343
Views
21
CrossRef citations to date
0
Altmetric
Reviews

Prognosis and management of acute myeloid leukemia in patients with Down syndrome

, &

References

  • Creutzig U, van den Heuvel-Eibrink MM, Gibson B, et al. Diagnosis and management of acute myeloid leukemia in children and adolescents: recommendations from an international expert panel. Blood 2012;120(16):3187-205
  • Rubnitz JE. How I treat pediatric acute myeloid leukemia. Blood 2012;119(25):5980-8
  • Roizen NJ, Patterson D. Down’s syndrome. Lancet 2003;361(9365):1281-9
  • Zeller B, Gustafsson G, Forestier E, et al. Acute leukaemia in children with Down syndrome: a population-based Nordic study. Br J Haematol 2005;128(6):797-804
  • Al-Ahmari A, Shah N, Sung L, et al. Long-term results of an ultra low-dose cytarabine-based regimen for the treatment of acute megakaryoblastic leukaemia in children with Down syndrome. Br J Haematol 2006;133(6):646-8
  • Creutzig U, Ritter J, Vormoor J, et al. Myelodysplasia and acute myelogenous leukemia in Down’s syndrome. A report of 40 children of the AML-BFM Study Group. Leukemia 1996;10(11):1677-86
  • Kudo K, Kojima S, Tabuchi K, et al. Results of the Japanese childhood acute myeloid leukemia 99 protocol for down syndrome and acute myeloid leukemia. ASH Annual Meeting Abstracts; Orlando, FL, USA; 2005. 106(11):276
  • Lange BJ, Kobrinsky N, Barnard DR, et al. Distinctive demography, biology, and outcome of acute myeloid leukemia and myelodysplastic syndrome in children with Down syndrome: children’s Cancer Group Studies 2861 and 2891. Blood 1998;91(2):608-15
  • Ravindranath Y, Abella E, Krischer JP, et al. Acute myeloid leukemia (AML) in Down’s syndrome is highly responsive to chemotherapy: experience on Pediatric Oncology Group AML Study 8498. Blood 1992;80(9):2210-14
  • Zipursky A, Thorner P, De Harven E, et al. Myelodysplasia and acute megakaryoblastic leukemia in Down’s syndrome. Leuk Res 1994;18(3):163-71
  • Kojima S, Matsuyama T, Sato T, et al. Down’s syndrome and acute leukemia in children: an analysis of phenotype by use of monoclonal antibodies and electron microscopic platelet peroxidase reaction. Blood 1990;76(11):2348-53
  • Langebrake C, Creutzig U, Reinhardt D. Immunophenotype of Down syndrome acute myeloid leukemia and transient myeloproliferative disease differs significantly from other diseases with morphologically identical or similar blasts. Klin Padiatr 2005;217(3):126-34
  • Zipursky A, Christensen H, De Harven E. Ultrastructural studies of the megakaryoblastic leukemias of Down syndrome. Leuk Lymphoma 1995;18(3-4):341-7
  • Xavier AC, Ge Y, Taub JW. Down syndrome and malignancies: a unique clinical relationship: a paper from the 2008 William Beaumont hospital symposium on molecular pathology. J Mol Diag 2009;11(5):371-80
  • Khan I, Malinge S, Crispino J. Myeloid leukemia in down syndrome. Crit Rev Oncog 2011;16(1-2):25-36
  • Chou ST, Opalinska JB, Yao Y, et al. Trisomy 21 enhances human fetal erythro-megakaryocytic development. Blood 2008;112(12):4503-6
  • Tunstall-Pedoe O, Roy A, Karadimitris A, et al. Abnormalities in the myeloid progenitor compartment in Down syndrome fetal liver precede acquisition of GATA1 mutations. Blood 2008;112(12):4507-11
  • Taub JW, Mundschau G, Ge Y, et al. Prenatal origin of GATA1 mutations may be an initiating step in the development of megakaryocytic leukemia in Down syndrome. Blood 2004;104(5):1588-9
  • Nikolaev SI, Santoni F, Vannier A, et al. Exome sequencing identifies putative drivers of progression of transient myeloproliferative disorder to AMKL in infants with Down syndrome. Blood 2013;122(4):554-61
  • Yoshida K, Toki T, Okuno Y, et al. The landscape of somatic mutations in Down syndrome-related myeloid disorders. Nat Genet 2013;45(11):1293-9
  • Klusmann J-H, Godinho FJ, Heitmann K, et al. Developmental stage-specific interplay of GATA1 and IGF signaling in fetal megakaryopoiesis and leukemogenesis. Genes Dev 2010;24(15):1659-72
