Blood & Marrow Transplantation for Children

MSK pediatric bone marrow transplant specialist Farid Boulad

Memorial Sloan Kettering’s world-renowned experts care for children, teens, and young adults who need stem cell and bone marrow transplants.

MSK was one of the nation’s first transplant centers. Our experts have been leaders in the field ever since. We pioneered innovative and effective types of transplants, including transplants from donors who are not related to the patient. We also developed and introduced several new treatments over the years that have made the process safer and more effective. Our goal is to get children back to leading full, healthy lives. We tailor treatment to each individual patient to give them the best possible chance for a cure, whether for cancer or other life-threatening illnesses.

If your child is in need of a stem cell or bone marrow transplant, you probably have many questions. Learning as much as you can about the transplant process can help you feel better prepared to speak with doctors about your next steps. We’ve compiled this list of FAQs to help get you started. If you have any questions, please reach out to us. We are here for you and want to help you in any way we can.

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Why does my child need a bone marrow or stem cell transplant?

A bone marrow transplant can be used to treat a number of different cancers or non-cancerous conditions in children. It is also known as a stem cell transplant. Sometimes, a child’s bone marrow — the spongy tissue found inside the bones — does not make the correct amounts of blood cells. Or there may be a problem with the cells that it makes. A bone marrow or stem cell transplant can replace your child’s bone marrow with donor stem cells to make it healthy again.

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What are stem cells?

Stem cells are the “factories” that produce all blood cells, including red blood cells, white blood cells, and platelets. Stem cells are found in the bone marrow and blood.

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Is a bone marrow transplant the same as a stem cell transplant?

Healthy stem cells can be collected from the bone marrow (where they normally are found) or from the bloodstream. When we collect the healthy stem cells from bone marrow, this is called a bone marrow transplant. When we collect the healthy stem cells from the bloodstream, this is called a peripheral blood stem cell transplant.

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Is your program nationally accredited?

Yes. Memorial Sloan Kettering’s stem cell transplant program is approved by the Children’s Oncology Group, the National Marrow Donor Program, the Bone Marrow Transplant Clinical Trials Network, the Pediatric Blood and Marrow Transplant Consortium, the Pediatric Immune Deficiency Treatment Consortium, and the Center for International Blood and Marrow Transplant Research. The program is certified by the Foundation for Accreditation of Cellular Therapy.

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What are the different types of transplants?

There are three different types of stem cell transplants. In an autologous transplant, we collect your child’s own stem cells and then transfer them back to him or her. In an allogeneic transplant, we collect stem cells from another person and then give them to your child. In a cord blood transplant, the stem cells come from the umbilical cord and placenta of a healthy newborn. Cord blood transplants can be a good choice for kids without a closely matched donor.

Learn more about Memorial Sloan Kettering’s research on cord blood transplantation.

Your team of doctors will pick which type of transplant is best for your child based on the specifics of his or her disease. We will also consider other factors, like what treatments your child has had and his or her general health. Your child’s transplant doctor will discuss all of this with you.

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What diseases are treated with a bone marrow or stem cell transplant?

Autologous transplants can be used to treat a child who is diagnosed with:

  • germ cell tumors (e.g., testicular cancer)
  • certain solid tumors (brain, eye, kidney)
  • certain types of lymphoma

Allogeneic and cord blood transplants can be used to treat a child who is diagnosed with:

  • leukemia
  • hemophagocytic lymphohistiocytosis (HLH)
  • myelodysplastic syndrome
  • inherited and acquired bone marrow failures
  • genetic immunodeficiencies (e.g., severe combined immunodeficiency, Wiskott-Aldrich syndrome, Omenns syndrome, CD40 ligand deficiency)
  • genetic autoimmune diseases (e.g., IPEX)
  • red blood cell disorders (e.g., sickle cell disease, thalassemia)
  • platelet disorders (e.g., amegakaryocytosis, congenital thrombocytopenia)
  • white blood cell disorders (e.g., severe congenital neutropenia, chronic granulomatous disease, leukocyte adhesion deficiency)
  • paroxysmal nocturnal hemoglobinuria
  • osteopetrosis
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How do I find a donor for an allogeneic transplant?

If your doctor tells you that your child needs an allogeneic transplant, finding a donor will be an important step. The team at MSK will help you find the best possible source for an allogeneic transplant, whether it is a sibling, relative, someone who is not related to your child, or cord blood.

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What are the different ways you can do a transplant?

We can do transplants in a number of different ways depending on your child’s specific needs.

