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, blood disorders, and autoimmune disorders. Blood-forming stem cells are vital because they grow into different types of blood cells. The main types of blood cells are:

• white blood cells, which are part of your immune system and help your body fight infection
• red blood cells, which carry oxygen throughout your body
• platelets, which help the blood clot and prevent bleeding

Diseases that may be treated with a bone marrow or cord blood transplant include:

• Leukemias and lymphomas
• Multiple myeloma and other plasma cell disorders
• Severe aplastic anemia and other marrow failure states
• Sickle cell disease and thalassemia
• Inherited immune system disorders, such as severe combined immunodeficiency (SCID) and Wiskott-Aldrich syndrome
• Inherited metabolic disorders, such as Hurler's syndrome and leukodystrophies
• Myelodysplastic syndromes

Your doctor recommends your treatment based on several factors, including the status of your disease and your health. Different treatments are better at different times for each disease.

Your doctor also looks at your overall health and risk factors. These things can change over time too.

Your doctor will consider both your disease status and your current health when deciding on a treatment method. In general, transplant works better when:

• The disease is in the early stages or in remission
• The disease gets better after chemotherapy
• You are as healthy as can be expected and your organs work well

Your doctor will decide what type of transplant is best for you:

1. Autologous transplant uses cells collected from your body.
2. Allogeneic transplant uses cells donated from a family member, an unrelated donor or an umbilical cord blood unit.

For an autologous transplant, the healthy cells for a transplant can come from 1 of 2 sources:

• Bone marrow
• Peripheral (circulating) blood stem cells (PBSC)

For an allogeneic transplant, the healthy cells for a transplant can come from 1 of 3 sources:

• Bone marrow
• Peripheral (circulating) blood stem cells (PBSC)
• Umbilical cord blood that is collected after a baby is born

If you need a transplant, your doctor will choose the best source of the cells for you. Your doctor makes this decision based on your disease, other treatments you have had, and your overall health.

If you need an allogeneic transplant, the cells you receive will come from someone else. Your doctor will look for a donor in your family who matches your human leukocyte antigens, or HLA, type. These are markers your body uses to know which cells belong in your body and which don't.

HLA tissue types are inherited, so your best chance of finding a match is with a brother or sister who have the same two biological parents. However, 70% of patients do not have a fully matched donor in their family. If you do not have a donor in your family, your doctor can search for a marrow donor or cord blood unit.

The registry of the C.W. Bill Young Cell Transplantation Program, also called the Be The Match Registry, is a listing of potential marrow donors and donated cord blood units. The registry is operated under Federal contracts by the National Marrow Donor Program (NMDP).

Find more information about finding a donor, including when your doctor may begin to search a listing of potential marrow donors and cord blood units throughout the world. NMDP/Be The Match has agreements with its global partners to provide access to more than 40 million potential marrow donors and more than 806,000 cord blood units within its search system.*

*Updated as of April 10, 2023. Current numbers can be found at World Marrow Donor Association.

It can take a few weeks to a few months or more to find a marrow donor or cord blood unit. However, sometimes a closely matched marrow donor or cord blood unit cannot be found. If your doctor cannot find a close match for you, they will look at other treatment options.

Sometimes, family and friends want to help by adding more donors to the registry. Be The Match can help with donor recruitment efforts.

If you're going to have a transplant, there are things you can do now to help you and your family prepare.

Consider the Transplant Center

First, you should check which transplant centers are covered by your health insurance.

Then, talk with your doctor about transplant centers that meet your specific needs. Consider asking the transplant center doctor:

• How many patients like me has this transplant center treated?
• How well do people treated at this center do after transplant?

You and your family will be spending a lot of time with the transplant team. Consider visiting the center and meeting the team.

Plan for Transplant Costs

Every health insurance plan is different, so you should talk to your insurance company as soon as you can. This way you can know ahead of time what your insurance will pay for and what you may have to pay. Some costs not covered by insurance may include, office visits, travel, housing costs if the transplant takes place far from home, and co-pays for medicines.

If you don't have insurance, talk to the social worker at your transplant center. You may be eligible for state or county programs, or other forms of financial help.

Prepare for Changes in Daily Life

Planning with family and friends before the transplant is important. Think about your responsibilities and who might be able to do them for you. Give people specific tasks to help you. Have a family member or friend organize your support. You also may find it helpful to talk with other transplant recipients for tips and emotional support.

