For children with leukaemia and Down’s syndrome
Children with Down’s syndrome have a 150 times higher risk of developing myeloid leukaemia (ML) at the age of up to 5 years compared to their peers without Down’s syndrome (DS). In comparison to children with cancer who do not suffer from Down’s syndrome, they have a better prognosis, but are much more sensitive to cytotoxic drugs. This increased risk of complications during therapy has led to deaths in the past and a generally higher mortality rate during treatment.
“The collaboration and exchange in an international research network is important, especially with particularly rare diseases such as ML-DS. Thanks to uniform therapy guidelines, quality-assured diagnosis and treatment as well as a uniform therapy standard for a rare disease can be ensured, even if the treating clinic has little experience with this specific disease so far.”
This is why the ML-DS 2018 trial aims to reduce the toxicity of chemotherapy without jeopardising the good results of the therapy. The therapy approach is to replace some of the conventional chemotherapy with the newly developed medication CPX-351. In this process, the active substances are packaged in tiny fat globules (liposomes) in order to take them specifically to the leukaemia cells and at the same time reduce the side effects of the substances. When patients react very well to the therapy at an early stage, their dosage is reduced in the last stage of chemotherapy. In this way, therapy-related mortality should ultimately be reduced without compromising the overall good prognosis.
Point your browser to the ML-DS 2018 trial for more information (in German).