A trial looking at treatment for haemophagocytic lymphohistiocytosis (HLH) in children and young people (HLH 2004)

This trial is looking at treatment for children and young people with haemophagocytic lymphohistiocytosis (HLH).

This trial is for children and young people up to and including the age of 18. We use the term ‘you’ in this summary, but of course if you are a parent, we are referring to your child.

HLH is a rare condition that means you produce too many white blood cells called lymphocytes and histiocytes. If it is not treated, these extra cells collect in organs in the body and damage them.

Although HLH is not cancer, it is similar to cancer and usually treated by cancer specialists. There are two different forms of HLH. One is called primary HLH, and is inherited (genetic). The other is called secondary HLH and develops because of a problem with the immune system, such as having an infection.

As so few people have HLH, it is difficult for doctors to develop standard treatments. A trial called ‘HLH 94’ looked at treatment with the chemotherapy drug etoposide and the steroid dexamethasone to begin with, followed by etoposide, dexamethasone and cyclosporin (a drug that dampens down the immune system) after that. This treatment worked quite well, but doctors are always keen to improve treatments.

They think that giving etoposide, dexamethasone and cyclosporin together from the beginning may be better. So that is the treatment in this trial. The aim of the trial is to find out how well this new treatment works.

Please note this trial is no longer recruiting patients.

Recruitment

Start 01/03/2006

End 19/10/2009

Phase

Phase 3

Who can enter

You can enter this trial if you

Have recently been diagnosed with either primary or secondary HLH
Are aged between 0 and 18 years old (inclusive)

You cannot enter this trial if you have had chemotherapy or cyclosporin for HLH before.

Trial design

This is a phase 3 trial. If you take part, you have etoposide as an injection into a vein, dexamethasone tablets and cyclosporin tablets for 8 weeks to begin with. This is called ‘initial therapy’. The aim is for the HLH to go into remission. You may also have methotrexate and hydrocortisone (a steroid) as an injection into your spine (intrathecal injection) if you need it.

After initial therapy, you have etoposide, dexamethasone and cyclosporin, but less often than before. This is called ‘continuation therapy’. The aim is to keep the HLH in remission.

You have continuation therapy until you have a stem cell transplant. The aim of this is to cure HLH in the long term. You have a stem cell transplant as soon as possible, but this depends on how quickly a ‘donor’ is available. The donor can either be a family member, or a non family member.

You are likely to have this treatment for HLH whether you take part in this trial or not. But doctors will collect specific information about you as part of this trial, so they can analyse it to see how well the treatment works.

Hospital visits

You will have some tests before you start treatment as part of this trial. The tests include

Blood tests
Chest X-ray
CT scan
MRI scan

You will be in and out of hospital for some time when you have treatment for HLH. This is true whether you take part in this trial or not. You have blood tests weekly for the first 8 weeks, then 2 weekly for about another 7 months.

You also have an X-ray, CT scan or MRI scan at about 2 months (9 weeks), 6 months (27 weeks), and 9 months (41 weeks).

During the initial therapy, you have treatment twice a week for 8 weeks. After that, during the continuation therapy, you have treatment once every 2 weeks. Your doctor will explain the exact treatment plan to you in more detail.

You will be in hospital for several weeks or so when you have the stem cell transplant. You may be in a single side room (in ‘isolation’) for a time during this stay.

After treatment you will see the doctors once a year. You will have blood tests and an X-ray, CT scan or MRI scan at each visit.

Side effects

As with all treatments, the treatments in this trial have some side effects. The most common side effects of etoposide are

A drop in blood cells causing an increased risk of infection, tiredness, shortness of breath and bleeding or bruising problems
Feeling or being sick
Hair loss
Loss of appetite

Dexamethasone can cause
Mood changes
Trouble sleeping (if taken at night)
Increase in appetite
Weight gain
Fluid retention

The most common side effects of cyclosporin are
High blood pressure
Temporary drop in kidney function
Headache
Feeling or being sick
Loss of appetite
Tiredness

The most common side effects of a stem cell transplant are
A big drop in blood cells causing an increased risk of infection, tiredness, shortness of breath and bleeding or bruising problems
Sickness and diarrhoea
Sore mouth
Tiredness

There is more information about the side effects of etoposide and the side effects of having a stem cell transplant on CancerHelp UK.