Award Recognizes Professor Khaled Musallam鈥檚 Valuable Contributions and Pioneering Work on Thalassemia Treatment听
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Khalifa University Adjunct Professor Khaled Musallam has won the 2025 Clinical Excellence Award from the European Hematology Association (EHA) for his achievements and commitment to clinical research鈥�, with valuable contributions and pioneering work.听
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The award was presented to him during the Opening Ceremony of the Association鈥檚 annual congress held in Milan, Italy. EHA is one of the largest hematology organizations in the world, promoting excellence in patient care, research, and education.听
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Professor Musallam is also Group Chief Research Officer, Burjeel Holdings, and is based in Burjeel Medical City in Abu Dhabi. He is also the Founding Director of the Center for Research on Rare Blood Disorders (CR-RBD), Director of the Thalassemia and Sickle Cell Center, and Head of the Innovative Trials Unit at the Genetics and Rare Disease Center. In addition, Professor Musallam is Global Research Lead at Burjeel Institute for Global Health in New York and Adjunct Professor at Weill Cornell Medicine in New York.听
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As a student, Professor Musallam focused his scientific inquiry on thalassemia, a genetic blood disorder characterized by chronic severe anemia. He has worked in both academia and industry across multiple organizations in Europe and the Middle East.听听
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Professor Musallam has fostered extensive collaborations both regionally and globally towards novel drug development. His work was mainly focused on setting up and leading large global datasets of individuals with thalassemia to identify the relationship between modifiable risk factors and long-term morbidity and mortality outcomes, which can be targeted by treatment optimization or novel therapies.听
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Professor Musallam鈥檚 work helped characterize the various forms of thalassemia with respect to transfusion dependence, leading to a shift in patient categorization globally. His work enabled the medical community to notice the detrimental effects of untreated anemia in non-transfusion-dependent patients (NTDT), including serious complications and early death. This was the basis for the development of two novel disease-modifying therapies 鈥� luspatercept and mitapivat, the first-ever to target the underlying disease in these patients. The therapies have been evaluated in global clinical trials with extensive involvement from Professor Musallam as principal investigator. He is also a member of the steering committee, and key advisor for regulatory filing with the US Food and Drug Administration, and the European Medicines Agency.听
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Professor Musallam鈥檚 studies were the first to provide clinical proof of principle on the correlation between iron overload and serious clinical complications of NTDT, affecting almost all organ systems. He identified thresholds which were later used on the design of oral iron chelation therapy (drugs that remove excess iron from the body) trials in NTDT and to define prescribing information and international management guideline recommendations for these agents. Collectively, his work provided a new treatment landscape for NTDT patients by uncovering these detrimental risk factors for poor patient outcomes and supporting the development of novel agents to address them.听
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Professor Musallam has made various practice-changing contributions to advance knowledge in transfusion-dependent individuals (TDT) that has helped quantify the risk of suboptimal treatment and its impact. His studies linking natural increases in fetal hemoglobin level and favorable patient outcomes supported clinical proof of principle for advances in gene editing, which has recently become a reality.听听
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A highly respected author with over 230 publications in leading peer-reviewed international medical journals, Professor Musallam鈥檚 expert contributions remain amongst the most recognized and cited in the field. He is also the Editor of the first-ever NTDT management guidelines by the Thalassemia International Federation and the latest TDT management guidelines, which are the prime resources used by treating physicians worldwide for patient care.听听
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Professor Musallam said: 鈥淭his award is a celebration of all the successful collaborations I have had throughout my career, especially with mentors and mentees who eventually became close colleagues and friends from all over the world. We were all joined by a single cause of improving the lives of patients living with a devastating disease. The support and resources made available to me in the UAE over the past five years have surely helped accelerate my research programs and allowed me to put our region on the international map of research and development. With the EHA award, we are inspired to keep the momentum going for advancing knowledge and discovering new treatment options.鈥澨�
Clarence Michael
English Editor – Specialist
