What is a bone marrow transplant?

Bone marrow transplant (BMT) is a type of therapy for people with certain types of cancer or other diseases. It is a type of treatment that replaces your unhealthy bone marrow with healthy cells. A BMT is done by taking cells that are made in the bone marrow called stem cells. These stem cells are filtered. They're then given back to the donor (autologous transplant) or to another person (allogeneic transplant). The goal of BMT is to put healthy stem cells into a person after their own unhealthy bone marrow has been treated to kill the abnormal cells.

BMT has been used since 1968 to treat diseases, such as:

• Leukemia

• Lymphoma

• Multiple myeloma

• Myelodysplatic syndromes

• Myeloproliferative neoplasms

• Aplastic anemia

• Immune deficiency disorders

Bone marrow is the soft, spongy tissue inside bones. It's where most of the body's blood cells develop and are stored. The cells that make other blood cells are called stem cells. It's the stem cells that are needed in BMT. The most basic of the stem cells is called the pluripotent stem cell. This is different than other blood cells in these ways:

• Renewal. It's able to reproduce another cell identical to itself.

• Differentiation. It's able to make one or more types of more mature cells.

Stem cells can either be collected from the circulating cells in the blood (the peripheral system) or from the bone marrow of a donor:

• Peripheral blood stem cells. Peripheral blood stem cells are collected by apheresis. This is the most common source of stem cells for transplantation. This is a process in which the donor is connected to a special cell separation machine by needles inserted into arm veins. Blood is taken from one vein and then circulated though the machine. This 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 needed to collect enough stem cells. This helps give a better chance that the transplanted cells will start to grow in the bone marrow and make healthy blood cells (called engraftment) in the recipient. A medicine may be given to the donor for about 1 week before apheresis. This medicine will cause the bone marrow to make more new stem cells. These new stem cells will be released from the marrow and into the blood system (stem cell mobilization). They can then be collected from the blood during apheresis.

• Bone marrow harvest. This is done by collecting stem cells with a needle placed into the donor's bone marrow. Most sites used for bone marrow harvesting are in the hip bones and the sternum. The procedure takes place in the operating room. The donor will be given medicine (general anesthesia) during the harvest. This puts them into a deep sleep so they won't feel the needle. In recovery, the donor may have some pain in the areas where the needle was inserted.

Why might I need a bone marrow transplant?

The goal of a bone marrow transplant is to cure many diseases and types of cancer. In some cases, the doses of chemotherapy (chemo) or radiation needed to cure a cancer are so high that a person's bone marrow stem cells will be permanently damaged or destroyed by the treatment. Then a bone marrow transplant may be needed. Bone marrow transplants may also be needed if the bone marrow has been destroyed by a disease.

A bone marrow transplant can be used to:

• Replace diseased bone marrow with healthy bone marrow for conditions, such as leukemia, aplastic anemia, and sickle cell anemia.

• Help build a new immune system that will fight leukemia or other cancers not killed by chemo or radiation.

• Replace the bone marrow and restore its normal function after high doses of chemo or radiation are given to treat cancer. This is often called rescue .

• Replace bone marrow with healthy bone marrow to prevent more damage from a genetic disease, such as Hurler's syndrome or adrenoleukodystrophy.

Talk with your healthcare provider and specialists in bone marrow transplants about risks and benefits before the procedure.

What are the risks of a bone marrow transplant?

Risks may vary, depending on:

• The type of transplant

• The type of disease that leads to transplant

• The way prep is done for the transplant

• Age and overall health of the recipient

• How well tissues match between donor and recipient

• Any severe complications

Possible complications that may happen with a bone marrow transplant include:

• Infections. Because white blood cells are made in the bone marrow, recent BMT patients are at high risk of infection. Infections are more likely in a recipient the lower the white cell count drops and the longer it stays low. Bacterial infections are the most common. Viral and fungal infections can be life-threatening. Any infection can cause an extended hospital stay. They can prevent or delay engraftment. They can also cause permanent organ damage. Antibiotics, antifungal medicines, and antiviral medicines are often given to try to prevent serious infection.

• Bleeding due to low platelet and low red blood cell counts. Thrombocytopenia (low platelets) and anemia (low red blood cells) can be dangerous and even life-threatening. These can happen when bone marrow isn't making new cells yet. Low platelets can cause dangerous bleeding in the lungs, gastrointestinal (GI) tract, and brain.

