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Acute Leukemia

Allogeneic stem cell transplantation for refractory acute myeloid leukemia in pediatric patients: the UK experience

Bone Marrow Transplantation volume 52, pages 825–831 (2017)Cite this article

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Abstract

We report outcomes for 44 children who underwent stem cell transplantation (SCT) for refractory AML in the UK between 2000 and 2012. Median age at SCT was 11.5 years. Twenty-three patients had primary refractory and 21 relapsed refractory AML. Refractory disease was confirmed by cytogenetics/molecular genetics in 24 cases. Median follow-up of the whole cohort is 6.8 years (2.1–14.9 years). Thirty patients (68%) achieved a CR following SCT. Transplant-related mortality at 1 year was 18%. Acute GVHD incidence was 52% (grade ⩾III 19%), chronic 7%. Relapse was the major cause of treatment failure and occurred in 32% of patients at a median of 61 days post SCT. Five-year overall survival and leukemia-free survival (LFS) were 43% (95% CI 31–61%). All patients with favorable cytogenetics (n=6) are alive in CR. Outcomes in patients with primary refractory disease were equivalent to those with relapsed refractory AML. Blast percentage ⩽30% in the BM pre-SCT, myeloablative conditioning and acute GVHD proved to be favorable prognostic features. We could stratify patients according to age ⩾10 years and >30% blasts in BM pre-SCT. Patients with none/one of these risk factors were highly salvageable (5 years LFS 53%) whereas those with both factors had a very poor prognosis (5 years LFS 10%). This may facilitate decision making on whether it is appropriate to consider transplant in such patients.

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Acknowledgements

We are grateful to the British Society of Bone Marrow Transplantation for providing scientific support to the present paper.

Author contributions

PO and KK collected and verified all the data; PO, GL, RP and PJA wrote the paper; RP provided statistical analysis; MC, PV, MP, SL, AV, RW, AP and JP critically revised the data, contributed to the discussion of the results and revised the paper.

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Author notes

  1. P O'Hare and G Lucchini: These authors contributed equally to this work.

Authors and Affiliations

  1. Bone Marrow Transplantation Department, Great Ormond Street Hospital, London, UK

    P O'Hare, G Lucchini, P Veys & P J Amrolia

  2. Pediatric Bone Marrow Transplant Department, Bristol Royal Children's Hospital, Bristol, UK

    M Cummins

  3. Hemato-Oncology Unit, Royal Marsden Hospital, London, UK

    M Potter

  4. Department of Haematology, Birmingham Children's Hospital, Birmingham, UK

    S Lawson

  5. Department of Paediatric Haematology, Sheffield Children's Hospital, Sheffield, UK

    A Vora

  6. Royal Manchester Children’s Hospital, Manchester, UK

    R Wynn

  7. Hematology Department, Oxford University Hospital, Oxford, UK

    A Peniket

  8. British Society for Blood and Marrow Transplantation Registry, Guy’s Hospital, London, UK

    K Kirkland, R Pearce & J Perry

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  1. P O'Hare
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on behalf of the British Society Of Bone Marrow Transplant (BSBMT) Clinical Trials Committee

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Correspondence to P J Amrolia.

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The authors declare no conflict of interest.

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O'Hare, P., Lucchini, G., Cummins, M. et al. Allogeneic stem cell transplantation for refractory acute myeloid leukemia in pediatric patients: the UK experience. Bone Marrow Transplant 52, 825–831 (2017). https://doi.org/10.1038/bmt.2017.3

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