A trial of blinatumomab for children and young people with a certain type of acute lymphoblastic leukaemia

Cancer type:

Acute leukaemia

Acute lymphoblastic leukaemia (ALL)

Blood cancers

Children's cancers

Leukaemia

Status:

Results

Phase:

Phase 2

This trial looked at a drug called blinatumomab for children and teenagers with a type of acute lymphoblastic leukaemia (ALL) called B cell precursor ALL.

More about this trial

Doctors usually treat acute lymphoblastic leukaemia (ALL) with chemotherapy and possibly a stem cell transplant. But sometimes these treatments don’t work or the leukaemia comes back after treatment.

Researchers wanted to find out if blinatumomab could help children and young people in this situation. Blinatumomab (MT103) is type of monoclonal antibody. It targets a protein called CD19 that is often found on leukaemia cells.

People in this trial had blinatumomab continuously into a vein for 4 weeks, and then no treatment for 2 weeks. Each 6 week period is called one cycle of treatment.

There were two parts to this study – phase 1 and phase 2.

In phase 1, the research team wanted to find the best dose of blinatumomab to give to people under 18 years old.

In phase 2, they wanted to find out how well blinatumomab worked for children and teenagers with ALL. It was for people with leukaemia that had not responded to or had come back after other treatment

Summary of results

The research team found the best dose of blinatumomab to give. And found it could be a useful treatment for children and teenagers with B cell precursor ALL.

They recruited patients between 2012 and 2014, and published the results in 2016.

Phase 1 results

This part of the trial recruited 49 people. The first few people had the lowest dose of blinatumomab. They didn’t have any serious side effects, so the next few people had a higher dose. And so on, until they found the best dose to give. This is called a dose escalation study.

The research team found the best dose to give, and this was used in the next part of the trial.

Phase 2 results

This part of the trial recruited 44 people. The research team looked at how well blinatumomab had worked for the people in phase 2. They also looked at 26 people from the phase 1 part of the trial who’d had the same dose.

They found that the leukaemia had gone away in 27 out of 70 people (39%) after 2 cycles (12 weeks) of treatment. This is called being in remission. Two of these people were still in remission 2 years after joining the trial.

Side effects

Blinatumomab did cause side effects. Some were mild or short lived, but some were more severe. The most common side effects were:

a drop in red blood cells, blood clotting cells (platelets) and white blood cells
a drop in blood potassium level, which can cause heart problems

10 people (14%) had a delay between cycles of treatment and 4 people (6%) stopped treatment early because of side effects they were having.

Other trials had shown that blinatumomab can cause a side effect called cytokine release syndrome (CRS). This happens when large amounts of proteins called cytokines are released into the blood. It causes problems such as fever, low blood pressure or a rash. CRS isn’t as common as some other side effects, but it can be quite serious if not treated quickly. In this trial, 8 out of 70 people (11%) had CRS.

Other possibly serious side effects are those that affect the brain and spinal cord (the central nervous system, or CNS). These include problems such as dizziness, having a fit or shaking that you can’t control (a tremor). In this trial, 17 out of 70 people (24%) had a CNS side effect. But most were mild or short term.

Conclusions

The research team concluded that they were able to find the best dose of blinatumomab to give. And that is was a useful treatment for children and teenagers with B cell precursor ALL that had come back or not responded to treatment. They suggest it is looked at further in other trials.

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.