• Pain. Pain related to mouth sores and GI irritation is common. High doses of chemo and radiation can cause severe mucositis. This is inflammation and ulceration of the mucosa. This includes the mouth and GI tract.

• Fluid overload. This is a complication that can lead to pneumonia, liver damage, and high blood pressure. The main reason for fluid overload is because the kidneys can't keep up with the large amount of fluid being given. The fluid is given in the form of IV (intravenous) hydration, medicines, nutrition, and blood products. The kidneys may also be damaged. This can happen from disease, infection, chemo, radiation, or antibiotics.

• Graft-versus-host disease (GVHD). This 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 or transplanted immune system can attack the recipient’s whole body and all of their organs. This is because the new cells don't see the tissues and organs of the recipient's body as native cells. Over time and with the help of medicines to suppress the new immune system, it will begin to accept its new body and stop attacking it. The most common sites for GVHD are GI tract, liver, skin, and lungs. GVHD can be mild to severe. It can be acute (develops within first 100 days after allogeneic transplantation). Or it can be chronic (develops usually more than 100 days after the day of transplant).

• Respiratory distress. The respiratory system may suffer during transplant. This may be due to infection, inflammation of the airway, fluid overload, graft-versus-host disease, or bleeding. These are all possibly life-threatening problems that may happen in the lungs and pulmonary system.

• Organ damage. The liver and heart may be damaged during the transplant process. This may be temporary or permanent damage. It may be caused by infection, graft-versus-host disease, high doses of chemo and radiation, or fluid overload.

• Graft failure. In some cases, the graft (transplanted stem cells) may not take hold in the bone marrow and make new blood cells. Graft failure may happen as a result of infection or recurrent disease. It may happen if the stem cell count of the donated marrow wasn't enough to cause engraftment. It is rare in autologous stem cell transplants and more common in allogeneic transplants.

How do I get ready for a bone marrow transplant?

For the person getting the transplant, here is what happens before the procedure:

• Before the transplant, a full evaluation is done by the bone marrow transplant team. All other treatment choices are discussed and evaluated for risks and benefits.

• A complete medical history and physical exam are done. This includes tests to check the recipient’s blood and organ functions. For example, tests may assess the heart, kidney, liver, and lungs.

• A recipient will often come into the transplant center up to 10 days before transplant. They'll have hydration, evaluation, and other preparations. A flexible tube (catheter) is surgically placed in a vein in the chest area. This is called a central venous line. Blood products and medicines will be given through this line during treatment.

• For an allogeneic transplant, a suitable donor must be available. This means a donor who is tissue typed and matched. Finding a matching donor can be a difficult and lengthy process if a sibling match isn't available. Bone marrow donors are listed in several registries. A bone marrow search is done by searching these registries. The search checks for donors whose blood most closely matches the person needing the transplant.

The group of specialists involved in the care of people going through transplant is often called the transplant team. All team members work together to give the best chance for a successful transplant. The team consists of:

• Healthcare providers. These include healthcare providers who specialize in oncology, hematology, immunology, and bone marrow transplants.

• Bone marrow transplant nurse coordinator . This is a nurse who organizes all aspects of care before and after the transplant. They'll give information and manage the testing and follow-up care.

• Social workers. These are providers who will help you and your family deal with many issues that may come up. Issues may include lodging and transportation, finances, and legal issues.

• Dietitians. These are providers who will help you meet your nutritional needs before and after the transplant. They'll work closely with you and your family.

• Physical therapists. These are providers who will help you recover and become physically strong after the transplant.

• Spiritual care. Hospital chaplains can give spiritual care and support.

