Medications Used After Stem Cell Transplant

What happens after a stem cell transplant?

Your recovery and possible health issues from a stem cell transplant (also called a bone marrow transplant) depend on the type of transplant you had. Both autologous (auto) and allogeneic (allo) transplants cause your white blood cells to drop and your immune system to weaken, making you more at risk for serious infections. Your other blood counts also lower, such as platelets and red blood cells, raising your risk of bleeding and symptoms of anemia (shortness of breath, fatigue, and more). This all happens as your body works to accept and start using the newly transplanted cells (called engraftment).

Before your transplant, you will receive chemotherapy and, in some cases, radiation. If you receive radiation, you will likely get total body irradiation (TBI). Both chemotherapy and TBI are meant to weaken your immune system before you receive your new cells. This lowers certain levels of cells in your body so they do not reject the transplanted cells.

When your blood counts are low during this time, you will most likely need:

• Antibiotics, antifungals, and antivirals to prevent and/or treat infections (bacterial, fungal, and viral). These may be given orally (by mouth) or intravenously (IV, into a vein). You will also need some of these medications for up to a year after your transplant as your immune system works to accept the new cells.
• Transfusions of blood and platelets to raise levels of each type of cell. Your care team will monitor your blood levels very closely before, during, and after a transplant.

You can find more information about stem cell transplants in our “Bone Marrow Transplants” section.

What other medications will I need after my stem cell transplant?

The side effects from autologous and allogeneic stem cell transplants (and syngeneic transplants) are alike, except for the chance of Graft Versus Host Disease (GVHD) after an allo transplant. We will talk about GVHD and its treatment below.

Each cancer center has different protocols and procedures for stem cell transplants and the medications you will receive. Below is a general guideline. Talk with your care team about what medications you will be taking after your transplant and once you return home.

For auto and allo transplants, you will most likely need medications to help with the side effects of the radiation and/or chemotherapy you received before your transplant. You may need medications for:

• Nausea and/or vomiting caused by the chemotherapy or radiation you received before your transplant. Nausea and vomiting can last many weeks. You may be given medications such as aprepitant, fosaprepitant, ondansetron, dexamethasone, and olanzapine to help with this side effect.
• Diarrhea can be a side effect of chemotherapy and radiation. Your care team will give you tips on how to manage diarrhea. They may need to test your stool before you can take medications to lessen the diarrhea. Once you are home, always check with your care team before taking any new medications. If your diarrhea happens 30 days to 1 year after an allo transplant, you will be treated in a different way depending on what is causing the diarrhea (discussed below). Medications that help with diarrhea from chemotherapy may include loperamide and diphenoxylate and atropine.
• Mucositis (mouth sores) can also be caused by the chemotherapy. You may need pain medications to help with this side effect until your blood counts recover (raise). There are also mouthwashes that will help coat the sores and numb them. If there is a fungal or yeast infection in your mouth (called thrush), you will likely be given nystatin mouthwash to help with this effect.

Medications After Allogeneic Stem Cell Transplant

You will need more medications after an allo transplant because the cells come from another person. The goal is for your body to accept the new cells (to not reject them) and not let the new cells take over and attack your other cells. Graft Versus Host Disease (GVHD) is when stem cells that come from a donor start to attack the cells of the person who received the transplant. The graft is the donor cells and the healthy cells in your body are the host. Simply put, it is the donor cells versus your body’s cells and tissues.

Medications to prevent GVHD

Researchers have been working on ways to not only treat GVHD, but to prevent it. Most medications that are being used or studied to prevent GVHD are called immunosuppressants. These medications work in different ways to lower your immune system so that your body does not reject the new cells. Immunosuppressants can work on different parts of your immune system, most commonly T and B lymphocytes (also called T cells and B cells). These cells are types of white blood cells.

• The most common medications used for GVHD prevention are a combination of a calcineurin inhibitor (such as cyclosporine and tacrolimus) and a few doses of methotrexate (an antimetabolite and folate antagonist). Both cyclosporine and tacrolimus affect how T cells work by suppressing enzymes (slowing down certain proteins) that start an immune response. Methotrexate is able to kill donor T cells that are part of your newly-transplanted cells.
• Another combination that might be used to prevent GVHD is a calcineurin inhibitor with mycophenolate mofetil (MMF). MMF affects T and B cell lymphocytes.
• Sirolimus is another type of immunosuppressant that may be used. This medication slows down the actions of T cells and is generally less toxic to the kidneys than a calcineurin inhibitor.
• Cyclophosphamide is a chemotherapy medication often used before your transplant as “conditioning,” meaning it gets your body ready for your new cells. Cyclophosphamide can also be used after your transplant to help prevent GVHD. Cyclophosphamide used after transplant may also be called “in vivo T cell depletion,” meaning it kills T cells from the donor cells from inside of your body.
• Anti-thymocyte globulin (ATG) is a polyclonal antibody, or an immune globulin, also used to slow down or kill T cells from donor cells, making it another type of in vivo T cell depletion medication.

