Post by Jan-Henning Klusmann

🚨 New publication in Blood 🚨 Our research group is proud to share that the Phase III ML-DS 2018 trial evaluating CPX-351 in Down syndrome–associated myeloid leukemia (ML-DS) has just been published online in Blood Journals Portfolio: https://lnkd.in/efDNk9wT Children with ML-DS generally achieve excellent cure rates but are highly susceptible to treatment toxicity. The ML-DS 2018 study aimed to maintain these outcomes while further reducing therapy intensity. In this multicenter prospective trial, 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 a lower event-free survival compared with the previous ML-DS 2006 trial. 👉 Importantly, thanks to optimized relapse therapy, the overall survival remained comparable — demonstrating how advances in salvage strategies can sustain high cure rates even in this highly sensitive patient group. A big thank you to the entire study team — especially Stephanie Laszig, Emila Salzmann, and Antonia Diederichs — and all national and international collaborators for their tremendous work driving this study to publication. We are deeply grateful to all participating hospitals the study, and above all, to the patients and their families, whose trust and participation make this research possible. 💛 We are also deeply grateful to Jazz Pharmaceuticals and to the Hilfe für krebskranke Kinder Frankfurt e. V. für krebskranke Kinder Frankfurt e.V. for their invaluable support. 💛 #PediatricOncology #LeukemiaResearch #DownSyndrome #CPX351 #Blood Prof. Dr. Dirk Reinhardt Katharina Waack Helena Kerp José Gonçalves Dias Hasan Issa Wehner Sibylle Hermann Kreyenberg Melanie Bremm Sabine Hünecke Felicitas Thol Bianca Goemans Katarzyna Pawińska-Wąsikowska Goethe-Universität Frankfurt