Skip to main content
SpringerLink
Log in
Book cover

Hematopoietic Cell Transplantation in Children with Cancer pp 221–249Cite as

Acute Myeloid Leukemia

  • Chapter
  • First Online:

  • 1004 Accesses

Part of the book series: Pediatric Oncology ((PEDIATRICO))

Abstract

Great advances have been made in recent years in our understanding of the biology and epidemiology of acute myeloid leukemia (AML). These gains, in turn, have led to the identification of new prognostic markers, improved risk stratification, and more targeted chemotherapies. As outcomes with chemotherapy alone for children with newly diagnosed AML improve, the use of allogeneic hematopoietic cell transplantation (HCT) in the first complete remission has declined. HCT, however, remains the standard for children whose AML relapses. The approach to HCT has, likewise, evolved. The use of autologous transplantation has largely been abandoned; however, with advances in HLA matching in unrelated adult donor transplantation and the growing availability of unrelated cord blood units, the role of unrelated HCT has been greatly expanded. Efforts continue to be made to identify less toxic preparative regimens to abrogate late effects as well as acute transplant-related morbidity and mortality.

This is a preview of subscription content, log in via an institution.

Chapter
USD   29.95
Price excludes VAT (USA)
  • Available as PDF
  • Read on any device
  • Instant download
  • Own it forever
eBook
USD   129.00
Price excludes VAT (USA)
  • Available as EPUB and PDF
  • Read on any device
  • Instant download
  • Own it forever
Softcover Book
USD   169.99
Price excludes VAT (USA)
  • Compact, lightweight edition
  • Dispatched in 3 to 5 business days
  • Free shipping worldwide - see info
Hardcover Book
USD   199.99
Price excludes VAT (USA)
  • Durable hardcover edition
  • Dispatched in 3 to 5 business days
  • Free shipping worldwide - see info

Tax calculation will be finalised at checkout

Purchases are for personal use only

Learn about institutional subscriptions

References

  • Abrahamsson J, Clausen N, Gustafsson G, Hovi L, Jonmundsson G, Zeller B, Forestier E, Heldrup J, Hasle H, Nordic Society for Paediatric Haematology Oncology (2007) Improved outcome after relapse in children with acute myeloid leukaemia. Br J Haematol 136(2):229–236

    PubMed  Google Scholar 

  • Abrahamsson J, Forestier E, Heldrup J, Jahnukainen K, Jonsson OG, Lausen B, Palle J, Zeller B, Hasle H (2011) Response-guided induction therapy in pediatric acute myeloid leukemia with excellent remission rate. J Clin Oncol 29(3):310–315. doi:10.1200/JCO.2010.30.6829, JCO.2010.30.6829 [pii]

    PubMed  Google Scholar 

  • Alter BP, Giri N, Savage SA, Peters JA, Loud JT, Leathwood L, Carr AG, Greene MH, Rosenberg PS (2010) Malignancies and survival patterns in the National Cancer Institute inherited bone marrow failure syndromes cohort study. Br J Haematol 150(2):179–188. doi:10.1111/j.1365-2141.2010.08212.x, BJH8212 [pii]

    PubMed  Google Scholar 

  • Aoudjhane M, Labopin M, Gorin NC, Shimoni A, Ruutu T, Kolb HJ, Frassoni F, Boiron JM, Yin JL, Finke J, Shouten H, Blaise D, Falda M, Fauser AA, Esteve J, Polge E, Slavin S, Niederwieser D, Nagler A, Rocha V, Acute Leukemia Working Party of the European Group for Blood and Marrow Transplantation (2005) Comparative outcome of reduced intensity and myeloablative conditioning regimen in HLA identical sibling allogeneic haematopoietic stem cell transplantation for patients older than 50 years of age with acute myeloblastic leukaemia: a retrospective survey from the Acute Leukemia Working Party (ALWP) of the European Group for Blood and Marrow Transplantation (EBMT). Leukemia 19(12):2304–2312. doi:10.1038/sj.leu.2403967

    PubMed  CAS  Google Scholar 

  • Arceci RJ, Meshinchi S (2011) Biology of acute myeloid leukemia. In: Reaman G, Smith FO (eds) Childhood leukemia. Springer, Heidelberg.