• Other team members. Several other team members will assess you before the transplant. And they'll give follow-up care as needed. These include:

• • Pharmacists

  • Respiratory therapists

  • Lab technicians

  • Infectious disease specialists

  • Dermatologists

  • Gastroenterologists

  • Counselors and psychologists

A detailed assessment is done by the bone marrow transplant team. The decision for you to have a bone marrow transplant will be based on many factors. These include:

• Your age, overall health, and medical history

• Extent of the disease

• Availability of a donor

• Your tolerance for specific medicines, procedures, or therapies

• Expectations for the course of the disease

• Expectations for the course of the transplant

• Your opinion or preference

The preparations for a bone marrow transplant vary depending on the type of transplant, the disease that caused the need for a transplant, and your tolerance for certain medicines. Most often, high doses of chemo or radiation are used to treat the cancer and make room in the bone marrow for the new cells to grow. This therapy is often called ablative, or myeloablative, because of the effect on the bone marrow. The bone marrow makes most of the blood cells in our body. Ablative therapy prevents this process of cell production and the marrow becomes empty. An empty marrow is needed to make room for the new stem cells to grow and establish a new blood cell production system.

What happens during a bone marrow transplant?

After the chemo or radiation is provided, the stem cell transplant is given through the central venous catheter into the bloodstream. It isn't a surgical procedure to place the stem cells into the bone. It's more like receiving a blood transfusion. The stem cells find their way into the bone marrow and begin reproducing and growing new, healthy blood cells.

The days before transplant are counted as minus days. The day of transplant is considered day zero. Engraftment and recovery after the transplant are counted as plus days. For example, a recipient may enter the hospital on day -8 for preparative regimen. The day of transplant is numbered zero. Days +1, +2, etc., will follow. There are specific events, complications, and risks associated with each day before, during, and after transplant. The days are numbered to help the recipient and family understand where they are in terms of risks and discharge planning.

During the infusion of stem cells or bone marrow, the recipient may have:

• Pain

• Chills

• Fever

• Hives

• Chest pain

You will be closely watched during and after the infusion. Side effects will be treated as needed.

What happens after a bone marrow transplant?

After the transplant, supportive care is given to prevent and treat infections, side effects of treatments, and complications. The recipient may:

• Spend several weeks in the hospital

• Be very at risk of infection

• Have a lot of bleeding

• Need blood transfusions

• Be confined to a clean environment

• Take multiple antibiotics and other medicines

• Be given medicine to prevent GVHD—if the transplant was allogeneic. The transplanted new cells (the graft) tend to attack the recipient’s tissues (the host), even if the donor is a relative.

• Have lab tests frequently

• Have nausea, vomiting, diarrhea, mouth sores, and extreme weakness

• Have short-term mental confusion and emotional distress

After leaving the hospital, the recovery process continues for several months or longer. During this time the recipient can’t return to work or many activities. The recipient must also make frequent follow-up visits to the hospital or healthcare provider's office. Know how to contact your healthcare provider after office hours, during weekends, and on holidays. Know when to get immediate care.

Engraftment of the stem cells happens when the stem cells make their way to the marrow and begin making new blood cells. Engraftment usually happens around day +15 or +30. The timing depends on the type of transplant and the disease being treated. Blood counts will be checked often during the days after transplant. This is done to assess the progress of engraftment. Platelets are often the last blood cell to recover.

Engraftment can be delayed in the body. This may happen because of infection, medicines, low donated stem cell count, or graft failure. The new bone marrow may begin making cells in the first 30 days following transplant. But it may take months or years for the whole immune system to fully recover.

The long-term outlook for a bone marrow transplant varies. It depends on the:

• Type of transplant

• Type and extent of the disease being treated

• Disease response to treatment

• Genetics

• Your age and overall health

• Your tolerance of specific medicines, procedures, or therapies

• Severity of complications

As with any procedure, in bone marrow transplant the prognosis and long-term survival can vary greatly from person to person. The number of transplants being done for an increasing number of diseases, as well as ongoing medical developments, have greatly improved the outcome for bone marrow transplant in children and adults. Ongoing follow-up care is essential for the recipient after a bone marrow transplant. New methods to improve treatment and to decrease complications and side effects of a bone marrow transplant are continually being discovered.

Next steps

Before you agree to the test or procedure make sure you know:

• The name of the test or procedure

• The reason you're having the test or procedure

• What results to expect and what they mean

• The risks and benefits of the test or procedure

• What the possible side effects or complications are

• When and where you're to have the test or procedure

• Who will do the test or procedure and what that person’s qualifications are

• What would happen if you didn't have the test or procedure

• Any alternative tests or procedures to think about

• When and how you'll get the results

• Who to call after the test or procedure if you have questions or problems

• How much you'll have to pay for the test or procedure