  • Klusmann JH, Li Z, Böhmer K, et al. miR-125b-2 is a potential oncomiR on human chromosome 21 in megakaryoblastic leukemia. Genes Dev 2010;24(5):478-90
  • Li Z, Godinho FJ, Klusmann JH, et al. Developmental stage-selective effect of somatically mutated leukemogenic transcription factor GATA1. Nat Genet 2005;37(6):613-19
  • Wechsler J, Greene M, McDevitt MA, et al. Acquired mutations in GATA1 in the megakaryoblastic leukemia of Down syndrome. Nat Genet 2002;32(1):148-52
  • Xavier AC, Edwards H, Dombkowski AA, et al. A unique role of GATA1s in Down syndrome acute megakaryocytic leukemia biology and therapy. PLoS One 2011;6(11):e27486
  • Ge Y, Jensen TL, Stout ML, et al. The role of cytidine deaminase and GATA1 mutations in the increased cytosine arabinoside sensitivity of Down syndrome myeloblasts and leukemia cell lines. Cancer Res 2004;64(2):728-35
  • Ge Y, Stout ML, Tatman DA, et al. GATA1, cytidine deaminase, and the high cure rate of Down syndrome children with acute megakaryocytic leukemia. J Natl Cancer Inst 2005;97(3):226-31
  • Taub JW, Huang X, Ge Y, et al. Cystathionine-beta-synthase cDNA transfection alters the sensitivity and metabolism of 1-beta-D-arabinofuranosylcytosine in CCRF-CEM leukemia cells in vitro and in vivo: a model of leukemia in Down syndrome. Cancer Res 2000;60(22):6421-6
  • Taub JW, Huang X, Matherly LH, et al. Expression of chromosome 21-localized genes in acute myeloid leukemia: differences between Down syndrome and non-Down syndrome blast cells and relationship to in vitro sensitivity to cytosine arabinoside and daunorubicin. Blood 1999;94(4):1393-400
  • Taub JW, Matherly LH, Stout ML, et al. Enhanced metabolism of 1-beta-D-arabinofuranosylcytosine in Down syndrome cells: a contributing factor to the superior event free survival of Down syndrome children with acute myeloid leukemia. Blood 1996;87(8):3395-403
  • Bourquin JP, Subramanian A, Langebrake C, et al. Identification of distinct molecular phenotypes in acute megakaryoblastic leukemia by gene expression profiling. Proc Natl Acad Sci USA 2006;103(9):3339-44
  • Ge Y, Dombkowski AA, LaFiura KM, et al. Differential gene expression, GATA1 target genes, and the chemotherapy sensitivity of Down syndrome megakaryocytic leukemia. Blood 2006;107(4):1570-81
  • Malinge S, Izraeli S, Crispino JD. Insights into the manifestations, outcomes, and mechanisms of leukemogenesis in Down syndrome. Blood 2009;113(12):2619-28
  • Krischer JP, Epstein S, Cuthbertson DD, et al. Clinical cardiotoxicity following anthracycline treatment for childhood cancer: the Pediatric Oncology Group experience. J Clin Oncol 1997;15(4):1544-52
  • O’Brien MM, Taub JW, Chang MN, et al. Cardiomyopathy in children with Down syndrome treated for acute myeloid leukemia: a report from the Children’s Oncology Group Study POG 9421. J Clin Oncol 2008;26(3):414-20
  • van der Pal HJ, van Dalen EC, van Delden E, et al. High risk of symptomatic cardiac events in childhood cancer survivors. J Clin Oncol 2012;30(13):1429-37
  • Ravindranath Y, Steuber CP, Krischer J, et al. High-dose cytarabine for intensification of early therapy of childhood acute myeloid leukemia: a Pediatric Oncology Group study. J Clin Oncol 1991;9(4):572-80
  • Lie SO, Jonmundsson G, Mellander L, et al. A population-based study of 272 children with acute myeloid leukaemia treated on two consecutive protocols with different intensity: best outcome in girls, infants, and children with Down’s syndrome. Nordic Society of Paediatric Haematology and Oncology (NOPHO). Br J Haematol 1996;94(1):82-8
  • Rao A, Hills RK, Stiller C, et al. Treatment for myeloid leukaemia of Down syndrome: population-based experience in the UK and results from the Medical Research Council AML 10 and AML 12 trials. Br J Haematol 2006;132(5):576-83
  • Creutzig U, Reinhardt D, Diekamp S, et al. AML patients with Down syndrome have a high cure rate with AML-BFM therapy with reduced dose intensity. Leukemia 2005;19(8):1355-60