    Google Scholar 

  • Armenian SH, Sun CL, Kawashima T, Arora M, Leisenring W, Sklar CA, Baker KS, Francisco L, Teh JB, Mills G, Wong FL, Rosenthal J, Diller LR, Hudson MM, Oeffinger KC, Forman SJ, Robison LL, Bhatia S (2011) Long-term health-related outcomes in survivors of childhood cancer treated with HSCT versus conventional therapy: a report from the Bone Marrow Transplant Survivor Study (BMTSS) and Childhood Cancer Survivor Study (CCSS). Blood 118(5):1413–1420. doi:10.1182/blood-2011-01-331835

    PubMed  CAS  Google Scholar 

  • Aversa F, Terenzi A, Tabilio A, Falzetti F, Carotti A, Ballanti S, Felicini R, Falcinelli F, Velardi A, Ruggeri L, Aloisi T, Saab JP, Santucci A, Perruccio K, Martelli MP, Mecucci C, Reisner Y, Martelli MF (2005) Full haplotype-mismatched hematopoietic stem-cell transplantation: a phase II study in patients with acute leukemia at high risk of relapse. J Clini Oncol 23(15):3447–3454. doi:10.1200/JCO.2005.09.117, JCO.2005.09.117 [pii]

    Google Scholar 

  • Bader P, Hancock J, Kreyenberg H, Goulden NJ, Niethammer D, Oakhill A, Steward CG, Handgretinger R, Beck JF, Klingebiel T (2002) Minimal residual disease (MRD) status prior to allogeneic stem cell transplantation is a powerful predictor for post-transplant outcome in children with ALL. Leukemia 16(9):1668–1672. doi:10.1038/sj.leu.2402552

    PubMed  CAS  Google Scholar 

  • Bajwa R, Skeens M, Garee A, Miao Y, Soni S, Pietryga D, Gross T, Termuhlen A (2012) Metabolic syndrome and endocrine dysfunctions after HSCT in children. Pediatr Transplant 16(8):872–878. doi:10.1111/petr.12002

    PubMed  Google Scholar 

  • Balgobind BV, Zwaan CM, Pieters R, Van den Heuvel-Eibrink MM (2011) The heterogeneity of pediatric MLL-rearranged acute myeloid leukemia. Leukemia. doi:10.1038/leu.2011.90

    PubMed  Google Scholar 

  • Barker JN, Weisdorf DJ, DeFor TE, Blazar BR, McGlave PB, Miller JS, Verfaillie CM, Wagner JE (2005) Transplantation of 2 partially HLA-matched umbilical cord blood units to enhance engraftment in adults with hematologic malignancy. Blood 105(3):1343–1347. doi:10.1182/blood-2004-07-2717

    PubMed  CAS  Google Scholar 

  • Barnard DR, Lange B, Alonzo TA, Buckley J, Kobrinsky JN, Gold S, Neudorf S, Sanders J, Burden L, Woods WG (2002) Acute myeloid leukemia and myelodysplastic syndrome in children treated for cancer: comparison with primary presentation. Blood 100(2):427–434

    PubMed  CAS  Google Scholar 

  • Beatty PG, Mori M, Milford E (1995) Impact of racial genetic polymorphism on the probability of finding an HLA-matched donor. Transplantation 60(8):778–783

    PubMed  CAS  Google Scholar 

  • Becton D, Dahl GV, Ravindranath Y, Chang MN, Behm FG, Raimondi SC, Head DR, Stine KC, Lacayo NJ, Sikic BI, Arceci RJ, Weinstein H, Pediatric Oncology G (2006) Randomized use of cyclosporin A (CsA) to modulate P-glycoprotein in children with AML in remission: Pediatric Oncology Group Study 9421. Blood 107(4):1315–1324. doi:10.1182/blood-2004-08-3218

    PubMed  CAS  Google Scholar 

  • Bornhauser M, Storer B, Slattery JT, Appelbaum FR, Deeg HJ, Hansen J, Martin PJ, McDonald GB, Nichols WG, Radich J, Woolfrey A, Jenke A, Schleyer E, Thiede C, Ehninger G, Anasetti C (2003) Conditioning with fludarabine and targeted busulfan for transplantation of allogeneic hematopoietic stem cells. Blood 102(3):820–826. doi:10.1182/blood-2002-11-3567

    PubMed  Google Scholar 

  • Bornhauser M, Illmer T, Schaich M, Soucek S, Ehninger G, Thiede C (2007) Improved outcome after stem-cell transplantation in FLT3/ITD-positive AML. Blood 109(5):2264–2265; author reply 2265

