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A Collective Journey Toward Innovation in Myelodysplastic Syndromes

December 7, 2024
Gray Magee, MD, @GrayMageeMD, and Manisha Bhutani, MD, @manisha_bhutani
Atrium Health Levine Cancer Institute, Charlotte, NC

It’s often said that the power of one reflects the strength of many. Since its inception in 2005, the joint symposium of ASH and the European Hematology Association (EHA) has served as a bridge for transcontinental collaboration, uniting expertise across the Atlantic to present a cohesive and unified approach — exemplifying the principle that we achieve more together than we ever could apart. Today’s annual ASH-EHA Joint Symposium (12:30 p.m. - 1:30 p.m., Convention Center, Hall B) will focus on the prognostication, risk stratification, and management of myelodysplastic syndromes (MDS). Co-chaired by ASH President Mohandas Narla, DSc, and EHA President Antonio Median Almeida, MD, PhD, the session aims to spotlight the advances that could transform our understanding and treatment of MDS.  

In recent years, advances in massively parallel DNA sequencing have deepened our understanding of the mutation-driving genes and biological pathways underlying the onset and progression of MDS. By integrating mutational analysis into clinical practice, the classification of this disease has been refined, defining clinical entities with distinct molecular profiles and outcomes. This approach has not only improved individual prognostic assessments but also laid the groundwork for personalized treatment strategies for both lower-risk and higher-risk disease groups.  

“The substantial advances in basic science, translational research, and new therapeutics make MDS an exciting area to explore,” said Dr. Narla. “We believe this topic will be of broad interest to attendees of the annual meeting.”  

Matteo Giovanni Della Porta, MD, will discuss the evolving standard of care for the treatment of lower-risk MDS and explore some of the most promising next-generation agents under investigation. While existing scoring systems categorize patients into multiple risk groups, treatment decisions are typically based on two broad categories — lower-risk and higher-risk MDS. “Striking the right balance between efficacy and quality of life is crucial in treatment selection,” said Dr. Della Porta. “The goal is to improve anemia, reduce transfusion dependence, enhance quality of life, and delay progression to leukemia — all while minimizing the need for intensive treatments.”  

“After years of limited therapies, the approval of targeted treatments like azacitidine and decitabine for higher-risk MDS — and lenalidomide, luspatercept, and imetelstat for lower-risk MDS — has provided new hope for improved patient outcomes,” Dr. Della Porta added. 

Luspatercept, which targets ligands of the transforming growth factor beta pathway to promote red cell maturation, has replaced erythropoiesis-stimulating agents (ESAs) as a first-line treatment for severe anemia. Imetelstat, which inhibits telomerase, offers durable transfusion independence for heavily transfused patients who are unresponsive to or ineligible for ESAs and is now approved for this indication. 

Amy E. DeZern, MD, will discuss the latest breakthroughs in MDS prognostication and risk stratification, demonstrating how a personalized approach to assessing risk can help improve patient outcomes. “A precise characterization of both the genomic complexity of the malignant clone and immune dysfunction in each patient will significantly improve the prediction of disease evolution, clinical outcomes, and drug sensitivity — ultimately improving clinical management and fostering new therapeutic approaches for patients with unmet needs,” Dr. DeZern said. 

In this rapidly evolving treatment landscape, Dr. DeZern also stressed the importance of incorporating quality-of-life measures and patient-reported outcomes. “Evaluating the risk-benefit profile of new therapies requires considering not just survival, but how well patients live,” she said. “A modern, personalized approach must prioritize patient-centered care, ensuring that therapies not only improve longevity but also enhance quality of life, with careful monitoring of treatment benefits at an individual level.” 

The integration of large-scale data initiatives and multicenter clinical trials will be key to accelerating discovery and translating new therapeutic paradigms into clinical practice. This session is not just an opportunity to learn — it’s also a call to action for the future of MDS treatment. 

"We anticipate that the relationship will continue to thrive, as hematology knows no regional boundaries," Dr. Narla said. "The cooperation between the two societies, built on mutual respect and the recognition of the talented individuals on both sides, reinforces our belief that collaboration, rather than competition, not only benefits both organizations but also advances global hematology efforts." 

When the power of one is united with the power of many, we can transform the trajectory of MDS research and patient care. 

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