Stem Cell Transplant

Stem cell transplants are life-saving procedures that can treat serious blood cancers, blood disorders, and some autoimmune diseases by replacing damaged or diseased blood cells with healthy ones.

Table of contents

• What is a Stem Cell Transplant?
• Conditions Treated with Stem Cell Transplants
• How Stem Cell Transplants Work
• Types of Stem Cell Transplants
• The Transplant Process
• How Blood-Forming Stem Cells Are Matched
• Success Rates and Factors
• Risks and Complications
• Recovery and Life After Transplant
• How Long Treatment Takes
• Cost Considerations

What is a Stem Cell Transplant?

A stem cell transplant, also known as a bone marrow transplant or hematopoietic stem cell transplant, is a medical procedure that replaces damaged or diseased blood cells with healthy ones. The procedure involves giving a patient healthy blood-forming stem cells to help restore the body’s ability to produce blood cells^[1].

Stem cells are special cells found in bone marrow—a spongy tissue located in the center of some bones—that can develop into different types of blood cells^[5]. These blood-forming stem cells are vital because they create all the components your blood and immune system need to function properly^[3].

The stem cells used in transplants can come from three main sources: the bloodstream (called peripheral blood stem cells), bone marrow taken directly from inside bones, or umbilical cord blood collected when a baby is born^[1]. When stem cells come from the blood, the procedure may be called a peripheral blood stem cell transplant (PBSCT). When they come from bone marrow, it’s called a bone marrow transplant (BMT), and when from cord blood, it’s called a cord blood transplant^[1].

Conditions Treated with Stem Cell Transplants

Stem cell transplants are most commonly used to treat cancers and disorders that affect blood cells. The procedure is typically considered when other treatments haven’t worked, when a condition comes back after treatment, or when very high doses of chemotherapy or radiation are needed to destroy cancer cells^[2].

Blood cancers that may be treated with stem cell transplants include leukemia (a cancer affecting white blood cells), lymphoma (another type of cancer affecting white blood cells), multiple myeloma, and myelodysplastic syndromes^[1]. The procedure may also be used for neuroblastoma, Ewing sarcoma, brain tumors that have returned in children, germ cell tumors, and testicular cancer^[1].

Beyond cancers, stem cell transplants can treat various blood disorders and other conditions. These include severe aplastic anemia (bone marrow failure), sickle cell disease, thalassemia, severe combined immunodeficiency (SCID), Hurler syndrome, and certain autoimmune diseases^[1][5].

How Stem Cell Transplants Work

Stem cell transplants typically work in an indirect way against cancer. Rather than directly destroying cancer cells, they restore your body’s ability to produce new blood cells after you’ve received very high doses of chemotherapy or radiation therapy designed to kill cancer cells^[1].

The high doses of chemotherapy and radiation used before a transplant are strong enough to destroy cancer cells, but they also damage or destroy the normal stem cells in your bone marrow. After this treatment, the transplanted healthy stem cells are given to you through a needle in your vein, similar to a blood transfusion^[1]. The new stem cells travel through your bloodstream to your bone marrow, where they begin to grow and produce healthy blood cells—a process called engraftment^[7].

In some blood cancers, particularly leukemia, stem cell transplants can work directly against the disease through an effect called graft-versus-tumor or graft-versus-leukemia. This occurs when white blood cells from a donor attack any remaining cancer cells in your body, improving the chances of success^[1].

Types of Stem Cell Transplants

There are two main types of stem cell transplants, classified based on where the stem cells come from^[2]:

Autologous stem cell transplant uses your own healthy stem cells. Before you receive high-dose chemotherapy or radiation, doctors collect and store your stem cells. After the treatment destroys your diseased cells, your own stored stem cells are returned to your body^[2]. This type of transplant is slightly more common and may allow some patients to receive part or all of their treatment on an outpatient basis^[2].

Allogeneic stem cell transplant uses stem cells donated by another person. The donor could be a close family member (especially a brother or sister), another relative, an unrelated donor found through a registry, or cord blood from a newborn baby^[13][5]. This type of transplant generally requires a longer hospital stay and has more potential complications than an autologous transplant^[2].

Over 22,000 people in the United States have diseases that a stem cell transplant could potentially cure^[2].

The Transplant Process

A stem cell transplant involves several carefully planned stages that typically include^[5][14]:

Tests and examinations come first, where doctors assess your overall health and determine whether you’re strong enough for the procedure. These evaluations help your medical team plan your specific treatment^[5].

Harvesting is the process of collecting the stem cells. For patients using their own cells, or for donors providing cells to someone else, stem cells are typically collected through a painless process called apheresis. During apheresis, blood is drawn from a vein and circulated through a machine that separates and collects the stem cells, then returns the remaining blood components back to the person^[14]. When stem cells are collected from bone marrow rather than blood, the donor undergoes a procedure in an operating room where a needle is used to withdraw marrow from inside the hipbone. The donor may experience some pain afterward^[14].

Conditioning is the preparatory treatment given before the transplant. You receive high doses of chemotherapy and sometimes radiation therapy to destroy diseased cells and create space in your bone marrow for the new stem cells^[14].

Transplanting the stem cells is usually a painless procedure that feels much like receiving medication through a tube in your vein^[2]. Your healthcare provider may first give you fluids and medication to help prevent side effects. The process of receiving the stem cells may take several hours, during which your medical team monitors you closely for any reactions^[2].