    PubMed  Google Scholar 

  • Brice L, Weiss R, Wei Y, Satwani P, Bhatia M, George D, Garvin J, Morris E, Harrison L, Cairo MS, Sands SA (2011) Health-related quality of life (HRQoL): the impact of medical and demographic variables upon pediatric recipients of hematopoietic stem cell transplantation. Pediatr Blood Cancer 57(7):1179–1185. doi:10.1002/pbc.23133

    PubMed  Google Scholar 

  • Brochstein JA, Kernan NA, Groshen S, Cirrincione C, Shank B, Emanuel D, Laver J, O’Reilly RJ (1987) Allogeneic bone marrow transplantation after hyperfractionated total-body irradiation and cyclophosphamide in children with acute leukemia. N Engl J Med 317(26):1618–1624. doi:10.1056/NEJM198712243172602

    PubMed  CAS  Google Scholar 

  • Brown P, McIntyre E, Rau R, Meshinchi S, Lacayo N, Dahl G, Alonzo TA, Chang M, Arceci RJ, Small D (2007) The incidence and clinical significance of nucleophosmin mutations in childhood AML. Blood 110(3):979–985

    PubMed  CAS  Google Scholar 

  • Bunin NJ, Davies SM, Aplenc R, Camitta BM, DeSantes KB, Goyal RK, Kapoor N, Kernan NA, Rosenthal J, Smith FO, Eapen M (2008) Unrelated donor bone marrow transplantation for children with acute myeloid leukemia beyond first remission or refractory to chemotherapy. J Clin Oncol 26(26):4326–4332. doi:10.1200/JCO.2008.16.4442

    PubMed  Google Scholar 

  • Confer DL (2001) The National Marrow Donor Program. Meeting the needs of the medically underserved. Cancer 91(1 Suppl):274–278. doi:10.1002/1097-0142(20010101)91:1+<274::AID-CNCR18>3.0.CO;2-E [pii]

    PubMed  CAS  Google Scholar 

  • Cooper TM, Franklin J, Gerbing RB, Alonzo TA, Hurwitz C, Raimondi SC, Hirsch B, Smith FO, Mathew P, Arceci RJ, Feusner J, Iannone R, Lavey RS, Meshinchi S, Gamis A (2012) AAML03P1, a pilot study of the safety of gemtuzumab ozogamicin in combination with chemotherapy for newly diagnosed childhood acute myeloid leukemia: a report from the Children’s Oncology Group. Cancer 118(3):761–769. doi:10.1002/cncr.26190

    PubMed  CAS  Google Scholar 

  • Creutzig U, Ritter J, Zimmermann M, Reinhardt D, Hermann J, Berthold F, Henze G, Jurgens H, Kabisch H, Havers W, Reiter A, Kluba U, Niggli F, Gadner H (2001) Improved treatment results in high-risk pediatric acute myeloid leukemia patients after intensification with high-dose cytarabine and mitoxantrone: results of Study Acute Myeloid Leukemia-Berlin-Frankfurt-Munster 93. J Clin Oncol 19(10):2705–2713

    PubMed  CAS  Google Scholar 

  • Davies SM, Wang D, Wang T, Arora M, Ringden O, Anasetti C, Pavletic S, Casper J, Macmillan ML, Sanders J, Wall D, Kernan NA (2009) Recent decrease in acute graft-versus-host disease in children with leukemia receiving unrelated donor bone marrow transplants. Biol Blood Marrow Transplant 15(3):360–366. doi:10.1016/j.bbmt.2008.12.495

    PubMed  Google Scholar 

  • de Lima M, Couriel D, Thall PF, Wang X, Madden T, Jones R, Shpall EJ, Shahjahan M, Pierre B, Giralt S, Korbling M, Russell JA, Champlin RE, Andersson BS (2004) Once-daily intravenous busulfan and fludarabine: clinical and pharmacokinetic results of a myeloablative, reduced-toxicity conditioning regimen for allogeneic stem cell transplantation in AML and MDS. Blood 104(3):857–864. doi:10.1182/blood-2004-02-0414

    PubMed  Google Scholar 

  • Dohner K, Schlenk RF, Habdank M, Scholl C, Rucker FG, Corbacioglu A, Bullinger L, Frohling S, Dohner H (2005) Mutant nucleophosmin (NPM1) predicts favorable prognosis in younger adults with acute myeloid leukemia and normal cytogenetics: interaction with other gene mutations. Blood 106(12):3740–3746. doi:10.1182/blood-2005-05-2164, 2005-05-2164 [pii]

