High-Dose Chemotherapy and Stem Cell Transplant for Retinoblastoma

Doctors are studying the use of this type of treatment in children with retinoblastoma that has spread outside the eye and who are unlikely to be cured with other treatments.

A stem cell transplant (SCT) lets doctors give higher doses of chemotherapy (chemo) than could safely be given otherwise). In the past, this type of treatment was commonly referred to as a bone marrow transplant.

The bone marrow is the soft, inner part of some bones where new blood cells are made. Chemo drugs can affect quickly dividing cells like those in the bone marrow. Even though higher doses of these drugs might be more effective in treating tumors, they can’t be given because they would cause severe damage to bone marrow cells, leading to life-threatening shortages of blood cells.

To try to get around this problem, the doctor may treat the child with high-dose chemo (sometimes along with radiation therapy) and then use a stem cell transplant to “rescue” the bone marrow.

How is it done?

The first step in a SCT is to collect, or harvest, the child’s own blood-making stem cells to use later. (These are the cells that make the different types of blood cells.)

In the past, the stem cells were often taken from the child’s bone marrow, which required a minor operation. But doctors have found that these cells can be taken from the bloodstream during a procedure similar to donating blood. Instead of going into a collecting bag, the blood goes into a special machine that filters out the stem cells and returns the rest of the blood to the child’s body. The stem cells are then frozen until the transplant. This process may need to be done more than once.

Once the stem cells have been stored, the child gets high-dose chemotherapy in the hospital, sometimes along with radiation therapy. When the treatment is finished (a few days later), the stem cells are thawed and returned to the body in a process similar to a normal blood transfusion. The stem cells travel through the blood and settle in the bone marrow.

Over the next few weeks, the stem cells start to make new blood cells. Until this happens, the child is at high risk of infection because of a low white blood cell count, as well as bleeding because of a low platelet count. To avoid infection, protective measures are taken, such as using special air filters in the hospital room and having visitors wear protective clothing. Blood and platelet transfusions and treatment with IV antibiotics may also be used to prevent or treat infections or bleeding problems.

The child can usually leave the hospital once their blood cell counts return to a safe level. They may then need to make regular visits to the outpatient clinic for about 6 months, after which time their care may be continued by their regular doctors.

Practical points

A SCT is a complex treatment that can cause life-threatening side effects. If the doctors think your child can benefit from a transplant, the best place to have it done is at a nationally recognized cancer center where the staff has experience with the procedure and with managing the recovery period.

A stem cell transplant often requires a long hospital stay and can be very expensive, often costing well over $100,000. Be sure to get a written approval from your insurer before treatment if it is recommended for your child. Even if the transplant is covered by your insurance, your co-pays or other costs could easily amount to many thousands of dollars. It’s important to find out what your insurer will cover before the transplant to get an idea of what you might have to pay.

Possible side effects

The possible side effects from SCT are generally divided into early and long-term effects.

Short-term, early side effects: The early complications and side effects are basically the same as those listed in the Chemotherapy for Retinoblastoma section, but they can be more severe because the drug doses are higher. Side effects can include:

  • Low blood cell counts (with fatigue and an increased risk of infection and bleeding)
  • Nausea and vomiting
  • Loss of appetite
  • Mouth sores
  • Diarrhea
  • Hair loss

One of the most common and serious short-term effects is an increased risk of serious infections. Antibiotics are often given to try to keep this from happening. Other side effects, like low red blood cell and platelet counts, may require blood product transfusions or other treatments.

Long-term and late side effects: Some complications and side effects can last for a long time or might not occur until months or years after the transplant. These can include:

  • Radiation damage to the lungs
  • Problems with the thyroid or other hormone-making glands
  • Problems with fertility
  • Damage to bones or problems with bone growth
  • Development of another cancer (including leukemia) years later

Be sure to talk to your child’s doctor before the transplant to learn about possible long-term effects your child might have. For more on the long-term effects of this and other treatments, see the section Late and Long-term Effects of Treatment for Retinoblastoma.

To learn more about stem cell transplants, see Stem Cell Transplant for Cancer.

The American Cancer Society medical and editorial content team
Our team is made up of doctors and master's-prepared nurses with deep knowledge of cancer care as well as journalists, editors, and translators with extensive experience in medical writing.

Last Medical Review: March 12, 2015 Last Revised: March 12, 2015

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