A trial of eltrombopag with azacitidine for myelodysplastic syndrome (ELASTIC)

Cancer type:

Blood cancers
Myelodysplastic syndrome (MDS)




Phase 1

This trial looked at combining eltrombopag with azacitidine for people with myelodysplastic syndrome (MDS).

This trial was open for people to join between 2014 and 2018. The team reported the results in 2021.

More about this trial

Doctors can treat myelodysplastic syndrome (MDS) with azacitidine. This works but doctors are always looking for ways to improve treatment.

Eltrombopag is a drug that increases the number of cells called platelets Open a glossary item in your blood. We knew from research that a drug like eltrombopag taken with azacitidine helped people with MDS.

The researchers wanted to find out if eltrombopag taken with azacitidine can do the same. To do this, they first needed to find the best dose to give.

The main aims of this trial were to find out:

  • the highest, safest dose of eltrombopag to give with azacitidine
  • what the side effects are of having these 2 drugs together

Summary of results

The team found the best dose of eltrombopag to give with azacitidine. 

Trial design
This was a phase 1 Open a glossary item  study. Everyone had eltrombopag. 31 people took part. 

The first few people had a low dose of eltrombopag with azacitidine. If they didn’t have any serious side effects, the next few people had a higher dose. And so on, until they found the best dose to give. 

Overall 17 people of the 31 people completed their treatment of eltrombopag and azacitidine. And 14 people didn’t. 

Reasons for not completing the treatment were:

  • their cancer getting worse
  • side effects
  • that some people died due to their cancer 

The team found that they could increase the dose of eltrombopag to the highest one without any of the side effects becoming too bad. 

Side effects
The side effects reported most often were:

  • constipation or diarrhoea
  • feeling or being sick
  • a drop in blood cells
  • chest pain
  • tummy (abdominal) pain as well as other problems with their stomach and bowels
  • tiredness and lack of energy (fatigue)
  • reactions at the injection site such as pain and redness
  • swollen legs and arms
  • high temperature (fever)
  • lung infection, skin infection and other infections
  • dizziness, confusion and falls
  • high level of potassium in the blood
  • shortness of breath
  • nose bleeds
  • skin rash, itching

The team concluded that the combination of eltrombopag and azacitidine was safe and acceptable. 

They noted that a  large phase 3 trial Open a glossary item looked at this combination and found some concerns about how safe it is and how well it works. Their conclusion was it shouldn’t be standard treatment Open a glossary item  for people with MDS.  

Where this information comes from    
We have based this summary on information from the research team. As far as we are aware, the information they sent us has not been reviewed independently (peer reviewed Open a glossary item) or published in a medical journal yet. The figures we quote above were provided by the research team. 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 Alex Sternberg

Supported by

Blood Cancer UK
Experimental Cancer Medicine Centre (ECMC)
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

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"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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