BONE MARROW TRANSPLANT FOR CHILDREN: THE TREATMENT PLAN FOR BONE MARROW CANCER AND OTHER DISEASES

Bone marrow transplant is the treatment plan of choice if your child has cancer or an immune disease that affects the bone marrow. Indeed, words like “leukemia” and “non-Hodgkin lymphoma” can be painfully linked to children with cancer.

As a responsible parent, you should be well informed about the therapeutic options available, with their benefits and risks included.

So, the first step is to fully understand the disease and the stem cell transplant process.

WHAT IS THE BONE MARROW?

The bone marrow is the spongy tissue found inside the bones. It specializes in producing special cells called hematopoietic stem cells (blood stem cells) that can turn into different types of blood cells.

Blood stem cells can also be found in the blood that is moving throughout your body. But its origin is in the bone marrow.

Stem cells are unique cells that can make copies of themselves and also change into different types of cells that your body needs. The rest of our body cells are differentiated and can only make copies of themselves.

However, blood stem cells are unspecialized and can turn into red blood cells, white blood cells, and platelets.

These blood cells are essential, and each one has a different role:

  • Red blood cellsare responsible for transporting oxygen throughout the body.
  • White blood cellsare the core of the immune system.
  • Plateletsare the blood cells that form clots to stop bleeding.

WHAT IS A BONE MARROW TRANSPLANT?

A bone marrow transplant also called stem cell transplant, replaces diseased blood cells with healthy ones within the bone marrow.

The replacement cells can come from your child’s own body or a matching donor (parent, sibling, or unrelated volunteer).

Written by Dr Osama Bahsas Zaky
Updated on September 9, 2021

What Is the Bone Marrow?

What Is a Bone Marrow Transplant?

Why May Your Child Need a Transplant?

The Goals of a Transplant.

Different Types of Stem Cell Transplants.

How Are a Donor and Recipient Matched for ALLO Transplants?

Child Preparation Before a Transplant.

How Are the Stem Cells Collected?

The Procedure.

After a Stem Cell Transplant.

Complications and Risks.

How to Know If the Transplant Was Successful.

Bone Marrow Transplant Cost.

Why May Your Child Need a Bone Marrow Transplant?

Your child may need a stem cell transplant to treat conditions in which the bone marrow is diseased and can no longer produce healthy blood cells.

A transplant can be used to treat certain types of bone marrow cancer, such as:

  • Leukemia is the more common bone marrow cancer in children and teens. The good news is that almost 90% of children with acute lymphocytic leukemia can be cured after bone marrow transplants.
  • Myeloma is bone marrow cancer that affects plasma cells. Plasma cells are blood cells that produce antibodies in your body to fight infections. Myeloma cells interfere with the normal production of antibodies, leaving the body’s immune system susceptible and weakened.
  • Lymphoma is cancer that begins in infection-fighting blood cells called lymphocytes. It usually appears in lymph nodes, but it can also affect the bone marrow. The most common in children is non-Hodgkin lymphoma.

There are other blood and immune system diseases that affect the bone marrow and can be successfully treated with a stem cell transplant, such as:

  • Aplastic anemia
  • Immune deficiency such as Wiskott-Aldrich syndrome or severe combined immunodeficiency disorder
  • Genetic diseases such as thalassemia, sickle cell disease, Hurler syndrome, Diamond Blackfan anemia, or adrenoleukodystrophy disorder.

What are the goals of a bone marrow transplant?

The main goal is to cure many immune system diseases and types of cancer.

Other goals:

  • Replace diseased bone marrow with healthy stem cells, or if a disease has destroyed bone marrow. This is the case of conditions such as leukemia, sickle cell anemia, and aplastic anemia.
  • Replace bone marrow after high doses of chemotherapy or radiation needed to cure cancer, like non-Hodgkin lymphoma and neuroblastoma. In this case, the child’s bone marrow stem cells will be damaged or permanently destroyed by the treatment. This procedure is called rescue instead of transplant.
  • Replace damaged or diseased marrow with new stem cells that can effectively help kill the recipient’s cancer cells directly.
  • Replace the existing bone marrow to prevent more damage from a genetic disease. This is done for conditions such as Hurler syndrome and adrenoleukodystrophy disorder.

In any case, a stem cell transplant will only be carried out if the other therapeutic options have not helped, and the benefits of the transplant outweigh the potential risks.

What Are the Different Types of Bone Marrow Transplants?

Depending on the stem cells donor, there are different modalities with precise indications and benefits.

Autologous transplant

Stem cells for an autologous transplant are extracted from your own child’s body.

Sometimes, cancer is treated with high-dose chemotherapy or radiation therapy. This type of treatment can permanently damage stem cells. Hence, doctors remove your child’s stem cells from the blood or bone marrow before the cancer treatment begins.

Then, after your child completes the protocol, the stem cells are returned to his/her body, restoring the immune system. This procedure is also called an AUTO transplant or stem cell rescue.

