PROGRAMS

A multi-centre randomised controlled trial of prophylactic granulocyte-macrophage colony-stimulating factor (GM-CSF) to reduce sepsis in pre-term infants

Can a substance called GM-CSF help reduce the risk of infection in premature babies?

What was this study about?

Premature babies are very vulnerable to infection (neonatal sepsis) and the risk is greatest in babies whose growth, in the womb, has fallen behind. Infections can cause disability or death in premature babies.

The PROGRAMS trial aims to find out whether a substance called GM-CSF (granulocyte-macrophage colony stimulating factor) can reduce infection and improve later health for these premature babies. GM-CSF is a naturally occurring substance that increases the number of white blood cells essential to fight infection.

In this trial, once parents had given their consent, premature babies who were at high risk of developing sepsis were divided at random into two groups. One group of babies were given GM-CSF by injection to see if it could prevent sepsis occurring. The second group were not given a placebo injection. Endpoints were assessed by an independent committee of experts who did not know which group each baby was from - so this was single-blind study.

This trial was undertaken in partnership with the National Perinatal Epidemiology Unit in Oxford.

What difference did this study make?

The results show that a fifth of babies enrolled in the PROGRAMS study had a very low number of white blood cells that fight infection. These babies were at particularly high risk of infection. Babies who received GM-CSF rapidly increased the number of these blood cells, but surprisingly this did not seem to reduce the number of infections. The number of infections was the same in babies who received GM-CSF and those who did not. The same number of babies in the group who received GM-CSF and the group that did not, went home, and their health was similar.

This means that giving GM-CSF to very small premature babies does not provide any benefit in the short term nor does it do any harm. Using GM-CSF will not reduce infections and so it should not be used unnecessarily in small babies. The information gathered from the PROGRAMS study does not provide a solution for the problem of infection, but has pointed the way to other lines of research. Each of these small achievements is a small but important step towards ultimately being able to prevent infection in the future.

Type of study

Randomised trial

Contact details

mrcctu.ctuenquiries@ucl.ac.uk

Who funded the study?

This study received funding from two UK charities - Action Medical Research and the Wellcome Trust.

When did it take place?

This trial began recruiting in 2000. The trial closed to recruitment in 2006.

Where did it take place?

The study took place in 26 hospitals across the UK.

Who was included?

280 premature babies who were at risk of sepsis. They were in 26 hospitals across the UK.