While children with ML-DS generally achieve excellent cure rates, they are much more susceptible to treatment toxicities than other patients. To overcome this the ML-DS 2018 trial aimed to main current treatment outcomes while reducing therapy intensity and with that also treatment related toxicities. In this study CPX-351 replaced conventional induction therapy.

While CPX-351 showed excellent tolerability and no treatment-related mortality, the relapse rate was unexpectedly higher, leading to lower EFS than in the previous ML-DS 2006 trial.

Importantly due to optimized relapse therapy, the OS remained comparable, demonstrating how advances in salvage strategies can sustain high cure rates even in highly sensitive patient groups

Find the publication at:

Laszig, S., Diedrichs A. et al., CPX-351 in Down Syndrome-associated Myeloid Leukemia: Results and Prognostic Factors from the Phase III ML-DS 2018 Trial, Blood 2025; nlood.2025030775. doi: Https://doi.org/10.1182/blood.2025030775