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EBMT 2025 | Post-transplant monitoring and follow-up in the evolving hemoglobinopathy transplant journey
Mattia Algeri, MD, Bambino Gesù Children’s Hospital, Rome, Italy, comments on the post-transplant monitoring and follow-up during the stem cell transplantation (SCT) or gene therapy journey in patients with hemoglobinopathies. Dr Algeri highlights the differences between these therapeutic approaches and notes that long-term follow-up for autologous gene therapies requires more specific guidelines, which presents an area of future research. This interview took place at the 51st Annual Meeting of the EBMT in Florence, Italy.
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Transcript
Post-transplant monitoring and follow-up in hemoglobinopathy patients during the transplant journey is crucial for several reasons. The main differences between allogeneic transplantation and autologous gene therapies is that in one case we have myeloablation plus immunoablation, in another case myeloablation alone and this determines several consequences in what is the expected burden of post-transplant complications...
Post-transplant monitoring and follow-up in hemoglobinopathy patients during the transplant journey is crucial for several reasons. The main differences between allogeneic transplantation and autologous gene therapies is that in one case we have myeloablation plus immunoablation, in another case myeloablation alone and this determines several consequences in what is the expected burden of post-transplant complications. Therefore, this influences also the intensity of the anti-infectious prophylaxis, while the type of toxic complications namely cytopenia, mucositis, VOD risk, this is almost superimposable.
For what concerns the long-term follow-up, we do have limited information in the patients treated with autologous gene therapies and for the moment we must adhere to the frameworks derived from allogeneic stem cell transplantation, but certainly one of the tasks for the future is to develop specific guidelines for patients given autologous gene therapies.
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Disclosures
Vertex Pharmaceuticals, Novartis.