    PubMed  Google Scholar 

  • Dokal I (2000) Dyskeratosis congenita in all its forms. Br J Haematol 110(4):768–779. doi:bjh2109 [pii]

    PubMed  CAS  Google Scholar 

  • Donadieu J, Leblanc T, Bader Meunier B, Barkaoui M, Fenneteau O, Bertrand Y, Maier-Redelsperger M, Micheau M, Stephan JL, Phillipe N, Bordigoni P, Babin-Boilletot A, Bensaid P, Manel AM, Vilmer E, Thuret I, Blanche S, Gluckman E, Fischer A, Mechinaud F, Joly B, Lamy T, Hermine O, Cassinat B, Bellanne-Chantelot C, Chomienne C (2005) Analysis of risk factors for myelodysplasias, leukemias and death from infection among patients with congenital neutropenia. Experience of the French Severe Chronic Neutropenia Study Group. Haematologica 90(1):45–53

    PubMed  Google Scholar 

  • Douer D, Preston-Martin S, Chang E, Nichols PW, Watkins KJ, Levine AM (1996) High frequency of acute promyelocytic leukemia among Latinos with acute myeloid leukemia. Blood 87(1):308–313

    PubMed  CAS  Google Scholar 

  • Duval M, Klein JP, He W, Cahn JY, Cairo M, Camitta BM, Kamble R, Copelan E, de Lima M, Gupta V, Keating A, Lazarus HM, Litzow MR, Marks DI, Maziarz RT, Rizzieri DA, Schiller G, Schultz KR, Tallman MS, Weisdorf D (2010) Hematopoietic stem-cell transplantation for acute leukemia in relapse or primary induction failure. J Clin Oncol 28(23):3730–3738. doi:10.1200/JCO.2010.28.8852

    PubMed  Google Scholar 

  • Dvorak CC, Agarwal R, Dahl GV, Gregory JJ, Feusner JH (2008) Hematopoietic stem cell transplant for pediatric acute promyelocytic leukemia. Biol Blood Marrow Transplant 14(7):824–830. doi:10.1016/j.bbmt.2008.04.015, S1083-8791(08)00181-X [pii]

    PubMed  Google Scholar 

  • Eapen M, Horowitz MM, Klein JP, Champlin RE, Loberiza FR Jr, Ringden O, Wagner JE (2004) Higher mortality after allogeneic peripheral-blood transplantation compared with bone marrow in children and adolescents: the Histocompatibility and Alternate Stem Cell Source Working Committee of the International Bone Marrow Transplant Registry. J Clin Oncol 22(24):4872–4880

    PubMed  Google Scholar 

  • Eapen M, Rubinstein P, Zhang MJ, Camitta BM, Stevens C, Cairo MS, Davies SM, Doyle JJ, Kurtzberg J, Pulsipher MA, Ortega JJ, Scaradavou A, Horowitz MM, Wagner JE (2006) Comparable long-term survival after unrelated and HLA-matched sibling donor hematopoietic stem cell transplantations for acute leukemia in children younger than 18 months. J Clin Oncol 24(1):145–151. doi:10.1200/JCO.2005.02.4612, 24/1/145 [pii]

    PubMed  Google Scholar 

  • Eapen M, Rubinstein P, Zhang MJ, Stevens C, Kurtzberg J, Scaradavou A, Loberiza FR, Champlin RE, Klein JP, Horowitz MM, Wagner JE (2007) Outcomes of transplantation of unrelated donor umbilical cord blood and bone marrow in children with acute leukaemia: a comparison study. Lancet 369(9577):1947–1954

    PubMed  Google Scholar 

  • Flomenberg N, Baxter-Lowe LA, Confer D, Fernandez-Vina M, Filipovich A, Horowitz M, Hurley C, Kollman C, Anasetti C, Noreen H, Begovich A, Hildebrand W, Petersdorf E, Schmeckpeper B, Setterholm M, Trachtenberg E, Williams T, Yunis E, Weisdorf D (2004) Impact of HLA class I and class II high-resolution matching on outcomes of unrelated donor bone marrow transplantation: HLA-C mismatching is associated with a strong adverse effect on transplantation outcome. Blood 104(7):1923–1930. doi:10.1182/blood-2004-03-0803, 2004-03-0803 [pii]

