Who can enter
- Children with Down syndrome and myeloid leukemia
- Age: 6 months up to 6 years
Goal
The goal of this study is to demonstrate that two modifications of standard treatment provide equally good treatment outcomes with fewer side effects for patients.
Background
Myeloid leukemia is a form of leukemia with a relatively high incidence in young children with Down syndrome. Previous studies on the treatment of leukemia in children with Down syndrome have shown that the leukemia is well curable. But children with Down syndrome also appear to be more susceptible to the side effects of treatment compared to children without Down syndrome.
Currently, children with myeloid leukemia and Down syndrome are treated according to the ML-DS 2006 protocol. The treatment consists of four cycles, in which different drugs are given. In total, the treatment lasts about 16 weeks.
This treatment protocol has given good results, but also still causes many side effects. Therefore, we want to investigate whether we can moderate the treatment so that there are fewer side effects, while the result of the treatment remains just as good. In the ML-DS 2018 study, we are adapting the treatment in two ways:
- We are using a new drug during cycle 1 and cycle 2.
- We give children who respond well to the first cycle a lower dose during the fourth cycle.
1) The drug CPX-351, also known by the brand name Vyxeos ®, is a new drug. It is registered and approved in the Netherlands to treat adult patients with myeloid leukemia. It is not yet registered for the treatment of children.
CPX-351 is a combination of two well-known agents (daunorubicin and cytarabine) that were previously used separately in the treatment of children with leukemia. These drugs are now "packaged" together in very small fatty spheres (liposomes). This packaging of the drugs in liposomes allows them to enter the blood much more gradually. This may allow them to have a longer-lasting effect in the body and cause fewer or less serious side effects. In particular, we expect CPX-351 to cause fewer infections and fewer side effects to the heart.
2) It has been shown that children with a good response to the first cycle have a much better chance of being cured. To do this, after cycle 1, the very small amount of residual disease in the child is measured. This is also called the "Minimal Residual Disease" or "MRD" . In addition, we have seen that many serious side effects are caused by the high dose of the drug cytarabine during the fourth cycle.
Therefore, in this study, we give children who respond well to the first cycle a lower dose of the drug cytarabine during the fourth cycle.