Prof. Khaled Musallam MD PhD is currently the Group Chief Research Officer at Burjeel Holdings in Abu Dhabi, UAE where he founded the clinical research function and operations across the Group’s large hospital network. He is also Chief of Cell & Gene and Hematology, Director of the Thalassemia & Sickle Cell Center, and Founding Director of the Center for Research on Rare Blood Disorder (CR-RBD) at Burjeel Cancer Institute, Burjeel Medical City. Beyond his executive roles, he also leads various clinical trials and real-world evidence studies to offer comprehensive and innovative treatment options for patients with hemoglobinopathies and other hematologic disorders in Abu Dhabi and the wider region.
He is also Head of the Innovative Trials Unit at the Genetics and Rare Disease Center at Burjeel Medical City. Prof. Musallam is Co-Chair of Burjeel Holdings’ Institutional Review Board/Ethics Committee (IRB/EC) and Global Research Lead at the Burjeel Institute for Global Health in New York, USA. He is also Adjunct Clinical Professor at the College of Medicine and Health Sciences at Khalifa University and an external member of the Abu Dhabi Health Research and Technology Committee at the Department of Health in Abu Dhabi, UAE where he supports decisions on clinical trials and grant application approvals. Prof. Musallam is also Adjunct Professor of Hematology Research in Pediatrics (Courtesy) at Weill Cornell Medicine in New York on the pathway recognizing excellence in investigation.
Prof. Musallam received his medical degree from the American University of Beirut in Lebanon and his PhD from the Leiden University Medical Center in the Netherlands. He completed hematology/oncology research fellowships at the American University of Beirut Medical Center and the University of Milan. He is a member of Alpha Omega Alpha Honor Medical Society and Sigma Xi, The Scientific Research Honor Society.
Prof. Musallam has a globally renowned research portfolio. He collaborated with numerous investigators and research groups from the Middle East, Asia, Europe, and the US where he led large multinational projects investigating hematological disorders (hemoglobinopathies, iron overload and deficiency, thrombosis, bleeding disorders).
He has authored several book chapters and international management guidelines and over 230 articles in leading international peer-reviewed journals including the New England Journal of Medicine, Lancet, Nature, Blood, among others. His h-index is 63. He has also presented over 85 abstracts at leading international congresses like ASH and EHA.
Prof. Musallam’s research expertise primarily revolves around establishment and analysis of large observational databases to investigate pathologic mechanisms and (modifiable) risk factors for adverse morbidity and mortality outcome sand his work reshaped thalassemia management over the past two decades. He has led several research programs primarily focused on non-transfusion-dependent thalassemia (NTDT) and uncovered the clinical profile of hypercoagulability and iron overload in these patients, which supported the development of oral iron chelation therapy trials (deferasirox) and their first regulatory approvals in this indication. His work on ineffective erythropoiesis/anemia and their detrimental impact on morbidity and mortality outcomes has also supported the development of several first-in-class novel disease-modifying agents (e.g., luspatercept, mitapivat) that are expected to reshape the natural history of thalassemia in the next 5 years; with him serving as principal investigator or member of the steering committee for the majority of such global clinical trial programs and supporting their regulatory filings. In transfusion-dependent patients (TDT), his work has mostly focused on establishing the link between hematologic and iron parameters with morbidity and mortality outcomes to inform optimal management decisions, as well as development of new iron chelators and novel agents targeting transfusion burden. Moreover, his work provided clinical proof of principle for the benefit of reactivating fetal hemoglobin, the core principle of gene editing techniques in thalassemia. He is also leading various initiatives on the use of repurposed drugs in thalassemia syndromes. His work is now also extending to alpha-thalassemia, a thalassemia subtype with considerable limitation in available literature on clinical manifestations and management needs, through leading the first Alpha Thalassemia Global Cohort (ATGC) study. His contributions to understanding the disease and its management led to transformative changes in international management guidelines, with him being an Editor for both the NTDT and TDT Thalassaemia International Federation (TIF) management guidelines used by thalassemia care providers globally.
Prof. Musallam has also designed several studies evaluating hematologic and other risk factors of poor surgical outcomes and led a series of publications in top tier journals using the American College of Surgeons NSQIP database of over a million patients. He also acts as Subject Matter Expert in the Improving Estimates of Anemia, Global Burden of Disease Project with the Institute for Health Metrics and Evaluation at the University of Washington. Prof. Musallam also led the development of several registries in Europe focused on hemophilia and bleeding disorders which paved the way to the development of scoring systems that aid clinical decision making.
Prof. Musallam received the prestigious European Haematology Association (EHA) Clinical Excellence Award in 2025 in recognition for his clinical research journey. He also received the Khalifa University Adjunct Faculty Research Excellence Award in 2025. He was ranked as World Expert and named among the top 0.1% scholars (top 10 in anemia and top 3 in beta-thalassemia worldwide) by EXPERTSCAPE since 2019 and is recognized among the world top 2% most-cited scientists for ‘Cardiovascular System & Hematology, Immunology, Clinical Medicine’ in Elsevier’s “Updated science-wide author databases of standardized citation indicators” by Stanford University since 2017.
Prof. Musallam is a frequent reviewer for over 50 medical journals and international congresses and assumed editorial roles for the European Journal of Internal Medicine, World Journal of Medical Education and Research, Thalassemia Reports, International Journal of Molecular Sciences, and Faculty of 1000 Medicine. He is a frequent chair and speaker at international congresses and has led several international training workshops on essentials in clinical research and continues to mentor several young scholars to kick start their research journey.
In addition to his academic activities, Prof. Musallam has senior level pharmaceutical industry experience in Switzerland (Novartis Pharma AG) where he led large, multicenter clinical trials and US/EU regulatory submissions for several hematology/oncology and rare disease products. He is also founder of Evida Medical and was Global Medical Lead at AMICULUM, an independent global cluster of agencies that offered healthcare consultancy and medical communication services for pharmaceutical companies throughout the life cycle of innovative medicines.
