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LeukemiaReview

09 Jul 2025

The hallmarks of hematopoietic stem cell transplantation for pediatric acute myeloid leukemia.

Abstract

Allogeneic hematopoietic stem cell transplantation (HSCT) has significantly improved the outcome of children with high-risk (HR) acute myeloid leukemia (AML).

Implementing allogeneic HSCT depends on numerous factors, including adverse cytogenetics, molecular abnormalities, poor response to first-line treatment, or relapsed or primary refractory disease.

In HR AML, allogeneic HSCT is considered to be the consolidation strategy of choice in first complete remission (CR1) and offers the best chance of cure for patients with relapsed disease.

Advances in donor/recipient typing, conditioning regimens, graft-versus-host-disease (GvHD) management, and supportive care have contributed to this improvement in overall-and transplant-outcome.

This review will comprehensively discuss indications for HSCT and its modalities in pediatric AML by examining past, current, and future strategies for disease- and response-related stratification.

We will examine the key importance of low/negative measurable residual disease (MRD) before transplantation and discuss conditioning regimens and graft variables, as well as novel approaches to harness the graft-versus-leukemia (GvL) effect, including targeted immunotherapy.

The review will also address toxicities associated with HSCT, GvHD prophylaxis, and the management of treatment failure. Ultimately, this review seeks to inform clinical practice and highlights how improved outcomes have been achieved through the collective efforts of international study groups.

COI Statement

Competing interests: KK acknowledges research support from Jazz Pharmaceuticals, travel grants from Sobi and Medac, and receives honoraria from Vertex. BG declares honoraria from Vertex. JHK acknowledges advisory roles with Bluebird Bio, Novartis, Roche, and Jazz Pharmaceuticals. FL participated in advisory boards for Amgen, Sanofi, and Vertex; speaker’s bureau for Amgen, Gilead, Miltenyi, Novartis, Sanofi, and SOBI. All other authors declare that the manuscript was written in the absence of any commercial or financial relationships that could be perceived as a potential conflict of interest.

References:

  • Egan G, Chopra Y, Mourad S, Chiang KY, Hitzler J. Treatment of acute myeloid leukemia in children: a practical perspective. Pediatr Blood Cancer. 2021;68:e28979.
  • Bleakley M, Shaw PJ, Nielsen JM. Allogeneic bone marrow transplantation for childhood relapsed acute lymphoblastic leukemia: comparison of outcome in patients with and without a matched family donor. Bone Marrow Transplant. 2002;30:1–7.
  • Bleakley M, Lau L, Shaw PJ, Kaufman A. Bone marrow transplantation for paediatric AML in first remission: a systematic review and meta-analysis. Bone Marrow Transplant. 2002;29:843–52.
  • Niewerth D, Creutzig U, Bierings MB, Kaspers GJ. A review on allogeneic stem cell transplantation for newly diagnosed pediatric acute myeloid leukemia. Blood. 2010;116:2205–14.
  • Zwaan CM, Kolb EA, Reinhardt D, Abrahamsson J, Adachi S, Aplenc R, et al. Collaborative efforts driving progress in pediatric acute myeloid leukemia. J Clin Oncol. 2015;33:2949–62.

Article info

Journal issue:

  • Volume: not provided
  • Issue: not provided

Doi:

10.1038/s41375-025-02685-5

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