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EBMT 2025 | Comparing unrelated UCB versus unmanipulated haploidentical HSCT using PTCy in pediatric AML
Benedetta Elena Di Majo, MD, Fondazione IRCCS San Gerardo dei Tintori, Monza, Italy, discusses the findings of a retrospective study on behalf of the EBMT Paediatric Diseases Working Party (PDWP) and Cellular Therapy & Immunobiology Working Party (CTIWP), which compared single unrelated umbilical cord blood (UCB) versus unmanipulated haploidentical hematopoietic stem cell transplantation (HSCT) using post-transplant cyclophosphamide (PTCy) in pediatric patients with acute myeloid leukemia (AML). Both approaches yielded comparable outcomes, but with a notable difference in the incidence of chronic graft-versus-host disease (GvHD), which was significantly higher in haploidentical transplant recipients. This interview took place at the 51st Annual Meeting of the EBMT in Florence, Italy.
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Transcript
So as we know, AML in pediatric patients has an incidence of approximately seven cases per million children under the age of 15, so this is a very important issue in kids. And what we know is that when we are talking about transplanting this disease, we have the indication to transplant patients in CR1 for high-risk patients and in all patients for CR2. But importantly, most of them, 70% of them, can’t find a haploidentical donor, nor 40% of them can find a matched unrelated donor in international registries...
So as we know, AML in pediatric patients has an incidence of approximately seven cases per million children under the age of 15, so this is a very important issue in kids. And what we know is that when we are talking about transplanting this disease, we have the indication to transplant patients in CR1 for high-risk patients and in all patients for CR2. But importantly, most of them, 70% of them, can’t find a haploidentical donor, nor 40% of them can find a matched unrelated donor in international registries. So our rationale was to compare the use of mismatched grafts in these kind of patients.
And what we found, comparing the outcomes of UCB and haplo with PTCy in more than 250 patients was that there were comparable outcomes in both of the groups. And we also had chronic GVHD, which was actually the only outcome that was not comparable, as it was significantly different between the two groups. And what we found with a p-value of 0.019 difference between the two groups was that haplo recipients with PTCy had a high risk of chronic GVHD. So of course, we will have to further implement prophylaxis for these kind of patients, because as we know, chronic GVHD in pediatric patients is a very important issue. And so we have to prevent that in order to have a better quality of life. But still we had the comparable outcomes in these very useful resources of different donors and we can have cost-effective and ready-to-use different kinds of transplantation in this very bad disease.
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