Skip to main content

Thank you for visiting You are using a browser version with limited support for CSS. To obtain the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles and JavaScript.

  • Original Article
  • Published:

Acute Leukemia

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


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.

This is a preview of subscription content, access via your institution

Access options

Buy this article

Prices may be subject to local taxes which are calculated during checkout

Figure 1
Figure 2
Figure 3
Figure 4

Similar content being viewed by others


  1. Rubnitz JE, Inaba H . Childhood acute myeloid leukaemia. Br J Haematol 2012; 159: 259–276.

    Article  PubMed  PubMed Central  Google Scholar 

  2. Pession A, Masetti R, Rizzari C, Putti MC, Casale F, Fagioli F et al. Results of the AIEOP AML 2002/01 multicenter prospective trial for the treatment of children with acute myeloid leukemia. Blood 2013; 122: 170–178.

    Article  CAS  PubMed  Google Scholar 

  3. Creutzig U, Zimmermann M, Dworzak MN, Gibson B, Tamminga R, Abrahamsson J et al. The prognostic significance of early treatment response in pediatric relapsed acute myeloid leukemia: results of the international study Relapsed AML 2001/01. Haematologica 2014; 99: 1472–1478.

    Article  PubMed  PubMed Central  Google Scholar 

  4. Quarello P, Fagioli F, Basso G, Putti MC, Berger M, Luciani M et al. Outcome of children with acute myeloid leukaemia (AML) experiencing primary induction failure in the AIEOPAML 2002/01 clinical trial. Br J Haematol 2015; 171: 566–573.

    Article  PubMed  Google Scholar 

  5. Bonanomi S, Connor P, Webb D, Ancliff P, Amrolia P, Rao K et al. Successful outcome of allo-SCT in high-risk pediatric AML using chemotherapy-only conditioning and post transplant immunotherapy. Bone Marrow Transplant 2008; 42: 253–257.

    Article  CAS  PubMed  Google Scholar 

  6. Creutzig U, van den Heuvel-Eibrink MM, Gibson B, Dworzak MN, Adachi S, de Bont E et al. Diagnosis and management of acute myeloid leukemia in children and adolescents: recommendations from an international expert panel. Blood 2012; 120: 3187–3205.

    Article  CAS  PubMed  Google Scholar 

  7. Locatelli F, Masetti R, Rondelli R, Zecca M, Fagioli F, Rovelli A et al. Outcome of children with high-risk acute myeloid leukemia given autologous or allogeneichematopoietic cell transplantation in the AIEOP AML-2002/01 study. Bone Marrow Transplant 2015; 50: 181–188.

    Article  CAS  PubMed  Google Scholar 

  8. Bunin NJ, Davies SM, Aplenc R, Camitta BM, De Santes KB, Goyal RK et al. Unrelated donor bone marrow transplantation for children with acute myeloid leukemia beyond first remission or refractory to chemotherapy. J Clin Oncol 2008; 26: 4326–4332.

    Article  PubMed  PubMed Central  Google Scholar 

  9. Duval M, Klein JP, He W, Cahn JY, Cairo M, Camitta BM et al. Hematopoietic stem-cell transplantation for acute leukemia in relapse or primary induction failure. J Clin Oncol 2010; 28: 3730–3738.

    Article  PubMed  PubMed Central  Google Scholar 

  10. Gorman MF, Ji L, Ko RH, Barnette P, Bostrom B, Hutchinson R et al. Outcome for children treated for relapsed or refractory acute myelogenous leukemia (rAML): a Therapeutic Advances in Childhood Leukemia (TACL) Consortium study. Pediatr Blood Cancer 2010; 55: 421–429.

    Article  PubMed  Google Scholar 

  11. Craddock C, Labopin M, Pillai S, Finke J, Bunjes D, Greinix H et al. Factors predicting outcome after unrelated donor stem cell transplantation in primary refractory acute myeloid leukaemia. Leukemia 2011; 25: 808–813.

    Article  CAS  PubMed  Google Scholar 

  12. Hemmati PG, Terwey TH, Na IK, Jehn CF, le Coutre P, Vuong LG et al. Allogeneic stem cell transplantation for refractory acute myeloid leukemia: a single center analysis of long-term outcome. Eur J Haematol 2015; 95: 498–506.

    Article  PubMed  Google Scholar 

  13. Jabbour E, Daver N, Champlin R, Mathisen M, Oran B, Ciurea S et al. Allogeneic stem cell transplantation as initial salvage for patients with acute myeloid leukemia refractory to high-dose cytarabine-based induction chemotherapy. Am J Hematol 2014; 89: 395–398.

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  14. Skalska-Sadowska J, Wachowiak J, Zając-Spychała O, Niewiadomska-Wojnałowicz I, Januszkiewicz-Lewandowska D, Balwierz W et al. Outcome of refractory and relapsed acute myeloid leukemia in children treated during 2005–2011—experience of the Polish Pediatric Leukemia/Lymphoma Study Group (PPLLSG). Contemp Oncol (Pozn) 2014; 18: 48–53.

    Google Scholar 

  15. Thol F, Schlenk RF, Heuser M, Ganser A . How I treat refractory and early relapsed acute myeloid leukemia. Blood 2015; 126: 319–327.

    Article  CAS  PubMed  Google Scholar 

  16. Swerdlow SH, Campo E, Harris NL, Jaffe ES, Pileri SA, Stein H et al. WHO Classification of Tumors of Haematopoetic and Lymphoid Tissues, 4th edn. International Agency for Research on Cancer: Lyon, France, 2008.

    Google Scholar 

  17. Giralt S, Ballen K, Rizzo D, Bacigalupo A, Horowitz M, Pasquini M et al. Reduced-intensity conditioning regimen workshop: defining the dose spectrum. Report of a workshop convened by the center for international blood and marrow transplant research. Biol Blood Marrow Transplant 2009; 15: 367–369.

    Article  PubMed  PubMed Central  Google Scholar 

  18. Kaplan EL, Meier P . Nonparametric estimation from incomplete observations. J Am Statist Assoc 1958; 53: 457–481.

    Article  Google Scholar 

  19. Fine JP, Gray RJ . A proportional hazards model for the subdistribution of a competing risk. J Am Statist Assoc 1999; 94: 496–509.

    Article  Google Scholar 

  20. Chiesa R, Gilmour K, Qasim W, Adams S, Worth A, Zhan H et al. Omission of in vivo T-cell depletion promotes rapid expansion of naïve CD4+ cord blood lymphocytes and restores adaptive immunity within 2 months after unrelated cord blood transplant. Br J Haematol 2012; 156: 656–666.

    Article  CAS  PubMed  Google Scholar 

Download references


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.

Author information

Authors and Affiliations



Corresponding author

Correspondence to P J Amrolia.

Ethics declarations

Competing interests

The authors declare no conflict of interest.

Additional information

Supplementary Information accompanies this paper on Bone Marrow Transplantation website

Supplementary information

Rights and permissions

Reprints and permissions

About this article

Check for updates. Verify currency and authenticity via CrossMark

Cite this article

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).

Download citation

  • Received:

  • Revised:

  • Accepted:

  • Published:

  • Issue Date:

  • DOI:

This article is cited by


Quick links