Pediatric Bone Marrow Transplant

Your child is their own donor. Stem cells can be collected from your child in two different ways:

• Peripheral blood stem cells (PBSCs). Stem cells are taken from your child by apheresis. Apheresis is a process of collecting stem cells that float in the blood (peripheral blood stem cells). The cells are then given back to your child after intensive treatment.
• Bone marrow harvest. Stem cells are collected from your child by a needle placed into the soft center of the bone. Most sites used for bone marrow harvesting are in the hip bones because they have a larger number of stem cells. 

The donor is another person who shares the same or similar genetic type as your child. This is often a brother or sister. Finding a matching donor can be a lengthy process. In some cases, a parent may be a donor. Or the donor may be a matched unrelated donor (MUD) found on a bone marrow registry. Stem cells are taken from the donor either by apheresis or bone marrow harvest.

Stem cells are taken from an umbilical cord right after a baby’s birth.

These stem cells grow into mature blood cells quicker and more effectively than stem cells from the bone marrow of another child or adult. The stem cells are tested, typed, counted, and frozen until they are needed for a transplant.

Your child’s transplant is managed by a team of specialists who care for children having BMTs. This team includes:

• Doctors. Healthcare providers who specialize in oncology, hematology, immunology and bone marrow transplantation.
• Transplant nurse coordinator. This professional organizes all aspects of care given to your child before and after the transplant. They will give you information and arrange testing and follow-up care.
• Social worker. This professional will help your family deal with issues, such as lodging and transportation, finances and legal issues. They can also help arrange different ways to help your child continue with schooling.
• Psychologist (PhD) or licensed clinical social worker (LCSW). These professionals can provide counseling and emotional support to you and your family.
• Dietitian. A provider who will help your child with nutritional needs before and after the transplant.
• Physical therapist. A provider who will help your child regain strength after the transplant.
• Pastoral care. This includes people trained to give spiritual care and support.

Other team members who may evaluate your child before and after BMT include:

• Pharmacist
• Respiratory therapist
• Lab technicians
• Infectious diseases specialist
• Dermatologist
• Gastroenterologist
• Psychologist
• Child life specialist

The goal of BMT is to cure many diseases and certain types of cancer. It can be used to:

• Replace diseased bone marrow with healthy bone marrow. This is done for conditions, such as leukemia, aplastic anemia, and sickle cell anemia.
• Replace bone marrow after high doses of chemotherapy or radiation are given to treat cancer. This process is often called rescue instead of transplant. This is done for diseases like lymphoma and neuroblastoma.
• Replace bone marrow with healthy bone marrow to prevent more damage from a genetic disease. This is done for diseases such as Hurler syndrome and adrenoleukodystrophy disorder.

Diseases that can be treated with BMT include:

• Cancers, such as leukemia, lymphoma, neuroblastoma, rhabdomyosarcoma, brain tumors or kidney cancer
• Aplastic anemia
• Immune deficiency, such as severe combined immunodeficiency disorder or Wiskott-Aldrich syndrome
• Genetic diseases, such as sickle cell disease, thalassemia, Diamond Blackfan anemia, Hurler syndrome or adrenoleukodystrophy disorder

A bone marrow transplant has risks, some of which are life-threatening. Your child’s healthcare provider and BMT team will discuss risks and benefits with you before the procedure.

Risks and complications may vary depending on:

• The type of bone marrow transplant
• The type of disease the transplant is treating
• What medicines are used before the transplant
• Your child’s age and overall health
• The differences of the tissue match between donor and recipient

Possible complications include:

• Infections. Infections are likely with severe bone marrow suppression. This is when not enough blood cells are made in the bone marrow. Medicines are often given to prevent and treat infection.
• Low levels of platelets (thrombocytopenia) and red blood cells (anemia). These can be dangerous and even life-threatening. Most children will need multiple blood product transfusions.
• Pain. Pain caused by mouth sores and gastrointestinal (GI) irritation is common from chemotherapy and radiation. Pain medicine and good mouth care can help.
• Diarrhea, nausea and vomiting.  These may happen with chemotherapy, radiation and GI irritation. Calories and protein may be given through an IV (intravenous) line until your child can eat again and the diarrhea has stopped.
• Fluid overload. This most often occurs when the kidneys can’t keep up with the large amount of IV fluids being given. Your child will be watched closely for fluid overload. They will be weighed often, have blood tests and their urine will be measured often. Medicines may be given to help kidney function and get rid of excess fluid.
• Breathing problems. Serious lung problems can be caused by infection, airway inflammation, fluid overload, graft-versus-host disease and bleeding. Your child may need supplemental oxygen to breathe better until problems are treated.
• Organ damage. Short-term (temporary) or long-lasting (permanent) liver and heart damage may be caused by infection, graft-versus-host disease, chemotherapy and radiation or fluid overload. Your child's blood and vital signs will be checked carefully to look for organ damage.
• Graft failure. The transplanted cells (graft) may fail to grow in the marrow. This may happen from infection, the disease coming back or a stem cell count in the donated marrow that was too low. Graft failure may be treated with another bone marrow transplant.
• Graft-versus-host disease (GVHD). GVHD can be serious and life-threatening. It happens when the donor’s immune cells attack the recipient's healthy cells. Your child will be watched closely for GVHD. Signs can include diarrhea, fever, rash, skin changes, stomach pain, breathing problems and decreased liver function. Your child will be given medicines before the transplant to reduce this risk.