Treatments for high-risk neuroblastoma

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The following are treatment options for high-risk neuroblastoma. The healthcare team will suggest treatments based on your child's needs and work with you to develop a treatment plan.

Treatment for high-risk neuroblastoma is very intensive. It usually includes a combination of therapies, including chemotherapy, surgery, radiation therapy, high-dose chemotherapy with stem cell rescue, retinoids and immunotherapy. Treatment is divided into the following 3 phases.

Induction is done to try to get the cancer into remission by destroying or removing as much of the cancer as possible. Combinations of different chemotherapy drugs will be used. Surgery may be done after chemotherapy to remove as much of the tumour as possible.

Consolidation is done to try to get rid of any cancer cells that are still in the body. High-dose chemotherapy is given and then followed with a stem cell rescue to allow the bone marrow to recover. Radiation therapy may be given to the primary tumour before, during and after high-dose chemotherapy and stem cell rescue.

Maintenance is done to try to lower the chance that the neuroblastoma will come back (recur). Maintenance includes retinoids and immunotherapy.


Chemotherapy is often used to treat high-risk neuroblastoma. Chemotherapy uses anticancer drugs to destroy cancer cells. It is usually the first treatment given after surgery to diagnose neuroblastoma. It is used to shrink the tumour, including where it has grown into or is touching nearby organs and structures, and to clear disease that has spread elsewhere in the body.

Chemotherapy for induction usually involves different combinations of drugs given one after the other. These drugs include:

  • cyclophosphamide
  • vincristine
  • doxorubicin
  • etoposide
  • cisplatin
  • topotecan
High-dose chemotherapy combinations used in the consolidation phase include:

  • carboplatin, etoposide and melphalan
  • busulfan and melphalan
  • thiotepa and cyclophosphamide

Children may receive 1 or 2 rounds of high-dose chemotherapy depending on the circumstances. Find out more about chemotherapy for neuroblastoma.


Surgery is often done after initial chemotherapy to remove as much of the tumour as possible. Find out more about surgery for neuroblastoma.

High-dose chemotherapy and stem cell rescue

Before treatment begins, the healthcare team will harvest and store some of your child's stem cells. They are usually collected from the blood during induction chemotherapy. Later in treatment, the child is then given high-dose chemotherapy to destroy any cancer cells in the body.

After high-dose chemotherapy, the child is given the stem cells that were collected before chemotherapy (called an autologous peripheral blood stem cell transplant) in order to allow their bone marrow to recover.

Find out more about stem cell rescue for neuroblastoma.

Radiation therapy

Radiation therapy may be used to treat high-risk neuroblastoma. Radiation therapy uses high-energy rays or particles to destroy cancer cells. It may be given to treat emergencies, such as spinal cord compression, or to treat areas where the cancer has spread (called metastatic sites) that did not respond well to other therapies.

Radiation therapy is typically given to the area where the tumour was removed (called the tumour bed), even if the entire tumour was completely removed. This is done to destroy any cancer cells that may be left behind after surgery. Radiation therapy to the tumour bed is usually given after high-dose chemotherapy and stem cell rescue.

Sometimes radioisotope therapy with radioactive metaiodobenzylguanidine (MIBG) is used to treat high-risk neuroblastoma. This treatment is only available in a few Canadian centres and is only given within a clinical trial. Stem cell rescue may be needed afterwards to help the bone marrow to recover.

Find out more about radiation therapy for neuroblastoma.

Retinoids and immunotherapy

Retinoids and immunotherapy may be given after a stem cell transplant to treat high-risk neuroblastoma as part of the maintenance phase of treatment. Retinoids are a form of vitamin A that help some cancer cells mature (differentiate) into normal cells. Immunotherapy uses an antibody called dinutuximab (Unituxin) to target any remaining neuroblastoma cells so that they are then killed by the body's own immune system. This antibody treatment is usually given together with granulocyte-macrophage colony-stimulating factors (GM-CSFs) such as sargramostim (Leukine) to help to boost the immune response.

Find out more about retinoids and immunotherapy for neuroblastoma.

Targeted therapy

Targeted therapy may be used to treat neuroblastoma. Targeted therapy uses drugs to target specific molecules (such as proteins) on or inside cancer cells to stop the growth and spread of cancer and limit harm to normal cells. ALK inhibitors (such as crizotinib) target neuroblastoma cells that have an abnormal ALK gene. ALK inhibitors may be used to treat neuroblastoma that is no longer responding to other treatments or as part of initial treatment in a clinical trial.

Find out more about targeted therapy for neuroblastoma.

Clinical trials

Children with cancer may be treated in a clinical trial. Clinical trials look at new and better ways to prevent, find and treat cancer. Find out more about clinical trials.

Expert review and references

  • Meredith Irwin, MD
  • Daniel Morgenstern, MD
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  • Brodeur GM, Hogarty MD, Bagatell R, Mosse YP, Maris JM. Neuroblastoma. Pizzo PA, Poplack DG, eds.. Principles and Practice of Pediatric Oncology . 7th ed. Philadelphia, PA: Wolters Kluwer; 2016: 30:772–797.
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