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A hematopoietic cell transplant is a medical procedure used to treat a variety of diseases, including childhood cancer. It replaces damaged or destroyed blood-forming (hematopoietic) cells with healthy blood-forming cells.
Since the main source of blood-forming cells is the bone marrow, the procedure has traditionally been referred to as a bone marrow transplant. As advances have been made, the term hematopoietic cell transplant has grown in popularity. The terms are often used in place of each other. Some people may also call the process a stem cell transplant.
A transplant can successfully treat certain childhood cancers, but it can have serious side effects and late effects. It is an option to consider carefully. Having a transplant is not an easy process. It is taxing physically and emotionally for both the patient and family caregivers. But there are many people on the transplant care team to support patients and families along the way. Many children and teenagers who have had transplants are now living active, cancer-free lives.
Bone marrow is a soft, spongy material that is in the center of most of the body’s bones. Large numbers of blood-forming (hematopoietic) cells live in the bone marrow.
Hematopoietic cells are the parents of all other blood cells. They mature into cells that eventually become:
The bone marrow works like a blood cell factory, constantly making new hematopoietic cells so that the red blood cells, white blood cells, and platelets circulating in the blood can do their jobs.
All red blood cells and platelets are manufactured in the bone marrow, along with about 70 percent of white blood cells. (The other 30 percent are produced by the spleen, lymph nodes, and thymus gland.)
Very small numbers of hematopoietic cells can also can be found in the peripheral (circulating) blood. Umbilical cord blood is also a source of blood-forming cells.
The goal of transplant is to:
In pediatric cancer, transplant is primarily used to treat leukemia, usually when standard treatment for the cancer has failed. Leukemia, cancer of the blood and bone marrow, causes the body to produce damaged white blood cells, which makes the patient very sick.
Transplants may also be used to treat patients with cancers that involve tumors of the soft tissue or brain that require very high doses of chemotherapy or radiation to treat their disease.
There are basically two types of transplants:
An allogeneic transplant replaces damaged or destroyed blood cells with healthy ones from another person. These cells may come from a donor or umbilical cord blood. The donor may be a sibling, another family member, or an unrelated donor.
Blood-forming cells are harvested from the bone marrow, umbilical cord blood or peripheral (circulating) blood of the donor.
The patient receiving the donor cells will receive therapy to suppress his or her immune system. This therapy is called immunosuppressive therapy. It involves high doses of chemotherapy with or without radiation. Immunosuppressive therapy fights the cancer, destroys the patient’s existing immune system, and makes room for the donor cells to grow in the patient’s bone marrow.
Donor cells are given through a vein in a process much like a blood transfusion. The cells find their way through the bloodstream to the center of the long bones. This process is required to restore the patient’s ability to make healthy red blood cells, white blood cells, and platelets.
Patients receive supportive care with blood products, antibiotics, anti-viral drugs, and, in some cases, immunosuppressive medications to prevent graft versus host disease (GVHD) to help them recover.
An autologous transplant uses the patient’s own cells to restore his or her own ability to make red blood cells, white blood cells, and platelets after receiving high doses of chemotherapy with or without radiation required to treat their cancer. The patient’s cells are collected ahead of time from the peripheral (circulating) blood or bone marrow and cryopreserved (frozen) for later use.
There are two ways to collect blood-forming cells from the patient: apheresis or bone marrow harvest.
They can be mobilized from the bone marrow to the peripheral bloodstream by using chemotherapy followed by granulocyte colony-stimulating factor (G-CSF). Sometimes patients may have these cells mobilized after receiving only G-CSF. The patient will then undergo a procedure called apheresis to collect these cells.
A bone marrow harvest may also be performed to collect blood forming cells directly from the bone marrow. This can be done at any time after the white blood cell counts have reached normal levels.
After the cells are collected by apheresis or bone marrow harvest, they are frozen for future use when the transplant is indicated by the medical team.
Before having the transplant, the patient will be checked for infectious diseases and that he or she is medically able to have the transplant.
During the transplant, the patient receives high doses of chemotherapy with or without radiation to destroy cancerous cells, which in turn destroy the patient’s ability to make blood cells. Without the transplant, the patient would not be able to recover production of their own red blood cells, white blood cells, and platelets.
Through the vein, the patient receives his or her own cells back in a process much like a blood transfusion. The cells find their way through the bloodstream to the center of the long bones. This process is required to restore the patient’s ability to make their own red blood cells, white blood cells, and platelets.
The patient may receive blood products, antibiotics, and anti-viral drugs while they are waiting on their blood-forming cells to engraft.
Transplant is a physically challenging medical procedure. The medical team will first determine if the patient is a suitable candidate by considering the patient’s:
To ensure the patient can tolerate a transplant, the patient will also have tests on his or her heart, lungs, kidneys, and other vital organs. These tests typically include:
If they don’t already have one, patients will receive a central line so they won’t have frequent needle sticks.
