Drugs used for treating GVHD | Cancer Research UK
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Drugs used for treating GVHD

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This page tells you about the drugs used to treat graft versus host disease (GVHD). There is information about


How the drugs work

Most of these drugs work by damping down your immune system and so stopping the donated cells attacking your body. If you have GVHD, you are at a greater risk of getting an infection because it weakens your immune system. Treatments for GVHD further increase this risk. Your doctor and nurses will keep a close eye on you, but you should report any signs of infection as soon as you notice them, so you can start any necessary treatment.



Although steroids are made naturally in the body they can also be made artificially and used to treat various conditions. Steroids suppress your immune response and reduce inflammation. The most common types of steroids used for GVHD are prednisolone, which you take as a tablet, and methylprednisolone which you usually have as an injection into a vein. You can find out about the side effects of steroids in our section about cancer drugs.

Steroids are available as creams to reduce skin GVHD. They are also available as eye drops if you have GVHD affecting the eye. 

Once your GVHD is under control, your doctor will gradually reduce the dose. You might have steroids on their own, or with one of the other drugs or treatments listed here.



Ciclosporin is also known as Neoral, Deximune, Sandimmum. This drug works by reducing the number of T cells in your body. It comes as a capsule which you take daily or as a liquid that you drink. You must not have grapefruit or grapefruit juice within an hour of taking ciclosporin because it can interfere with how the drug works. You will have regular blood tests to check for side effects to your kidneys or liver.

The common side effects of ciclosporin include

  • Feeling sick
  • Indigestion
  • Tiredness
  • Dizziness
  • Raised blood pressure
  • Headaches
  • Low levels of red blood cells (anaemia)
  • Shaking hands (tremor)
  • Kidney problems
  • Excess growth of body and facial hair in women (hirsutism)
  • Swollen gums


Tacrolimus is also known as Prograf, Modigraf and Advagraf. It dampens (suppresses) your immune system to treat GVHD. It comes as capsules which you usually take twice a day. It is also available as a liquid that you drink. You can also have it by drip (intravenously).

The common side effects include

  • Shaking hands
  • High blood sugar
  • Feeling sick and loss of appetite
  • Diarrhoea
  • Stomach pain
  • Difficulty in sleeping

Your doctor may prescribe Protopic ointment (tacrolimus) if you have GVHD affecting the eyes.


Light treatment (ECP)

This is also known as extracorporeal photopheresis or light therapy. Photopheresis can improve skin, liver and mouth GVHD. Your doctor may also suggest it for chronic GVHD of the lung.

Doctors normally give photopheresis after other treatment hasn’t worked or has stopped working. Photopheresis is a complicated process and it may take up to 6 months before you see any real improvement in symptoms. If your doctors think photopheresis may help treat your GVHD, they will refer you to a skin specialist (dermatologist) at a centre that offers this treatment.

To have the treatment your nurse will connect you to a machine by a drip. Your blood then goes through the machine, which separates off some of your white blood cells. You then have your blood, minus the white blood cells, given back into your vein. The separated white blood cells are treated with a special drug and then exposed to ultraviolet light. This light activates the drug so that it is able to destroy the abnormal white blood cells. After this process your nurse gives back the treated white blood cells into your vein.

Each treatment takes between 3 and 4 hours, and you would usually have it every 2 weeks for between 6 and 12 months. Having this procedure does not hurt, but you will usually need to have a central line. Some people may feel a bit weak or dizzy during or after the treatment. After the treatment your skin and eyes may be more sensitive to sunlight for about 24 hours, so you need to protect yourself from the sun and wear sunglasses.


Biological therapies

There are a number of different types of biological therapy. As well as using biological therapies to treat cancer, doctors may use some types to help treat GVHD. These include

Monoclonal antibodies

Monoclonal antibodies are made in the laboratory and are designed to target specific proteins on the surface of body cells. You have them through a drip (intravenously) into your bloodstream. Several types of monoclonal antibody are being used to treat GVHD. There is information here about

Alemtuzumab (Campath)

Alemtuzumab works by targeting a protein on the surface of certain white blood cells and destroying them. This suppresses your immune system and helps prevent or treat GVHD.

Infliximab (Remicade)

Infliximab has been used to treat acute GVHD. It targets a type of chemical called a cytokine, which causes inflammation of body tissues. Infliximab binds with the cytokine to stop it from working.

Rituximab (Mabthera)

Rituximab acts on white blood cells called B cells. We still don’t completely understand how GVHD develops. In the past doctors thought only the T cells were involved. Over the last couple of years they have been looking at whether B lymphocytes (another type of white blood cell) might also be involved. These lymphocytes make antibodies which destroy foreign cells. Doctors have found that giving rituximab may help treat chronic GVHD. But we need more research to confirm this.