  • Abildgaard L, Ellebaek E, Gustafsson G, et al. Optimal treatment intensity in children with Down syndrome and myeloid leukaemia: data from 56 children treated on NOPHO-AML protocols and a review of the literature. Ann Hematol 2006;85(5):275-80
  • Sorrell AD, Alonzo TA, Hilden JM, et al. Favorable survival maintained in children who have myeloid leukemia associated with Down syndrome using reduced-dose chemotherapy on Children’s Oncology Group trial A2971: a report from the Children’s Oncology Group. Cancer 2012;118(19):4806-14
  • Gamis AS, Woods WG, Alonzo TA, et al. Increased age at diagnosis has a significantly negative effect on outcome in children with Down syndrome and acute myeloid leukemia: a report from the Children’s Cancer Group Study 2891. J Clin Oncol 2003;21(18):3415-22
  • Blink M, Zimmermann M, von Neuhoff C, et al. Normal karyotype is a poor prognostic factor in myeloid leukemia of Down syndrome: a retrospective, international study. Haematologica 2014;99(2):299-307
  • Hasle H, Abrahamsson J, Arola M, et al. Myeloid leukemia in children 4 years or older with Down syndrome often lacks GATA1 mutation and cytogenetics and risk of relapse are more akin to sporadic AML. Leukemia 2007;22(7):1428-30
  • Hasle H, Alonzo TA, Auvrignon A, et al. Monosomy 7 and deletion 7q in children and adolescents with acute myeloid leukemia: an international retrospective study. Blood 2007;109(11):4641-7
  • von Neuhoff C, Reinhardt D, Sander A, et al. Prognostic impact of specific chromosomal aberrations in a large group of pediatric patients with acute myeloid leukemia treated uniformly according to trial AML-BFM 98. J Clin Oncol 2010;28(16):2682-9
  • Blink M, Van den Heuvel-Eibrink MM, de Haas V, et al. Myeloid leukemia of Down syndrome: the results of an international retrospective study. ASH Annual Meeting Abstracts; Orlando, FL, USA; 2010. 116(21):2718
  • Kudo K, Kojima S, Tabuchi K, et al. Prospective study of a pirarubicin, intermediate-dose cytarabine, and etoposide regimen in children with down syndrome and acute myeloid leukemia: the Japanese childhood AML cooperative study group. J Clin Oncol 2007;25(34):5442-7
  • Klusmann JH, Creutzig U, Zimmermann M, et al. Treatment and prognostic impact of transient leukemia in neonates with Down syndrome. Blood 2008;111(6):2991-8
  • Loew TW, Gamis A, Smith FO, et al. Down syndrome patients with relapsed acute myelogenous leukemia. ASH Annual Meeting Abstracts;Orlando, FL, USA; 2004. 104(11):4526
  • Loew TW, Gamis A, Smith FO, et al. Induction therapy failures in down syndrome patients with acute myelogenous leukemia. ASH Annual Meeting Abstracts;Orlando, FL, USA;2004. 104(11):4527
  • Taga T, Saito AM, Kudo K, et al. Clinical characteristics and outcome of refractory/relapsed myeloid leukemia in children with Down syndrome. Blood 2012;120(9):1810-15
  • Meissner B, Borkhardt A, Dilloo D, et al. Relapse, not regimen-related toxicity, was the major cause of treatment failure in 11 children with Down syndrome undergoing haematopoietic stem cell transplantation for acute leukaemia. Bone Marrow Transplant 2007;40(10):945-9
  • Hitzler JK, He W, Doyle J, et al. Outcome of transplantation for acute myelogenous leukemia in children with Down syndrome. Biol Blood Marrow Transplant 2013;19(6):893-7
  • Muramatsu H, Sakaguchi H, Taga T, et al. Reduced intensity conditioning in allogeneic stem cell transplantation for AML with Down syndrome. Pediatr Blood Cancer 2014;61(5):925-7
  • Kojima S, Kato K, Matsuyama T, et al. Favorable treatment outcome in children with acute myeloid leukemia and Down syndrome. Blood 1993;81(11):3164-4
  • Kojima S, Sako M, Kato K, et al. An effective chemotherapeutic regimen for acute myeloid leukemia and myelodysplastic syndrome in children with Down’s syndrome. Leukemia 2000;14(5):786-91
  • Kudo K, Hama A, Kojima S, et al. Mosaic Down syndrome-associated acute myeloid leukemia does not require high-dose cytarabine treatment for induction and consolidation therapy. Int J Hematol 2010;91(4):630-5