Patient families will also meet with a social worker or psychologist to discuss emotional health, as well as a financial counselor to help with insurance approvals and financial issues.
The transplant team at the hospital will coordinate the donor search. A sibling with the same biological parents is usually the first choice because the donor’s and patient’s hematopoietic cells must have similar genetic markers. These markers are proteins called human leukocyte antigens (HLA). Determining whether or not someone is an HLA match requires an HLA test, which involves taking a sample of the patient and potential donor’s blood. In some cases, an inner cheek swab sample can be used for this test. The cells are then sent to a laboratory for testing.
Since these genetic markers are inherited from parents, a brother or sister is the most likely match. If the patient and sibling have the same biological parents, each brother and sister has a 25 percent chance of being an HLA match to the patient. Because of this, about 70% of patients will not have a matched sibling donor available.
If there is no matched sibling, then the care team will search for an unrelated donor or cord blood unit (if the transplant center performs cord blood transplants) through the Be the Match® Registry of the National Donor Marrow Program. About 30 percent of patients find a matched unrelated donor.
If a perfect match cannot be found, the doctor may suggest using a mismatched donor, which is a donor who is a close, but not exact, HLA match. Mismatched donor transplants are fairly common, and many are successful.
For patients who can’t find a suitable donor who is a close match, in some cases it is possible to use bone marrow or peripheral hematopoietic cells from a family member who is a “half-match.” This type of transplant is called a haploidentical (half-matched) bone marrow transplant.
Donors must also be medically capable of serving as a donor. The donor center, through Be the Match, will screen the potential donor for certain health conditions:
Blood-forming cells can be collected from bone marrow, peripheral (circulating) blood, and from the blood of donated umbilical cords. This process is called harvest.
If the cells for transplant are obtained from the bone marrow, the collection procedure is called a “bone marrow harvest.” The donor is given either general anesthesia, which allows the person to sleep during the procedure, or regional anesthesia, which causes loss of feeling below the waist. Needles are inserted through the skin over the pelvic (hip) bone and into the bone marrow to draw the blood forming cells out of the bone. Harvesting the marrow takes about an hour.
The harvested bone marrow is then processed to remove blood and bone fragments. For autologous transplant, harvested cells from the bone marrow can be combined with a preservative and frozen to keep the cells alive until they are needed. This technique is known as cryopreservation. Blood-forming cells can be cryopreserved for many years.
If the blood-forming cells needed for transplant are obtained from the bloodstream, the process is called apheresis or leukapheresis. For several days before apheresis, the donor or patient may be given a medication called granulocyte colony stimulating factor—G-CSF to stimulate the bone marrow to make more hematopoietic cells and release them into the bloodstream.
In apheresis, blood is removed through a large vein in the arm or from a central venous catheter (a flexible tube that is placed in a large vein in the neck, chest, or groin area). The blood goes through a machine that removes the hematopoietic cells. The remaining blood is then returned to the donor. Apheresis typically takes 4 to 6 hours.
For autologous transplant, the patient’s collected cells will be frozen until they are needed. For allogeneic transplant, the donor cells are then processed and given fresh to the patient as soon as possible after collection.
Blood-forming cells also may be retrieved from umbilical cord blood. For this to occur, the mother must contact a cord blood bank before the baby’s birth. The cord blood bank may request that she complete a questionnaire and give a small blood sample.
Cord blood banks may be public or private. Public cord blood banks accept donations of cord blood and may provide the donated cells to another matched individual in need of a transplant. Private cord blood banks will store the cord blood for the family, in case it is needed later for the child or another family member. The family must pay for the collection and storage of the cord blood. The 2017 American Academy of Pediatrics statement on Cord Blood Banking for Potential Future Transplantation noted that “the cost and value of the maintenance of private cord blood banks is not supported by the evidence for use at the present time.”
After the baby is born and the umbilical cord has been cut, blood is retrieved from the umbilical cord and placenta. If the mother agrees, the umbilical cord blood is processed and frozen for storage by the cord blood bank.
Many patients think a transplant is a complicated surgical procedure, but the transplant itself is a relatively simple process. It is much like a blood transfusion. Donor cells are in a bag or syringe that is connected through a tube to the patient’s central line. It only takes a few minutes to a few hours and is not painful.
After entering the bloodstream, the hematopoietic cells travel to the bone marrow, where they begin to divide and become white blood cells, red blood cells, and platelets in a process known as engraftment. Engraftment usually happens in 2-4 weeks (longer if the source was an umbilical cord.) Complete recovery of the immune system may take several months for autologous (using patient’s own cells) transplant to 6 months or a year for allogeneic (using related or unrelated donor cells) transplant.
