Bone marrow is the spongy tissue found inside bones. It contains stem cells that produce the body’s blood cells. A bone marrow transplant, also called a stem cell transplant or blood and marrow transplant, replaces cancer cells or unhealthy stem cells that can no longer produce blood cells with healthy cells.
Stem cells can be harvested for transplant from bone marrow, umbilical cord blood or from peripheral blood (the blood that circulates throughout the body). While commonly called a bone marrow transplant, the procedure can also be called a peripheral blood stem cell transplant or a cord blood transplant, depending on the source of the stem cells.
In most cases, your transplant will be allogeneic or autologous. The difference between allogeneic and autologous transplants is the source of the stem cells. The stem cells in an allogeneic transplant come from a donor (matched related to you or matched unrelated), while autologous transplants use your own stem cells. Your doctor chooses the one that is best to treat your condition.
Before your transplant, you will have a detailed medical evaluation. You may undergo chemotherapy or radiation therapy or both. When you are in remission or partial remission, your doctor may recommend a transplant. Your doctor determines your treatment plan by considering many factors and listening to what you would like to do. Some of the factors your physician takes into account to determine if transplant is an option for you are:
- What kind of disease you have, including the cellular make-up or components) of your disease
- If there is a donor available
- Your age, weight, size, general health and past medical history
- How quickly you need a transplant
- Support systems and caregiver availability
- Quality of life
- Your desire and beliefs about having a transplant
Some of your risk factors may change over time making you a good candidate to have a transplant now, but not later and vice versa. For example, your heart function may decrease after getting chemotherapy, or your lung function may get better once the disease is in remission and the masses are gone.
Preparing for a Transplant
There are some treatments you may need before a bone marrow transplant, including:
- Myeloablative Therapy — High-dose chemotherapy and/or radiation therapy is given prior to the blood or marrow transplant to wipe out your immune system and dysfunctional bone marrow.
- Non-Myeloablative Therapy — You receive standard doses (lower intensity) of chemotherapy, followed by a blood or marrow transplant. It provides you with a new immune system, while delivering fewer chemotherapy side effects.
Autologous Bone Marrow Transplants
Autologous transplants are the most common and involve receiving your own stem cells. The procedure is used for multiple myeloma, lymphoma, amyloidosis, testicular cancer and several autoimmune diseases. Based on your response to chemotherapy, your physician determines the best time to collect the stem cells. Prior to transplant, stem cells are mobilized, collected and frozen. Specifically, they are mobilized (moved from the bone marrow into the blood) through a growth factor called granulocyte colony-stimulating factor (G-CSF).
- Chemotherapy following by G-CSF, usually Neupogen: You could be mobilized off your current treatment of chemotherapy or a day of chemotherapy given in our Day Hospital. We stimulate your bone marrow to produce excess stem cells and monitor blood counts. We collect your stem cells when your blood counts recover from the chemotherapy.
- G-CSF alone: You receive a high dose of Neupogen daily for four days to stimulate your bone marrow to produce excess stem cells. On the fifth day, you should be ready for your stem cell collection. During this time, your white blood cell count might be high. Do not be alarmed by this, as the Neupogen increases the count.
- G-CSF with Mozobil: You receive a high dose of Neupogen daily for three to four days to stimulate your bone marrow to produce excess stem cells. You will receive Mozobil the night before your stem cell collection. Ideally, you should get this injection 14 hours before the start of the stem cell collection.
Upon mobilization, the stem cells are collected using an apheresis machine. You will have an intravenous needle placed in a large vein in each arm. The needle in one arm draws blood into the apheresis machine to separate the stem cells from the rest of your blood. The blood returns to your body via the needle in your other arm. If your veins cannot be used for the procedure, we will insert a catheter for the collection.
You will need an anti-clotting drug during the procedure. This drug may cause a tingling sensation in the fingers, toes, and lips. Let the nurse know if you have any of these symptoms. We will slow down the speed of the collection and give TUMS (calcium carbonate) to help resolve the symptoms. A small amount of red blood cells and platelets are removed during the collection, so we monitor your blood counts closely. If you require blood or platelet transfusions, you will get them before you leave that day.
Stem cell collection takes about four hours each day for two to three days or until we collect the number of stem cells your transplant physician requested. A nurse monitors you throughout the procedure. You will receive Neupogen injections at the end of the collection process to keep you mobilized throughout the collection. You may bring a friend or family member with you, but they will need to leave for about 5 minutes when the nurse asks you questions.
Once collected, we take your stem cells to the cell processing lab for testing, counting and cryopreservation. To cryopreserve your stem cells, we add a preservative (DMSO) and freeze your stem cells until you are ready your transplant.
