NewYork-Presbyterian Morgan Stanley Children’s Hospital has one of the oldest and largest stem cell transplant programs in the nation, offering this treatment to children with cancer and noncancerous blood disorders. Some patients can have a less toxic reduced-intensity treatment.
With this approach to stem cell transplantation, we give low-dose chemotherapy plus immune therapy to allow the donor’s cells to grow while minimizing side effects.
What Is a Stem Cell Transplant?
Stem cells are found in the bone marrow, where they produce different types of blood cells. A stem cell transplant is a procedure in which healthy stem cells from a donor are given to a patient whose own stem cells have given rise to cancer or another blood disorder. Conditions that can develop from diseased stem cells include leukemia and sickle cell disease.
A stem cell transplant is sometimes called a bone marrow transplant.
What Does a Stem Cell Transplant Treat?
At NewYork-Presbyterian, we have superior expertise in using stem cell transplants to treat:
- Sickle cell disease
- Hemophagocytic lymphohistiocytosis — a disorder in which the immune system produces too many activated immune cells
- Leukemia that cannot be successfully treated with other therapies
- Severe combined immunodeficiency (SCID), as part of our New York State-certified SCID Program
- Rare blood disorders that can only be cured with a stem cell transplant
Types of Stem Cell Transplants
There are two main types of stem cell transplants:
- Autologous stem cell transplants use a patient’s own cells. Stem cells are removed from the patient’s blood and bone marrow, treated to filter out the damaged ones, and frozen. Later, the healthy cells are given back to the patient. An autologous stem cell transplant may be used to treat blood cancers such as Hodgkin’s lymphoma, non-Hodgkin’s lymphoma, and myeloma.
- Allogeneic stem cell transplants use stem cells from a donor, either a family member or someone unrelated. Healthy stem cells are isolated from the donor’s blood or from a donated umbilical cord. Later, the donated stem cells are infused into the patient. An allogeneic stem cell transplant may be used to treat chronic myeloid leukemia, acute myeloid leukemia, and myelodysplastic syndrome.
Donor stem cells must be a match for the patient’s immune system. Our team can help find a suitable donor through the National Marrow Donor Program for patients who do not have a family member who is a matched donor.
NewYork-Presbyterian’s stem cell transplant program is accredited by the Foundation for the Accreditation of Cellular Therapy (FACT) and the National Marrow Donor Program (NMDP) for autologous (self), allogeneic (donated), and umbilical cord blood transplantations.
How Is a Stem Cell Transplant Performed?
A stem cell transplant replaces unhealthy blood-forming cells with healthy ones. In some cases, healthy stem cells can be isolated from the patient (autologous transplant). More often, stem cells are donated by a family member or an unrelated donor (allogeneic transplant).
The basic steps of a stem cell transplant are as follows:
- Healthy stem cells are collected from the blood and bone marrow of either the patient or a stem cell donor.
- The patient receives chemotherapy to destroy diseased cells in the bone marrow and any cancer cells.
- The healthy blood stem cells collected beforehand are given to the patient intravenously (by vein).
- Once in the bloodstream, the healthy cells know where they belong in the body and settle in the bone marrow.
- In the bone marrow, the transplanted stem cells grow and produce healthy new red blood cells, white blood cells, and platelets. This process is called engraftment.
Transferring the stem cells takes only a few hours, and engraftment takes two to three weeks or more. Overall, patients can expect to stay in the hospital for eight to 12 weeks to receive the chemotherapy and the transplant.
Risks to Consider
Serious problems can occur after a stem cell transplant. The risks vary depending on the individual — for example, the disease being treated, the patient’s overall health, the type of transplant, and the chemotherapy regimen. Before going ahead with this procedure, talk with the doctor about your child’s individual risk, and about ways of dealing with potential complications.
Similar complications can arise from both allogeneic and autologous transplants. These include:
- Mouth sores
- Hair loss
- Iron-deficiency anemia
- Bleeding and bruising
- Nausea, vomiting, diarrhea
- Infections, such as pneumonia, shingles, or herpes simplex
- Recurrence, or relapse, of the disease that the transplant was used to treat
- Graft failure, meaning the new stem cells don’t work, or only work for a short time
- Graft-versus-host disease (GVHD), which can occur after an allogeneic transplant, causing the new stem cells to attack other cells in the body.
Preparing for a Stem Cell Transplant Procedure
Preparing for a stem cell transplant involves both medical procedures and logistical planning. A stem cell transplant is a lengthy process, and your child will need to be in the hospital for many weeks. We offer support, activities, education, and guidance during your child’s time with us, as well as afterward when it’s time to go home.
Here are the basic steps that take place before stem cells are transplanted:
- Medical testing to ensure that your child is healthy enough to undergo the procedure can include tests of lung and heart function and bone marrow biopsy.
- A tube similar to an intravenous line will be placed in the upper chest for infusing chemotherapy, stem cells, fluids, and other infusions.
- The patient or the donor receives medicine to stimulate the body to make more stem cells and release them from the bone marrow into the bloodstream.
- Blood containing stem cells is collected; the stem cells are filtered out, and the remaining blood is infused back into the patient or donor.
- Stem cells are frozen and stored until the patient is ready for transplant.
- The patient receives chemotherapy over a period of weeks to kill cancer cells and destroy the bone marrow. Some people also receive radiation. This process is called conditioning.
What to Expect After a Stem Cell Transplant
After the procedure, doctors watch to be sure the stem cell transplant has been accepted. Your child will also need time to recover from the side effects of the chemotherapy. This usually takes a few weeks. Then your child can leave the hospital.
After discharge, your child will continue to see the doctor often. We monitor and support patients on an outpatient basis for six to 12 months to reduce the risk of infections and complications. For most children, the return to regular activity and school takes approximately one year. Your doctor will give you more specific information about what to expect.
A stem cell transplant works by replacing unhealthy blood-forming cells with healthy ones.
Success rates vary depending on the age and health of the patient, the condition being treated, and the type of transplant. For some diseases, it offers a cure. It can put other diseases into remission.
Stem cell transplants are used when a person’s bone marrow is not producing healthy new blood cells. It is a treatment for leukemia and other diseases in which blood cells do not develop properly.
Choose NewYork-Presbyterian for Your Child’s Stem Cell Transplant
At NewYork-Presbyterian, we have one of the oldest and largest pediatric stem cell transplantation programs in the nation, and we achieve superior outcomes compared to other centers. Schedule an appointment to talk with a doctor and learn more about the treatment options available for your child’s condition.
Our patients receive state-of-the-art care from pediatric stem cell transplant physicians working with a team of other specialists devoted to pediatric conditions.