Patients need a parent or other adult family member to stay with them while in the hospital, which will be at least 4-6 weeks and perhaps longer. Patients may need to stay in a special part of the hospital reserved for transplant patients and perhaps other patients who have little or no immune system function. Infection control guidelines are different than other areas of the hospital.
It is best if parents can take turns taking care of their child, because it is draining both physically and emotionally to be a caregiver. If both parents aren’t available, consider another family member. Plan ahead because each caregiver must be trained.
If employed, parents should meet with their employer to explain the bone marrow transplant process and what will be required of them as the child’s primary caregiver.
During the first part of the hospital stay, patients will receive high-dose chemotherapy with or without radiation.
The chemotherapy (and radiation, if used) fights the cancer cells and also makes room for the healthy donor cells at the same time. It also weakens the recipient’s immune system in order to prevent rejection of the donor cells. Patients have to take many precautions to prevent infections such as wearing a mask and washing hands often.
In addition, anyone who is staying with or visiting the patient is screened for possible infections and contagious illnesses. Every transplant center is different, so families should ask for hospital-specific rules and guidelines.
All this may seem scary at first, but the care team is there to help children feel as comfortable as possible. Patients can bring some types of toys and other items to help them feel more at home. Child life specialists can provide fun activities such as games and crafts. Rehabilitation therapists will help patients exercise and give ideas about how to stay active.
Being apart from family, friends and normal activities can be difficult so children and teens are encouraged to keep in touch with others through texts, social media, video chats, e-mails, phone calls, and letters.
Transplant patients will not be able to attend school while in the hospital, but if they feel up to it they can keep up with some schoolwork. Caregivers should talk with their child’s school and also connect with the transplant center’s school program or school liaison.
Doctors evaluate the results of various blood tests to confirm that new blood cells are being produced (engraftment) and that the cancer has not returned. After the transplant, patients have blood drawn daily so it can be tested in the lab. Doctors will count how many red blood cells, white blood cells, and platelets are in the body. Since they are counted each day, doctors and patients can keep track of the progress. Because everyone is different, engraftment can happen at different times. Usually it takes two weeks to a month. White blood cells are the first to engraft, followed by red blood cells and then platelets. Patients may need transfusions of red blood cells and platelets to keep counts in a safe range while they wait on engraftment to take place.
Bone marrow aspiration (the removal of a small sample of bone marrow through a needle for examination under a microscope) can also help doctors determine how well the new blood-forming cells are working, and if the cancer is in remission.
When patients leave the transplant unit, they still have weakened immune systems and will have difficulty fighting infections. Some of these infections can be life-threatening. After discharge, patients will need a caregiver at home with them until their immune systems return to normal, a process that can take from a few months to a year.
If the family lives close by, the patient can return home. If not, the patient family will need to stay in a special housing facility near the hospital. Patients must return to the hospital frequently for appointments for the first few weeks. Families should be prepared to stay all day. The patient will have a physical exam and may need tests and treatments. Over time, patients won’t need to return to the hospital as frequently unless problems or complications occur. Parents should bring all of the patient’s medications to every clinic visit.
Even after patients go home, they have to take extra precautions because of the risk of infections.
The patient should keep all of the same infection control routines that were started on the transplant unit until the doctor says it is OK to stop. Every transplant center is different, so follow the procedures for your hospital.
Patients may be taking many medicines. These drugs are vital to the patient’s health. Follow these procedures:
Tell the transplant clinic staff immediately if the patient’s main caregiver will change. It’s good to plan the change in advance. A transplant staff member must train each caregiver.
The two conditions that cause the most serious side effects and late effects are graft rejection and graft vs. host disease (GVHD).
Graft rejection occurs when the immune system of the patient recognizes the donor cells as being different and destroys them. Since high-dose chemotherapy used during transplant destroys the patient’s blood-forming cells, they cannot regenerate on their own. Patients who experience graft rejection can become quite ill and, in some instances, die of complications from the treatment. To prevent graft rejection, the patient receives chemotherapy with or without radiation to destroy the immune system before the transplant occurs. If graft rejection occurs, another transplant or treatment may be an option.
Graft-versus-host Disease (GVHD) is a common complication following a transplant using donor cells. Between 20 and 50 percent of patients transplanted with donor cells develop graft-vs-host disease after transplant. GVHD is not an issue for patients who had a transplant using their own cells. GVHD is a complication that can occur after an allogeneic (related or unrelated donor) transplant. It occurs when white blood cells from the donor recognize the patient’s body as being different or foreign. These white blood cells then attack tissues in the patient’s body just as if they were attacking an infection.
Most cases are mild or moderate and resolve over time. However, GVHD, can be more severe, even life-threatening.
