EBMT 2025 | The selection of patients with transfusion-dependent thalassemia for alloSCT or gene therapy

Indication and selection of TDT thalassemia patients for transplant or gene therapy is a quite complicated topic because nowadays we are having luckily many curative options for these patients. Of course, the possibility of referring a patient for a curative option must be weighed with the other possibility of conservative conventional treatment, which anyway is giving improving results. Patients are now surviving up to their 50s or 60s or even more, although they start to experience some important complications from adolescence and adulthood. So we really need to weigh the potential of a cure through allogeneic stem cell and gene therapies with the risk associated with this type of treatment and compare this with the outcome that nowadays we have with conventional treatment. 

And the other aspect is to select the right treatment for the right patients. Young patients who have a matched sibling donor have excellent outcomes with allogeneic stem cell transplantation. So in this case, the patients can be referred for this type of option, but it must be carefully discussed with the families, especially regarding the risks of graft failure, GvHD, the immune-mediated risks, which are something that is specific to the allogeneic setting, and of course the risk of infertility. 

Patients above the age of 14 can be considered for autologous gene therapies, provided that this is approved and reimbursed in the country of origin of the patient. And in this case, of course, the patient is in an age range in which he or she may start experiencing some important complications or may have a higher iron overload as compared to childhood. So in this case, the selection can be a little bit more complicated because not all patients, despite having a treatment that has the possibility to cure, are not really eligible for that treatment because they already have some comorbidities or an excess of iron overload, which prevent them from being a true candidate for these therapies. So it’s very important to consider this factor in selecting the right patient for this therapy and improving also the conventional management already in these years in order to be able to treat as many patients as possible in the future. Otherwise, we will run the risk that some patients, despite having an approved therapy available, cannot be treated because of an excess of comorbidities.

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