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

Cancer type:

Blood cancers

Myelodysplastic syndrome (MDS)

Status:

Results

Phase:

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.

Results
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

Conclusion
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 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.