This trial showed that having etoposide, dexamethasone and cyclosporin as initial treatment was not better for people with haemophagocytic lymphohistiocytosis (HLH).
This trial recruited patients between 2004 and 2011, and the results were reported in 2017.
Results
The trial recruited 369 children and young people from 27 different countries. Of those taking part:
- 168 had primary HLH
- 201 had secondary HLH
Everyone taking part had etoposide, dexamethasone and cyclosporin as initial treatment. Some people went on to have a
stem cell transplant.
The research team looked at how many people were living 5 years after joining the trial. They found it was just over 6 out of 10 people (61%). When they looked at the different types of HLH they found it was:
- just under 6 out of 10 people (59%) who had primary HLH
- more than 6 out of 10 people (64%) who had secondary HLH
The number of people living at 5 years in the HLH 94 trial was more than 5 out of 10 (54%). So there was not much difference between the two.
Of the 188 people who went on to have a stem cell transplant, 121 (66%) lived for at least 5 years. This is the same percentage as the HLH 94 trial.
Side effects
The treatments used in this trial have all been used to treat cancer, and other conditions, for a number of years. The people taking part in this trial didn’t have any unexpected side effects.
A few people had serious side effects such as:
- problems with the brain which may cause fits or learning difficulties
- problems caused by high blood pressure
- liver problems
- infections
Conclusion
The trial team concluded that it was not better to include cyclosporin in initial treatment for HLH. They recommend that children and young adults with HLH continue to have etoposide and dexamethasone at the start of treatment.
We have based this summary on information from the research team. The information they sent us has been reviewed by independent specialists (
peer reviewed 
) 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.