Blood and Bone Marrow Transplants

Pediatric blood and bone marrow transplants involve transferring healthy blood-forming cells from one person to a child with a blood or bone marrow disorder. This procedure can treat conditions like leukemia, immune deficiencies or genetic disorders by replacing damaged or abnormal cells with healthy ones.

Our Transplant and Cellular Therapy team at the Lisa Dean Moseley Foundation Institute for Cancer and Blood Disorders at Nemours Children’s Hospital, Delaware makes our blood and bone marrow transplant program among the most respected in the world. Along with our renowned oncologists (cancer doctors), hematologists (blood specialists) and researchers, we rank among the very best in the country for both pediatric orthopedics and cancer care (U.S. News & World Report named Nemours Children’s Hospital, Delaware one of the “Best Hospitals”).

How Pediatric Blood and Bone Marrow Transplants Work

Blood has three main cell types: red blood cells (which carry oxygen), white blood cells (which fight infection) and platelets (which clot the blood). New blood cells (called “stem cells”) begin in the bone marrow — the red spongy material found inside the bones — and are released to the bloodstream when mature.

A blood and bone marrow transplant takes unhealthy stem cells out of the bone marrow and replaces them with healthy stem cells, with the intent to treat childhood cancers and nonmalignant blood and bone marrow diseases.

Treating Pediatric Cancers and Blood Disorders

A blood and bone marrow transplant procedure (also called “stem cell transplant”) is used to treat childhood cancers and blood disorders including:

• Cancers (leukemias, lymphomas, neuroblastoma, brain tumors, bone tumors like Ewing sarcoma, and soft tissue sarcomas like rhabdomyosarcoma)
• Blood disorders (sickle cell disease and other hemolytic anemias like thalassemia)
• Bone marrow disorders (myelodysplastic syndrome, severe aplastic anemia)
• Primary immune system disorders (hemophagocytic lymphohistiocytosis or “HLH”)
• Inherited metabolic diseases (called inborn errors in metabolism, or “IEM”)

Treating Nonmalignant Bone Marrow and Immune System Disorders

In addition to childhood cancers and blood disorders, Nemours treats nonmalignant bone marrow disorders, immune system deficiencies and some metabolic disorders with allogeneic blood and bone marrow transplantation.

Some of the nonmalignant conditions we treat include:

• Inherited bone marrow failure syndromes (Fanconi anemia, Diamond Blackfan anemia)
• Primary (inherited) immune disorders (severe combined immunodeficiency, Wiskott-Aldrich syndrome, leukocyte adhesion deficiency)
• Metabolic (lysomomal) storage diseases (Hurler syndrome, metachromatic leukodystropy, Krabbe disease, or “globoid leukodystrophy”)

Pediatric blood and bone marrow transplants require very specialized care from the first visit and throughout treatment, recovery and beyond. Transplants typically take up to three weeks for planning and preparation, and six weeks of inpatient hospital care in special blood and bone marrow transplant units staffed by highly trained physicians and nurses.

About the Transplant Process

The process differs depending on the type of pediatric blood and bone marrow transplant procedure, the condition we’re treating and other factors, but you generally can expect the following:

• Planning (consultation with your team, physical examination, medical testing and donor match)
• Conditioning (includes chemotherapy and/or radiation therapy to treat the disease and make room for healthy stem cells)
• Transplantation (stem cells are given intravenously, like a blood transfusion, while your child watches TV, plays games, creates art or works with Child Life specialists)
• Engraftment (occurs when stem cells mature and begin to produce blood cells, which can take two to four weeks)
• Recovery (includes a month of inpatient hospital monitoring to anticipate or treat side effects or complications that may arise as the immune system strengthens)
• Transition (help transitioning your child and family throughout the process and from hospital care to home care)
• Follow-up (includes maintaining frequent outpatient or inpatient follow-up care during the crucial first 100-day post-transplant mark, and also longer term)

Complications of Blood and Bone Marrow Transplants in Children

Blood and bone marrow transplants significantly impact the immune system, which increases the risk for infections and other complications that can be severe and life-threatening. It can take months or years for the immune system to recover entirely, so close monitoring is crucial.

At the Nemours Center for Cancer and Blood Disorders, we have the preventive measures, medicines, innovative medical therapies and support programs in place to help your child and family manage and treat possible issues after a transplant, including:

• Side effects during transplantation (e.g., nausea, hypertension, hives, chills, fever, pain)
• Graft-versus-host disease (when the immune system attacks the transplanted stem cells causing fever, rash, diarrhea, respiratory problems, decreased organ function)
• Graft failure (occurs when the body rejects the new stem cells)
• Anemia or thrombocytopenia (due to low blood counts)
• Respiratory problems (due to infections, fluid overload or graft-versus-host disease)
• Veno-occlusive disease (or “VOD,” when blood vessels that serve the liver become damaged, causing organ swelling and damage)

CAR-T Therapy Treatment for Leukemia

Nemours Children’s also offers Pediatric CAR-T (Chimeric Antigen Receptor T-cell) therapy, a specialized immunotherapy that harnesses a patient's own T-cells, genetically modifying them to express a receptor (CAR) that targets specific cancer cells. In pediatric cases, this treatment is used to combat certain types of leukemia and lymphomas, offering a promising approach to treat and potentially cure these cancers.

