Stem cell transplant (bone marrow transplant)

At Children’s Hospital Boston, we know how difficult it can be to go through a stem cell transplant, both for your child and for your whole family. That’s why our physicians are focused on family-centered care: From your first visit, you’ll work with a team of professionals who are committed to supporting all of your family’s physical and psychosocial needs.

Preparation for your child

Before your child receives the transplant, the following things happen:

• Your child’s bone marrow transplant team will complete an extensive evaluation. All other treatment options are discussed and evaluated for risk versus benefit.
   
• A suitable (tissue typed and matched) donor must be available. This can be a challenging and lengthy process. Voluntary marrow donors are registered in several national and international registries. A bone marrow search involves searching these registries for donors whose blood most closely resembles or matches your child’s blood.
   
• A complete medical history and physical examination of your child will be performed, including multiple tests to evaluate his blood and organ functions (such as the heart, kidney, liver and lungs).
   
• Your child will often come into the transplant center up to 10 days before transplant for hydration, evaluation, placement of the central venous line and other preparations. A catheter, also called a central venous line, is surgically placed in a vein in the chest area. Blood products and medications will be administered through the catheter.

How are a donor and recipient matched?

Matching involves looking at the donor and child’s HLA (human leukocyte antigen) tissue. There many HLA antigens—at least 100—but it is believed that there are a few major ones that determine whether it will be a good match. The others are considered “minor” and their effect on a successful transplant is not as well defined.

Medical research is still investigating the role all antigens play in the process of a stem cell transplant. The more antigens match, the better the transplant’s chance of success.

Once a potential donor is notified that she may be a match for your child, the donor will undergo additional tests related to their health, exposure to viruses, and complete genetic analysis to determine the extent of the match. The donor will be given instructions on how a bone marrow donation will be made.

Relatives may or may not elect to have their type registered for use with other recipients.

How are the stem cells collected?

Stem cells can either be collected from the bone marrow or the blood:

1. Peripheral blood stem cells (PBSCs)

Peripheral blood stem cells are collected by apheresis, a process in which blood is taken from a vein using a needle and circulated though a special cell separation machine. The machine removes the stem cells, and returns the remaining blood and plasma back to the donor through another needle inserted into the opposite arm.

• Several sessions may be required to collect enough stem cells to assure a chance of successful transplant for your child.
   
• The donor may take medication for about one week before the procedure that will make the bone marrow produce increased numbers of new stem cells that are released from the marrow into the blood.

2. Bone marrow harvest

Bone marrow harvesting involves collecting stem cells with a needle placed into the soft center of the bone, the marrow.

• Bone marrow is most often collected from the hipbones and the sternum. This procedure takes place in the operating room.
• The donor will be anesthetized during the procedure.
• In recovery, the donor may experience some pain in the areas where the needle was inserted.

3. Umbilical cord blood collection

Cord blood is collected after delivery, when the placenta and umbilical cord are separated from the infant.

• The blood that is contained in the cord is collected in a sterile container.
• The blood is then tested for type and the presence of viruses or disease, and the stem cells are counted.
• The cord blood is then stored in special freezers and is registered in a national registry for potential recipient matches.

The stem cell transplant procedure

Preparation for a bone marrow transplant varies depending on the type of transplant, the disease requiring transplant and your child's tolerance for certain medications. Here are some things to keep in mind:

• High doses of chemotherapy and/or radiation are usually part of preparation. This is necessary to make room in the bone marrow for the new cells to grow. This therapy is often called ablative, or myeloablative, because it prevents the production of cells, which essentially empties out the bone marrow. An empty marrow is needed to make room for the new stem cells to grow and establish a new production system.

• Children with bone marrow failure syndromes, such as Fanconi anemia and dyskeratosis congenita, may receive lower doses of chemotherapy or radiation in order to avoid complications. This is called reduced-intensity conditioning therapy.
   
• After chemotherapy and/or radiation, the stem cell transplant is given through a special catheter inserted into a large vein (called a central venous catheter). It is not a surgical procedure to place the marrow into the bone, and is similar to receiving a blood transfusion. The stem cells find their way into the bone marrow and begin reproducing and establishing new, healthy blood cells (engraftment).
   
• We give supportive care to your child to prevent and treat infections, side effects and complications. This includes frequent blood tests, close monitoring of vital signs, strict measurement of input and output, frequent weighing and providing a protected, sterile environment.

Counting the days

It’s helpful to understand how days are counted around your child’s transplant. The days are numbered to help the child and family understand where they are in terms of risks and discharge planning.

