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.

Before the transplant:

• The bone marrow transplant team will give your child an in-depth evaluation. The team will talk with you about the risks and benefits of transplant and other treatment choices.
• Your child will have physical exams and tests. The tests look at your child's blood. They check how well organs like the heart, kidney, liver and lungs function.
• Your child may need to go to the transplant center up to 10 days before the transplant. A thin, flexible tube (catheter) called a central venous line is surgically placed in a vein in your child’s chest. Blood products and medicines will be given through the catheter.

The days before the transplant are counted as minus days. The day of transplant is considered day 0. Engraftment and recovery following the transplant are counted as plus days. For example, a child may enter the hospital on day -8 to prepare for the BMT. Days +1, +2, and so on, will follow the transplant. There are possible events, complications and risks linked to each day before, during and after transplant. The days are numbered to help you and your child know where you are in terms of risks and discharge planning.

Once suitable stem cells are found, your child will get high doses of chemotherapy or radiation (sometimes both) to destroy existing bone marrow. This gives the new bone marrow cells room to grow. This may be called ablative or myeloablative therapy. It stops new blood cells from being made. The bone marrow becomes empty. An empty marrow is needed to make room for the new stem cells to grow and create a new system to make new blood cells.

Next, stem cells are given to your child through an IV in a large vein, often in the chest. This is called a central venous catheter. Getting the stem cells is like having a blood transfusion. The stem cells find their way into the bone marrow. They start growing and making new, healthy blood cells.

During infusion of the bone marrow, your child may have:

• Pain
• Chills
• Fever
• Hives
• Chest pain

After infusion, your child may:

• Be at risk for infection
• Have some bleeding
• Have nausea, vomiting, diarrhea, mouth sores and extreme weakness
• Have some emotional distress

Your child will get supportive care to prevent and treat infections, side effects and complications.

During this time, your child may:

• Spend several weeks in the hospital
• Be confined to a very clean environment to reduce the chance of infection
• Take multiple antibiotics and other medicines
• Need blood transfusions
• Be given medicine to prevent graft-versus-host disease
• Have daily blood tests

Engraftment of the stem cells happens when the donated cells make their way to the marrow and start making new blood cells. Engraftment usually happens between day +15 and +30. This depends on the type of transplant and the disease being treated. Blood tests will be done often to count blood cells after transplant. Platelets are usually the last type of blood cells to recover.

In some cases, engraftment can be delayed. This can be due to infection, medicines, low donated stem cell count or graft failure. The new bone marrow may start making cells in the first 30 days after the transplant. But it may take months or years for your child’s immune system to fully recover.

The transplant team will tell you when your child is ready to go home. This depends on many things, such as:

• Extent of engraftment
• Complications
• Your child's overall health
• How far you live from the facility

Before you go home, you'll be instructed on ways to reduce your child’s risk of infection. These include:

• Special air filtered rooms
• Diet restrictions
• Limiting visitors
• Strict hygiene
• Frequent bed linen changes

Your child will then need to see the transplant team often to:

• See how well the treatment is working
• Look for signs of problems such as infection
• Treat side effects, infection and other problems

Long-term success varies from child to child. Emotional support for your child and family is vital during all phases of the transplant. Your transplant team will help you manage the stress of this process and offer support when your child returns home. They'll teach you about medicines, how to care for your child and when and how to contact them with questions.

Your child’s transplant team will also talk with you about your child’s prognosis, follow-up care and future treatments.