A trial looking at venetoclax for acute myeloid leukaemia (VIALE-C)

Cancer type:

Acute leukaemia

Acute myeloid leukaemia (AML)

Blood cancers

Leukaemia

Status:

Results

Phase:

Phase 3

This trial looked at adding venetoclax to low dose cytarabine for acute myeloid leukaemia (AML).

It was for people who:

hadn’t had treatment and
weren’t suitable to have intensive chemotherapy to get rid of the AML

This trial was open for people to join between 2017 and 2018. The team published the results in 2020.

More about this trial

When this trial was done, the main treatment for AML was intensive chemotherapy. This may have included 2 chemotherapy drugs:

cytarabine
daunorubicin

Some people weren’t fit enough to have this treatment so they had low dose cytarabine instead. Doctors wanted to improve treatment for this group of people. In this trial they looked at adding a drug called venetoclax.

Venetoclax is a type of targeted drug Open a glossary item . It works by blocking a protein found in AML cells. This causes the AML cells to die.

In this trial, some people had low dose cytarabine and venetoclax. And some had low dose cytarabine and a dummy drug (placebo Open a glossary item ).

The main aims of this trial were to find out:

how well venetoclax and low dose cytarabine works
what the side effects are
how this combination of treatment affects quality of life

Summary of results

The trial team found that adding venetoclax to low dose cytarabine improved treatment results. It helped to destroy the leukaemia cells. This is called remission Open a glossary item . It also increased the length of time people lived compared to having low dose cytarabine on its own.

Trial design
This phase 3 trial took place worldwide. 211 people joined the trial and 210 had treatment. The median Open a glossary item age of people taking part was 76.

2 out of every 3 people had low dose cytarabine and venetoclax. 1 out of every 3 people had low dose cytarabine and a dummy drug. People were put into a treatment group at random:

142 had low dose cytarabine and venetoclax
68 people had low dose cytarabine and a dummy drug

viale-c.png

Results
The team looked at how well treatment worked. At a time where people are followed for one year on average, they looked at the length of time people lived. They found it was about:

7.2 months on average for those who had low dose cytarabine and venetoclax
4.1 months on average for those who had low dose cytarabine and the dummy drug

There is a difference between these two groups. The difference is not big enough for the trial team to say for sure whether it was because of the different treatments.

The team did another analysis 6 months later. They relooked at how long people lived. They found it was about:

8.4 months on average for those who had low dose cytarabine and venetoclax
4.1 months on average for those who had low dose cytarabine and the dummy drug

viale-c_1.png

The team also looked at who had no signs of AML in their bone marrow Open a glossary item . They found this was:

39 out of 143 people (27%) who had venetoclax and low dose cytarabine
5 out of 68 people (7%) who had the dummy drug and low dose cytarabine

They also looked at who had no signs of AML in their bone marrow but had abnormal blood counts Open a glossary item . This was:

68 out of 143 people (48%) who had venetoclax and low dose cytarabine
9 out of 68 people (13%) who had the dummy drug and low dose cytarabine and

The team also found that people who had venetoclax needed fewer blood transfusions.

Quality of life
The research team asked people taking part to fill out a quality of life Open a glossary item questionnaire before, during and after treatment.

People who had venetoclax and low dose cytarabine said their levels of tiredness got much better with treatment. Those who had only low dose cytarabine said their tiredness got a little bit better.

People who had venetoclax also said they had a better quality of life than those who didn’t.

Side effects
Most people who took part had at least one side effect. Some were mild or didn’t last very long. Some people had side effects that were more severe.

The most common severe side effects of venetoclax included:

a drop in the number of a specific type of white blood cell which can cause an increased risk of infection
an increased risk of bleeding

Similar numbers of people in both treatment groups had severe problems with:

a drop in a specific type of white blood cell which can cause an increased risk of infection (febrile neutropenia)
lung infections that may cause cough, fever, shortness of breath, sharp or stabbing chest pain, loss of appetite, low energy, and tiredness (fatigue) or confusion

The team say these side effects were manageable.

We have more information about the side effects of venetoclax.

Conclusion
The trial team concluded that adding venetoclax to low dose cytarabine improved treatment for people in this trial. And they found the side effects weren’t too bad.

Adding venetoclax got rid of the leukaemia in more people. It also increased the length of time this group of people lived. The team suggest that the combination might be a useful treatment option for people who can’t have intensive chemotherapy.

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.