• The days before transplant are counted as “minus” days (e.g. -8).
• The day of transplant is considered day zero (0).
• Engraftment and recovery following the transplant are counted as plus days (e.g. +1, +2, etc.).

For example, a child may enter the hospital on day -8 to begin preparation for the transplant. The day of transplant is usually numbered as day 0. Days +1, +2 (etc.) will follow. There are specific events, complications, and risks associated with each day before, during and after transplant.

During and after the transplant

A transplant is a serious procedure. During infusion of bone marrow, your child may experience any or all of the following symptoms:

• pain
• chills
• fever
• hives
• chest pain

After infusion, your child may:

• spend several weeks in the hospital
   
• experience excessive bleeding
   
• have blood transfusions
   
• experience nausea, vomiting, diarrhea, mouth sores and extreme weakness
   
• experience temporary emotional or psychological distress
   
• be very susceptible to infection
   
• take multiple antibiotics and other medications
   
• be confined to a sterile environment
   
• be given medication to prevent graft-versus-host disease (if the transplant came from a donor). The transplanted new cells (graft) tend to attack your child's tissues (the host), even if the donor is a relative.
   
• undergo frequent laboratory testing

Your child's physical and mental health is important in the success of a transplant. We take every measure to minimize complications and promote a healthy, happy and safe environment for your child.

When does engraftment occur?

Engraftment means that the donated cells have successfully traveled to the marrow and begun making new healthy blood cells. Depending on the disease and the type of transplant, this usually occurs between day +15 and +30.

• Engraftment can be delayed because of infection, medications or low donated stem cell count.
   
• Although the new bone marrow may begin making cells in the first 30 days following transplant, it may take months or years for the immune system to fully recover.

What complications and side effects may occur following transplant?

Complications may vary, depending on:

• type of marrow transplant
• type of disease requiring transplant
• preparative regimen
• your child’s age and overall health
• the strength of the tissue match between your child and the donor

While each child may experience complications differently, some of the most common include:

• infections - Any infection can cause an extended hospital stay, prevent or delay engraftment and/or cause permanent organ damage. Antibiotics, anti-fungal medications and anti-viral medications are often given to prevent serious infection. Preventative measures for common sources of infection are also a part of transplant, including:
  • special air-filtered rooms
     
  • diet restrictions
     
  • isolation requirements
     
  • restriction of visitors
     
  • strict hygiene regimen
     
  • frequent linen changes
     
• low platelets and low red blood cells - Thrombocytopenia (low platelets) and anemia (low red blood cells) can be dangerous and even life threatening. Most children receiving a transplant will require multiple blood transfusions.
   
• pain and GI irritation - Pain related to mouth sores and gastrointestinal (GI) irritation is common. Your child may also experience diarrhea, nausea and vomiting due to chemotherapy, radiation and/or GI irritation.
   
• fluid overload - Sometimes the kidneys can’t keep up with the large amount of fluid being given in the form of medications, and nutrition and blood products. The kidneys may also be damaged from disease, infection, chemotherapy, radiation or antibiotics. During transplant and recovery, we will watch for signs and symptoms of fluid overload, which can lead to pneumonia, liver damage and high blood pressure.
   
• respiratory distress - Infection, inflammation of the airway, fluid overload, graft-versus-host disease and bleeding are all potential life-threatening complications that may occur in the lungs and pulmonary system.
   
• organ damage - Temporary or permanent damage may be caused by infection, graft-versus-host disease, high doses of chemotherapy and radiation or fluid overload. Close monitoring of your child's blood work and vital signs is an important step in detecting and minimizing any organ damage that has occurred.
   
• graft failure - This may occur as a result of infection, recurrent disease or if the stem cell count of the donated marrow wasn’t high enough. If a source is available, this may be treated with an additional marrow transplant.
   
• graft-versus-host disease (GVHD) - GVHD can be a serious and life-threatening complication of a bone marrow transplant. GVHD occurs when the donor's immune system reacts against the recipient's tissue. The new cells do not recognize the tissues and organs of the recipient's body. Your child will be monitored closely for signs and symptoms of GVHD.

When will my child be discharged?

Your child’s discharge date will depend on many factors, including:

• the success of engraftment
   
• the presence of complications
   
• your child’s overall health
   
• your distance from the facility (Occasionally, a bone marrow transplant patient will be required to stay within a certain distance or travel time from the facility to ensure safety if complications arise.)

