Introduction This review summarizes the current state of evidence from peer-reviewed publications and relevant conference presentations from 2023–2025 and highlights the impact of patient selection, donor selection, conditioning, measures to prevent graft-versus-host disease (GvHD), maintenance therapy, monitoring of measurable residual disease (MRD), and supportive measures on the transplant outcome. Methods Narrative review with current randomized, large retrospective studies and important abstracts on adult acute myeloid leukemia (AML) patients who have undergone stem cell transplantation (allo-HCT). Conference contributions are included in the evaluation if mature data are still lacking evaluation. Results We see the greatest benefit of allo-HCT in patients with unfavorable genetics or MRD persistence after initial complete remission. MRD-negative patients with favorable genetics achieve long-term cure, in some cases even without transplantation [<CitationRef CitationID="CR1">1</CitationRef>, <CitationRef CitationID="CR2">2</CitationRef>]. In several studies, haploidentical transplants with posttransplant cyclophosphamide (PTCy) have achieved results similar to those of HLA-identical siblings or unrelated donors; however, registry data report higher nonrelapse mortality (NRM) or poorer overall survival (OS) in individual settings. In mismatched unrelated donor transplantation, PTCy was superior to rabbit antithymocyte globulin (rATG) for NRM and OS. With iomab‑B (radioimmunological approach), older patients with relapsed/refractory AML can receive targeted curative treatment. Sorafenib as maintenance therapy after allo-HCT improves relapse-free survival and OS in FLT3-ITD-mutated AML, while a benefit from gilteritinib after allo-HCT was particularly shown for MRD-positive patients. APR-246 (eprenetapopt): Phase II data support its use as maintenance therapy after allo-HCT and early pilot studies with enasidenib or ivosidenib report good tolerability and efficacy in small cohorts. MRD monitoring after HCT identifies molecular relapse and patients at high risk for overt relapse who may benefit from pre-emptive interventions; letermovir reduces CMV disease (not reactivation) to < 4%, higher gut microbiome diversity correlates with lower GvHD risk. Whether prophylactic fecal microbiota transfer (FMT) may reduce GvHD remains unproven in randomized trials. Conclusion In addition to ELN risk criteria and MRD status for accurate risk stratification, the wider availability of alternative donors thanks to PTCy-based GvHD prophylaxis, targeted maintenance therapy, and improved supportive care increase the curative potential of allo-HCT in AML.

Allogeneic hematopoietic stem cell transplantation (allo-HCT) remains a key curative treatment for acute myeloid leukemia (AML)

期刊论文
发表日期：

2025年12月11日

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Alexander Nikoloudis,
Sigrid Machherndl-Spandl,
Johannes Clausen

摘要

Introduction

This review summarizes the current state of evidence from peer-reviewed publications and relevant conference presentations from 2023–2025 and highlights the impact of patient selection, donor selection, conditioning, measures to prevent graft-versus-host disease (GvHD), maintenance therapy, monitoring of measurable residual disease (MRD), and supportive measures on the transplant outcome.

Methods

Narrative review with current randomized, large retrospective studies and important abstracts on adult acute myeloid leukemia (AML) patients who have undergone stem cell transplantation (allo-HCT). Conference contributions are included in the evaluation if mature data are still lacking evaluation.

Results

We see the greatest benefit of allo-HCT in patients with unfavorable genetics or MRD persistence after initial complete remission. MRD-negative patients with favorable genetics achieve long-term cure, in some cases even without transplantation [1, 2]. In several studies, haploidentical transplants with posttransplant cyclophosphamide (PTCy) have achieved results similar to those of HLA-identical siblings or unrelated donors; however, registry data report higher nonrelapse mortality (NRM) or poorer overall survival (OS) in individual settings. In mismatched unrelated donor transplantation, PTCy was superior to rabbit antithymocyte globulin (rATG) for NRM and OS. With iomab‑B (radioimmunological approach), older patients with relapsed/refractory AML can receive targeted curative treatment. Sorafenib as maintenance therapy after allo-HCT improves relapse-free survival and OS in FLT3-ITD-mutated AML, while a benefit from gilteritinib after allo-HCT was particularly shown for MRD-positive patients. APR-246 (eprenetapopt): Phase II data support its use as maintenance therapy after allo-HCT and early pilot studies with enasidenib or ivosidenib report good tolerability and efficacy in small cohorts. MRD monitoring after HCT identifies molecular relapse and patients at high risk for overt relapse who may benefit from pre-emptive interventions; letermovir reduces CMV disease (not reactivation) to < 4%, higher gut microbiome diversity correlates with lower GvHD risk. Whether prophylactic fecal microbiota transfer (FMT) may reduce GvHD remains unproven in randomized trials.

Conclusion

In addition to ELN risk criteria and MRD status for accurate risk stratification, the wider availability of alternative donors thanks to PTCy-based GvHD prophylaxis, targeted maintenance therapy, and improved supportive care increase the curative potential of allo-HCT in AML.