Engineering a Smarter, Stronger Immune System

The immune system is your body’s security system. It fights off anything that doesn’t belong, namely, viruses, bacteria, fungi and toxins.

White blood cells are an essential part of the immune system. They form in your bone marrow (the spongy material inside bones). One type of white blood cell is called a lymphocyte. There are two types of lymphocytes:

• B-cells: B-cells are the “brains” of the operation. They find invading enemies and send in reinforcements — the T-cells — to stop them.
• T-cells: The T-cells are the “muscle.” They destroy the targets that the B-cells found.

In acute lymphoblastic leukemia (ALL), cancer affects how these lymphocytes develop so they can’t do their job right. But with CAR T-cell therapy for leukemia, doctors reprogram the healthy T-cells to kill leukemia cells.

How CAR T-Cell Treatment for Leukemia Works

CAR T-cell therapy is a revolutionary new treatment for leukemia. It is a combination of gene therapy and immunotherapy.

During leukemia CAR T-cell therapy:

1. Doctors draw your child’s blood to get their T-cells.
2. Using gene therapy, they reprogram the T-cells to make the CAR protein. The CAR protein makes it possible for them to recognize and attack only cancer cells.

The pediatric Blood and Bone Marrow Transplant Program at Nemours Children’s Hospital, Delaware is one of only a handful in the United States that performs autologous and allogeneic transplants accredited by the Foundation for Accreditation of Cellular Therapies (FACT). We’re also recognized by the National Marrow Donor Program (NMDP) as an affiliated center for matched unrelated donor transplants.

Pediatric Blood and Bone Marrow Transplant Program Highlights

• Dedicated staff of board-certified pediatric blood and bone marrow specialists
  
• Transplantation specialists and experienced transplant nurses
• Onsite biobank/stem cell laboratory for stem cell cryopreservation, storage and infusion
• Advanced treatments and clinical trial participation as part of the Children’s Oncology Group (COG) (the world's largest organization devoted exclusively to pediatric cancer research)
• Specially equipped patient rooms and unit play room for our transplant patients
• Onsite chemotherapy infusion suite and pediatric day therapy suite
• Pediatric radiation therapy services through our affiliate, Christiana Care Health System’s Helen F. Graham Cancer Center
• Active Child Life and volunteer program offering play therapy, art therapy and more during treatment
• Compassionate transplant coordinators and social workers who provide treatment education, tips to manage side effects and resources for your child and family

Your Child’s Comprehensive Blood and Bone Marrow Transplant Care Team

In addition to an experienced, compassionate transplant coordinator (a nurse who provides your child and family transition support, education and care coordination), the Delaware Valley’s pediatric blood and bone marrow transplant teams include:

• Hematologists (blood disorder specialists)
• Oncologists (cancer doctors)
• Transplant physicians (stem cell transplant specialists)
• Geneticists (specialists in genetic counseling and testing)
• Immunologists (autoimmune disorder specialists)
• Pathologists (physicians who diagnose diseases by examining body tissues, fluids and organs)
• Radiation oncologists (specialists in radiation therapy treatment)
• Interventional radiologists (physicians who perform image-guided minimally-invasive procedures)
• Behavioral health specialists (psychologists and psychiatrists)
• Registered dieticians (nutrition professionals)
• Hematology-oncology nurses (registered nurses specializing in childhood blood disorders)
• Therapy services (physical therapy, occupational therapy, Child Life)
• Teachers (who work in our on-site school)

For your child’s overall health and wellness, we also prescribe physical medicine and rehabilitation, pain management services, palliative and care (to manage physical symptoms) and integrative medicine therapies (massage, hydrotherapy, biofeedback, yoga, meditation and more).

Support Services for Your Family

Nemours Children's provides an array of support services to help your child and family throughout the entire journey. Our supportive care begins on the very first day we meet, and continues beyond treatment.

Our services include: 

• Patient and family education
• Mental health counseling
• Support groups
• Survivorship programs
• Transition of care

We Make You Feel Right at Home

At Nemours Children’s Hospital, Delaware, we provide an environment where kids and families can find fun and normalcy during long-term hospital stays. Some of our amenities for blood and bone marrow patients and families include:

• Nearby family housing at the Ronald McDonald House of Delaware  
• Private patient rooms with ample outdoor lighting and a private bathroom
• Healing gardens
• Kid-friendly indoor play areas designated for children who received a transplant
• Playground for children recovering from a transplant
• Onsite learning and advocacy during the transition back to school
• Exercise equipment for children and their families on the transplant unit
• Weekly town-hall-style meetings to address concerns and answer questions that may come up from living in a communal or hospital-based environment
• Family advisory council of parents who can help you along the way

Learn More About All of Our Nemours Center for Cancer and Blood Disorders Facilities and Services in Delaware