In an unmodified stem cell transplant, all of the stem cells are given to your child without making any changes to them in a laboratory. This is also called a conventional stem cell transplant. Unmodified transplants are better for kids with certain noncancerous conditions or with certain types of cancer that put them at a very high risk of relapse. They also need to be able to take medications that prevent a complication known as graft-versus-host disease (GVHD).

We perform T cell-depleted transplants for children with a variety of conditions and cancers to prevent GVHD. In these transplants, the stem cells are separated from T cells — the cells that cause GVHD — in a laboratory. The remaining donated cells are then given to your child. Because T cell-depleted transplants have a much lower risk of GVHD, children are not required to take medications to prevent GVHD after the transplant.

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Will my child need chemotherapy or radiation before the transplant?

In most cases, chemotherapy or radiation is needed before the transplant to allow the transplanted cells to grow. In children with cancer, it is also important to remove the cancer cells. Before your child receives his or her transplant, we will give medications or in some cases radiation to wipe out their diseased bone marrow and make room for the new, healthy marrow. In autologous transplants, children receive chemotherapy to destroy any remaining non-bone marrow cancer cells, and then receive their own blood stem cells back.

For certain immunodeficiencies, intensive chemotherapy and radiation are not needed. As members of one of the most experienced transplant teams in the world, the specialists at MSK will customize the treatment your child receives before performing the transplant.

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How are stem cells collected?

Whether your child or someone else is donating stem cells, the process is the same. Doctors will first collect, or harvest, the blood-forming stem cells from the bone marrow or blood.

Stem cells taken from bone marrow are collected in an operating room while the donor is under general anesthesia. The most common place that bone marrow is collected from is the back of the hip bones. The donor will not need stitches after this minimally invasive procedure and will be sent home the same day.

Stem cells can also be collected using a procedure called apheresis. A few days before the procedure, donors are given a medication to encourage stem cells to move into the bloodstream. Blood is collected from the donor’s veins and a machine separates the stem cells from other blood cells. The remaining blood is then returned to the donor. The stem cells collected during the procedure are either used immediately or frozen and stored until they are needed.

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Once the stem cells are collected, when does the transplant occur?

We can give your child his or her new stem cells immediately after they are collected, or we can freeze them and store them for later use. Depending on the type of transplant, it can take as little as five minutes or as long as a few hours. The transplant is not a surgery. The stem cells are simply infused into your child’s bloodstream. The cells then travel to the bone marrow spaces in the large bones and begin producing normal blood cells within a few weeks.

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What happens after the transplant?

We’ll keep your child safe and protected in the hospital as his or her new marrow begins to produce normal blood cells. For two to four weeks after the transplant, before the new bone marrow starts working, patients’ risk for infection and excessive bleeding is very high. During these first few weeks, we’ll monitor your child carefully and give medicines and blood transfusions to help prevent infection. Your child will be isolated in the hospital during this period, but a parent is welcome to stay with him or her at all times. When enough healthy red blood cells, white blood cells, and platelets are made, your child is allowed to go home. This typically happens four to eight weeks after the transplant.

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How will we know if the transplant is a success?

Early after the transplant, while your child is still in the hospital, we will check his or her blood each day to determine if it is growing new, healthy cells. When we see new blood cells being made, we will check to make sure that these cells are from the healthy donor and that the issue they received the transplant for has been fixed. We will also monitor your child closely for any complications related to the transplant. 

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Who will be caring for my child?

We take a team approach to caring for our young patients and their families. Besides doctors, nurse practitioners, and nurses with special expertise in transplants, our team also includes a number of specialists. They are all here to support your child’s academic, emotional, and social development, as well as to help you and your family through the entire transplant process.

Learn more about our specialized staff and programs designed to meet you and your family’s needs.

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Will my child have to miss school during the transplant process?

We have full-time teachers on staff to help your child keep up with school.

Because of the high risk of infection, children who receive a transplant will not be able to return to school right away. However, MSK recognizes the importance of school in a child’s life. We have a school on-site as part of New York City’s Department of Education Hospital Schools program. Whether your child is staying with us during the transplant or needs to come in on an outpatient basis, our full-time teachers provide instruction for all grades, from pre-K through high school. They work closely with your child’s school or home instructors to maintain studies throughout their time at MSK. 

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What is the follow-up care after a stem cell transplant?

At MSK, a team dedicated to taking care of transplant patients will provide your child’s follow-up care. This team will care for all of your child’s needs in the time right after the transplant, as well as during the eventual transition back to a normal and healthy life. We also have a dedicated team that follows children after they have fully recovered from their transplant, to ensure our survivors remain healthy throughout their lives.

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