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About Pediatric Stem Cell Transplant

  • A stem cell transplant (also called bone marrow transplant) is the infusion of healthy stem cells into the body to stimulate new bone marrow growth. Stem cells are vital to a person's ability to fight infection. Stem cell transplants are performed on children whose stem cells have been damaged by disease or invasive treatments for cancer such as chemotherapy and/or radiation therapy. There are two types of stem cell transplant:

    • Autologous transplants, in which the patient's own stem cells are collected, stored at a special laboratory and re-introduced into the patient's system intravenously.
    • Allogeneic transplants, in which stem cells are collected from a tissue-matched donor (a sibling, an unrelated donor, or umbilical cord blood) and delivered intravenously.

    We perform both autologous and allogeneic stem cell transplants to treat a range of cancerous and non-cancerous conditions.

    Stem Cell Transplant Process Overview


  • Stem Cell Transplant in Children
    Leslie Lehmann, MD, Clinical Director of the Pediatric Stem Cell Transplant Program at Dana-Farber/Boston Children's Cancer and Blood Disorders Center, explains the stem cell transplant process, what to expect during and after treatment, and the variety of emotional and practical support resources available to pediatric patients and their families.

  • All children undergoing a stem cell transplant are given high-dose chemotherapy to make room in their bone marrow for the new stem cells, suppress their immune system to prevent graft rejection, and destroy cancer cells in their body. After this conditioning regimen, they are given a few days' rest before their transplant.

    Like a blood transfusion, stem cells are given to a child through an intravenous catheter. Children are awake through this painless process. It generally takes two to six weeks for the engraftment to "take" and for the stem cells to multiply and make new blood cells.

    Minor side effects, such as fever, chills, and shortness of breath can accompany the infusion of new stem cells. More significant complications, including graft rejection and graft-versus-host disease (a condition in which the donor's immune cells attack the patient's body), can occur following the transplant.

    We offer several support programs and services to children who are recovering from a stem cell transplant:

    • The David B. Perini Jr. Quality of Life Clinic helps survivors of childhood cancer address the long-term effects of treatment. This includes special programs to ease patients' transition to post-transplant life.
    • A multidisciplinary clinic, staffed by nutritionists, endocrinologists, physical therapists, dermatologists, psychologists and social workers for childhood cancer survivors of all ages, as well as a special follow-up clinic for stem cell transplant patients.
    • A clinic that addresses the needs of patients with graft-versus-host disease, a potentially serious complication following stem cell transplant. Patients meet with multiple medical experts during an outpatient visit to learn how to best manage the disease and its symptoms.

    Pediatric Stem Cell Transplant: An In-Depth Look at the Process

    Stem cell transplantation is a complex process that may take months to complete. Your doctors, nurses and the transplant patient coordinator will review information about your child's stem cell transplant during the consent meeting, daily rounds, and visits to the Jimmy Fund Clinic. A general description of the stem cell transplantation process follows.

    Pre-Transplant Appointments

    Children eligible for stem cell transplantation will have several preliminary appointments, including tests and blood work, scheduled before being admitted for transplant. These pre-transplant evaluations and appointments will be scheduled by our stem cell transplant Patient Coordinator, who acts as your liaison to the clinical team. You will also meet with a senior member of the transplant team to discuss the plan for your child in-depth, and to sign the transplant consent form.

    If your child is receiving an autologous (self) peripheral blood stem cell transplant, we will collect his/her stem cells before admitting your child for transplant. Stem cells are collected at the Apheresis Therapeutic Unit at Boston Children's Hospital. Stem cell collection is coordinated with your care provider and the stem cell transplant Patient Coordinator.

    Conditioning Therapy

    Conditioning therapy refers to the high-dose chemotherapy and/or radiation that children receive before their stem cell infusion. The day your child receives the bone marrow or stem cells is called Day Zero (Day 0). The days prior to Day 0 are the days that a patient receives the conditioning therapy. These days are referred to with negative numbers, for example Day -7, Day -6 or Day -5. There are several goals of conditioning therapy:

    • To eliminate cancer cells in the body in patients with a malignant disorder
    • To eliminate the normal cells in the marrow cavities of the bones so the new marrow will have room to grow

    If your child is receiving cells from another person, conditioning therapy has a third goal: To suppress your child's immune system so it will not reject the donor's cells.

    Day 0: Stem Cell Infusion

    Day 0 is the day the stem cells are infused. Stem cells are infused through the child's central venous line.

    Expected Side Effects

    After Day 0, the days are referred to with positive numbers, for example Day +1, Day +2 or Day +3. Patients may begin to have some of the expected side effects of the conditioning therapy such as nausea, vomiting, diarrhea, and mouth sores.

    Infections After Transplant

    Stem cell transplant patients are at high risk for infections because the immune system is suppressed from the conditioning therapy. Patients who receive stem cells from another person have an additional risk for infection since they take medications that suppress the new immune system. Patients need these immunosuppressive medicines to help prevent graft-versus-host disease (GVHD).

    Engraftment

    After the marrow has been infused, the number of white blood cells and neutrophils, a certain type of white blood cell, increase over the next two to four weeks. Engraftment occurs when the stem cell graft is making enough neutrophils to offer some protection against bacterial infections.

    Graft-Versus-Host Disease

    If your child receives stem cells from someone else, there is a risk of graft-versus-host disease (GVHD). GVHD may occur before or after stem cell engraftment. It is caused when the donor's (graft) T lymphocytes, a type of white blood cell, react against the cells of the patient (host). Therefore, the condition is called graft (donor)-versus-host (patient) disease. The patient is most likely to show the effects of GVHD in the skin, the gastrointestinal tract (digestive system) and the liver. In general, acute GVHD occurs before Day +100 and chronic GVHD occurs after Day +100.

    Other Side Effects

    The conditioning therapy and medications used during transplant may cause other side effects and toxicities, which will be discussed during your initial consent meeting. Our doctors and nurses will explain these problems, and the recommended treatments, if they occur. Blood tests, X-rays, CT scans, MRI scans, and ultrasounds or biopsies (tissue samples) of certain tissues may help in the diagnosis of such problems.

    Discharge

    A child may be discharged after engraftment has occurred. He or she must also be medically stable and able to take the required medications.

    Restrictions After Discharge

    Autologous (self) transplant patients should not attend school or visit other public indoor places for six months after transplant. Allogeneic (related and unrelated donor) transplant patients should not attend school or visit indoor public places for nine to 12 months after transplant, depending on the patient's status. We will provide you and your family with educational materials upon discharge that review common post-transplant issues.