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<p>Extramedullary disease (EMD) is a rare presentation in Acute Myeloid Leukaemia (AML). On behalf of PDWP/EBMT we studied the effect of EMD compared to isolated BM involvement in the outcomes following non-TBI conditioning hematopoietic stem cell transplantation (HCT) for AML in children. Patients were grouped into three categories: BM involvement only (Group A), BM + CNS involvement only (Group B) and BM+ other EMD+/− CNS (Group C). A total of 958 patients were included, 685 (71.5%) in Group A, 135 (14.1%) in Group B and 138 (14.4%) in Group C. The median post HCT follow-up was 5.4 years. Five years LFS, OS, RI and NRM were 62%, 68.4%, 26.4% and 11.6%, respectively. Multivariate analysis showed higher RI in Group C compared to Group A (HR = 1.45 (1.01–2.06) <i>p</i> = 0.04). In the multivariate analysis patients with EMD had no significant difference in LFS, OS and NRM. Our findings suggest that children with AML and BM with EMD+/− CNS involvement (group C) have a higher incidence of relapse after HCT compared to those with BM only or BM + CNS only disease. However, the presence of EMD did not have an impact on overall, leukaemia-free survival or non-relapse mortality.</p>

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The impact of pre-transplant extramedullary disease on the outcome of allogeneic hematopoietic cell transplantation for acute myeloid leukaemia in children- on behalf of PDWP/EBMT

  • 期刊论文

  • 发表日期：

    2026年5月19日

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  • Juliana Silva,
  • Jacques-Emmanuel Galimard,
  • Jean-Hugues Dalle,
  • Franco Locatelli,
  • Hawazen S. Alsaedi,
  • Miguel Angel Diaz,
  • Charlotte Jubert,
  • Cecile Renard,
  • Giuseppina Simone,
  • Peter Bader,
  • Franca Fagioli,
  • Vassiliki Kitra Roussou,
  • Caroline L. Furness,
  • Robert Wynn,
  • Birgit Burkhardt,
  • Alessandra Biffi,
  • Marc Bierings,
  • Cristina Díaz de Heredia,
  • Arcangelo Prete,
  • Amos Toren,
  • Katharine Patrick,
  • Wolfgang Holter,
  • Krzysztof Kalwak,
  • Arnaud Dalissier,
  • Kanchan Rao,
  • Selim Corbacioglu

摘要

Extramedullary disease (EMD) is a rare presentation in Acute Myeloid Leukaemia (AML). On behalf of PDWP/EBMT we studied the effect of EMD compared to isolated BM involvement in the outcomes following non-TBI conditioning hematopoietic stem cell transplantation (HCT) for AML in children. Patients were grouped into three categories: BM involvement only (Group A), BM + CNS involvement only (Group B) and BM+ other EMD+/− CNS (Group C). A total of 958 patients were included, 685 (71.5%) in Group A, 135 (14.1%) in Group B and 138 (14.4%) in Group C. The median post HCT follow-up was 5.4 years. Five years LFS, OS, RI and NRM were 62%, 68.4%, 26.4% and 11.6%, respectively. Multivariate analysis showed higher RI in Group C compared to Group A (HR = 1.45 (1.01–2.06) p = 0.04). In the multivariate analysis patients with EMD had no significant difference in LFS, OS and NRM. Our findings suggest that children with AML and BM with EMD+/− CNS involvement (group C) have a higher incidence of relapse after HCT compared to those with BM only or BM + CNS only disease. However, the presence of EMD did not have an impact on overall, leukaemia-free survival or non-relapse mortality.