A trial of azacitidine and lenalidomide for acute myeloid leukaemia or myelodysplasia that has come back (VIOLA)

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Cancer type:

Acute leukaemia
Acute myeloid leukaemia (AML)
Blood cancers
Leukaemia
Myelodysplastic syndrome (MDS)

Status:

Results

Phase:

Phase 1

This trial looked at the drugs azacitidine and lenalidomide for acute myeloid leukaemia or myelodysplasia that had come back after a stem cell transplant. 

It was for people who had a stem cell transplant using cells from somebody else. 

This trial was open for people to join between 2014 and 2016. The results were published in 2018. 

More about this trial

Doctors can treat acute myeloid leukaemia (AML) or myelodysplasia (MDS) with high doses of chemotherapy, followed by a stem cell transplant using cells from somebody else (an allogeneic transplant Open a glossary item).

But if AML or MDS comes back it can be more difficult to treat. Researchers are looking for ways to improve treatment. In this trial, they looked at 2 drugs called:

Azacitidine is already used to treat AML and MDS. It works by blocking the action of a protein that stops the cancer cells growing and dividing. This reduces the number of abnormal blood cells and helps to control cell growth.

Lenalidomide affects the way the immune system Open a glossary item works. But we don’t know exactly how it works yet.

The aims of the trial were to:

  • find the highest dose of lenalidomide to have with azacitidine
  • see how well the combination of treatment worked
  • learn more about the side effects 

Summary of results

The trial team found the best dose of lenalidomide to have with azacitidine. They also found that this combination of treatment worked well in this small trial. And the side effects weren’t too bad. 

About this trial
This was a phase 1 trial. 29 people took part. Of those:

  • 24 had AML
  • 5 had MDS

People had treatment in cycles. They had treatment in 6 week cycles. Every 6 weeks was 1 cycle of treatment. Everyone had azacitidine injections for 7 days. And then lenalidomide capsules every day for 3 weeks 

The doses of lenalidomide people had depended on when they joined the trial. 

People had treatment for up to 9 months if it was helping and the side effects weren’t too bad. 

Results
The researchers looked at the highest dose of lenalidomide people could cope with alongside azacitidine. They found this was 25mg each day. 15 people had this dose. 

They also looked at how well treatment worked in everyone who took part. They followed people up for an average of 23 months. 

They had the results for 15 people. They found:

  • in 3 people the AML or MDS went away completely
  • in 3 people the AML or MDS went away, but the blood counts hadn’t returned to normal
  • in 1 person it went away a little bit  
  • in 7 people the treatment didn’t work 

Of the 7 people for whom treatment worked:

  • 6 had AML 
  • 1 had MDS

Side effects
The most common side effects of treatment were:

  • infections
  • a serious blood infection (sepsis)
  • fever caused by a drop in the number of white blood cells Open a glossary item

Lenalidomide after a transplant can cause an increased chance of developing a condition called GVHD. So, the researchers kept a close eye on everyone who took part. But this happened in only 3 people. They had steroids to treat it and this worked well. 

Conclusion
This was a small trial that helped a few people whose AML or MDS had come back after a stem cell transplant. The trial team concluded that the combination of lenalidomide and azacitidine was safe. 

They say there needs to be a larger trial to find out more about this combination of treatment. Researchers hope to develop a new trial soon to carry on this important work. 

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

Professor Charles Craddock

Supported by

Bloodwise
Cancer Research UK Clinical Trials Unit - Birmingham
Celgene
Experimental Cancer Medicine Centre (ECMC)
NIHR Clinical Research Network: Cancer
University of Birmingham
TAP Network

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Last review date

CRUK internal database number:

11009

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

Around 1 in 5 people take part in clinical trials

3 phases of trials

Around 1 in 5 people diagnosed with cancer in the UK take part in a clinical trial.

Last reviewed:

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