GVHD is more easily prevented than treated. Preventive measures typically include the administration of medications that lower the immune system such as cyclosporine, tacrolimus, methotrexate, mycophenolate mofetil, or steroids after transplant. Some T-lymphocytes may also be removed from the donor cells before they are given.
There are two types of GVHD:
GVHD is categorized as acute when it occurs within the first 100 days after transplantation. The disease typically affects the liver, gastrointestinal tract, and skin. Symptoms of acute GVHD include rash, diarrhea, and yellow skin and eyes because of elevated levels of bilirubin.
Chronic GVHD usually occurs at about three months post-transplant, although in some cases it may not develop for a year or more after the transplant. It occurs in 10-40 percent of patients after transplant.
Symptoms vary more widely than those of acute GVHD and are similar to some autoimmune disorders. Chronic GVHD can be mild, moderate, or severe.
Chemotherapy and radiation used in preparation for the transplant can cause mucositis, which is sores throughout the gastrointestinal tract with symptoms such as mouth sores, esophagitis (trouble swallowing), stomach ulcers, or diarrhea with stomach cramps. Patients sometimes require intravenous pain medications and total parental nutrition (TPN) or a nasogastric tube for administration of nutrition.
Infection complications are one of the most serious side effects. Life-threatening infections can occur in about 30 percent of patients receiving allogeneic donor cells (related or unrelated donor.) The incidence is lower for patients receiving autologous (patient’s own blood-forming cell) transplants. Patients will be screening regularly for infections and will receive medication to reduce chances of infections. Allogeneic transplant patients will likely be placed on special diets. In some cases, surgical procedures may be needed to diagnose and treat complications.
A red, bumpy rash that involves the neck, ears, trunk, palms and soles can develop. It can form blisters. In severe cases, the skin will shred. The skin can be very itchy. Treatments are usually steroids such as prednisone.
Stomach problems may include abdominal pain, severe diarrhea, loss of appetite, and nausea/ vomiting.
The most common serious liver complication is veno-occlusive disease (VOD). Patients with prior liver injury, a history of hepatitis, or a high-risk disorder are at greatest risk of VOD, although the disease can develop in any patient after transplant. VOD is characterized by the elevated concentration of bilirubin (which results in the yellow appearance of the skin and eyes), an enlarged liver and fluid retention or weight gain. VOD is frequently treated with fluid restriction and a medication called defibrotide. Preventive measures may include giving the patient ursodiol or heparin and daily monitoring of weights and fluid balance while the patient is hospitalized. VOD can be severe, and in the worst cases, it can result in death.
Hair loss may occur several weeks after being admitted to the transplant unit because of the chemotherapy or as a result of graft-versus-host disease. It happens slowly over a few days.
Certain chemotherapy drugs can cause seizures. Patients who receive these types of drugs can be given medication to prevent seizures.
Pain is another complication. Mucositis (mouth sores) can be very painful. Cyclosporine and other medications given to prevent or treat GVHD can cause nerve pain. Pain medications and non-medication therapies can lessen pain and help patients cope.
Pneumonitis or lung inflammation may be caused by bacteria, viruses, or fungi. Patients are monitored closely and possible given preventive medication to prevent certain infections such as cytomegalovirus (CMV).
Fatigue is a common side effect. Patients should exercise regularly, rest often, and eat nutritious foods.
Caregivers should call the patient’s local doctor or their transplant unit’s doctor or nurse practitioner immediately if the patient has one or more of the following symptoms:
Because of the physical side effects and being separated from family, friends, and normal routines, patients may have periods of sadness or depression. It is OK to have a day or two of feeling sad, but if these feelings last longer patients should talk to a member of their care team. It could be their doctor or nurse or perhaps their social worker, chaplain, psychologist, or child life specialist.
Some of the complications associated with a transplant are known as late effects, because they are not apparent until several months or even years after treatment. Most of them resolve with time, but others may be permanent and need long-term attention.
Late effects of transplant may include:
No one experiences all of these problems. Risk for developing problems after transplant will depend on your disease, type of transplant you had, your age and prior treatment history. Many complications are preventable with proper screening and preventive measures. Regular doctor’s appointments are very important.
Patients are observed for a long time after transplantation to determine whether side effects are present. Patients who underwent total body irradiation to prepare for transplant are at risk of endocrine (gland) problems that include hypothyroidism, adrenal insufficiency or growth hormone insufficiency. It is important that the patient’s height and weight be recorded regularly and, if necessary, monitored by an endocrinologist.
In some cases, the cancer may come back (relapse) after the transplant. Relapse is most common in the first year after transplant and the risk decreases as time goes by. The transplant team will continue to care for you and will discuss other treatment options. These could include clinical trials or another transplant.
Reviewed: June 2018