A trial looking at treatment for children and young people with Langerhans Cell Histiocytosis (LCH-III)

Cancer type:

Children's cancers




Phase 3

This trial was trying to improve treatment for children and young people with Langerhans cell histiocytosis (LCH). The trial was for children and young people up to and including the age of 17.

LCH is a very rare condition that affects both children and adults. Langerhans cells are a type of white blood cell called a histiocyte Open a glossary item, They help to fight infection. Langerhans cells are normally found in the skin and the main airways. A person with LCH has too many Langerhans cells and they spread to different tissues and organs in the body, causing symptoms.

Although LCH is not cancer, it can behave in a similar way to cancer and is usually treated by cancer specialists. The behaviour of LCH and its treatment varies a lot. Some children do not need treatment while others may need chemotherapy, with or without steroids.

Doctors wanted to develop new treatments for all children with LCH. These could then be compared to other available treatments.

This trial was trying to find out if

  • Some children would benefit from treatment over a year, rather than 6 months
  • Adding methotrexate to treatment would help to stop LCH coming back in other children

Summary of results

Some early results from this trial suggest that Langerhans cell histiocytosis was less likely to come back in children who had treatment for a year than in those who had treatment for 6 months. In another group of children, the early results showed that adding methotrexate to treatment did not make a difference to the average length of time before LCH came back.

The trial recruited 2 different groups of children and young people. Those in group 1 were considered to be high risk, as the LCH was affecting body organs such as the liver Open a glossary item, spleen Open a glossary item, lungs Open a glossary item or bone marrow Open a glossary item.

The children in group 2 were considered to be low risk because although the LCH was affecting more than 1 part of their body, it was not in any of the organs listed above.

Children in group 1 were put into 2 further groups at random. This is called randomisation.

Early findings from this study show that children in both groups had similar responses to treatment. Adding methotrexate did not increase the initial response rate nor change the average length of time children with high risk LCH lived without the disease coming back.

All the children in group 2 had treatment with vinblastine and prednisolone. But they were also randomised to 2 further groups. Some had treatment for 6 months, some carried on for a year.

The early findings suggest that having treatment for a year may increase the amount of time before the LCH came back in these children with low risk LCH.

More studies are being planned to improve treatment for all children with Langerhans cell histiocytosis.

We have based this summary on information from the team who ran the trial.  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 trial team. We have not analysed the data ourselves.

Recruitment start:

Recruitment end:

How to join a clinical trial

Please note: In order to join a trial you will need to discuss it with your doctor, unless otherwise specified.

Please note - unless we state otherwise in the summary, you need to talk to your doctor about joining a trial.

Chief Investigator

Kevin Windebank

Supported by

Cancer Research UK Children's Cancer Trials Team
University of Birmingham
National Institute for Health Research Cancer Research Network (NCRN)

Questions about cancer? Contact our information nurses

Freephone 0808 800 4040

Last review date

CRUK internal database number:

Oracle - 760

Please note - unless we state otherwise in the summary, you need to talk to your doctor about joining a trial.

Katie took part in a new clinical trial

A picture of Katie cycling

"We believed that with the clinical trial, Katie had the best chance of recovery. Without these trials, amazing new treatments may never be found."

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