Prepare for Recovery

Recovering from transplant takes time, and complications are common. During the first several months after a transplant, your body will be weak. You may also be at higher risk for infections. To ease your recovery, follow the instructions from your transplant team. You will stay in or near the hospital during this time.

During recovery, most transplant recipients experience some side effects. These may last for several months or longer. Some are easy to manage. Others may be serious or painful. You can't always control whether you will have these long-term effects, but you and your doctor can take steps to treat and cope with them.

Help Your Child or Teenager Through Their Transplant

A transplant is a long and intense process that will affect the entire family. Learn what to expect to help you and your family cope with any challenges ahead. Talk with your child about what to expect at each phase during the transplant process including recovery. Share information tailored to their age and developmental stage. Give your child opportunities to express feelings, participate in decisions and ask questions.

Teens going through transplant may have needs for privacy and independence. Talk with the transplant team about ways your teen can participate in the treatment plan.

Caregiving

Learn how to comfort and provide support to a friend or family member undergoing a bone marrow or cord blood transplant.

A bone marrow or cord blood transplant is a long process with many steps.

1. The Cells are Collected

Autologous Transplant: Your own blood forming cells are collected from the circulating blood (peripheral blood stem cells) or marrow. Most patients use PBSC for an autologous transplant. But your doctor will decide which is best for you.

• PBSC collection: Blood-forming cells are collected from the bloodstream. This process is called apheresis. Before apheresis you receive shots to increase the number of blood-forming cells in your bloodstream. During apheresis, blood is removed from your vein through an intravenous (IV) line, passed through a machine, and put back into your vein. The machine takes out the blood-forming cells that will be used for your transplant.
• Marrow collection: Blood-forming cells are collected from the pelvic, or hip, bone through surgery. You receive anesthesia so you are comfortable during the process. A doctor uses a special needle to remove the blood-forming cells from your bone marrow.

Allogeneic Transplant: Your doctor will look for a marrow donor or cord blood unit that matches your HLA type. HLA stands for human leukocyte antigen. This is a marker your body uses to know which cells belong in your body and which don’t.

If you don’t have a donor in your family, your doctor will search for an unrelated donor or cord blood unit through the registry of the C.W. Bill Young Cell Transplantation Program, also called the Be The Match Registry®. The registry is a listing of potential marrow donors and donated cord blood units.

NMDP/Be The Match has agreements with its global partners to provide access to more than 40 million potential marrow donors and more than 806,000 cord blood units within its search system.*

*Updated as of April 10, 2023. Current numbers can be found at the World Marrow Donor Association.

2. Your Body is Prepared for a Transplant

Before your body can receive the new cells, you get chemotherapy, with or without radiation. The treatment is called a preparative, or conditioning, regimen. The preparative regimen destroys the diseased cells and makes space in your body for the new cells to go.

3. You get the Cells (Transplant Day)

On transplant day, the cells are put into your body through an intravenous (IV) catheter. The cells move into the spaces inside your bones where they grow and make new blood cells. They make healthy new red blood cells, white blood cells, and platelets.

4. Recovery

Recovery after transplant can last weeks to months. You may need to stay in the hospital or near the transplant center for the first 100 days after transplant. Then you may need more time for recovery at home. Throughout your recovery, doctors, nurses and social workers closely care for you to prevent and treat any side effects or complications.

If you have a disease that may be treated with a bone marrow or cord blood transplant, talk to your doctor. Ask questions about your disease, your risk factors, and treatment options. Every question you have is important.

Some questions you may want to ask your doctor about treatment options are:

• What treatment do you recommend and why?
• Is the goal to cure my disease, stop the disease progression, or control symptoms?
• What are other possible treatments?
• Will a bone marrow or cord blood transplant be an option at some point in my treatment?

Questions specific to a bone marrow or cord blood transplant might include:

• When do you recommend a transplant?
• When do you begin searching for a marrow donor or cord blood unit?
• What are the risks of waiting or trying other treatments first?
• What do you think the chances are that my transplant will be successful?

As a stem cell transplant patient, you have new opportunities for a healthy and full life. Stem cell transplants have many benefits, but they also have risks. Because stem cell transplants destroy and rebuild your immune system, they increase your risk for fungal infections.

There are two main types of stem cell transplants:

• Stem cells from your own body (also called an autologous transplant).
• Stem cells from a donor (also called an allogeneic transplant). The donor can be a family member or someone unrelated to you.

