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

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  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. 

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

One person died because of the side effects of treatment. 

Conclusion
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 ) 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.