Retinoids and immunotherapy

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Retinoids and immunotherapy are sometimes used to treat neuroblastoma. Retinoids are a form of vitamin A that help some cancer cells mature (differentiate) into normal cells. Immunotherapy helps to direct the body's own immune system to kill any remaining neuroblastoma cells.

Retinoids and immunotherapy may be used to treat high-risk or recurrent neuroblastoma. They are given after high-dose chemotherapy and stem cell rescue, and may be called maintenance therapy. Retinoids and immunotherapy drugs are used to:

  • kill any remaining cancer cells
  • stop cancer cells from growing and spreading
  • lower the risk that the cancer will come back (recur)
There are 3 drugs commonly used together to treat neuroblastoma: a retinoid called isotretinoin (Accutane) and 2 immunotherapy drugs. The healthcare team will consider your child's personal needs to plan the drugs, doses and schedules of retinoids and immunotherapy. Your child may also receive other treatments.

Retinoid drugs used for neuroblastoma

The retinoid used for high-risk or recurrent neuroblastoma is isotretinoin. It is given by mouth for 6 months after high-dose chemotherapy and stem cell rescue.

Immunotherapy drugs used for neuroblastoma

There are different immunotherapy drugs used to treat neuroblastoma.

Monoclonal antibodies are a type of targeted immunotherapy used to treat neuroblastoma. Dinutuximab (Unituxin) is a monoclonal antibody that targets a protein called GD2, which is expressed on the surface of neuroblastoma cells. It works by attaching to neuroblastoma cells that have the GD2 protein and then signalling to the body's immune system to attack and kill the neuroblastoma cells. It is usually given along other immunotherapies.

Granulocyte growth factors (such as G-CSF) may be used to lessen some of the side effects of cancer treatments. They stimulate the bone marrow to make more white blood cells. This helps reduce the risk of infection after chemotherapy. For immunotherapy, a different growth factor called GM-CSF (sargramostim) is used to stimulate the immune system. It is given by an injection under the skin.

Interleukins are a group of hormone-like substances made by white blood cells. They stimulate the immune system to recognize and destroy cancer cells. Until recently, interleukin-2 (Aldesleukin, Proleukin) was used as part of immunotherapy for neuroblastoma. But evidence from studies in Europe now suggests that the addition of interleukin-2 does not appear to be as important, so it is no longer used routinely for most patients receiving immunotherapy for high-risk neuroblastoma.

Side effects

Side effects can happen with any type of treatment for neuroblastoma, but every child’s experience is different. Some children have many side effects. Others have few or none at all.

Side effects of retinoids and immunotherapy will depend mainly on the type of drug or drug combination, the dose and your child's overall health.

Some common side effects of retinoids include:

  • skin problems
  • dry lips, mouth, nose and eyes
  • muscle and joint pains
  • changes in nails
Some common side effects of immunotherapy include: Side effects can develop months or years after treatment for neuroblastoma. Find out more about late effects for neuroblastoma.

Tell the healthcare team if your child has these side effects or others you think might be from retinoids or immunotherapy. The sooner you tell them of any problems, the sooner they can suggest ways to help your child deal with them.

Information about specific cancer drugs

Details on specific drugs change quite regularly. Find out more about sources of drug information and where to get details on specific drugs.

Questions to ask about immunotherapy

Find out more about immunotherapy. To make the decisions that are right for your child, ask the healthcare team questions about immunotherapy.

Expert review and references

  • Meredith Irwin, MD
  • Daniel Morgenstern, MD
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