High-dose chemotherapy and stem cell rescue for neuroblastoma

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High-dose chemotherapy and stem cell rescue is sometimes used to treat neuroblastoma. It uses particularly strong doses of chemotherapy to kill remaining neuroblastoma cells. To allow the bone marrow to recover from this chemotherapy, stem cells will be given back afterwards. These stem cells will be collected from your child's blood during initial chemotherapy and stored for later use. This is sometimes called a stem cell transplant. A stem cell transplant is risky and complex, and the procedure must be done in a specialized transplant centre or hospital.

Your child may be offered a stem cell transplant to treat neuroblastoma if it:

  • is high risk
  • has spread to other parts of the body
  • has come back (recurred) after treatment
The following chemotherapy drugs may be used for high-dose chemotherapy:

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

Your child may receive 1 or 2 rounds of high-dose chemotherapy and stem cell rescue depending on the individual circumstances.

Side effects

Side effects can happen any time during, immediately after or a few days or weeks after a stem cell transplant. Everyone's experience is different. Sometimes late side effects develop months or years later. Most side effects go away on their own or can be treated, but some may last a long time or become permanent.

Side effects of a stem cell transplant will depend mainly on the type of chemotherapy drug or drug combination given, if radiation therapy was given, the type of transplant and your child's overall health. Common side effects of a stem cell transplant 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 side effects you think might be from a stem cell transplant. The sooner you tell them of any problems, the sooner they can suggest ways to help your child deal with them.

Questions to ask about stem cell transplant

Find out more about stem cell transplant and side effects of stem cell transplant. To make the decisions that are right for you, ask your healthcare team questions about stem cell transplant.

Expert review and references

  • Meredith Irwin, MD
  • Daniel Morgenstern, MD
  • American Society of Clinical Oncology. Neuroblastoma. 2018: https://www.cancer.net/cancer-types/neuroblastoma-childhood/view-all.
  • Lacayo NJ. Pediatric Neuroblastoma. eMedicine/Medscape; 2017: https://emedicine.medscape.com/.
  • Cancer Research UK. Neuroblastoma. Cancer Research UK; 2014.
  • PDQ® Pediatric Treatment Editorial Board. Neuroblastoma Treatment (PDQ®)–Health ProfessionalVersion. Bethesda, MD: National Cancer Institute ; 2020: https://www.cancer.gov/.
  • 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.
  • PDQ® Pediatric Treatment Editorial Board. Neuroblastoma Treatment (PDQ®)–Patient Version. Bethesda, MD: National Cancer Institute; 2021: https://www.cancer.gov/types/neuroblastoma/patient/neuroblastoma-treatment-pdq.
  • American Cancer Society. Treating Neuroblastoma. 2020: https://www.cancer.org/.
  • Bartholomew, J . Neuroblastoma. Baggott C, Fochtman D, Foley GV & Patterson Kelly, K (eds.). Nursing Care of Children and Adolescents with Cancer and Blood Disorders. 4th ed. APHON; 2011: 30: pp. 1038-1053.
  • Lacayo NJ. Medscape: Pediatric Neuroblastoma Treatment and Management. WebMD LLC; 2012.
  • Cancer Research UK. Neuroblastoma. Cancer Research UK; 2014.