Many other medications are being used and tested in different combinations to prevent GVHD, such as:

• Etancercept affects the role of tumor necrosis factor (TNF), a protein in your body that helps in the inflammation process that leads to GVHD.
• Infliximab is a chimeric monoclonal antibody against tumor necrosis factor (TNF).
• Bortezomib affects nuclear factor (NF) in T cells, slowing down or stopping T cells from starting an immune response.
• Marviroc blocks the CCR5 coreceptor on the surface of certain immune cells, lessening the immune response.
• Sitagliptin slows down or stops dipeptidyl peptidase 4 (DPP-4, also known as CD26), a receptor on T cells, to slow down the immune response.
• Basiliximab and daclizumab are interleukin-2 receptor antagonists. Both are a type of monoclonal antibody that affect how T cells work.
• Abatacept slows or stops T cell activation during an immune response.
• Tocilizumab is a human monoclonal antibody against Interleukin 6 (IL-6) which, in combination with standard GVHD prevention, has shown to lower risk of GVHD.
• “Statin” medications used to lower cholesterol, such as atorvastatin, are also being studied to prevent GVHD. They are thought to be able to help control an immune response and lessen inflammation in the body.
• Vorinostat is a histone deacetylase inhibitor that has been shown to affect inflammation and immune responses.
• Rituximab is a monoclonal antibody that targets CD20. When given after stem cell transplant, rituximab has been found to lower the chance of chronic GVHD that requires systemic steroid use.
• Alemtuzumab is a monoclonal antibody that targets CD52. This medication can also be used for in vivo T cell depletion.

Medications to treat GVHD

Treatment for GVHD depends on:

• If the GVHD is:
  • Acute: Starts 10 to 90 days after your transplant and the symptoms often go away after some time.
  • Chronic: Starts about 90 to 600 days after your transplant and can last for many years or your whole life.
• The symptoms you are having.
• The body part that is affected by the GVHD.

The goal of GVHD treatment is to manage symptoms and to prevent more damage to your organs. The most important part of GVHD treatment is immunosuppression, which suppresses or lowers your immune system to lessen the attack from the donor cells.

The most common medications to treat GVHD are corticosteroids. Lower-grade (less serious) cases of GVHD can be treated with topical steroids that you put on your skin. Topical tacrolimus is also an option if your body does not respond well to steroids. Higher-grade (more serious) GVHD may need systemic (IV, into a vein) steroids, such as methylprednisolone. If your GI tract is affected by GVHD, you may be given budesonide or beclomethasone, which are not absorbed (taken up) by the GI tract.

Many of the medications being used and studied to prevent GVHD are also used to treat it, such as:

• Cyclosporine.
• Tacrolimus.
• Methotrexate.
• Mycophenolate mofetil (MMF).
• Etanercept.
• Antithymocyte globulin (ATG).
• Infliximab.
• Basiliximab.
• Daclizumab.
• Sirolimus.
• Tocilizumab.

 Other medications that may be used to treat GVHD are:

• Ruxolitinib is approved by the FDA for steroid-refractory acute GVHD (does not respond or comes back after systemic steroid use) and chronic GVHD after failure of one or two types of systemic (goes throughout your whole body) therapy.
• Ibrutinib is approved by the FDA for the treatment of chronic GVHD after failure of one or more types of systemic therapy.
• Belumosudil is FDA approved for treatment of chronic GVHD after failure of at least two prior types of systemic therapy.
• Axatilimab-csfr is FDA approved for the treatment of chronic graft-versus-host disease (cGVHD) after failure of at least two prior types of systemic therapy

Some medications may be used “off-label” to treat GVHD, meaning they are FDA approved for different reasons but have been found to also help treat GVHD in certain cases:

• Alpha-1-antitrypsin is a protease inhibitor that is thought to slow down the inflammation process in GVHD.
• Hydroxychloroquine (HCQ) is a 4-aminoquinoline antimalarial drug that has been found to slow down the immune response in GVHD.
• Azathioprine is an immunosuppressant that can help slow down your immune response.
• Pentostatin is an adenosine deaminase (ADA) inhibitor that has been found to slow down your immune response.

Another treatment option may be extracorporeal photopheresis (ECP), which uses a photosensitive medication that makes your white blood cells more sensitive to light and UV light. 

This is not a complete list of medications that may be used for the prevention or treatment of GVHD. Talk with your provider about which medications may be used at your cancer center. New medications are always being studied, so be sure to ask your care team about Clinical Trials for the prevention and treatment of GVHD. 

If you are going to have or have had a stem cell transplant, your care team will watch you very closely for at least 1 year after your transplant for all of these issues and side effects. It is important to tell your provider about any new signs or symptoms you have right away so that treatment can begin as soon as possible.

Your care team will also watch for relapse (the cancer coming back after transplant) very closely and may give you medications to prevent relapse. Talk with your provider about any questions or concerns you may have about medications and follow-up care you will need after your transplant.