Allogenic transplant 

Stem cells for an allogenic transplant come from another person, called a donor. This is also called an ALLO transplant.

The donor’s stem cells are injected into the patient after completing chemotherapy or radiation therapy.

The benefit of an ALLO transplant is that your child may have a “graft-versus-cancer cell effect.” In this case, the new stem cells target and destroy the recipient’s cancer cells that are still in the body. This is the primary mode ALLO transplants work to treat cancer.

Umbilical cord blood transplant.

In this type of allogeneic transplant, stem cells from umbilical cord blood are used. The umbilical cord connects the fetus and mother before birth. After the baby is born, the umbilical cord is cut, and mothers can donate it. Then, the blood from these cords is frozen and stored in a cord blood bank and can later be used.

How Are a Donor and Recipient Matched for ALLO Transplants?

Finding a “donor match” is an essential step for an ALLO transplant.

A match is a healthy donor whose white blood proteins, called human leukocyte antigens (HLA), closely match your child’s. This unique genetic marker must be identical or very similar to that of the child receiving the bone marrow transplant.

There are nearly 100 HLA antigens. However, there are a few key antigens that determine whether or not a donor and recipient match.

This process is also called HLA typing. The best chance of getting a perfect match is from a sister, a brother, or sometimes a close family member like the parents.

However, cells from a parent or sibling are not always a perfect match for a child’s HLA type, but they are 50% identical. Examples of this transplant are parent-child transplant and haplotype mismatched transplant.

Also, another family member or an unrelated volunteer can be a match too. In this last case, a volunteer registry of unrelated donors is used.

Bone marrow transplant procedure - Hematopoietic stem cell transplantation

How Can I Prepare My Child Before a Bone Marrow Transplant?

An extensive evaluation and several pre-transplant tests must be completed before undergoing the transplant process.

The main goal is to assess the child’s general health to ensure that he/she is physically prepared for the procedure.

Also, the decision for a child to have a bone marrow transplant will ultimately depend on many factors, including:

  • The child’s age, health status, and medical history.
  • Severity of the disease.
  • Donor’s availability.
  • Expectations for the course of the disease and the transplant
  • The parent’s opinion

In general, after the decision is made, the process includes collecting the donor stem cells, the patient receiving a protocol to prepare their body for the transplant, the actual transplant, and the recovery process.

To aid the procedure, approximately ten days before the stem cell transplant, a surgeon or radiologist will implant a long tube (intravenous catheter) into a large vein in your child’s chest or neck.

The catheter, also called a central line, must remain in place for the duration of the treatment. The transplant team will use the central line to infuse the new stem cells, blood products, and special medications.

How are the stem cells collected for a bone marrow transplant?

There are three ways to collect the stem cells.

Apheresis 

The child or the donor will receive daily injections of a medication called growth factor to increase stem cell production. Then, these stem cells move into the circulating blood so that they can be collected.

During this process, blood is drawn from an arm vein and circulated through a special machine.

The machine separates the blood into its different components, including stem cells. The stem cells are collected and frozen for future use in the transplant.

Then, the remaining blood is put back into the child’s or donor’s body through another needle inserted into the opposite arm.

Cord Blood Transplant

The stem cells are collected from the blood of the umbilical cords (cord blood transplant).

Harvesting 

In this case, the stem cells are coming directly from the bone marrow. The child or the donor is taken to a hospital’s operating room (under sedation) to directly collect the stem cells with a needle placed into the soft center of the bone, aka the bone marrow. The most common harvest sites are the hip bones and sternum.

How is the bone marrow transplant procedure?

Step 1: Pre-transplant or conditioning treatment

This step takes 5 to 10 days. It is also called ablative or myeloablative therapy.

Your child will get a high round of chemotherapy or radiation therapy. This will damage his/her immune system so that the body can accept the transplant without attacking the stem cells.

Also, this step allows preparing the child’s bone marrow for the new stem cells, giving them room to grow. It stops new blood cells from being produced so that the bone marrow becomes empty.

Step 2: Getting the new stem cells in 

This step is transplant day.

It takes nearly 30 minutes for each dose of stem cells to be infused. Getting the stem cells infusion is like having a blood transfusion. Your child may need more than one infusion.

The new stem cells don’t need to be injected directly into the bones, though.

The health care team injects the stem cells into the child’s bloodstream using the central line. Then, the transplanted stem cells find their way to the marrow, where they begin producing new, healthy blood cells.

Over time, the donor’s or own child’s previously harvested stem cells replace the cells in the bone marrow. This process is also called engraftment.

During infusion of bone marrow, the child may experience some side effects:

  • Fever
  • Hives
  • Pain
  • Chills
  • Chest pain

What Will Happen to My Child After a Bone Marrow Transplant?

The procedure can be an intensive and challenging experience. Your child will be hospitalized for a month or more.

Engraftment of the stem cells happens when the donated cells find their way to the bone marrow and start producing new blood cells. Depending on the transplant and the disease, engraftment usually happens around 15 days or a month after the transplant.

