Pioneering discoveries take center stage at EHA2025
As hematologists from across the globe gather in Milan for the 30th Congress of the European Hematology Association (EHA2025), six studies selected for this year’s press briefing underscore the event’s reputation as a platform for transformative research.
From novel immunotherapy targets and clinical trial data to practice-changing insights into rare diseases, these studies capture the diversity and innovation that define modern hematology.
SLAMF6 blockade reinvigorates T Cell response
To date, immune checkpoint blockade in acute myeloid leukemia (AML) has failed to deliver durable clinical responses, but new research from Lund University and Skåne University Hospital may change that. Dr. Carl Sandén and colleagues identified SLAMF6 as a dominant immune checkpoint active in 60% of AML cases. In preclinical models, blocking SLAMF6 using the antibody TNC-1 unleashed potent T cell responses without harming normal stem cells.
“Our study shows that SLAMF6 blockade could make AML finally responsive to immunotherapy,” said Dr. Sandén.
These results position SLAMF6 as a next-generation immune checkpoint target with potential to expand the effectiveness of immunotherapy in AML.
Largest study to date sheds light on rare MPL-mutant essential thrombocythemia
Essential thrombocythemia (ET) is a rare myeloproliferative neoplasm mostly driven by mutations in JAK2 or CALR. However, 3–5% of patients carry mutations in MPL and consequently, studies on MPL-mutant ET have been limited to small cohorts. A landmark multinational study led by Prof. Steffen Koschmieder (RWTH Aachen University) has filled that gap by profiling outcomes in over 300 patients across 37 institutions. While survival was comparable to other ET subtypes, the study found distinct clinical features and risk profiles.
“This collaborative effort brings clarity to a rare and poorly understood group of patients,” said Prof. Koschmieder.
These data lay the foundation for new risk scoring systems and treatment guidelines specifically for MPL-mutant ET patients.
A new standard for relapsed/refractory DLBCL
Treatment options are limited for patients with relapsed or refractory diffuse large B-cell lymphoma (R/R DLBCL). The global Phase III POLARGO trial led by Prof. Matthew Matasar (Rutgers Cancer Institute) offers a major advance and showed that the Pola-R-GemOx regimen (polatuzumab vedotin + chemotherapy) cut the risk of death by 40% versus standard R-GemOx.
“This represents a meaningful step forward for patients with few alternatives,” said Prof. Matasar.
With consistent efficacy across DLBCL subtypes and a manageable safety profile, Pola-R-GemOx may emerge as a go-to strategy for transplant-ineligible patients.
Clinical trial establishes post-transplant cyclophosphamide as superior GVHD prophylaxis in matched sibling stem cell transplants
The Australasian BM12 CAST trial, presented by Prof. David Curtis (Alfred Health and Monash University), has upended assumptions about graft-versus-host disease (GVHD) prophylaxis. Post-transplant cyclophosphamide (PTCy) combined with cyclosporin significantly outperformed the standard methotrexate-based regimen in matched sibling transplants.
“This combination significantly prolonged GVHD-free, relapse-free survival and established a new benchmark for matched related donor transplantation,” said Prof. Curtis.
The simplicity and efficacy of this regimen may have immediate implications for clinical practice and position it to become the new standard of care for GVHD prophylaxis in matched sibling transplants.
Minimal risk of blood complications from common diabetes and heart failure medications
Drug safety is an important topic with the widespread use of SGLT-2 inhibitors in diabetes, heart failure, and kidney disease. A real-world Korean study led by Dr. Ji Yun Lee (Seoul National University Bundang Hospital) found that while 17% of patients developed erythrocytosis, related complications like thrombosis were rare.
“These findings provide important reassurance to clinicians and patients using SGLT-2 inhibitors,” said Prof. Lee.
Further studies are needed to explore long-term risks, but current data support the continued use of SGLT-2 inhibitors without undue concern about erythrocytosis-related complications.
Mitapivat expands its promise to rare anemias
Mitapivat is approved for pyruvate kinase deficiency and is now showing potential in other rare hereditary anemias. The SATISFY Phase 2 study, presented by Dr. Thomas Doeven (University Medical Center Utrecht), showed significant hemoglobin gains and reduced hemolysis in patients with hereditary spherocytosis, xerocytosis, and CDA II.
“Mitapivat’s ability to enhance red cell metabolism and energy production has now shown real promise beyond its previously approved indications, particularly for these patients who face limited treatment choices,” said Dr. Doeven.
The SATISFY trial is part of a broader effort under EuroBloodNet to advance therapies for rare hematological diseases. Further studies, including a sibling study in Canada, are planned to expand and confirm these findings.
A Congress that reflects a changing field
EHA2025 serves as a vital meeting point for scientific discovery and strategic advancement, bringing together experts from diverse disciplines to shape the future of hematology. The event highlights the growing intersection of rare diseases, groundbreaking clinical trials, and innovative treatments, reinforcing EHA’s commitment to improving lives worldwide.
With more than 185 sessions, 2,000 posters, and dedicated spaces such as the EHA Theater and Meeting Hub, this year’s Congress emphasizes collaboration and the rapid translation of research into patient benefits. By fostering discussion and connection, EHA2025 ensures that new scientific findings swiftly impact clinical practice and improve patient care.