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<p>Core-binding factor acute myeloid leukemia (CBF-AML) generally has a favorable prognosis, with allogeneic hematopoietic stem cell transplantation (allo-SCT) recommended for relapsed/ refractory (R/R) cases achieving second complete remission (CR). However, clinical outcomes remain suboptimal for patients who relapse or fail to achieve CR following induction chemotherapy. Allo-SCT in non-CR is a potential strategy for such patients, though supporting evidence in CBF-AML is limited. To assess outcomes and prognostic factors of allo-SCT in R/R CBF-AML with active disease, we conducted a retrospective analysis of 610 patients with CBF-AML in non-CR undergoing allo-SCT from 2010 to 2021 across 174 centers within the European Society for Blood and Marrow Transplantation. Graft sources included matched sibling (MSD, <i>n</i> = 151), unrelated (UD, <i>n</i> = 368), and haploidentical donors (Haplo, <i>n</i> = 91). Among patients, 124 had inv(16), and 486 had t(8;21). Two-year overall survival (OS) and leukemia-free survival (LFS) were 53.6% and 42.7%, respectively. Haplo-SCT showed inferior OS compared to MSD (HR 1.79, <i>p</i> = 0.003) and UD (HR 1.64, <i>p</i> = 0.004) and reduced chronic graft-versus-host disease. Patients with t(8;21) exhibited higher relapse incidence (HR 2.04, <i>p</i> = 0.002) and poorer survival outcomes than those with inv(16). These findings confirm the therapeutic role of allo-SCT in R/R CBF-AML in non-CR, supporting its favorable risk profile.</p>

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Allogeneic stem cell transplantation in de novo core-binding factor acute myeloid leukemia in active disease: a study from the Acute Leukemia Working Party of the European Society for Blood and Marrow Transplantation

  • 期刊论文

  • 发表日期：

    2025年4月23日

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  • Sara Tarantino,
  • Myriam Labopin,
  • Robert Zeiser,
  • Matthias Stelljes,
  • Thomas Schroeder,
  • Nicolaus Kröger,
  • Wolfgang Bethge,
  • Jakob Passweg,
  • Martin Bornhäuser,
  • Christoph Schmid,
  • Johanna Tischer,
  • Matthias Eder,
  • Eolia Brissot,
  • Jordi Esteve,
  • Arnon Nagler,
  • Mohamad Mohty,
  • Fabio Ciceri

摘要

Core-binding factor acute myeloid leukemia (CBF-AML) generally has a favorable prognosis, with allogeneic hematopoietic stem cell transplantation (allo-SCT) recommended for relapsed/ refractory (R/R) cases achieving second complete remission (CR). However, clinical outcomes remain suboptimal for patients who relapse or fail to achieve CR following induction chemotherapy. Allo-SCT in non-CR is a potential strategy for such patients, though supporting evidence in CBF-AML is limited. To assess outcomes and prognostic factors of allo-SCT in R/R CBF-AML with active disease, we conducted a retrospective analysis of 610 patients with CBF-AML in non-CR undergoing allo-SCT from 2010 to 2021 across 174 centers within the European Society for Blood and Marrow Transplantation. Graft sources included matched sibling (MSD, n = 151), unrelated (UD, n = 368), and haploidentical donors (Haplo, n = 91). Among patients, 124 had inv(16), and 486 had t(8;21). Two-year overall survival (OS) and leukemia-free survival (LFS) were 53.6% and 42.7%, respectively. Haplo-SCT showed inferior OS compared to MSD (HR 1.79, p = 0.003) and UD (HR 1.64, p = 0.004) and reduced chronic graft-versus-host disease. Patients with t(8;21) exhibited higher relapse incidence (HR 2.04, p = 0.002) and poorer survival outcomes than those with inv(16). These findings confirm the therapeutic role of allo-SCT in R/R CBF-AML in non-CR, supporting its favorable risk profile.