Recovery has many stages, beginning with intensive medical monitoring after the transplant day. Then, your child will slowly transition to a schedule of regular medical check-ups in the coming months and years.

Since the immune system will take some time to work again, your child will be at risk of getting an infection right after the procedure.

Hence, the doctors will administer antibiotics and immunosuppressive medications to reduce the child’s immune system reaction against the new stem cells.

Things that you should expect after your child’s bone marrow transplant:

  • Several weeks in the hospital.
  • Possible infections.
  • Excessive bleeding or bruising.
  • Periodic blood transfusions until the bone marrow begins producing enough of those cells on its own.
  • The child’s confinement in a clean environment.
  • Multiple antibiotics and other intravenous medications.
  • Continual laboratory testing to see how the body and immune system are responding to the donor cells.
  • Nausea, vomiting, mouth sores, diarrhea, and extreme fatigue or weakness.
  • Temporary mental confusion.
  • Emotional or psychological distress.

What are complications and risks of a bone marrow transplant?

It’s important that, as a parent, you’re aware of both the risks and possible benefits before treatment begins. Always talk with the transplant team and treating physician about what to expect before, during, and after the procedure.

One of the most feared complications is graft-versus-host disease (GVHD). This can be a serious and life-threatening complication of a stem cell transplant.

GVHD occurs when the child’s immune system reacts against the recipient’s tissue in allogeneic transplants. Then, the transplanted cells start to attack the other cells in the body.

GVHD risk is greater if the stem cells come from an unrelated donor, but it can happen to any patient who gets a bone marrow transplant from a donor. Acute GVHD usually happens earlier, during the first months after the transplant. It typically affects the skin, digestive tract, or liver. Chronic GVHD typically develops later and can affect many organs.

How to Know If the Bone Marrow Transplant Was Successful?

To starters, your child’s blood counts should be back to safe levels after a few weeks. At first, the transplant makes these numbers very low for 1 to 2 weeks.

This is one way to know if the transplant was a success. Also, bone marrow cancer can be cured.

A cure may be possible for certain bone marrow cancers, such as leukemia and lymphoma.

For other diseases, remission of the cancer is the best possible outcome.

Remission is having reduced signs or symptoms of cancer.

How much does bone marrow transplant cost?

Sant Joan de Déu Children’s Hospital (Barcelona, Spain) is a world reference center for bone marrow transplant with a great team of qualified specialists. The hospital has a high success rate for bone marrow transplants, so you can rest assured that your child will get the best care.

The price can range from 180,000 € to 260,000 € depending on each patient.

If you would like to receive a detailed quote, please fill in the contact form and send the complete medical file. We will transfer this data to the medical team so that we can offer you the solution that best suits your needs.

We have qualified staff who will advise you in your language and accompany you throughout the hospital process.

BONE MARROW TRANSPLANT SPECIALISTS AT SANT JOAN DE DÉU CHILDREN’S HOSPITAL (BARCELONA, SPAIN)

Doctor Izaskun Elorza Álvarez

Doctor Izaskun Elorza Álvarez

Hematopoietic stem cell transplant specialist
Doctor Isabel Badell Serra

Doctor Isabel Badell Serra

Specialist in acute leukemia and Fanconi anemia.
Doctor Susana Rives Sola

Doctor Susana Rives Sola

Specialist in pediatric acute lymphoblastic leukemia. Immunotherapy in acute leukemia
Doctor Albert Català Temprano

Doctor Albert Català Temprano

Specialist in oncological hematology and bone marrow failure
Doctor Rubén Berrueco Moreno

Doctor Rubén Berrueco Moreno

Specialist in non-oncological hematology, in particular bleeding disorders and hemostasis in children.
Doctor Júlia Marsal Ricomà

Doctor Júlia Marsal Ricomà

Pediatric hematologist.

Bibliography – More recent publications:

Allogeneic stem cell transplantation in the era of novel therapies for acute lymphoblastic leukaemia. Barba P, Elorza I.

Reduced-intensity conditioning haematopoietic stem cell transplantation in genetic diseases: Experience of the Spanish Working Group for Bone Marrow Transplantation in Children. Badell I.

Survival analysis of hematopoietic stem cell transplantation in children with primary immunodeficiency in Spain. Badell I, González M, Martínez AM, Sánchez de Toledo J, Olivé MT, González ME, Elorza I, Díaz de Heredia C.

Results of hematopoietic stem cell transplantation in hemoglobinopathies: thalassemia major and sickle cell disease. Elorza I, Olivé T, Dapena JL, Llort A, Sánchez de Toledo J, Díaz de Heredia C.

Subsequent malignancies after long-term follow-up of pediatric hematopoietic stem cell transplantation. Sisinni L, Gich I, Torrent M, Badell I.

Hematopoietic stem cell transplantation: An experience of the Spanish Working Group for Bone Marrow Transplantation in Children Badell I.

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    Sant Joan de Deu Children’s Hospital. Barcelona, Spain. Pediatric Cancer Center Barcelona

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