The path to these results began more than a decade ago. From 2005 to 2011, a pilot study for the first time investigated whether treatment could be administered in a targeted manner when molecular blood tests indicate an impending relapse of the blood cancer at an early stage, even before clinical symptoms appear. This concept of MRD-guided treatment was initially tested in AML patients following allogeneic stem cell transplantation and was later expanded to include patients with NPM1-mutated AML who had undergone conventional therapy.
The findings from these early studies ultimately led to the multi-center RELAZA2 trial, which was designed to evaluate the efficacy of this treatment approach. The first results were published in 2018. Now, comprehensive long-term data are available for the very first time.
"When we began with the first MRD-guided approaches twenty years ago, it was still unclear whether this would actually lead to a new therapeutic pathway," said Prof. Uwe Platzbecker, Medical Director of the University Hospital Dresden (UKD) and one of the program's initiators. "The RELAZA2 long-term data now shows that early intervention based on molecular markers has the potential to significantly influence the course of the disease."
A key component of the research program was precise molecular diagnostics. "The ability to reliably detect even minimal residual disease has fundamentally changed the treatment of AML and MDS," explained Prof. Christian Thiede, laboratory director of the study at Medical Clinic I at UKD "Today, MRD is no longer just a prognostic marker, but can be used to guide therapeutic decisions."
The project also presented an extraordinary scientific and organizational challenge for the study coordinators. "The RELAZA studies represent a long-term, close collaboration among many centers," said Dr. Anne‑Sophie Platzbecker, the study's first author. "Without the dedication of the participating teams and, above all, without the trust of the patients, this development would not have been possible."
The studies were conducted primarily within the framework of the Study Alliance Leukemia (SAL) , a network of leading German leukemia centers. "This work impressively demonstrates how clinical research can yield new treatment strategies through cooperation over many years," emphasized Prof. Martin Bornhäuser, Director of Medical Clinic I at the UKD and one of the Managing Directors of the National Center for Tumor Diseases (NCT/UCC) Dresden . "It provides an essential foundation for future studies and personalized treatment approaches."
Prof. Esther Troost, Dean of the Faculty of Medicine at TUD Dresden University of Technology , explained: "Oncology has always been one of the core research areas at the Faculty of Medicine at TU Dresden. Studies such as RELAZA2 serve as a prime example of how closely basic research, translational approaches, and clinical application are intertwined at our institution. "Since 2015, the NCT/UCC Dresden has had this unique structure, whose success is demonstrated by studies such as these."
The publication of the long-term data marks the conclusion of a significant chapter in translational leukemia research. However, the researchers do not see this as a final conclusion, but rather as a starting point for further studies intended to further develop the concept of MRD-guided therapy and integrate it even more precisely into clinical practice. Prevention has arrived in leukemia therapy.
Background on the disease:
Myelodysplastic syndrome (MDS) is a precursor to leukemia and is one of a group of bone marrow disorders in which the production of healthy blood cells is impaired. Acute myeloid leukemia (AML) is a particularly aggressive form of blood cancer that usually progresses rapidly if swift and intensive treatment is not applied.
About the study:
The study project was designed under the sponsorship of TUD Dresden University of Technology and in cooperation with Dresden's Coordination Center for Clinical Studies by the Clinical Studies Department of the Medical Clinic and Polyclinic I at Dresden University Medicine and implemented in various centers in Germany as part of the academic study group Study Alliance Leukemia (SAL). With over 50 participating centers in Germany and Austria, SAL is one of the two large German AML study groups and is coordinated by its study center in Dresden.
Publication:
"Azacitidine to treat measurable residual disease in patients with MDS/AML: final long-term results of the RELAZA2 trial": https://doi.org/10.1182/blood.2025030816
Editorial: https://www.sciencedirect.com/science/article/pii/S0006497126000601
