maximally treating their cancer is a challenge. Overall, maintaining quality of life and keeping track of development is a challenge.

Please expand on the importance of recognizing the fertility issues that can arise for patients who have suffered from childhood cancer.

Then there are the challenges of supportive care. Again, we are very much focused on the future and survivorship. One of the issues that are very important to survivors is fertility preservation, and that’s something that we didn’t talk about when I first started my training. We just wanted to cure as many kids as possible, and if they lived, got married, and wanted to have children, then they could deal with it.

Now, when we expect that so many of our patients will be long-term survivors, we’re thinking ahead 10 to 20 years when they may want to have children of their own. What can we do, if anything, at the very beginning, before any chemotherapy or radiation is given to try to preserve their fertility for the future?

A lot of research has been done and a lot of effort has been put into this. I established a program for fertility preservation for our patients about 5 years ago, and we can offer options for most of our patients, which is excellent. The problem is that insurance companies in general don’t pay for it. We’re talking about meeting with a new family, saying their child has cancer, this is what the therapy is going to be like, and that there is also the possibility that their 5-year-old may not be able to have children in the future. It’s terrible to tell a family that, and then we’ll say, we do have some options to try to preserve your child’s fertility. However, you may have to pay $2,000 out of pocket because your insurance company won’t pay for it. That's a very sad thing.

We try very hard to find resources to help families and we’re successful much of the time, but I would say that the biggest barrier for some people in terms of fertility preservation is the cost.

That’s something we want to increase awareness of, increase philanthropic efforts for, and advocate on a legislative level to try to get some of these insurance laws changed. This is so that insurance companies are mandated to pay for it. However, you can imagine the pace of that kind of change is very slow.

Could you share insights into recent advancements in pediatric oncology treatment options or research that give hope for improved outcomes?

Both adults and kids have benefited in recent years from advancements in immunotherapy.

For decades, we were stuck with surgery, radiation, and chemotherapy as the only way to treat and cure patients with cancer. Chemotherapy medicines have a lot of potential AEs in the short term while the patient is going through it as well as in the long term.

There’s always been research interest in trying to use our own immune system, which normally should be patrolling the body trying to fight off infections. However, the immune system can be harnessed to kill cancer cells if you figure out the right way to do it. [We want] to get the immune system in contact with the cancer cell so that the patient’s immune system can recognize that the cancer cell doesn’t belong and destroy it.

There have been amazing advances in this field of immunotherapy in the past decade. There are other procedures, almost like a bone marrow transplant, but not quite. Instead, it [involves] using the patient’s own cells in [a treatment known as] CAR T-cell therapy.

[CAR T-cell therapy] was pioneered in the past decade and that’s an amazing thing where you basically engineer the patient’s own cells to fight the cancer cells. The great thing about that is it’s a one-time thing. When you reinfuse the patient’s own re-engineered cells, they keep multiplying. The expectation is that those engineered cells will keep destroying any new cancer cells that appear. It’s an ongoing process.

[People in] the field of pediatric and adult oncology are trying to figure out the best way to use that technology, the CAR T technology, in terms of diseases and targets. However, we’re only at the beginning of that field and it’s very exciting, especially in the context of new medicines that are considered immunotherapies.

For many years, we were sort of rearranging the same old chemotherapy drugs, trying to figure out how to recombine them, and how to change the doses. However, these immunotherapies have given us a whole new world of medicines, and we’re trying to figure out if we add them to our regular chemotherapy medicines to improve cure rates or take some of these immunotherapies and substitute them for regular chemotherapy drugs that have so many AEs. That’s being done more and more.

There are some cancers where patients are not getting these old-fashioned chemotherapy drugs that are so toxic; they are being treated with immunotherapies alone.

Parents often ask me what the long-term AEs of these immunotherapies are in children. We don’t have answers for that. This is a new category of drugs and that’s always a challenge that we don’t know what the long-term AEs are of anything until we cure enough patients and study them for decades.

We certainly know that [these agents are] well tolerated in kids. We’re hoping that they don’t have the same type of long-term effects on the heart and other tissues [our historical interventions do].

How do you support families and children emotionally and psychologically through their cancer journey?

The field of pediatric oncology has recognized for years that you need to provide robust psychosocial support for the patient and their entire family. Again, that’s not unique to our program. We’re very fortunate that we have a strong psychosocial support team here at Hackensack; we have a group of social workers who are dedicated to pediatric hematology oncology patients.

Moreover, we have child life therapists who are specifically trained to work with the kids at their developmental level. We also have psychologists and an educational liaison whose role is to help families navigate the school system, whether the child is in kindergarten, or they’re in high school, and to help parents advocate for the resources their children need to keep up. What we don’t want is for children to be left back [because of their disease].

We expect they’re going to keep up. Sometimes kids even do better because they’re getting one-on-one teaching for some amount of time. But our educational liaison is there to advocate for families. So, what I think we do in pediatric oncology is recognize these needs and try to make the experience for the entire family as tolerable as possible.

This is so disruptive to the life of the child and the entire family, as very often one parent has to stop working temporarily to be the primary caretaker. How does that affect the family financially? How does it affect them emotionally? What is going on with the siblings?

These are all things that we think about. There’s no way to provide the type of care that we do medically without providing this kind of support.