  • Tchernia G, Lejeune F, Boccara JF, et al. Erythroblastic and/or megakaryoblastic leukemia in Down syndrome: treatment with low-dose arabinosyl cytosine. J Pediatr Hematol Oncol 1996;18(1):59-62
  • Tandonnet J, Clavel J, Baruchel A, et al. Myeloid leukaemia in children with Down syndrome: report of the registry-based French experience between 1990 and 2003. Pediatr Blood Cancer 2010;54(7):927-33
  • Hama A, Yagasaki H, Takahashi Y, et al. Acute megakaryoblastic leukaemia (AMKL) in children: a comparison of AMKL with and without Down syndrome. Br J Haematol 2008;140(5):552-61
  • Athale UH, Razzouk BI, Raimondi SC, et al. Biology and outcome of childhood acute megakaryoblastic leukemia: a single institution’s experience. Blood 2001;97(12):3727-32
  • Barnard DR, Alonzo TA, Gerbing RB, et al. Comparison of childhood myelodysplastic syndrome, AML FAB M6 or M7, CCG 2891: report from the Children’s Oncology Group. Pediatr Blood Cancer 2007;49(1):17-22
  • O’Brien MM, Cao X, Pounds S, et al. Prognostic features in acute megakaryoblastic leukemia in children without Down syndrome: a report from the AML02 multicenter trial and the Children’s Oncology Group Study POG 9421. Leukemia 2013;27(3):731-4
  • Reinhardt D, Diekamp S, Langebrake C, et al. Acute megakaryoblastic leukemia in children and adolescents, excluding Down’s syndrome: improved outcome with intensified induction treatment. Leukemia 2005;19(8):1495-6
  • Rubnitz JE, Inaba H, Dahl G, et al. Minimal residual disease-directed therapy for childhood acute myeloid leukaemia: results of the AML02 multicentre trial. Lancet Oncol 2010;11(6):543-52
  • Roberts I, Alford K, Hall G, et al. GATA1-mutant clones are frequent and often unsuspected in babies with Down syndrome: identification of a population at risk of leukemia. Blood 2013;122(24):3908-17
  • Hirai H, Iwasawa Y, Okada M, et al. Small-molecule inhibition of Wee1 kinase by MK-1775 selectively sensitizes p53-deficient tumor cells to DNA-damaging agents. Mol Cancer Ther 2009;8(11):2992-3000
  • Caldwell JT, Edwards H, Buck SA, et al. Targeting the wee1 kinase for treatment of pediatric Down syndrome acute myeloid leukemia. Pediatr Blood Cancer 2014;31(10):1797-73
  • Porter CC, Kim J, Fosmire S, et al. Integrated genomic analyses identify WEE1 as a critical mediator of cell fate and a novel therapeutic target in acute myeloid leukemia. Leukemia 2012;26(6):1266-76
  • Tibes R, Bogenberger JM, Chaudhuri L, et al. RNAi screening of the kinome with cytarabine in leukemias. Blood 2012;119(12):2863-72
  • Van Linden AA, Baturin D, Ford JB, et al. Inhibition of wee1 sensitizes cancer cells to antimetabolite chemotherapeutics in vitro and in vivo, independent of p53 functionality. Mol Cancer Ther 2013;12(12):2675-84
  • Goldenson B, Crispino JD. The aurora kinases in cell cycle and leukemia. Oncogene 2014. [Epub ahead of print]
  • Cervantes A, Elez E, Roda D, et al. Phase I Pharmacokinetic/pharmacodynamic study of MLN8237, an investigational, oral, selective aurora a kinase inhibitor, in patients with advanced solid tumors. Clin Cancer Res 2012;18(17):4764-74
  • Friedberg JW, Mahadevan D, Cebula E, et al. Phase II study of alisertib, a selective aurora a kinase inhibitor, in relapsed and refractory aggressive b- and t-cell non-Hodgkin lymphomas. J Clin Oncol 2014;32(1):44-50
  • Wen Q, Goldenson B, Silver Serena J, et al. Identification of Regulators of Polyploidization Presents Therapeutic Targets for Treatment of AMKL. Cell 2012;150(3):575-89
  • Malinge S, Chlon T, Dore LC, et al. Development of acute megakaryoblastic leukemia in Down syndrome is associated with sequential epigenetic changes. Blood 2013;122(14):e33-43
  • Stankov MV, El Khatib M, Kumar Thakur B, et al. Histone deacetylase inhibitors induce apoptosis in myeloid leukemia by suppressing autophagy. Leukemia 2014;28(3):577-88
  • Maroz A, Stachorski L, Emmrich S, et al. GATA1s induces hyperproliferation of eosinophil precursors in Down syndrome transient leukemia. Leukemia 2014;28(6):1259-70

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.