Reduced therapy for Down syndrome myeloid leukemia

It is well known that young children with Down syndrome (DS) have both a greatly increased risk of acute myeloid leukemia (ML-DS) and a decreased tolerance of intensive chemotherapy. In our latest clinical study – a large, multicentered, international, nonrandomized trial reducing treatment intensity in 170 patients of ML-DS – we saw that reduced therapy preserved the excellent outcomes of this cohort observed with historical treatment approaches. Meanwhile, poor early treatment response and gain of chromosome 8 were identified as independent prognostic factors indicating a worse event-free survival. These findings build on international experience demonstrating that most young children with ML-DS can be cured with a reduced therapy, that also reduces the treatment-related cytotoxic effects that these children suffer. In addition, this trial confirms that there remains a subset of patients for whom we have limited treatment options. The findings of this study have been published in Blood Journal: “Therapy reduction in patients with Down syndrome and myeloid leukemia: the international ML-DS 2006 trial”.


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