NCCN Guidelines Give Framework for Childhood Neuroblastoma Treatment

Newly crafted guidelines could help guide the treatment for patients with pediatric neuroblastoma and offer patients and their families a framework for important discussions to have with clinicians, according to an expert.

The National Comprehensive Cancer Network (NCCN), comprised of a group of experts from leading cancer centers, recently published its Clinical Practice Guidelines in Oncology for Neuroblastoma.

Avoiding Long-Term Side Effects

Notably, the guidelines provide information on risk stratification, which is crucial in the treatment of the childhood disease, as according to the American Cancer Society, patients with low- or intermediate-risk disease have average survival rates of 90% and above, while those with high-risk disease have a survival rate of about 50%.

“There are children with neuroblastoma who don't need any therapy at all, they just get monitored, and they're fine. And then there are children with neuroblastoma who have the most aggressive kind of disease that need basically every kind of cancer therapy that we can think of, and the outcomes are still poor,” Dr. Rochelle Bagatell said in an interview with CURE®. “So, it's very important to understand how to approach neuroblastoma, how to assign a risk group for a patient and then how to use the treatment that's appropriate for patients in that risk group.”

Bagatell is a professor of Pediatrics and Solid Tumor Section Chief at Children's Hospital of Philadelphia, and Chair of the NCCN Clinical Practice Guidelines in Oncology (NCCN Guidelines®) Panel for Neuroblastoma.

She explained that these guidelines can be beneficial to patients, families and clinicians in determining risk and treatment plan, especially when it comes to minimizing unneeded aggressive treatments that may lead to long-term side effects in patients with low-risk disease.

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“If you have a little baby with neuroblastoma diagnosed on day of life three, and you follow the guidelines, and you don't overdo it with that child, and that patient grows up and is a productive citizen and doesn't have a lot of long-term effects from what you did, that's a huge amount of life gained,” Bagatell said.

For patients with high-risk disease, Bagatell said, “the therapy has really changed over the last few decades, and it's become much more complex and makes use of use of many more tools in our toolbox.”

A Framework for Insurance Coverage, Patient-Provider Discussions

The NCCN Guidelines for neuroblastoma could also provide reasoning for coverage when insurers do not cover certain treatments, according to Bagatell.

“What these guidelines may potentially do — since they're designed by thought leaders and really built by consensus — is they can serve as backup for us when we go to the insurance provider and say, ‘This patient needs this scan or this therapy.’ And this is a consensus in the field that may be very helpful in advocating for our patients,” she explained.

Bagatell also warned that “medicine is not done by cookbook, or in this case, algorithm,” and that each patient is nuanced, and their care should be planned and personalized as such. The neuroblastoma guidelines are not intended to be hard-and-fast rules that cannot be deviated from.

“There may be specific cases where the nuances of a particular patient's case means that you have to adjust your thinking from what's written on those nice, clear lines,” Bagatell said. “But the general guidance about how to think about the risk of recurrence, what general type of therapy would be appropriate, how much chemotherapy when to do surgery. Those are the kinds of things that patients and families can look at and bring to their doctor and discuss.”

The Future of Neuroblastoma Treatment

Looking forward, Bagatell said that she’d like to see continued improvements in the approach of non-high-risk patients, so that, “we can really identify the population with certainty and back off on therapy and maintain the excellent outcomes.”

For high-risk patients, Bagatell said, “even though the relapse-free survival of children with high-risk neuroblastoma has doubled. In the time that I've been a pediatric oncologist. It's not good enough. And there are still children who experience relapse and die from their disease that we really need to think about and treat in a more effective way.”

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