Your child’s length of stay can vary significantly depending on these factors. On average, children stay in the hospital for about 30 to 40 days.

Frequent follow-up visits to your child's transplant team are necessary to determine the effectiveness of treatment, quickly detect any complications or recurrent disease and manage the late effects associated with this procedure. The transplant team will tell you how frequent these visits should be.

What is the long-term outlook for a stem cell transplantation?

Your child’s prognosis will greatly depend on the following:

• type of marrow transplant
• your child’s age and overall health
• type and extent of the disease being treated
• disease response to treatment
• genetics
• severity of complications
• your child's tolerance of specific medications, procedures or therapies

The number of transplants occurring for an increased number of diseases and medical developments has greatly improved the outcome for bone marrow transplant in both children and adults, but continuous follow-up care is essential. New methods to improve treatment and decrease complications and side effects are continually being discovered.

Post-transplant care

Following a successful transplant, your child will require specialized care and precautions as her immune system recovers.

Rebuilding your child's immune system

Your child needs a stem cell transplant because her bone marrow has either been damaged by disease or been suppressed by intensive chemotherapy. Since bone marrow cells produce all forms of blood cells, including infection-fighting white blood cells, your child's immune system will also be impaired.

Once the donor stem cells start working, your child's bone marrow will begin to produce healthy white blood cells. However, it will take time for these new cells to become strong enough to fight off infection. It’s important you and your child understand that rebuilding your child's immune system is a process that will take several months.

Protecting your child at home

At Children's, your child is protected from bacteria, viruses and fungi by special air filtering and infection-control precautions. When your child returns home, we will ask your family to take several precautions to provide as safe a recovery environment as possible.

Some of the steps we will ask you to take include:

• removing houseplants
• adopting stringent hand washing habits
• avoiding certain foods, like raw fish, which may lead to infection

Although we require all transplant patients to observe certain precautions against infection after transplant, some of the post-transplant restrictions vary based on the type of transplant performed. An example of this is the time period we ask a patient to wait before visiting public places and returning to school.

• In general, we ask autologous transplant patients (those who receive a transplant of their own stem cells) to avoid common areas like these, which heighten the risk of infection, for at least six months after transplant.
   
• In comparison, we generally ask allogeneic transplant patients (those who receive donor stem cells) to wait nine to 12 months.

We can’t predict exactly when it will be safe for your child to visit public places or return to school. Our first priority is protecting your child's health, and we will ask you to keep your child at home for a longer period of time if necessary.

Post-transplant vaccinations  

In addition to rebuilding itself naturally over time, your child's immune system will also require vaccinations to help produce disease-specific antibodies.

Although your child may have been vaccinated, her new immune system needs to be refreshed through another vaccination. Even if your child received routine immunizations prior to transplant, her new immune system will have no memory of those vaccinations. For this reason, your child must be re-vaccinated after a bone marrow transplant.

We recommend waiting a year after the transplantation before your child receives any vaccinations. However, each child is different, and we will adjust this recommendation if necessary. We also suggest your child receive all vaccinations from the same physician. This makes it easier to track vaccination records for things like school, sport or camp applications.

Your child can be vaccinated by Jimmy Fund Clinic staff members or by her primary care provider. In either case, we’ll provide a helpful list to track your child's post-transplant vaccinations.

Our physicians will explain more about immune system reconstitution during your new patient consultation.

Long-term follow-up

We offer several programs for your child after her stem cell transplant.

• Dana-Farber's David B. Perini, Jr. Quality of Life Clinic helps survivors of childhood cancer address the long-term effects of their therapy. The Clinic includes special programs to ease patients' transition to post-transplant life, such as the School Liaison Program.
   
• In addition, we offer a multidisciplinary clinic staffed by nutritionists, endocrinologists, physical therapists, dermatologists, psychologists and social workers for childhood cancer survivors of all ages, as well as a special follow-up clinic for stem cell transplant patients.
   
• Dana-Farber Cancer Institute's GVHD Clinic is a special program within the Jimmy Fund Clinic that addresses the needs of transplant patients with graft-versus-host disease (GVHD), a serious potential complication of stem cell transplant. At the Clinic, patients meet with multiple medical experts during one outpatient visit in order to manage the disease and its symptoms.

Read more about our pediatric support services.

Resources and support

We understand that you may have a lot of questions if your child needs to have a stem cell transplant. How will this affect my child long-term? What do we do next? We’ve tried to provide some answers to those questions in these pages, but there are also a number of resources and support services to help you and your family through this difficult time.