Possible side effects of monoclonal antibodies

Treatment with any monoclonal antibody can make you more prone to infections. So, as with GVHD generally, look out for any signs of infection and report them immediately to your doctor. The most common side effect of all monoclonal antibodies is an allergic reaction to the drug. This reaction is most likely to happen when you first have the treatment. You will have paracetamol and an antihistamine drug before you have the treatment to prevent a reaction. If you have a reaction, your doctor or nurse can usually control it by slowing down or stopping the drip for a while.

You can find out about the general side effects of monoclonal antibodies in the biological therapy section. You can find information about the side effects of individual biological therapy drugs in the cancer drugs section.

mTOR inhibitors

mTOR is a type of protein that can make cells produce chemicals to trigger cell growth. So mTOR inhobitors stop cells from growing and dividing, and dampens the immune system. There are different types of mTOR inhibitor. These include

Sirolimus (Rapamune) is a drug that trials have shown can help to prevent GVHD. Doctors may also use it to treat severe acute GVHD. It can also sometimes help to control chronic GVHD when steroids and ciclosporin are not working. You take it as tablets or a solution that you drink. You must not have grapefruit or grapefruit juice within an hour of taking sirolimus because it can interfere with how the drug works. Rapamune liquid contains soya oil. Patients allergic to peanut or soya must not take this medicine. You will have regular blood tests to check your liver and kidney function.

Some of the most common side effects include

  • Low levels of platelets or red blood cells
  • A high temperature
  • High blood pressure
  • Low levels of some important chemicals in the blood (potassium and phosphate)
  • Pain in the abdomen, diarrhoea or constipation

You may have sirolimus with other drugs that suppress your immune system.

Everolimus is a drug that doctors may use instead of sirolimus. You take everolimus as a tablet. Common side effects include

We have more information about the side effects of everolimus.

Tyrosine kinase inhibitors (TKIs)

Tyrosine kinase inhibitors block messengers (enzymes) which send growth signals in cells. So they stop the cells growing and dividing. One type of TKI doctors may use for chronic GVHD, particularly if it's affecting the skin or lung, is imatinib (Glivec). You take this as a tablet. Some of the common side effects of imatinib include

We have more information about the side effects of imatinib.


Thalidomide is another cancer growth blocker and works in different ways. As with the other drugs, it dampens the immune system. It has been tested as a treatment for GVHD. Some research has shown that it can help to control chronic GVHD. Doctors do not use it very often and usually only if other treatments are not controlling the GVHD. You take thalidomide as a tablet. The main side effects include

  • A drop in the number of blood cells
  • Drowsiness
  • Constipation
  • Numbness of the fingers and toes (peripheral neuropathy)
  • Increased risk of blood clots

It is essential to avoid pregnancy while taking this drug and for a number of weeks afterwards. We have more information about the side effects of thalidomide.


Etanercept blocks a chemical called tumour necrosis factor (TNF) from causing damage to tissue. You have it as an injection under your skin. Doctors are more likely to use it for acute GVHD. Etanercept may be particularly helpful for GVHD affecting the gut. Side effects may include

  • Infection
  • High temperature
  • Itchy skin
  • Reaction around the injection site, such as pain, swelling and bruising



Mycophenolate mofetil (MMF)

Mycophenolate is also known as CellCept. It is another drug doctors use to damp down your immune system. You take it by mouth as a tablet, capsule or liquid, usually twice a day. The main side effects are diarrhoea, sickness and a higher risk of infections because it lowers your white blood count.


Anti lymphocytic or anti thymocytic globulin (ALG or ATG)

ALG or ATG destroy T cells. Doctors mainly use ALG or ATG before transplant to prevent your body rejecting the donor cells and reduce your risk of GVHD. But you might also have one of them if you develop acute GVHD. You have ALG or ATG as a drip (infusion) over about 4 hours.

You may react to ALG or ATG. This may cause symptoms such as fever, shakiness, skin rash, sickness, headache and changes to your blood pressure. Your nurse will probably give you medicines beforehand to help prevent a reaction.



Doctors may use chemotherapy drugs to help treat GVHD. These include

Pentostatin (Nipent) can be used against acute or chronic GVHD because it breaks down the T cells. You have it through a drip in your arm (intravenously). This drug puts you at a higher risk of infections. So, as with GVHD generally, look out for any signs of infection and report them immediately to your doctor. You can find information about pentostatin in the cancer drugs section.

Methotrexate stops T cells from dividing and so keeps the numbers down. You often have low doses of methotrexate after your transplant to try to prevent GVHD. Doctors may also use it to treat GVHD when other treatments have not worked. We have more information about methotrexate and its side effects.


Newer GVHD drugs

Newer drugs being used for GVHD include

  • Clofazamine (Lamprene) – a drug usually used to treat the skin condition leprosy. It reduces swelling (inflammation)
  • Halofuginone – a drug that helps treat fibrosis caused by chronic GVHD
  • Hydroxychloroquine – a drug usually used to treat malaria. It may be useful for chronic GVHD

Doctors are still looking at how well these treatments work for GVHD. Your doctor may offer you one of these treatments if other treatments have not controlled your GVHD.

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Updated: 16 December 2014