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EBMT 2025 | Troubleshooting the transplant process in hemoglobinopathies
In this video, Mattia Algeri, MD, Bambino Gesù Children’s Hospital, Rome, Italy, briefly outlines the key takeaways from a discussion on troubleshooting the transplant process in hemoglobinopathies. Dr Algeri highlights the importance of patient preparation for the conditioning process, emphasizing that while certain aspects of preparation are similar, others are specific to patients undergoing autologous gene therapies. Dr Algeri also discusses the ongoing debate around busulfan conditioning, suggesting that Q6 administration may be associated with reduced veno-occlusive disease (VOD) risk. This interview took place at the 51st Annual Meeting of the EBMT in Florence, Italy.
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Transcript
Well, there were several takeaways, especially in how to prepare the patient in the best way. The preparation is, again, similar for certain aspects and different for others. The preparation of the patients for the conditioning before the conditioning is almost the same, hypertransfusion, reduction of the hemoglobin S percentage. But of course, patients given autologous gene therapies also have to undergo mobilization and collection of the cells...
Well, there were several takeaways, especially in how to prepare the patient in the best way. The preparation is, again, similar for certain aspects and different for others. The preparation of the patients for the conditioning before the conditioning is almost the same, hypertransfusion, reduction of the hemoglobin S percentage. But of course, patients given autologous gene therapies also have to undergo mobilization and collection of the cells. So this part is crucial to optimize the transplant outcome and this is something that is specific for those patients given autologous gene therapies.
We provided many insights on how to prepare the patient for the collection and also how to correctly administer conditioning. It has been discussed whether busulfan should be infused Q6, Q24. This is an ongoing debate. It has not been solved yet and it has not been solved either yesterday, but in any case, the evidence suggests that Q6 administration is associated with a reduced VOD risk and probably this is the better way to adjust the dose and target the desired cumulative area under the curve.
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Disclosures
Vertex Pharmaceuticals, Novartis.
