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Stem cell transplants, also referred to as bone marrow transplants, are intricate medical procedures aimed at replacing diseased or damaged bone marrow with healthy stem cells. Stem cells, known as the body's "master cells," possess the unique ability to differentiate into various types of specialized cells, including blood cells (National Cancer Institute, 2022).

The process typically involves several steps:

1. Preparation: Prior to the transplant, the patient may undergo conditioning therapy, which often involves high doses of chemotherapy or radiation. This treatment aims to eradicate the diseased cells in the bone marrow and create space for the incoming healthy stem cells (National Cancer Institute, 2022).

2. Stem Cell Collection: The healthy stem cells used in the transplant are either collected from the patient themselves (autologous transplant) or obtained from a compatible donor (allogeneic transplant). Stem cell collection may involve harvesting cells from the patient's own bone marrow or peripheral blood, or from a donor's bone marrow or blood (National Cancer Institute, 2022).

3. Transplantation: Once the stem cells are collected and prepared, they are infused into the patient's bloodstream, similar to a blood transfusion. These cells then travel to the bone marrow, where they settle and begin to regenerate (National Cancer Institute, 2022).

4. Engraftment: The process of the transplanted stem cells establishing themselves and beginning to produce new blood cells is called engraftment. This phase is critical for the patient's recovery, as it marks the restoration of the body's ability to generate healthy blood cells (National Cancer Institute, 2022).

5. Recovery and Monitoring: Following the transplant, patients require careful monitoring and supportive care to manage potential complications such as infections, graft-versus-host disease (in allogeneic transplants), and other side effects of the procedure. Recovery can vary in duration and may involve a period of immune suppression to prevent rejection of the donor cells (National Cancer Institute, 2022).

Stem cell transplants are utilized in the treatment of various hematological malignancies, including leukemia, lymphoma, and multiple myeloma, as well as certain non-cancerous conditions such as aplastic anemia and inherited metabolic disorders (National Cancer Institute, 2022).

(National Cancer Institute, 2022)

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The National Cancer Institute's webpage on "Blood Stem Cell Transplants" provides comprehensive information about stem cell transplants, including their purpose, procedures, and usage in treating various conditions (National Cancer Institute, 2022).

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Types of cancer treated with stem cell transplants

Stem cell transplants are procedures that restore blood-forming stem cells in people who have had theirs destroyed by the high doses of chemotherapy or radiation therapy that are used to treat certain cancers.

Stem cell transplants are most often used to treat people with cancers that affect blood cells, such as leukemia, lymphoma, multiple myeloma, and myelodysplastic syndromes. They may also be used for neuroblastoma, Ewing sarcoma, brain tumors that have come back in children, germ cell tumors, and testicular cancer.

Stem cell transplants are also used for other blood disorders, such as aplastic anemia, sickle cell disease, and autoimmune diseases.

Stem cell transplants for other types of cancer are being studied in clinical trials, which are research studies involving people. To find a study that may be an option for you, see Find a Clinical Trial.

How stem cell transplants work against cancer

Stem cell transplants do not usually work against cancer directly. Instead, they restore your body's ability to produce new blood cells after treatment with the very high doses of chemotherapy and maybe other treatments, such as radiation therapy, that are used to destroy cancer cells.

But in leukemia, the stem cell transplant may work against cancer directly. This happens because of an effect called graft-versus-tumor or graft-versus-leukemia, which can occur after transplants that use stem cells from a donor. This effect occurs when white blood cells from your donor (the graft) attack any cancer cells that remain in your body (the tumor or leukemia cells). This effect improves the chances of success of the transplant.

Types of stem cell transplants

In a stem cell transplant, you receive healthy blood-forming stem cells through a needle in your vein. Most of the blood-forming stem cells that are used in transplants come from the bloodstream. But they can also come from the bone marrow or umbilical cord, which is blood collected when a baby is born. Once they enter your bloodstream, the stem cells travel to the bone marrow, where they take the place of the cells that were destroyed by treatment. Transplants can be:

autologous, which means the stem cells come from you, the person with cancer

allogeneic, which means the stem cells come from someone else. The donor may be a blood relative or someone who is not related, if the cells are a close enough match to yours

syngeneic, which means the stem cells come from your identical twin

There are benefits and risks to both autologous and allogeneic stem cell transplants. With autologous transplants, the transplanted cells will match. But there is a small risk that cancer cells will be transplanted.

With allogeneic transplants, it is important that the cells match closely enough that your immune system won't see the transplanted blood stem cells as foreign and destroy them.

Mini-transplants are a type of allogeneic transplant that use lower doses of cancer treatment than a regular transplant. They do not kill all your blood-forming stem cells, but they still kill some of the cancer cells. This type of allogeneic transplant can prevent rejection of the donor's stem cells by suppressing your immune system.

Tandem transplants are a type of autologous transplant. During a tandem transplant, you receive a round of high-dose chemotherapy followed by a stem cell transplant. Then after many weeks or months, you have another round of high-dose chemotherapy followed by another stem cell transplant.

Whether a stem cell transplant is right for you and which type you might have depends on many factors, such as:

the type of cancer you have

how advanced your cancer is

if you can use your own stem cells

if matching donor stem cells are available

if there are other treatments that are likely to work for your cancer

if you can tolerate high doses of chemotherapy

if you have other serious health problems

other treatments you've had in the past

Your doctor will carefully weigh these issues with the risks and benefits of each type of stem cell transplant and discuss them with you.

How blood-forming stem cells are matched

To decide if the stem cells from a donor are a match for you, they will be tested for their HLAs (which stands for human leukocyte antigens). HLAs are sets of proteins, or markers, that you have on most cells in your body. Each person has a different set of HLAs. The more HLAs that you and the donor have in common, the better the chance that your body will accept the donor's stem cells.

Most often, the best match for an allogeneic stem cell transplant is a brother or sister.

See Donating Blood Stem Cells for Stem Cell Transplants to learn about stem cell donation.

Stem cell transplant side effects

The high doses of cancer treatment that you have before a stem cell transplant can cause problems such as:

Stem cell transplants may cause both short- and long-term problems. Short-term problems may include:

Long-term problems of stem cell transplants may include:

infertility

cataracts (clouding of the lens of the eye, which causes loss of vision)

new secondary cancers

liver, kidney, lung, or heart damage

bone and muscle weakness

Talk with your doctor or nurse about side effects that you might have, how serious they might be, and what to do about them.

Tell your doctor or nurse if you have trouble eating during stem cell transplant. You might find it helpful to speak with a dietitian.

For more information about side effects and how to manage them, see Side Effects of Cancer Treatment.

Graft-versus-host disease (GVHD)

If you have an allogeneic transplant, you might develop a serious problem called graft-versus-host disease. Graft-versus-host disease can occur when white blood cells from your donor (the graft) see cells in your body (the host) as foreign and attack them. This problem can cause damage to your skin, liver, intestines, and many other organs.

Graft-versus-host disease can be acute or chronic. Acute graft-versus-host disease occurs within the first 3 months after transplant. Chronic graft-versus-host disease occurs 3 months after a transplant or later.

Graft-versus-host disease can be treated with steroids or other drugs that suppress your immune system.

There are a few ways that the risk of graft-versus-host disease can be reduced.

The closer your donor's stem cells match yours, the less likely you are to have graft-versus-host disease.

Your doctor may give you drugs to suppress your immune system.

Donated stem cells can be treated to remove the white blood cells (called T cells) that cause graft-versus-host disease. This process is called T-cell depletion.

How much stem cell transplants cost

Stem cells transplants are complicated procedures that are very expensive. They require long hospital stays at special treatment centers and require the services of many health care providers. If you do not live nearby, you will need to stay in a hotel or apartment when you are not in the hospital. If you have no problems, you can go home 100 days after you've received the donor stem cells. But you will need to be closely followed by a doctor who has experience in taking care of people who have had a stem cell transplant.

Transplants can cause serious side effects that can be expensive to manage.

If you need to travel for treatment, you might have extra costs for transportation, housing, and childcare.

Most insurance plans cover some of the costs of transplants for certain types of cancer. Talk with your health plan about which services it will pay for. The business office of your treatment center may help you understand all the costs involved.

To learn about groups that may be able to provide financial help, go to the National Cancer Institute database, Organizations that Offer Support Services, and search "financial assistance." Or contact NCI's Cancer Information Service for information about groups that may be able to help.

Where you go for a stem cell transplant

When you need an allogeneic stem cell transplant, you will need to go to a hospital that has a specialized transplant center. The National Marrow Donor Program® maintains a list of transplant centers in the United States.

How long it takes to have a stem cell transplant

A stem cell transplant can take a few months to complete. The process begins with treatment with high doses of chemotherapy and maybe radiation therapy. This treatment goes on for a week or two. Once you have finished, you will have a few days to rest.

Next, you will receive the blood stem cells. The day you receive your stem cells is often called "day zero." The stem cells will be given to you through an intravenous (IV) catheter. This process is like receiving a blood transfusion. It takes 1 to 5 hours to receive all the stem cells.

After receiving the stem cells, you begin the recovery phase. During this time, doctors will follow the progress of the new blood cells by checking your blood counts often. As the new stem cells produce blood cells, your blood counts will go up.

Even after your blood counts return to normal, it takes much longer for your immune system to fully recover-several months for autologous transplants, and 1 to 2 years for allogeneic or syngeneic transplants.

How you may feel after a stem cell transplant

Stem cell transplants affect people in different ways. How you feel depends on:

the type of transplant that you have

the doses of treatment you have before the transplant

how you respond to the high-dose treatments

your type of cancer

how advanced your cancer is

how healthy you were before the transplant

Since people respond to stem cell transplants in different ways, your doctor or nurses cannot know for sure how the procedure will make you feel.

Working during a stem cell transplant

Whether or not you can work during a stem cell transplant may depend on the type of job you have. The process of a stem cell transplant, with the high-dose treatments, the transplant, and recovery, can take many months. You will be in and out of the hospital during this time. Even when you are not in the hospital, sometimes you will need to stay near it, rather than staying in your own home.

