Aim of the webinar:
This webinar will provide a clinically practical and transfusion-focused update on the rapidly evolving therapeutic landscape for thalassemia. It will place emerging disease-modifying and potentially curative approaches alongside the continuing need for safe, individualized transfusion support and iron-overload management.
1. Describe the unmet needs in transfusion-dependent and non–transfusion-dependent β-thalassemia, including lifelong red-cell exposure, iron overload, alloimmunization, and treatment burden.
2. Compare the mechanisms, clinical roles, expected benefits, and key limitations of emerging pharmacologic approaches, including erythroid maturation agents and pyruvate kinase activation.
3. Explain the principles of autologous gene addition and gene editing, including fetal hemoglobin reactivation, conditioning requirements, and the distinction between disease modification and potentially curative treatment.
Level of education required:
Intermediate (practical application), with selected advanced content on cellular and gene-based therapies.
Prior knowledge required:
Participants should have a basic understanding of β-thalassemia pathophysiology, the distinction between transfusion-dependent thalassemia (TDT) and non–transfusion-dependent thalassemia (NTDT), regular red-cell phenotyping/genotyping, and the fundamentals of hematopoietic stem-cell transplantation will be helpful but is not essential.
Attendees are encouraged to review the following articles before the webinar:
-
Cappellini MD, Viprakasit V, Taher AT, et al. A Phase 3 Trial of Luspatercept in Patients with Transfusion-Dependent β-Thalassemia. New England Journal of Medicine. 2020;382:1219–1231. doi:10.1056/NEJMoa1910182.
-
Makis A, Voskaridou E, Papassotiriou I, Hatzimichael E. Novel Therapeutic Advances in β-Thalassemia. Biology (Basel). 2021;10(6):546. doi:10.3390/biology10060546.
-
Malay J, Salama RAA, Qureshi GSA, et al. Gene Therapy: A Revolutionary Step in Treating Thalassemia. Hematology Reports. 2024;16(4):656–668. doi:10.3390/hematolrep16040064.
-
Hu J, Zhong Y, Xu P, et al. β-Thalassemia gene editing therapy: Advancements and difficulties. Medicine. 2024;103(18):e38036. doi:10.1097/MD.0000000000038036.
-
Gamaleldin M, Abdelhalim S, Abraham I. Recent Advances in Thalassemia Management: From Curative Therapies to Artificial Intelligence. Thalassemia Reports. 2026;16(2):7. doi:10.3390/thalassrep16020007.
Target audience:
Transfusion medicine physicians, blood-bank medical directors and practitioners, hematologists, pediatric hematologists, hemoglobinopathy and thalassemia specialists, hematopoietic stem-cell transplantation and cellular-therapy teams, transfusion laboratory scientists and technologists, nurses, pharmacists, and trainees involved in the care of patients with thalassemia.
The Speaker:
Fatma Burcu Belen Apak
Professor of Pediatric Hematology and Oncology
Baskent University Faculty of Medicine
Pediatric Hematopoietic Stem Cell Transplantation Unit, Ankara, Turkey
The Moderators:
Ratti Ram Sharma
Head of the Department of Transfusion Medicine at Postgraduate Institute of Medical Education and Research, Chandigarh, India.
Richard Gammon
Are you an ISBT member? Then we have great news! Your CPD points and webinar certificates are automatically saved in My Account – My CPD. Simply log in to the ISBT website using your member credentials to access them. To download your certificate, click on the webinar title in the My CPD area and it will be saved directly to your computer.
Did you know you can also record your own CPD activities here? You’re welcome to use this space to track all your educational activities — even those not directly related to ISBT.
If you enjoyed this webinar, be sure to keep an eye on the ISBT events calendar for upcoming ISBT webinars and Live Journal Clubs. We’d love to welcome you again at future events!
If you’re unable to attend a live session or would like to rewatch a webinar, visit the ISBT webcast library, where you’ll find recordings of all ISBT webinars, Live Journal Clubs, and ISBT congress webcasts. Use the filters to easily find topics that interest you.
