Bone Marrow Transplant for Children
A bone marrow transplant (BMT) is a treatment for children with certain types of cancer
or other diseases. The goal of BMT is to replace a child’s diseased bone marrow with
healthy bone marrow.
Bone marrow is the spongy material in the center of the bones that makes all types
of blood cells, including young blood cells called stem cells. In BMT, stem cells
are filtered, and then put back into the same child. Or they are put into another
sick child. These stem cells will grow and become new and healthy bone marrow. Transplanted
stem cells are called the graft.
There are different types of BMT based on how the healthy stem cells are collected.
Autologous bone marrow transplant
Your child is their own donor. Stem cells can be collected from your child in 2
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.
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
Allogeneic bone marrow transplant
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.
Umbilical cord blood transplant
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:
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.
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
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.
A provider who will help your child regain strength after the
This includes people trained to give spiritual care and
Other team members who may evaluate your child before and after BMT include:
- Respiratory therapist
- Lab technicians
- Infectious disease specialist
- Child life specialist
The goal of BMT is to cure many diseases and certain types of cancer. It can be used
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
Diseases that can be treated with BMT include:
such as leukemia, lymphoma, neuroblastoma, rhabdomyosarcoma, brain tumors, or kidney
- Aplastic anemia
deficiency, such as severe combined immunodeficiency disorder or Wiskott-Aldrich
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
Risks and complications may vary depending on:
- The type of BMT
- 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
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.
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.
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
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
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:
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
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 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.
During the Test
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
needed to make room for the new stem cells to grow and create a new system to make
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
transfusion. The stem cells find their way into the bone marrow. They start growing
making new, healthy blood cells.
During infusion of the bone marrow, your child may have:
- Chest pain
After the Test
After infusion, your child may:
- Be at
risk for infection
nausea, vomiting, diarrhea, mouth sores, and extreme weakness
some emotional distress
child will get supportive care to prevent and treat infections, side effects, and
physical and emotional complications.
During this time, your child may:
several weeks in the hospital
confined to a very clean environment to reduce the chance of infection
multiple antibiotics and other medicines
- Need blood transfusions
- Be given
medicine to prevent GVHD
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
type of blood cells to recover.
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
the first 30 days after the transplant. But it may take months or years for your child’s
immune system to fully recover.
transplant team will tell you when your child is ready to go home. This depends on
things, such as:
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:
air filtered rooms
bed linen changes
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
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
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
Before you agree to the test or the procedure for your child make sure you know:
- The name of the test or procedure
- The reason your child is having the test or procedure
- What results to expect and what they mean
- The risks and benefits of the test or procedure
- When and where your child is to have the test or procedure
- Who will do the procedure and what that person’s qualifications are
would happen if your child didn't have the test or procedure
- Any alternative tests or procedures to think about
and how you'll get the results
- Who to call after the test or procedure if you have questions or your child has problems
much you'll have to pay for the test or procedure