    PubMed  CAS  Google Scholar 

  • Gale RE, Green C, Allen C, Mead AJ, Burnett AK, Hills RK, Linch DC (2007) The impact of FLT3 internal tandem duplication mutant level, number, size and interaction with NPM1 mutations in a large cohort of young adult patients with acute myeloid leukemia. Blood 111(5):2776–2784

    PubMed  Google Scholar 

  • Gibson BE, Wheatley K, Hann IM, Stevens RF, Webb D, Hills RK, De Graaf SS, Harrison CJ (2005) Treatment strategy and long-term results in paediatric patients treated in consecutive UK AML trials. Leukemia 19(12):2130–2138. doi:10.1038/sj.leu.2403924

    PubMed  CAS  Google Scholar 

  • Gibson BE, Webb DK, Howman AJ, De Graaf SS, Harrison CJ, Wheatley K (2011) Results of a randomized trial in children with Acute Myeloid Leukaemia: medical research council AML12 trial. Br J Haematol 155(3):366–376. doi:10.1111/j.1365-2141.2011.08851.x

    PubMed  CAS  Google Scholar 

  • Goemans BF, Tamminga RY, Corbijn CM, Hahlen K, Kaspers GJ (2008) Outcome for children with relapsed acute myeloid leukemia in the Netherlands following initial treatment between 1980 and 1998: survival after chemotherapy only? Haematologica 93(9):1418–1420. doi:10.3324/haematol.12807

    PubMed  Google Scholar 

  • Greaves MF, Maia AT, Wiemels JL, Ford AM (2003) Leukemia in twins: lessons in natural history. Blood 102(7):2321–2333. doi:10.1182/blood-2002-12-3817, 2002-12-3817 [pii]

    PubMed  CAS  Google Scholar 

  • Gupta V, Tallman MS, He W, Logan BR, Copelan E, Gale RP, Khoury HJ, Klumpp T, Koreth J, Lazarus HM, Marks DI, Martino R, Rizzieri DA, Rowe JM, Sabloff M, Waller EK, DiPersio JF, Bunjes DW, Weisdorf DJ (2010) Comparable survival after HLA-well-matched unrelated or matched sibling donor transplantation for acute myeloid leukemia in first remission with unfavorable cytogenetics at diagnosis. Blood 116(11):1839–1848. doi:10.1182/blood-2010-04-278317, blood-2010-04-278317 [pii]

    PubMed  CAS  Google Scholar 

  • Gurney JG, Severson RK, Davis S, Robison LL (1995) Incidence of cancer in children in the United States. Sex-, race-, and 1-year age-specific rates by histologic type. Cancer 75(8):2186–2195

    PubMed  CAS  Google Scholar 

  • Hasle H, Mellemgaard A, Nielsen J, Hansen J (1995) Cancer incidence in men with Klinefelter syndrome. Br J Cancer 71(2):416–420

    PubMed  CAS  Google Scholar 

  • Hasle H, Olsen JH, Nielsen J, Hansen J, Friedrich U, Tommerup N (1996) Occurrence of cancer in women with Turner syndrome. Br J Cancer 73(9):1156–1159

    PubMed  CAS  Google Scholar 

  • Hasle H, Arico M, Basso G, Biondi A, Cantu Rajnoldi A, Creutzig U, Fenu S, Fonatsch C, Haas OA, Harbott J, Kardos G, Kerndrup G, Mann G, Niemeyer CM, Ptoszkova H, Ritter J, Slater R, Stary J, Stollmann-Gibbels B, Testi AM, van Wering ER, Zimmermann M (1999a) Myelodysplastic syndrome, juvenile myelomonocytic leukemia, and acute myeloid leukemia associated with complete or partial monosomy 7. European Working Group on MDS in Childhood (EWOG-MDS). Leukemia 13(3):376–385

    PubMed  CAS  Google Scholar 

  • Hasle H, Wadsworth LD, Massing BG, McBride M, Schultz KR (1999b) A population-based study of childhood myelodysplastic syndrome in British Columbia, Canada. Br J Haematol 106(4):1027–1032. doi:bjh1645 [pii]

    PubMed  CAS  Google Scholar 

  • Hasle H, Clemmensen IH, Mikkelsen M (2000) Risks of leukaemia and solid tumours in individuals with Down’s syndrome. Lancet 355(9199):165–169. doi:10.1016/S0140-6736(99)05264-2, S0140-6736(99)05264-2 [pii]