There are different reasons for receiving either type of transplant. You and your healthcare provider will discuss your options and choose the treatment that’s best for you.

What you need to know about fungal infections

A transplant using stem cells from a donor increases your risk for fungal infection more than a transplant that uses stem cells from your own body. If your stem cells come from another person, you will need to take anti-rejection medication. This medication weakens your immune system.

Chemotherapy and radiation lower your white blood cell count. As you recover from your transplant, your white blood cell count can become very low, also known as neutropenia [PDF – 4 pages].  During this time, your body will have trouble fighting infections, including fungal infections.

Graft-versus-host disease (GVHD). If you receive stem cells from a donor, the transplanted stem cells may attack your body. This is known as graft-versus-host disease, which can increase your risk for fungal infection. The steroid medications given to treat graft-versus-host disease can also increase the chance of getting a fungal infection.

Fungal infections can range from mild to life-threatening. Some fungal infections are mild skin rashes, but others can be deadly, like fungal pneumonia. Because of this, it’s important to seek treatment as soon as possible to try to avoid serious infection.

Fungal infections can look like bacterial or viral infections. If you’re taking medicine to fight an infection and you aren’t getting better, ask your doctor about testing you for a fungal infection.

Fungal infections can happen any time after your transplant. Fungal infections can happen days, weeks, or months after the stem cell transplant. If you develop GVHD, you’re at risk for a longer period of time.

Some types of fungal infections are more common than others in stem cell transplant patients. Aspergillosis is the most common type of fungal infection in stem cell transplant patients, followed by Candida infection and mucormycosis, but other types of fungal infections are also possible.

Your hospital stay matters. After your transplant, you may need to stay in the hospital for a long time. While there, you may need to have procedures that can increase your chance of getting a fungal infection. Please see the following sites for more information:

• Types of healthcare-associated infections
• Hospitalized patients and fungal infections

Where you live (geography) matters. Some disease-causing fungi are more common in certain parts of the world. If you live in or visit these areas, you may be more likely to get these infections than the general population.

Indoor mold. You may be at higher risk for getting sick from indoor mold.

Preventing fungal infections in stem cell transplant patients

Fungi are difficult to avoid because they are a natural part of the environment. Fungi live outdoors in soil, on plants, trees, and other vegetation. They are also on many indoor surfaces and on your skin. However, there may be some ways for you to lower the chances of getting a serious fungal infection.

Learn about fungal infections. There are different types of fungal infections. Learning about them can help you and your healthcare provider recognize the symptoms early, which may prevent serious illness.

Get additional medical care if necessary. Fungal infections often resemble other illnesses. Visiting your healthcare provider may help with faster diagnosis and may prevent serious illness.

Testing for early infection can help lower your risk for some types of fungal infection. Some patients may benefit from blood tests to detect fungal infections like aspergillosis before symptoms appear. Talk to your doctor to determine if this type of test is right for you.

Antifungal medication. Your healthcare provider may prescribe medication to prevent fungal infections. Scientists are still learning about which transplant patients are at highest risk and how to best prevent fungal infections.

Protect yourself from the environment. As you recover after surgery and start doing your normal activities again, there may be some ways to lower your chances of getting a serious fungal infection by trying to avoid disease-causing fungi in the environment. It’s important to note that although these actions are recommended, they have not been proven to prevent fungal infections.

• Try to avoid areas with a lot of dust like construction or excavation sites.
• Stay inside during dust storms.
• Stay away from areas with bird and bat droppings. This includes places like chicken coops and caves.
• Wear gloves when handling materials such as soil, moss, or manure.
• Wear shoes, long pants, and a long-sleeved shirt when doing outdoor activities such as gardening, yard work, or visiting wooded areas.

Researchers hope stem cells will one day be effective in the treatment of many medical conditions and diseases. But unproven stem cell treatments can be unsafe—so get all of the facts if you’re considering any treatment.

Stem cells have been called everything from cure-alls to miracle treatments. But don’t believe the hype. Some unscrupulous providers offer stem cell products that are both unapproved and unproven. So beware of potentially dangerous procedures—and confirm what’s really being offered before you consider any treatment.

The facts: Stem cell therapies may offer the potential to treat diseases or conditions for which few treatments exist. Sometimes called the body’s “master cells,” stem cells are the cells that develop into blood, brain, bones, and all of the body’s organs. They have the potential to repair, restore, replace, and regenerate cells, and could possibly be used to treat many medical conditions and diseases.

