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Neuroblastoma
Neuroblastoma is a tumor of the sympathetic nervous system that primarily affects infants and young children. Its course varies widely: some forms may regress spontaneously, while high-risk forms require a series of intensive treatments.
This tumor develops from immature cells of the sympathetic nervous system. The primary tumor most often arises in an adrenal gland or along the sympathetic chains, from the base of the neck to the pelvis. In France, approximately 130 to 150 new cases are diagnosed each year, mainly before the age of 5.
Key Takeaways
Age, extent of disease, histology, chromosomal and genomic characteristics—including MYCN status—as well as response to treatment determine the risk group and the intensity of therapy.
Why does the disease occur?
In most cases, no cause is identified. There is no proven link to diet, lifestyle, or psychological factors. Rare forms are associated with a genetic predisposition, particularly constitutional abnormalities in ALK or PHOX2B, which may lead to a recommendation for genetic counseling.
Symptoms
Symptoms vary depending on the location and extent of the disease: abdominal mass or pain, constipation, shortness of breath, bone pain, difficulty walking, fatigue, pallor, fever, bruising around the eyes, or symptoms related to spinal cord compression. In rare cases, opsoclonus-myoclonus-ataxia syndrome may be associated with neuroblastoma.
When to Seek Immediate Medical Attention
Weakness in the legs, a sudden difficulty walking, urinary problems, severe pain, difficulty breathing, or a rapid deterioration in overall health require urgent medical evaluation.
Diagnosis and Staging
Ultrasound, CT scans, and MRI are used to locate the tumor and assess its relationship to surrounding organs, blood vessels, and the spinal canal. An MIBG scan is used to identify tumor sites when the tumor takes up this tracer. Measuring catecholamine metabolites in urine or blood aids in the diagnosis.
A biopsy confirms the disease and allows for the evaluation of MYCN, ploidy, segmental chromosomal abnormalities, and other biological characteristics. Bone marrow samples may be taken to complete the evaluation.
Tumor biology guides treatment
MYCN amplification is a significant risk factor, but risk stratification is never based solely on this finding.
Very different behaviors
Some forms of the disease in infants may regress spontaneously or evolve into a less aggressive tumor, which may justify closely monitored observation. Low-risk forms may be managed with surgery alone or limited treatment. Intermediate-risk forms often require chemotherapy and surgery. High-risk forms require intensive multimodal treatment.
Treatments
Surgery
The goal is to remove the tumor without damaging nearby organs, blood vessels, or nerves. In advanced cases, surgery is often performed after debulking chemotherapy.
Chemotherapy
It is used when the tumor is not immediately operable, when the disease is metastatic, or when the risk group warrants systemic treatment.
High-Risk Forms
Treatment generally combines induction chemotherapy, surgery, high-dose chemotherapy with autologous stem cell support, radiation therapy, followed by maintenance therapy with retinoids and GD2-targeted immunotherapy.
Radiation Therapy and Therapeutic MIBG
Radiation therapy targets the primary site and, depending on the situation, certain persistent lesions. Iodine-131-labeled MIBG may be considered in selected refractory or relapsed cases and in strategies evaluated through research.
The Forecast
The prognosis varies considerably, ranging from localized forms or those affecting infants—which generally have a very favorable outlook—to high-risk forms that are much more difficult to treat. An average figure masks this diversity and does not allow us to predict a child’s course of the disease.
Research and Therapeutic Innovation
HR-NBL2 is an international Phase III trial focused on high-risk neuroblastoma. It compares strategies during induction therapy, high-dose chemotherapy, and radiation therapy. The French registry indicates that the trial is open for enrollment and is being conducted by Gustave Roussy.
VERITAS compared two intensification strategies in metastatic patients who responded inadequately to induction therapy. The study has been completed, and the available results did not allow for a determination of which of the two strategies was more effective.
A preclinical program at Gustave Roussy
Research is being conducted to investigate the combination of CAR-T cells and modified macrophages in aggressive neuroblastoma. This approach is experimental and is not a validated or routinely available treatment.
Follow-up After Treatment
Follow-up care is designed to monitor for recurrence and late effects. It may include monitoring of hearing, kidney function, heart function, endocrine function, growth, fertility, and development, as well as psychological, educational, nutritional, and social support.
Patient Care at Gustave Roussy
The Department of Pediatric and Adolescent Oncology coordinates patient care with the teams in imaging, partner surgical departments, pathology, molecular biology, nuclear medicine, radiation oncology, transplant, genetics, and supportive care teams. The case is discussed during a multidisciplinary team meeting.
Frequently Asked Questions
No. Neuroendocrine tumors (NETs) exhibit a wide range of behaviors. Some well-differentiated tumors grow slowly, while others grow more rapidly. Poorly differentiated neuroendocrine carcinomas constitute a distinct category and are generally more aggressive.
The difference lies primarily in the appearance and organization of the tumor cells. A TNE is well-differentiated, while a neuroendocrine carcinoma is poorly differentiated. Their course and treatments differ, which is why an expert histopathological analysis is so important.
No. Some tumors produce hormones without causing any symptoms. A tumor is considered functional when hormone secretion leads to a clinical syndrome. Test levels should be tailored to the symptoms and the tumor’s location.
This test checks for the presence of receptors on tumor cells. It can contribute to the staging of the disease and help determine whether treatment targeting these receptors—such as targeted internal radiation therapy—can be considered.
Yes, in certain well-defined TNE cases that are stable and do not progress, and that do not present life-threatening symptoms. This monitoring is active and planned. It allows treatments to be reserved for the time when their expected benefit outweighs their risks.
Most are not. However, some may be part of a predisposition syndrome. An oncogenetic consultation is offered when the characteristics of the disease or family history warrant it.
Yes. A second opinion can confirm the diagnosis, help prioritize treatment options, or assess eligibility for a clinical trial. The case file must include the medical and imaging records requested by the dedicated platform.