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EBMT 2026 | Key considerations when selecting patients with hemoglobinopathies for transplantation
In this video, Mohammed Essa, MD, Ministry of National Guard Health Affairs, Riyadh, Saudi Arabia, discusses the key considerations for selecting patients with hemoglobinopathies for stem cell transplantation, highlighting the importance of assessing disease severity, organ function, and the availability of a matched related donor. This interview took place at the 52nd Annual Meeting of the EBMT in Madrid, Spain.
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Transcript
That’s a great question and actually the answer to this question may be variable at different times and maybe in six months from now or a year it might be a little bit different. And also it depends where you are in different countries around the world given the accessibility in addition to the number of patients who have this devastating disease. So we really rely on the severity of the disease, the availability, whether these patients have a matched related donor to go for allogeneic transplantation or to go for further treatment, including alternative donor transplantation or gene therapy...
That’s a great question and actually the answer to this question may be variable at different times and maybe in six months from now or a year it might be a little bit different. And also it depends where you are in different countries around the world given the accessibility in addition to the number of patients who have this devastating disease. So we really rely on the severity of the disease, the availability, whether these patients have a matched related donor to go for allogeneic transplantation or to go for further treatment, including alternative donor transplantation or gene therapy. And that depends as well on organ function and whether they could tolerate their conditioning regimen. That is, until now, it’s myeloablative in case of gene therapy, but maybe in certain situations where we could give a reduced intensity in the case of alternative donor transplantation, knowing the risks of other side effects or adverse events such as graft-versus-host disease. In addition, another major factor, such as those patients who were excluded from the trial initially, especially the ones that we are still waiting and we need to learn more about, like patients with CNS vasculopathy for example in patients with sickle cell disease.
This transcript is AI-generated. While we strive for accuracy, please verify this copy with the video.
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Disclosures
Advisory board: Vertex.