Engraftment and recovery is the period when the transplanted stem cells begin to settle in your bone marrow and start producing new blood cells. For bone marrow or blood stem cell transplants, engraftment typically occurs between two and three weeks after the transplant. For cord blood transplants, it takes three to five weeks^[14]. During this critical time, most patients remain in the hospital to protect them from infection and monitor for side effects^[14].

To help patients and families understand what to expect, many transplant centers use a numbering system. The days before transplant are counted as negative numbers (day -1 being the day before transplant), the transplant itself is “day zero,” and recovery days are counted as positive numbers starting with day +1^[23].

How Blood-Forming Stem Cells Are Matched

When you need an allogeneic transplant using someone else’s stem cells, finding a compatible donor is crucial for success. Donated stem cells must carry a special genetic marker called human leukocyte antigen (HLA) that is identical or very similar to yours^[5].

The best chance of finding a match comes from a brother or sister, or sometimes another close family member. If no suitable donors are found among close relatives, a search of donor registries is conducted^[5]. Most people eventually find a donor through these registries, though a small number may find it very difficult or impossible to locate a suitable match^[5].

Success Rates and Factors

The success of a stem cell transplant depends on several important factors. These include the type of disease you have, whether it’s early-stage or advanced, whether you’ve had previous cancer treatments, your age, and your body’s overall healing capacity^[2].

Because every person’s situation is unique, it’s difficult to calculate exact survival rates or predict life expectancy. Your healthcare provider can discuss what survival rates mean specifically for your individual case^[2].

Stem cell transplants may offer the possibility of a cure for some patients with certain diseases, while for others they provide a longer period of disease-free survival^[3]. While cancer can return after a stem cell transplant, the risk becomes lower as more time passes^[2].

Risks and Complications

Stem cell transplants are complex procedures that carry significant risks. It’s important to understand both the potential benefits and the possible complications before deciding to proceed with treatment^[5].

The greatest risk is that the transplant may not successfully slow or eliminate your illness^[2]. Common problems during and after the transplant process include nausea, vomiting, diarrhea, mouth sores, hair loss, and extreme tiredness^[2].

More serious complications can include a reduced number of blood cells, which may lead to anemia (causing tiredness and weakness), excessive bleeding or bruising, and an increased risk of infections. Severe, potentially life-threatening infections can occur after a stem cell transplant^[2][6]. You will need to take antibiotics for several months to help prevent infections^[22].

A particularly important complication of allogeneic transplants is graft-versus-host disease (GVHD). This happens when the transplanted cells from the donor start to attack other cells in your body. Allogeneic transplants have more potential complications than autologous transplants because of this risk^[2][5].

The specific complications you might face vary based on your overall health, age, and previous treatments. Your healthcare team will carefully explain potential complications so you can weigh the risks against the potential benefits^[2].

Recovery and Life After Transplant

Recovery from a stem cell transplant is a gradual process that requires patience and careful attention to your health. After leaving the hospital, you will still need to stay close to the transplant center—typically within an hour’s drive—for one to three months. During this time, you’ll have frequent follow-up appointments to monitor your recovery and watch for complications^[23].

Initially, follow-up visits may be weekly, then gradually become less frequent. These appointments allow your medical team to check your general recovery, monitor your blood cell counts, and assess how well the treatment worked^[20].

Feeling extremely tired is normal after a transplant and affects different people to varying degrees. It may take several months before you truly feel your strength returning^[20]. Taking things slowly when you first return home is important. Getting plenty of rest, building up activity gradually, and being patient with yourself are key to recovery^[20].

Your immune system takes time to rebuild after a transplant, which means you remain at higher risk for infections during recovery. You may need to continue taking medicines to prevent infections, and you’ll need to be careful about exposure to germs^[20]. For the first 30 days at home, many patients need to limit their activities and social interactions. Some people carry a card indicating that any blood transfusions they receive must be specially treated (irradiated) to prevent reactions—typically for about a year after transplant^[20].

Recovery after an allogeneic (donor) transplant takes much longer than an autologous transplant, and the risk of complications remains higher for an extended period^[20].

Some patients describe reaching the 100-day milestone after transplant as a major turning point, when they feel stronger and freer to resume more normal activities. However, full recovery can take up to a year or even longer before you really feel you’re back to normal^[1][21].

How Long Treatment Takes

The length of time involved in a stem cell transplant varies depending on the type of transplant and your individual situation. Generally, you can expect to spend 30 to 60 days in the hospital or at an outpatient clinic for the transplant itself^[23].

For autologous transplants using your own cells, you may not need to stay in the hospital longer than three weeks. Some patients may even be able to receive part or all of their treatment on an outpatient basis^[22].

For allogeneic transplants using donor cells, hospital stays are typically longer—often four weeks or more. About one in four people need to be readmitted to the hospital within the first three months due to complications^[22].

Having a stem cell transplant is an intensive and challenging experience. Even after leaving the hospital, the full transplant process—including recovery—can take up to a year or longer before you feel fully recovered^[5].

Cost Considerations

Stem cell transplants are expensive procedures. Understanding the costs involved and how they’ll be covered is an important part of planning for treatment^[1]. Your healthcare team can help you understand your specific situation regarding insurance coverage and costs. Many transplant centers have financial counselors or social workers who can help you navigate insurance questions and explore options for financial assistance if needed.