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EBMT 2025 | The impact of donor types in alloSCT with PTCy for severe aplastic anemia
Juan Montoro, MD, La Fe University and Polytechnic Hospital, Valencia, Spain, discusses the impact of donor types in allogeneic stem cell transplantation (alloSCT) with post-transplant cyclophosphamide (PTCy) for severe aplastic anemia, highlighting the findings from a comparative study of the Severe Aplastic Anemia Working Party (SAAWP) of the EBMT. Dr Montoro notes that while matched sibling donors remain the gold standard, haploidentical family and unrelated donors are increasingly used to improve donor availability. This study elucidated that PTCy is equally effective for preventing graft-versus-host disease (GvHD) between donor types. However, the study highlighted the challenges associated with haploidentical transplants, including higher non-relapse mortality (NRM) and poorer survival outcomes. This interview took place at the 51st Annual Meeting of the EBMT in Florence, Italy.
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Transcript
I would like to thank the scientific committee for the opportunity to present our study at this EBMT, which evaluates the impact of donor type on allogeneic transplant outcomes in severe aplastic anemia using post-transplant cyclophosphamide. As we know, HLA-matched sibling donors remain the gold standard for younger patients undergoing allo. However, many patients lack a suitable matched sibling donor, and while matched unrelated donors are a common alternative, finding an appropriate match can be challenging, leading to the exploration of mismatched unrelated donors and haploidentical family donors...
I would like to thank the scientific committee for the opportunity to present our study at this EBMT, which evaluates the impact of donor type on allogeneic transplant outcomes in severe aplastic anemia using post-transplant cyclophosphamide. As we know, HLA-matched sibling donors remain the gold standard for younger patients undergoing allo. However, many patients lack a suitable matched sibling donor, and while matched unrelated donors are a common alternative, finding an appropriate match can be challenging, leading to the exploration of mismatched unrelated donors and haploidentical family donors. And despite the increasing use of PTCy to prevent GvHD, its impact on severe aplastic anemia patients has been scarcely evaluated and studied. So this is why we wanted to investigate this issue.
So our study, conducted on behalf of the Severe Aplastic Anemia Working Party of the EBMT, analyzed 348 patients who underwent their first allogenic transplant from 2010 to 2022 with a matched sibling, unrelated donor, or haploidentical donors, all receiving PTCy for GvHD prophylaxis.
And the conclusion of this study, recognizing certain inherent limitations, as is the case with other registry-based analyses, our study highlights the efficacy of PTCy in achieving low rates of acute and chronic GvHD in patients with severe aplastic anemia, undergoing haploidentical, matched sibling donor and unrelated donor stem cell transplantation with no significant differences between donor types. However, the higher non-relapse mortality and poorer survival outcomes in haploidentical transplants underscore the challenges associated with increased HLA disparity.
Among donor types, matched sibling donor transplant resulted in the most favorable outcomes, including the lowest non-relapse mortality and best survival, reinforcing their status as the preferred donor type when available. In the absence of a matched sibling donor, alternative transplantation from an unrelated donor also demonstrated better outcomes compared to haploidentical. Finally, conducting prospective controlled studies is crucial for advancing our understanding and improving outcomes for patients with severe aplastic anemia undergoing allogenic transplant.
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