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EBMT 2026 | Hematologists’ perspectives on gene therapy for sickle cell disease in the post-approval era
Alexander Ngwube, MD, Baylor College of Medicine, Houston, TX, discusses the results of a survey conducted in the United States to evaluate hematologists’ knowledge and attitudes towards gene therapy for sickle cell disease (SCD) in the post-approval era. Dr Ngwube notes that while enthusiasm for gene therapy was high among physicians, with 98% willing to discuss it with patients, generalists lacked knowledge on the differences between approved products and long-term complications compared with transplant and SCD specialists. This interview took place at the 52nd Annual Meeting of the EBMT in Madrid, Spain.
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Transcript
So we did a survey last year where we tried to evaluate the knowledge and the attitude towards gene therapy in patients and amongst physicians that were trained to take care of patients. And we divided these hematologists into three separate groups. We had those who were specific in taking care of patients with sickle cell disease. We had those that were generalists...
So we did a survey last year where we tried to evaluate the knowledge and the attitude towards gene therapy in patients and amongst physicians that were trained to take care of patients. And we divided these hematologists into three separate groups. We had those who were specific in taking care of patients with sickle cell disease. We had those that were generalists. And then we had those that were transplant physicians. And the one thing we noticed is that the transplant group and the sickle cell group were both prepared and had more knowledge compared to the general hematology oncologist. But the most important thing is that enthusiasm was high. Almost 98% of those physicians were ready to discuss it. But like I said, those that were generalists kind of didn’t know necessarily the difference between the different products. They were kind of a little bit… their knowledge about the long-term complications of gene therapy or the long-term outcomes of gene therapy and sickle cell disease was a bit lacking compared to hematologists that were sickle cell focused or transplant focused. So that was kind of like where the big differences were. But one thing I was certain is that everybody wanted to offer this to their patients.
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Disclosures
Vertex Therapeutics (paid consultant one-time, ended November 2025) and Beam Therapeutics (paid consultant one-time, ended December 2025).
