EBMT 2024 | A global view of transfusion-dependent thalassemia: standard of care, outcomes, and unmet needs
Ali Taher, MD, PhD, FRCP, American University of Beirut Medical Center, Beirut, Lebanon, summarizes a talk on the current standard of care, outcomes, and unmet needs for patients with transfusion-dependent thalassemia (TDT) worldwide. Blood transfusion and iron chelation therapy have significantly decreased the morbidity and mortality of patients with TDT. However, Prof. Taher highlights that the prevalence of TDT is high in areas of the world where access to therapy is low, meaning that 70-80% of patients globally do not have access to safe transfusion and iron chelation. Additionally, the lack of psychological support provided to patients must be addressed. This interview took place at the 50th Annual Meeting of the EBMT in Glasgow, Scotland.
These works are owned by Magdalen Medical Publishing (MMP) and are protected by copyright laws and treaties around the world. All rights are reserved.
Transcript (edited for clarity)
So this talk was given to a bone marrow transplant team. So the way was to introduce transfusion-dependent thalassemia, which as you all know, is a genetic disease where the patients would require continuous blood transfusion for survival, otherwise they will not survive. And I have discussed the current standards, not the novel therapies, which means the blood transfusion and chelation therapy...
So this talk was given to a bone marrow transplant team. So the way was to introduce transfusion-dependent thalassemia, which as you all know, is a genetic disease where the patients would require continuous blood transfusion for survival, otherwise they will not survive. And I have discussed the current standards, not the novel therapies, which means the blood transfusion and chelation therapy. Both of these blood transfusion and chelation therapy have affected the impact of survival, decreased morbidity and mortality in this group of patients. However, these are an unmet need because this disease is common and prevalent in areas of the world where of limited resources. So that was an interesting point where we have discussed what can we do for those countries, and how can we relate them to the transplant ideas and transplant work?
I mean, as you know, thalassemia is prevalent in areas of limited resources. Actually, 70% and 80% of patients do not even have good blood transfusion, safe blood transfusion, and chelation therapy. So this remains to be an unmet need. The presence of an MRI that detects iron overload in this group of patients also remains to be an unmet need. The psychological aspects of these patients and their quality of life. So we’re talking about novel therapy, modern therapy, yet the basic is not there. So it’s really very important to look at those unmet needs, try to improve them at a time when we could give the best novel modern therapy to all patients.
We didn’t have a lot of time to discuss this, but it’s really very important that the quality of life of patients are affected by this disease, and this would lead to a lot of anxiety, depression, sometimes suicidal attempt. So always when we look at these patients, we really need to look at the medical component of them, as well as the mental health that is affected by this disease. And more and more, we’re trying to look at it more carefully and integrate it in the management of our patients.
Read more...
Disclosures
Novo Nordisk: Consultancy; Bristol-Myers Squibb (Celgene): Consultancy, Research funding; Vifor: Consultancy, Research Funding; Pharmacosmos: Consultancy, Research Funding; Agios: Consultancy, Research Funding.
