The Thalassaemia International Federation (TIF) has launched the third edition of its Non-Transfusion-Dependent Beta-Thalassemia (NTDT) guidelines. The updated manual is used by medical practitioners around the world to treat and manage the disease that affects thousands of people around the world. The latest edition features novel approaches for the management and new therapies for the treatment of NTDT, a genetic blood disorder that affects the production of hemoglobin, which had been underestimated in clinical significance for many years. The guidelines provide a comprehensive framework for the diagnosis and use of newer therapies in the ongoing care of patients with this complex disease.
These guidelines have been developed based on the latest research and clinical experience, and they offer a more comprehensive and up-to-date approach to the treatment of NTDT. It is co-authored by Prof. Ali Taher from the American University of Beirut, Abu Dhabi-based Dr. Khaled Musallam, Group Chief Research Officer at Burjeel Holdings, and Prof. Maria D. Cappellini from the University of Milan. As the authors are recognized for being the top three global experts in thalassemia, the guidelines are primarily driven by their extensive research and work in the field.
One of the key areas of focus and novelty in the guidelines is the focus on ineffective erythropoiesis (the body’s process of making red blood cells, which is defective in thalassemia) and subsequent anemia. This was primarily driven by several scientific discoveries by the three authors in the last few years on the detrimental role of anemia in these patients and its association with increased morbidity and mortality. This has led to several clinical trials of novel therapies aiming to ameliorate anemia in this patient population including the BEYOND trial of the drug ‘luspatecept’, co-authored by Dr. Musallam; and the newer phase 3, randomized trial (ENERGIZE) of the drug mitapivat, currently ongoing at Burjeel Medical City.
“Our understanding of NTDT has swiftly evolved in the past two decades, and it is now established that the disease carries far more morbidity than previously recognized – further highlighting the need for developing innovative therapies,” said Dr. Musallam, who has also recently won a DoH grant for a collaborative project to expand the field of knowledge of thalassemia in the UAE.
Saeed, a thalassemia patient and member of the Emirates Thalassemia Society, is optimistic that the new guidelines will help improve the condition of patients. “Thalassemia is a lifelong disease and requires individualized care that can only be attainable through such practical guidelines that match patient needs with available and new therapies,” said Saeed.
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