Newly Diagnosed With Neuroblastoma

You will hear a stage. You will hear more about risk group, and that is the one that decides treatment.

NCI sorts neuroblastoma into low, intermediate, or high risk. The split is built from stage, the child's age, how the tumour looks under the microscope, and genetic features of the tumour itself. Two children with the same stage can land in different groups and get very different plans.

The gap between those groups is wide. In a Children's Oncology Group study of more than 5,000 children, five-year overall survival was 98 percent for low-risk disease and about 95 percent for intermediate-risk disease. For high-risk disease, NCI reports a five-year survival rate of 62 percent.

Across all infants and children under 15 diagnosed from 2014 through 2020, NCI puts five-year relative survival at about 85 percent. That single number hides the spread, which is why the risk group is the thing to ask for.

Several of these can run at the same time, so ask which are already booked.

• Urine catecholamines. Neuroblastoma cells release chemicals that show up as VMA and HVA in urine, measured against creatinine. A random sample works; NCI says a 24-hour collection is not needed.
• Imaging of the tumour. Usually CT or MRI with contrast. NCI specifies MRI for tumours beside the spine that could press on the spinal cord.
• MIBG scan. A tracer that about 90 percent of neuroblastomas take up. NCI calls it a critical part of the standard workup, for the main tumour and for spread. If a tumour does not take it up, an FDG-PET scan is used instead.
• Bone marrow testing, to look for tumour cells.
• Tumour tissue. This is needed for the pathology classification, for MYCN copy number, and for chromosome changes. NCI notes core-needle biopsy gives similar results to open surgery with fewer complications.

MYCN is the genetic result families hear about most. Extra copies of that gene generally point to more aggressive disease.

This is the part that sounds impossible when you first hear it.

NCI reports that spontaneous regression is well described in infants, particularly in the pattern called 4S or MS. It generally happens in tumours without MYCN amplification.

In a Children's Oncology Group study, 83 carefully chosen infants under 6 months old with small adrenal masses of 3.1 cm or less were watched without even a biopsy. Surgery was held in reserve for growth or rising urine markers. Eighty-one percent never had surgery, and all were alive at two years.

A German trial followed 340 infants with localised neuroblastoma and no MYCN amplification. Of the 93 watched with visible tumour still present, 44 either shrank on their own or stopped growing.

Observation of this kind is offered to a narrow group. If it is offered to your child, ask what would end it.

For low-risk disease, NCI describes surgery followed by observation, or observation with or without a biopsy. Chemotherapy is held back for children with symptoms. Radiation is used only as emergency treatment.

For intermediate-risk disease, chemotherapy usually comes before surgery. The drugs are doxorubicin, cyclophosphamide, a platinum drug, and etoposide. The number of cycles depends on the tumour's biology and how it responds. NCI notes that trials have since cut the duration and intensity for several subsets with no loss of results.

For the 4S or MS pattern, asymptomatic infants with favourable biology may simply be observed with supportive care. Chemotherapy is used for babies who have symptoms, for unfavourable biology, and for infants under 3 months.

If your child is high risk, the plan will span most of a year. NCI describes three phases.

Induction is dose-intensive chemotherapy with surgery. The backbone alternates cisplatin and etoposide with vincristine, cyclophosphamide, and doxorubicin, and adds topotecan with cyclophosphamide.

Consolidation is two rounds of high-dose therapy with a stem cell transplant, plus radiation to the original tumour site and to remaining spots of spread. A randomised trial found two rounds gave better three-year event-free survival than one.

Postconsolidation is immunotherapy alongside GM-CSF and isotretinoin. The antibody used is dinutuximab. Its FDA label covers children with high-risk neuroblastoma who reached at least a partial response to earlier treatment. That label carries a boxed warning for serious infusion reactions and for nerve pain, which is why an opioid is given before, during, and after each infusion.

Follow the emergency instructions your child's team has given you. Otherwise:

• Call 911 or go to an emergency department if your child's legs go weak, they stop being able to walk, or they lose control of bladder or bowel. A tumour beside the spine can press on the cord, and that is a same-hour problem. Seizures, or breathing that is fast and laboured, belong here too.
• Call your child's cancer team at once, day or night, if their temperature reaches 100.4°F (38°C) or higher during chemotherapy, or they shake with chills. CDC treats fever on chemotherapy as a medical emergency, and a child's infection can turn serious within hours. If you cannot reach the team quickly, take them to an emergency department and say straight away that your child is on chemotherapy.
• Call your child's team the same day if pain will not settle with the prescribed medicines, vomiting keeps going, or their belly keeps growing.
• Call your child's team within a day or two if their eyes dart about, their movements turn jerky, they have become clumsy, or they look pale and bruise easily.

Neuroblastoma, Childhood Cancer: An Overview for Families, Clinical Trials for Children With Cancer, and Childhood Cancer Survivorship and Follow-Up Care.

• https://www.cancer.gov/types/neuroblastoma/hp/neuroblastoma-treatment-pdq
• https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=d66bdf0d-9d65-45de-ae5b-a58617c27492
• https://www.cdc.gov/cancer-preventing-infections/patients/fever.html