Bone Marrow Transplant

Nemours’ Blood and Bone Marrow Transplant (BBMT) program offers children and families like yours the hope of a potential cure for a wide range of diseases, including cancers (like leukemia, lymphoma, brain tumors, and other solid tumors), blood disorders, immune system diseases, as well as many inherited genetic diseases and bone marrow failure syndromes.

A BBMT or stem cell transplant – also called a ”hematopoietic progenitor cell (or HPC) transplant” – replaces diseased or damaged cells in children whose normal blood cells either aren’t working correctly or have been crowded out by abnormal cells. Hematopoietic (pronounced: hee-ma-toe-poy-et-ic) stem cells are immature cells that are capable of developing into healthy blood cells when transplanted.

How It Works

Hematopoietic stem cells can be found in:

• bone marrow (the spongy tissue inside bones where our bodies manufacture blood cells)
• the circulating blood in our bodies (this is known as “peripheral blood”)
• umbilical cord blood (donated by the mother after a baby’s birth)

There are two main types of stem cell transplants:

• an autologous transplant, using the child’s own blood or bone marrow
• an allogeneic transplant, using new bone marrow from another person (donor) whose tissue closely matches the patient (This might be a close relative or an anonymous donor found through an international listing of volunteers or from stored cord blood units. A syngeneic transplant is when an identical twin is the donor.)

The type of transplant needed will depend on the child’s specific medical condition and the availability of a matching donor.

After the stem cells are collected, they’re introduced (or infused) into the bloodstream through an intravenous (IV) line, much like a blood transfusion. Once in the body, they can produce healthy new blood and immune system cells.

A stem cell transplant is used to treat a wide range of conditions, such as:

• leukemia
• solid tumors (like neuroblastoma)
• bone marrow failure syndromes (like aplastic anemia)
• immunodeficiency states (like severe combined immunodeficiency disorders)
• blood disorders (like sickle cell diseases)
• non-blood-related genetic disorders (like mucopolysaccharidosis)

Your Caring Care Team – Helping You Every Step of the Way

Every child who needs a stem cell transplant will be assigned their own personal transplant coordinator, who will take care of all the details so you don’t have to. That means:

• coordinating appointments
• communicating with insurance companies and your doctors
• scheduling personnel who’ll work with you and your child during your visit
• making sure all paperwork and details have been taken care of by the time you visit us
• helping you solve any problems that may arise in regard to transplant

A social worker will also be involved with you and your family from the time you come in for your child’s consultative visit until being discharged from the BBMT service. The social worker will help you prepare for your child’s inpatient stay for the transplant. Like the transplant coordinator, the social worker can help you with a variety of resources including travel, lodging, financial support, and learning about the area. Social workers are also available to provide emotional support for you and your child, as well as family or individual counseling, if needed.

Working together to make each transplant successful, the rest of your team will include an entire family of experts, such as:

• pediatric transplant physicians (doctors specializing in the care of children getting stem cell transplants)
• radiation oncologists (doctors specially trained in radiation therapy, if needed)
• a large group of nurses
• pharmacists
• physical and occupational therapists
• laboratory staff
• dieticians
• teachers
• child life specialists
• psychologists
• clergy

To help both you and your child cope with the process and side effects of transplants, our team of expert, loving caregivers works to take care of the “whole child” and all of his or her needs. For example, our child life specialists and physical and occupational therapists will visit with your child – encouraging play and movement to make sure your child is emotionally and physically strong prior to leaving the hospital. Psychologists will look for any learning problems that may be related to treatment and address them as early as possible – offering counseling to help your child adjust to treatment, manage pain, comply with taking medications, and cope with depression and anxiety.

Our child life therapists, in particular, are a key part of your child’s transplant journey. Their goal is to help children – and their families – make a positive adjustment to the experience of illness and hospitalization. Child life specialists (also sometimes called “child life therapists”) are child development professionals specially trained to work with kids who are sick or disabled and are receiving treatment in a hospital setting. They provide children with the opportunity to participate in play and recreational activities that support their normal development.

And, because we recognize the importance of the parents’ role in the care and healing of the child, we strive to involve you every step of the way. A transplant is a team effort, with the patient and their primary caregiver being the most important members of the team.

What to Expect - Before, During, and After

Before

All children referred to our program will meet with one of our transplant physicians, the transplant coordinator, and other members of our transplant team to find out if a transplant is the best possible treatment for their condition. Each treatment plan is personalized, based on each child’s individual medical history and overall condition.

The transplant procedure begins with either taking stem cells from the child’s own blood or bone marrow or by finding a suitable, healthy matched donor to provide cells.

For autologous transplant, if the cells will be taken from the blood that circulates through the body (this is called “peripheral blood”) the collection takes a few hours and may be repeated over 2-3 days. The child or the donor is able to watch TV, read, participate in bedside art, or play games during the collection. After each collection is completed, the child is free to explore the hospital floor and playroom. And a special  IV line is kept in place so that a new needle stick isn’t needed each day.

If the transplant will come from bone marrow, the child or the donor will need to have it drawn with a needle from the hip bone on the back of the body. This is done under sedation or anesthesia and usually takes less than an hour. The child (or donor) typically spends one night in the hospital.

During

Before a child can get the transplant, they need a treatment called “conditioning therapy.” In order to make room for new bone marrow, the body must be rid of its present bone marrow. This is accomplished through high-dose chemotherapy and sometimes, radiation therapy, too. Most of this treatment will happen in the hospital BBMT unit, but sometimes conditioning can be started on an outpatient basis before the child is admitted to the unit.

The purpose of conditioning is to treat the child’s disease. If the stem cells are coming from a donor, the treatment also prepares the child’s immune system to accept the donor’s cells. However, conditioning will destroy or damage both abnormal and normal cells. That’s why, after conditioning, the child is given healthy stem cells through the transplant – which is performed the same way as a blood transfusion, through the patient’s central line.

After

Following the transplant, the stem cells grow in the child’s bone marrow and generate healthy, disease-free blood cells and bone marrow. But it normally takes a few weeks for engrafting to take place (this is the process of new stem cells manufacturing new blood cells) so your child will need to stay in the hospital until then. The good news: Nemours’ average length of stay is generally less than the national average, which means you can have your child back home again sooner rather than later.

While the marrow is settling in, though, red blood cells and platelet counts may run low, which can put kids at risk for infections, bleeding, and anemia. So they may need red blood cell and/or platelet transfusions until their marrow can produce these on its own.

We take the time to educate all of our patients and their families about post-transplant care. One of our top priorities is ensuring that you and your child feel confident and able to manage the rest of the healing process in the comfort of your own home.

Once transplant patients are discharged from the hospital, they’ll have frequent visits at Nemours and/or their referring clinic to monitor their progress for months after the transplant. The visits will become further apart as the time from the transplant increases, but we prefer that our patients have follow-up appointments for years after the transplant and even into adulthood.