    PubMed  CAS  Google Scholar 

  • Hasle H, Abrahamsson J, Arola M, Karow A, O’Marcaigh A, Reinhardt D, Webb DK, van Wering E, Zeller B, Zwaan CM, Vyas P (2008) 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 22(7):1428–1430. doi:10.1038/sj.leu.2405060, 2405060 [pii]

    PubMed  CAS  Google Scholar 

  • Hjalgrim LL, Rostgaard K, Schmiegelow K, Soderhall S, Kolmannskog S, Vettenranta K, Kristinsson J, Clausen N, Melbye M, Hjalgrim H, Gustafsson G (2003) Age- and sex-specific incidence of childhood leukemia by immunophenotype in the Nordic countries. J Natl Cancer Inst 95(20):1539–1544

    PubMed  Google Scholar 

  • Ho P, Alonzo TA, Gerbing RB, Pollard JA, Stirewalt D, Hurwitz CA, Heerema NA, Hirsch B, Raimondi SC, Lange B, Franklin J, Radich J, Meshinchi S (2008) CEBPA mutations predict favorable prognosis in pediatric AML. ASH Ann Meet Abstr 112(11):142

    Google Scholar 

  • Ho PA, Alonzo TA, Gerbing RB, Pollard J, Stirewalt DL, Hurwitz C, Heerema NA, Hirsch B, Raimondi SC, Lange B, Franklin JL, Radich JP, Meshinchi S (2009) Prevalence and prognostic implications of CEBPA mutations in pediatric acute myeloid leukemia (AML): a report from the Children’s Oncology Group. Blood 113(26):6558–6566. doi:10.1182/blood-2008-10-184747, blood-2008-10-184747 [pii]

    PubMed  CAS  Google Scholar 

  • Ho PA, Kutny MA, Alonzo TA, Gerbing RB, Joaquin J, Raimondi SC, Gamis AS, Meshinchi S (2011) Leukemic mutations in the methylation-associated genes DNMT3A and IDH2 are rare events in pediatric AML: a report from the Children’s Oncology Group. Pediatr Blood Cancer 57(2):204–209. doi:10.1002/pbc.23179

    PubMed  Google Scholar 

  • Hollink IH, Zwaan CM, van den Heuvel-Eibrink MM, Zimmerman M, Arentsen-Peters S, Pieters R, Cloos J, Kaspers GJ, De Graaf SS, Creutzig U, Reinhardt D, Thiede C (2007) Nucleophosmin gene mutations identify a favorable risk group in childhood acute myeloid leukemia with a normal karyotype. ASH Ann Meet Abstr 110(11):366

    Google Scholar 

  • Horan JT, Alonzo TA, Lyman GH, Gerbing RB, Lange BJ, Ravindranath Y, Becton D, Smith FO, Woods WG (2008) Impact of disease risk on efficacy of matched related bone marrow transplantation for pediatric acute myeloid leukemia: the Children’s Oncology Group. J Clin Oncol 26(35):5797–5801

    PubMed  Google Scholar 

  • Horan JT, Logan BR, Agovi-Johnson MA, Lazarus HM, Bacigalupo AA, Ballen KK, Bredeson CN, Carabasi MH, Gupta V, Hale GA, Khoury HJ, Juckett MB, Litzow MR, Martino R, McCarthy PL, Smith FO, Rizzo JD, Pasquini MC (2011) Reducing the risk for transplantation-related mortality after allogeneic hematopoietic cell transplantation: how much progress has been made? J Clin Oncol 29(7):805–813. doi:10.1200/JCO.2010.32.5001

    PubMed  Google Scholar 

  • Horowitz MM, Gale RP, Sondel PM, Goldman JM, Kersey J, Kolb HJ, Rimm AA, Ringden O, Rozman C, Speck B et al (1990) Graft-versus-leukemia reactions after bone marrow transplantation. Blood 75(3):555–562

    PubMed  CAS  Google Scholar 

  • Huang X, Liu D, Liu K, Xu L, Chen H, Han W, Chen Y, Wang Y, Zhang X (2009) Haploidentical hematopoietic stem cell transplantation without in vitro T cell depletion for treatment of hematologic malignancies in children. Biol Blood Marrow Transplant 15(1 Suppl):91–94. doi:10.1016/j.bbmt.2008.10.019, S1083-8791(08)00469-2 [pii]