But the U.S. Food and Drug Administration is concerned that some patients seeking cures and remedies are vulnerable to stem cell treatments that are illegal and potentially harmful. And the FDA is increasing its oversight and enforcement to protect people from dishonest and unscrupulous stem cell clinics, while continuing to encourage innovation so that the medical industry can properly harness the potential of stem cell products.

To do your part to stay safe, make sure that any stem cell treatment you are considering is either:

• FDA-approved, or;
• Being studied under an Investigational New Drug Application (IND), which is a clinical investigation plan submitted and allowed to proceed by the FDA.

Stem Cell Uses and FDA Regulation

The FDA has the authority to regulate stem cell products in the United States.

Today, doctors routinely use stem cells that come from bone marrow or blood in transplant procedures to treat patients with cancer and disorders of the blood and immune system.

With limited exceptions, investigational products must also go through a thorough FDA review process as investigators prepare to determine the safety and effectiveness of products in well-controlled human studies, called clinical trials. The FDA has reviewed many stem cell products for use in these studies.

As part of the FDA’s review, investigators must show how each product will be manufactured so the FDA can make sure appropriate steps are being taken to help assure the product’s safety, purity, and strength (potency). The FDA also requires sufficient data from animal studies to help evaluate any potential risks associated with product use. (You can learn more about clinical trials on the FDA’s website.)

That said, some clinics may inappropriately advertise stem cell clinical trials without submitting an IND. Some clinics also may falsely advertise that FDA review and approval of the stem cell therapy is unnecessary. But when clinical trials are not conducted under an IND, it means that the FDA has not reviewed the experimental therapy to help make sure it is reasonably safe. So be cautious about these treatments.

About FDA-approved Products Derived from Stem Cells

The only stem cell-based products that are FDA-approved for use in the United States consist of blood-forming stem cells (hematopoietic progenitor cells) derived from cord blood.

These products are approved for limited use in patients with disorders that affect the body system that is involved in the production of blood (called the “hematopoietic” system). These FDA-approved stem cell products are listed on the FDA website. Bone marrow also is used for these treatments but is generally not regulated by the FDA for this use.

Safety Concerns for Unproven Stem Cell Treatments

All medical treatments have benefits and risks. But unproven stem cell therapies can be particularly unsafe.

For instance, attendees at a 2016 FDA public workshop discussed several cases of severe adverse events. One patient became blind due to an injection of stem cells into the eye. Another patient received a spinal cord injection that caused the growth of a spinal tumor.

Other potential safety concerns for unproven treatments include:

• Administration site reactions,
• The ability of cells to move from placement sites and change into inappropriate cell types or multiply,
• Failure of cells to work as expected, and
• The growth of tumors.

Note: Even if stem cells are your own cells, there are still safety risks such as those noted above. In addition, if cells are manipulated after removal, there is a risk of contamination of the cells.

Advice for People Considering Stem Cell Therapies

Know that the FDA plays a role in stem cell treatment oversight. You may be told that because these are your cells, the FDA does not need to review or approve the treatment. That is not true.

Stem cell products have the potential to treat many medical conditions and diseases. But for almost all of these products, it is not yet known whether the product has any benefit—or if the product is safe to use.

If you're considering treatment in the United States:

• Ask if the FDA has reviewed the treatment. Ask your health care provider to confirm this information. You also can ask the clinical investigator to give you the FDA-issued Investigational New Drug Application number and the chance to review the FDA communication acknowledging the IND. Ask for this information before getting treatment—even if the stem cells are your own.
• Request the facts and ask questions if you don’t understand. To participate in a clinical trial that requires an IND application, you must sign a consent form that explains the experimental procedure. The consent form also identifies the Institutional Review Board (IRB) that assures the protection of the rights and welfare of human subjects. Make sure you understand the entire process and known risks before you sign. You also can ask the study sponsor for the clinical investigator’s brochure, which includes a short description of the product and information about its safety and effectiveness.

If you're considering treatment in another country:

• Learn about regulations that cover products in that country.
• Know that the FDA does not have oversight of treatments done in other countries. The FDA typically has little information about foreign establishments or their stem cell products.
• Be cautious. If you’re considering a stem cell-based product in a country that may not require regulatory review of clinical studies, it may be hard to know if the experimental treatment is reasonably safe.