EBMT 2025 | Investigating post-transplant infusion of rivo-cel in patients with inborn errors of immunity

Rivo-cel was an approach based on the post-transplant infusion of donor-derived T-cells transduced with inducible Caspase-9 suicide gene, which is a construct that is able to induce the apoptosis of the allogeneic T-cells in case of uncontrolled GvHD. These lymphocytes were infused 14 plus or minus 4 days after an haploidentical transplant, a haploidentical allograft, to improve the recovery of the adaptive immunity. In this trial, the data that I will present will refer specifically to the European and UK patients treated for the inborn error of immunity and we observe excellent outcome in a patient with severe combined immune deficiency with all the patients alive and cured – 19 patients – and very good outcomes also in patients with complex immune deficiencies. Although in this setting, what we have to say is that, those patients with severe organ damage and severe pre-transplant infection were not entirely cured by this approach. So even the post-transplant infusion of titrated number of allogeneic T-cells with a suicide gene, a safety suicide gene, in any case, is not able to completely overcome the increased risk of morbidity and mortality associated with severe organ damage and severe pre-transplant infection in inborn errors of immunity.

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