Allogeneic Bone Marrow Transplants
Allogeneic transplants are used for acute and chronic leukemia, non-Hodgkin or Hodgkin lymphoma, aplastic anemia, multiple myeloma, myelodysplasia, and several other malignant and benign disorders.
The preferred donors for allogeneic transplant include a matched sibling donor or a volunteer matched unrelated donor. Our blood and marrow and cellular therapy experts also specialize in alternative donor transplants (when a matched sibling or unrelated donor is not available) with a haploidentical (half matched) donor, or transplantation using cord blood units (see below).
Finding a Donor
Patients and families often have questions about finding a donor. The Blood and Marrow Transplant and Cellular Therapy Program works with your family members to screen them for eligibility. If there is no match, a donor search coordinator will review the international Be The Match® registry to identify any potential matches. The process of finding a donor takes about four to six weeks for a related donor, and an additional six weeks for an unrelated donor.
Allogeneic transplants start with chemotherapy that lowers your blood cell counts. You may also have radiation treatments, but not everyone does. During this, the myelo-suppression phase, your blood cell counts will remain low until the transplanted cells begin to function and start producing new cells. If you have a condition that requires specialized critical care, we will admit you to the intensive care unit. The most common side effects during this time are:
- Nausea, vomiting and/or diarrhea
- Mouth sores
- Blood or platelet transfusions
- Increased risk for bleeding
After the transplant, your bone marrow starts the recovery process called engraftment. White blood cells are the first to recover – usually within two to four weeks. Red blood cells and platelets take longer to recover, and you may require transfusions several months after your transplant.
When your cells start to engraft you may experience a group of symptoms called engraftment syndrome. The most common symptoms of engraftment syndrome are:
The treatment for engraftment syndrome requires early identification and a short course of high-dose steroids.
Graft vs. Host Disease
About 50 percent of patients who receive an allogeneic transplant develop a complication called graft vs. host disease (GVHD), in which the donor’s cells see the recipient’s body as foreign and attack it, causing damage. Although the frequency and severity of GVHD is reduced in patients with closely matched donors, even "perfectly" matched transplants can result in some degree of GVHD.
Depending on the symptoms, GVHD can be acute, usually occurring within the first few months after transplant, or chronic, which can develop after an acute case and can last for months or years. Acute GVHD symptom include:
- Rash with burning and redness of the skin. The skin may blister and, in severe cases, the exposed surface of the skin may flake off.
- Nausea, vomiting, diarrhea, abdominal cramps and loss of appetite.
- Yellowing of the skin caused by lower liver function.
Chronic GVHD affects more organs, making it a challenge to manage. Symptoms of chronic GVHD include:
- Dry itchy rash, changes in skin color or tightening of the skin
- Mouth dryness or sores
- Dryness and infection of the eyes
- Chronic cough, shortness of breath, frequent respiratory infections
- Vaginal dryness, strictures
Prevention is the best treatment for GVHD. Take your medications as directed and protect yourself from the sun, as exposure to the sun's ultraviolet rays increases your risk for GVHD. Most importantly, watch for symptoms and report them as soon as possible. Early treatment is key to your recovery.
In our special GVHD Clinic, we monitor patients for symptoms and provide essential treatment if GVHD occurs. If GVHD occurs, steroids are the first medication of choice. Other options, including clinical trials, are also available.
Chronic GVHD Consortium
We form one of nine primary study sites of the Chronic GVHD Consortium, an integrated group of academic medical centers, patient support organizations and clinical research resources around the country dedicated to conducting clinical research in GVHD and improving the care and medical outcomes of patients who experience it. Our association with the consortium gives you access to the latest clinical trials in the fight against GVHD.
For some diseases, like myeloma and testicular cancer, a multiple (or tandem) transplant approach may be preferable. These transplants are specific to the disease and are rare. Options include:
- Autologous/Autologous Transplant — You have one transplant using your own cells, followed by another autologous transplant within three to six months following recovery from the first transplant. This is usually done in a clinical trial setting.
- Autologous/Allogeneic Transplant — An autologous transplant is followed several months later by an allogeneic transplant.
Umbilical Cord Blood Transplants
Donated umbilical cord blood has several times the amount of stem cells we can collect from your bone marrow or blood. It is an alternative if we can’t find a related or unrelated matched donor. This procedure is used more often with children.
Another option is a haploidentical or "haplo" (half-matched) transplant using the stem cells of a donor — usually a sibling, parent or child — who is not a complete match for the patient. These procedures are usually part of a clinical trial.