A trial of givinostat for polycythaemia vera

Cancer type:

Blood cancers

Polycythaemia

Status:

Results

Phase:

Phase 1/2

This trial looked at a drug called givinostat to treat a blood disorder called polycythaemia vera.

Polycythaemia vera is a myeloproliferative disorder Open a glossary item . This means a condition in which the bone marrow produces too many blood cells. In polycythaemia, this is too many red blood cells .

This trial was open for people to join between 2014 and 2017. The team published the results in 2020 and 2021.

More about this trial

Givinostat is a drug that blocks proteins (enzymes Open a glossary item ) in the body called histone deacetylases (pronounced dee-as-et-isle-azes). Cells need these to grow and divide.

Blocking them may stop too many red blood cells being made. Drugs that block these enzymes are called histone deacetylase inhibitors or HDAC inhibitors.

We know from research that givinostat can help people with myeloproliferative disorders such as polycythaemia.

The aims of this trial were to find out:

the best dose of givinostat to treat polycythaemia
how well givinostat works for people with polycythaemia
what the side effects are

Summary of results

The team found that givinostat helped people with polycythaemia.

Trial design
This was a phase 1/2 trial. 54 people took part. The majority of the people had polycythaemia. The trial also included people with other types of myeloproliferative disorder (MPN).

This trial had 2 parts. In the first part the team found what the best dose of givinostat was for people with polycythaemia. The best dose is the highest dose with the fewest side effects.

In the second part everyone had the best dose of givinostat.

Results
After an average follow up of 4 years the team looked at how well givinostat had worked. They looked at the number of people whose MPN:

had gone away (a complete response ) or
had got a bit better (a partial response )

They found that over 8 out of every 10 people (80%) had either a complete response or a partial response.

The team also looked at the side effects. They found that:

the majority were mild to moderate
some were severe
none were life threatening

The most common side effects were:

a drop in red blood cells or white blood cells
a drop in the platelets
problems with the digestive system such as a dry mouth or indigestion
diarrhoea
changes to blood test results that show how well the kidneys are working
skin problems such as itching

The team considered the majority of side effects reported were not due to taking givinostat long term.

Conclusion
The team concluded that givinostat helped people with polycythaemia and other types of MPN. They say that the side effects were acceptable.

They also conclude that these results support the use of long term givinostat for these people.

More detailed information
There is more information about this research in the references below.

Please note, these articles are not in plain English. They have been written for health care professionals and researchers.

Journal articles
Safety and efficacy of the maximum tolerated dose of givinostat in polycythemia vera: a two-part Phase Ib/II study

Alessandro Rambaldi, Alessandra Iurlo and others.

Leukemia. Year 2020. Volume 34. Pages 2234–2237.

Long-term safety and efficacy of givinostat in polycythemia vera: 4-year mean follow up of three phase 1/2 studies and a compassionate use program

Alessandro Rambaldi, Alessandra Iurlo and others.

Blood Cancer Journal. Year 2021. Volume 11. Issue 53. Pages 1-7.

Where this information comes from
We have based this summary on the information in the articles above. These have been reviewed by independent specialists (peer reviewed) Open a glossary item and published in medical journals. We have not analysed the data ourselves. As far as we are aware, the links we list above are active and the articles are free and available to view.