Low-dose cytarabine reduces mortality in TMD patients

Approximately 5-10% of children with Down syndrome (DS) are diagnosed with transient myeloproliferative disorder (TMD). The majority of these children are asymptomatic and go into spontaneous remission without therapeutic intervention. However, 20% die within 6 months (early death), and another 20% progress into myeloid leukemia (ML-DS) within their first 4 years of life. The aim of the TMD Prevention 2007 trial was to evaluate whether low-dose cytarabine treatment can prevent ML-DS in patients with TMD. The results of this study have now been published in Blood Advances: “Low-dose cytarabine to prevent myeloid leuemia in children with Down syndrome: TMD Prevention 2007 study“. A key finding was that low-dose cytarabine reduced mortality in symptomatic TMD patients when compared to the historical control. However, this treatment did not prevent progression to ML-DS.

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