    PubMed  Google Scholar 

  • Kadan-Lottick NS, Kawashima T, Tomlinson G, Friedman DL, Yasui Y, Mertens AC, Robison LL, Strong LC (2006) The risk of cancer in twins: a report from the childhood cancer survivor study. Pediatr Blood Cancer 46(4):476–481. doi:10.1002/pbc.20465

    PubMed  Google Scholar 

  • Kaspers GJ, Zimmermann M, Reinhardt D, Gibson BE, Tamminga RY, Aleinikova O, Armendariz H, Dworzak M, Ha SY, Hasle H, Hovi L, Maschan A, Bertrand Y, Leverger GG, Razzouk BI, Rizzari C, Smisek P, Smith O, Stark B, Creutzig U (2013) Improved outcome in pediatric relapsed acute myeloid leukemia: results of a randomized trial on liposomal daunorubicin by the International BFM Study Group. J Clin Oncol 31(5):599–607. doi:10.1200/JCO.2012.43.7384

    PubMed  CAS  Google Scholar 

  • Kelly LM, Kutok JL, Williams IR, Boulton CL, Amaral SM, Curley DP, Ley TJ, Gilliland DG (2002a) PML/RARalpha and FLT3-ITD induce an APL-like disease in a mouse model. Proc Natl Acad Sci U S A 99(12):8283–8288

    PubMed  CAS  Google Scholar 

  • Kelly LM, Yu JC, Boulton CL, Apatira M, Li J, Sullivan CM, Williams I, Amaral SM, Curley DP, Duclos N, Neuberg D, Scarborough RM, Pandey A, Hollenbach S, Abe K, Lokker NA, Gilliland DG, Giese NA (2002b) CT53518, a novel selective FLT3 antagonist for the treatment of acute myelogenous leukemia (AML). Cancer Cell 1(5):421–432

    PubMed  CAS  Google Scholar 

  • Kelly LM, Horan J, Alonzo TA (2012) Hematopoietic cell transplant versus chemotherapy as consolidation treatment for pediatric AML with poor-risk cytogenetics. American Society of Hematology Annual Meeting, Atlanta

    Google Scholar 

  • Kernan NA, Bartsch G, Ash RC, Beatty PG, Champlin R, Filipovich A, Gajewski J, Hansen JA, Henslee-Downey J, McCullough J et al (1993) Analysis of 462 transplantations from unrelated donors facilitated by the National Marrow Donor Program. N Engl J Med 328(9):593–602

    PubMed  CAS  Google Scholar 

  • Kersey JH, Ramsay NK, Kim T, McGlave P, Krivit W, Levitt S, Filipovich A, Woods W, O’Leary M, Coccia P, Nesbit ME (1982) Allogeneic bone marrow transplantation in acute nonlymphocytic leukemia: a pilot study. Blood 60(2):400–403

    PubMed  CAS  Google Scholar 

  • Kirstetter P, Schuster MB, Bereshchenko O, Moore S, Dvinge H, Kurz E, Theilgaard-Monch K, Mansson R, Pedersen TA, Pabst T, Schrock E, Porse BT, Jacobsen SE, Bertone P, Tenen DG, Nerlov C (2008) Modeling of C/EBPalpha mutant acute myeloid leukemia reveals a common expression signature of committed myeloid leukemia-initiating cells. Cancer Cell 13(4):299–310. doi:10.1016/j.ccr.2008.02.008, S1535-6108(08)00044-5 [pii]

    PubMed  CAS  Google Scholar 

  • Kletzel M, Jacobsohn D, Duerst R (2006) Pharmacokinetics of a test dose of intravenous busulfan guide dose modifications to achieve an optimal area under the curve of a single daily dose of intravenous busulfan in children undergoing a reduced-intensity conditioning regimen with hematopoietic stem cell transplantation. Biol Blood Marrow Transplant 12(4):472–479. doi:10.1016/j.bbmt.2005.12.028

    PubMed  CAS  Google Scholar 

  • Klusmann JH, Reinhardt D, Zimmermann M, Kremens B, Vormoor J, Dworzak M, Creutzig U, Klingebiel T (2012) The role of matched sibling donor allogeneic stem cell transplantation in pediatric high-risk acute myeloid leukemia: results from the AML-BFM 98 study. Haematologica 97(1):21–29. doi:10.3324/haematol.2011.051714

    PubMed  CAS  Google Scholar 

  • Knechtli CJ, