A trial of azacitidine for chronic graft versus host disease (AZTEC)

Cancer type:

Acute leukaemia
Acute lymphoblastic leukaemia (ALL)
Acute myeloid leukaemia (AML)
Blood cancers
High grade lymphoma
Hodgkin lymphoma
Non-Hodgkin lymphoma




Phase 2

This trial looked at using a drug called azacitidine for people who have chronic GvHD. 

This trial was open for people to join between 2016 and 2019. The team published the results in 2021.

More about this trial

Graft versus host disease (GvHD) is a possible complication of high dose cancer treatment. It happens after a bone marrow or stem cell transplant from another person. 

Chronic GvHD (cGvHD) starts more than 100 days after your transplant. It may affect your skin, gut, liver or mouth. Chronic GvHD may be mild or severe, and for some people can go on for several months or even years.

Steroids are a common treatment for chronic GvHD. Some people:

  • become dependent on the steroids and their doctors aren’t able to reduce the dose
  • take steroids but they only work for a short time or not at all
  • can’t have steroids

When this trial was done there was no standard treatment Open a glossary item for people in this situation. So doctors wanted to improve treatment. In this trial they looked at using a chemotherapy drug called azacitidine. Some research suggested that it could be a useful treatment. 

The main aims of the trial were to find out:

  • if azacitidine works for people with chronic GvHD
  • more about the side effects 

Summary of results

In this small trial, the researchers found that azacitidine could be a promising treatment for chronic GvHD. 

Trial design
This was a phase 2 trial. It was in 2 parts. A few people had azacitidine in part 1. In part 2 the trial team planned to look at azacitidine in a larger group. 

14 people joined part 1. Everyone was on steroid treatment when they joined the trial but either:

  • it wasn’t working or
  • the doctors couldn’t reduce the dose

Everyone had up to 6 cycles Open a glossary item of azacitidine as long as it was working and the side effects weren’t too bad. 7 people completed all 6 cycles and some had treatment for a bit longer. 

The trial team looked at how well treatment worked within 6 months of starting azacitidine. They looked at symptoms to see if they had improved. They found:

  • the GvHD was better in 1 person
  • the GvHD got a bit better in 6 people
  • treatment didn’t work for 7 people

The team also looked at how well treatment was working 6 months after starting trial treatment. At this timepoint:

  • the GvHD was better in 5 people
  • the GvHD got a bit better in 3 people
  • the treatment didn’t work for 6 people 

The team also found that:

  • azacitidine took about 5 months to work if it was going to
  • 6 out of 7 people who completed six or more cycles reduced their steroid dose

Quality of life and GvHD symptoms 
Everyone filled in a questionnaire before, during and after treatment. It asked about side effects and how people had been feeling. This is called a quality of life study.

Before joining the trial the most common symptoms of GvHD were skin and mouth problems. The trial team asked people to:

  • rate their symptoms before, during and after the treatment
  • report if their symptoms got better or worse during treatment and afterwards

Those whose treatment worked said their symptoms improved and so did their quality of life. 

Side effects
Most people had side effects. Some of the side effects were mild but others were more severe. The most common of the more severe side effects included: 

  • a drop in white blood cells Open a glossary item
  • a drop in blood cells that help the blood clot (platelets) Open a glossary item
  • infections
  • low levels of potassium in the blood
  • high blood pressure 

One person died because of the side effects of treatment. 

The team concluded that azacitidine might be a useful treatment for chronic GvHD. The first part of this trial showed that it worked for enough people to be looked at in part 2. 

Azacitidine is being looked at in other trials. Researchers think it could be a useful treatment for people with acute myeloid leukaemia or myelodysplasia whose steroid treatment has stopped working. Researchers think azacitidine might work to treat chronic GvHD in this group of people.  

Where this information comes from
We have based this summary on information from the research team. The information they sent us has been reviewed by independent specialists (peer reviewed Open a glossary item) and published in a medical journal. The figures we quote above were provided by the trial team who did the research. We have not analysed the data ourselves.

Recruitment start:

Recruitment end:

How to join a clinical trial

Please note: In order to join a trial you will need to discuss it with your doctor, unless otherwise specified.

Please note - unless we state otherwise in the summary, you need to talk to your doctor about joining a trial.

Chief Investigator

Dr Ram Malladi

Supported by

Blood Cancer UK 
NIHR Clinical Research Network: Cancer
University of Birmingham

If you have questions about the trial please contact our cancer information nurses

Freephone 0808 800 4040

Last review date

CRUK internal database number:


Please note - unless we state otherwise in the summary, you need to talk to your doctor about joining a trial.

Wendy took part in a new trial studying the possible side effect of hearing loss

A picture of Wendy

"I was delighted to take part in a clinical trial as it has the potential to really help others in the future.”

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