You will be more tired and your ability to concentrate on work may be affected. You will be visiting the hospital two or three times a week after discharge. You may need to spend a few hours in the hospital for blood or platelet transfusions or replacing minerals in your body.

So, if your job allows, you may want to arrange to work remotely part-time. Many employers are required by law to change your work schedule to meet your needs during cancer treatment. Talk with your employer about ways to adjust your work during treatment. You can learn more about these laws by talking with a social worker.

For more information about working with cancer and your legal rights, see Going Back to Work.

Stem cell transplants in clinical trials

If you are interested in finding a stem cell transplant clinical trial, use the advanced clinical trials search form or contact NCI's Cancer Information Service.

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Stem Cell and Bone Marrow Transplants for Cancer

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Stem cell transplants are procedures that restore blood-forming stem cells in cancer patients who have had theirs destroyed by very high doses of chemotherapy or radiation therapy. Learn about the types of transplants and side effects that may occur.

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April 17th, 2023: The FDA announced the approval of a cell therapy for patients with blood cancers, intended to lower the risk of infection following stem cell transplants. This approval signifies a significant advancement in the management of blood cancers and highlights the importance of innovative treatments in improving patient outcomes post-transplantation (FDA, 2023).

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FDA Approves Cell Therapy for Patients with Blood Cancers to Reduce Risk of Infection Following Stem Cell Transplantation

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FDA approves Omisirge, a nicotinamide modified allogeneic (donor) cord blood-based cell therapy to reduce the risk of infection following stem cell transplant.

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(Cancer.net, 2021)

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Bone Marrow and Stem Cell Transplantation for Cancer – An Introduction

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1. Treatment for Various Diseases: Stem cell transplants are used to treat a variety of diseases, including leukemia, lymphoma, multiple myeloma, and certain genetic disorders. They offer a potential cure or long-term remission for these conditions.

2. Replacing Damaged Cells: Stem cell transplants replace damaged or diseased cells with healthy ones, promoting the regeneration of healthy tissues and organs.

3. Reduced Risk of Rejection: In autologous transplants, where a patient's own stem cells are used, there's no risk of rejection or graft-versus-host disease (GVHD), a common complication of allogeneic transplants.

4. Increased Survival Rates: For some diseases, such as certain types of leukemia and lymphoma, stem cell transplants have significantly increased survival rates and long-term remission compared to other treatments.

5. Potential for Immunotherapy: Stem cell transplants can be combined with other treatments, such as immunotherapy, to enhance their effectiveness in fighting cancer or other diseases.

(American Society of Hematology).

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1. Risk of Complications: Stem cell transplants carry risks of various complications, including infection, bleeding, graft failure, GVHD (in allogeneic transplants), organ damage, and development of new cancers.

2. Side Effects of Conditioning Therapy: The high-dose chemotherapy or radiation used in conditioning therapy prior to the transplant can cause severe side effects, including nausea, vomiting, hair loss, fatigue, and increased risk of infection.

3. Limited Availability: Finding a suitable donor for allogeneic transplants can be challenging, particularly for individuals from ethnically diverse backgrounds, reducing the availability of this treatment option.

4. Long Recovery Period: Recovery from a stem cell transplant can be lengthy and challenging, involving a prolonged period of immune suppression, frequent medical monitoring, and potential long-term effects on physical and emotional well-being.

5. Cost: Stem cell transplants are expensive procedures, involving hospitalization, intensive medical care, and follow-up treatments, which can pose financial burdens on patients and healthcare systems.

(American Society of Hematology).

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Opinion: Stem cell transplants represent a groundbreaking advancement in medical science, offering hope and potential cures for a wide range of diseases.

Rationale: Stem cell transplants have revolutionized the treatment landscape by providing personalized and targeted therapies for conditions that were previously considered untreatable or had limited treatment options. By harnessing the regenerative potential of stem cells, these transplants can replace damaged or diseased cells, restore normal physiological function, and even eradicate certain cancers. Moreover, ongoing research and technological advancements continue to expand the scope and efficacy of stem cell transplants, making them increasingly accessible and effective for patients worldwide. Overall, stem cell transplants hold immense promise for improving patient outcomes, extending survival rates, and ultimately, transforming the future of medicine (National Cancer Institute, 2022).

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American Society of Hematology. "Stem Cell Transplantation." Retrieved from https://www.hematology.org/education/patients/blood-disorders/stem-cell-transplantation

FDA. (2023). FDA approves cell therapy for patients with blood cancers to reduce risk of infection following stem cell transplant. Retrieved from https://www.fda.gov/news-events/press-announcements/fda-approves-cell-therapy-patients-blood-cancers-reduce-risk-infection-following-stem-cell

Hamadani, M., & Craig, M. (2019). Autologous hematopoietic cell transplantation for T-cell lymphoma: single-center series and systematic review. Leukemia & Lymphoma, 60(3), 666–674. DOI: 10.1080/10428194.2018.1476163

National Cancer Institute (2022). "Blood Stem Cell Transplants." Retrieved from https://www.cancer.gov/about-cancer/treatment/types/stem-cell-transplant

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