Pediatric Stem Cell Transplants
Bone Marrow Transplants in Children
Duke’s pediatric blood and bone marrow transplant program is internationally recognized for its innovative use of stem cells to treat childhood leukemia, lymphoma, inherited primary immunodeficiency disease, inherited metabolic diseases, and sickle cell disease.
Treating Disease with Stem Cell Transplant
Stem cell transplants, also known as bone marrow transplants, may be used to treat:
- High-risk cancers as well as relapsed or recurrent cancers
- Rare inherited disorders that prevent young bodies from working as they should
- Sickle cell disease and thalassemia, conditions where red cells are not correctly produced by the body
We are one of the few programs in the world to offer bone marrow transplants for congenital immune disorders that prevent the body from fighting infection, as well as congenital metabolic diseases such as Hurler syndrome, metachromatic leukodystrophy, Tay-Sachs disease, and adrenoleukodystophy (ALD). Children born with these rare conditions typically have a defective gene that prevents the body from producing properly functioning enzymes.
As leaders in the research and development of new stem cell transplantation techniques, we can extend these lifesaving therapies to many more people -- from infants to young adults, and even to those considered by other centers to be too sick for treatment. We have successfully performed transplants for infants as young as one month and young adults as old as 26 years.
How Bone Marrow Transplant Treats Disease
A bone marrow transplant infuses healthy stem cells in an effort to replace stem cells harmed by disease or treatments such as chemotherapy or radiation. The new cells make healthy blood cells and regenerate the immune system. Stem cells can be removed from the bone marrow, from blood cells circulating through the bloodstream (doctors call this peripheral blood) or umbilical cord blood that remains in the placenta or umbilical cord after birth.
There are three different types of stem cell transplants:
- An autologous transplant uses your child’s own stem cells. This would involve peripheral blood and may be recommended for cancers such as neuroblastoma or brain tumors.
- Allogeneic transplant uses cells from a matched family member or unrelated donor.
- Syngeneic transplant uses stem cells from an identical twin.
Your child’s transplant team will perform a comprehensive evaluation to find a donor match and to determine which type of transplant will be most beneficial for your child.
Choosing a Pediatric Stem Cell Transplant Center
If your child needs a stem cell transplant, you will want an experienced, compassionate team to guide your family along this journey. As a National Cancer Institute-designated Comprehensive Cancer Center, we are dedicated to improving patient outcomes. We are also part of the National Comprehensive Cancer Network (NCCN), an alliance of the nation’s leading cancer centers dedicated to improving patient care.
- Our pediatric bone marrow transplant specialists are pioneers in blood and marrow transplantation. In 1993, Duke performed the first umbilical cord blood transplant, and we are still one of the world’s leading centers for this approach. Our physician-scientists also are investigating the potential of umbilical cord blood as a regenerative therapy for children with disorders such as autism and cerebral palsy.
- Duke’s pediatric blood and bone marrow transplant team is led by board-certified pediatric hematology-oncology specialists. We work closely with other Duke pediatric specialists to provide special care for children with additional medical problems, or who experience complications after transplantation.
- We provide a comfortable, family-friendly setting with access to all the services and experts your child needs. We have a dedicated isolation floor for children who are at a higher risk for infection, as well as a day hospital for children undergoing outpatient procedures, such as infusions or certain types of stem cell transplants.
- All of our inpatient nurses have chosen to work exclusively with pediatric blood and marrow transplant patients. They are a highly skilled, highly educated team of caregivers who regularly share their nursing research results at international conferences.
- Our 16-bed blood and marrow transplant unit has a dedicated pharmacist whose job is to carefully manage dose adjustments and monitor for potential drug interactions. She has more than 15 years of experience working with pediatric transplant patients.
- Our pediatric nutrition specialist helps your child maintain their optimal nutritional status during the transplant process by using both intravenous nutrition as well as oral nutrition supplements.
- Our dedicated discharge planning nurse helps patients transition from the inpatient unit to the outpatient clinic. She visits families at the Ronald McDonald house or other local housing facilities to make sure parents are managing the many medications that are required after transplant.
- Duke is home to the Carolinas Cord Blood Bank, one of the largest public cord blood banks in the world. The extensive inventory comes from a racially diverse group of donors. This improves the chances of finding a suitable match for your child. The bank allows “directed donations,” in which the cord blood can be saved for a family member with certain serious medical conditions including sickle cell disease, congenital immunodeficiency disorder, congenital or acquired marrow failure syndrome, or an inborn error of metabolism.
- Your child may have access to the latest therapies through our innovative research programs and clinical trials. One of our current studies, for example, is investigating a novel use of white blood cells for transplant patients with life-threatening infections. Our researchers also have developed a cell therapy, DUOC-01, to treat children undergoing standard cord blood transplants for congenital metabolic diseases. It is hoped that this therapy will speed up the delivery of the missing proteins to these children with inherited deficiencies.
- Our donor search coordinator, financial coordinators, housing coordinator, and developmental psychologists will guide your family through the process and help you support your child’s recovery.
- Our Family Support Program, made possible by generous donors, provides resources and services for the entire family. These include patient incentive programs, activities and gifts for siblings, and community meals for caregivers.
PEDIATRIC BONE MARROW TRANSPLANT
Preparing for a Bone Marrow Transplant
We will search for a stem cell donor match based on your child’s human leukocyte antigen (HLA) typing. This blood test will be done at our lab to verify your child’s typing and is necessary even if it was done previously at your home doctor’s office or the American Red Cross. Potential donors will also undergo HLA typing to ensure they are a suitable match for your child.
During the two weeks before transplantation, your child will have a complete medical evaluation at Duke, including any necessary blood work and imaging tests. This will help us uncover any previously unknown medical problems that should be addressed before transplantation. Our medical team also will educate you about your child’s condition and about the transplant procedure.
Your child may be in the hospital for several months, depending on their disease and the type of transplant they undergo. Options for housing, if needed, include a temporary apartment rental or hotel room, the nearby Ronald McDonald House or assistance from local nonprofit organizations. Our transplant coordinator can help you with the process.
PEDIATRIC BONE MARROW TRANSPLANT
Your child’s transplant team will determine which type of transplant to perform. If a donor transplant is recommended, a search to identify a donor will take place with immediate family members and the National Marrow Donor Program. Arrangements will be made for the cells to be harvested. If an autologous transplant is recommended, your child’s stem cells will be harvested and frozen for use after the conditioning regimen.
Chemotherapy alone, or chemotherapy with radiation therapy, kills any cancer cells in your child’s body. The conditioning regimen also makes room in the bone marrow space for the transplanted cells to grow. Your child may experience side effects related to the chemotherapy and radiation treatments. The stem cell transplant can take place after the conditioning regimen is complete.
Following the transplant, we will monitor your child’s progress and manage side effects and complications appropriately. Your child will need to stay at Duke, or live locally, for one to eight months -- depending on their condition -- after the transplant to ensure a safe recovery.
Once your child’s treatment is complete, he or she will be discharged back to the care of your referring or home doctor. Your primary Duke transplant specialist will remain in close contact with your home team and will always be available if their advice is needed. Your child will need to return to Duke every three months for one year and then once a year to undergo tests to monitor his or her response to therapy and major organ function.