Stem cell transplant for retinoblastoma

A stem cell transplant is sometimes used to treat retinoblastoma. It is used to replace stem cells when stem cells or bone marrow are damaged. A stem cell transplant is very complex, and the procedure must be done in a specialized transplant centre or hospital to decrease risk and increase chances for a successful outcome.

Stem cells are found in the bone marrow, the blood and umbilical cords. They are basic cells that develop into different types of cells that have different jobs. All our blood cells develop from blood stem cells.

A stem cell transplant uses high-dose chemotherapy to kill all of the cells in the bone marrow. This includes both healthy cells and the cancer cells. After high-dose chemotherapy, healthy stem cells are given to replace the ones in the bone marrow that were destroyed.

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

  • the cancer has spread to other parts of the body
  • the cancer has not responded to other treatments
  • the cancer has come back (recurred)

Your child may be offered high-dose chemotherapy with an autologous stem cell transplant for extraocular retinoblastoma that has spread to the brain and spinal cord (called the central nervous system, or CNS) or other distant sites. In this type of transplant, the stem cells are taken from your child’s own bone marrow or blood.

Side effects

Side effects can happen with any type of treatment for retinoblastoma, but every child’s experience is different.

If your child develops side effects, they can happen any time during, immediately after or a few days or weeks after a stem cell transplant. Sometimes late side effects develop months or years after a stem cell transplant. Most side effects go away on their own or can be treated, but some side effects 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:

  • infection
  • bone marrow suppression (low blood cell counts)
  • graft-versus-host disease (GVHD)
  • digestive system problems

Side effects can develop months or years after treatment for retinoblastoma. Find out more about late effects of treatments for retinoblastoma.

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 your child, ask the healthcare team questions about stem cell transplant.

Expert review and references

  • American Cancer Society. Treating Retinoblastoma. 2015.
  • Bollard CM, Krance RA & Heslop HE . Hematopoetic stem cell transplantation in pediatric oncology. Pizzo, P. A. & Poplack, D. G. (Eds.). Principles and Practice of Pediatric Oncology. 6th ed. Philadelphia: Lippincott Williams & Wilkins; 2011: 16: pp. 467-490.
  • Canadian Retinoblastoma Society . National Retinoblastoma Strategy Canadian Guidelines for Care. Canadian Journal of Ophthalmology. NRC Research Press; 2009.
  • Cancer Research UK. Eye cancer (retinoblastoma). Cancer Research UK; 2015.
  • Retinoblastoma. Children's Hospital Boston. Children's Hospital Boston. Boston, MA: Children's Hospital Boston; 2011.
  • Hurwitz RL, Shields CL, Shields JA, et al . Retinoblastoma. Pizzo, P. A. & Poplack, D. G. (Eds.). Principles and Practice of Pediatric Oncology. 6th ed. Philadelphia: Lippincott Williams & Wilkins; 2011: 27: pp. 809-837.
  • Isidro MA. Medscape Reference: Retinoblastoma Treatment & Management. 2016.
  • Macmillan Cancer Support. Retinoblastoma in children. 2016.
  • Nambayan AG & Gafford E . Retinoblastoma. St. Jude Children's Research Hospital. Cure4Kids. Memphis, TN: St. Jude Children's Research Hospital; 2006.
  • National Cancer Institute. Retinoblastoma Treatment (PDQ®) - Health Professional Version. Bethesda, MD: National Cancer Institute; 2011.
  • National Cancer Institute. RetinoblastomaTreatment (PDQ®) Patient Version. 2016.
  • National Cancer Institute. Retinoblastoma Treatment (PDQ®) Health Professional Version. 2016.
  • Penn Medicine. All About Retinoblastoma. University of Pennsylvania; 2017.
  • Retinoblastoma: The Basics. University of Pennsylvania. OncoLink. Reviewed ed. University of Pennsylvania; 2008.

Medical disclaimer

The information that the Canadian Cancer Society provides does not replace your relationship with your doctor. The information is for your general use, so be sure to talk to a qualified healthcare professional before making medical decisions or if you have questions about your health.

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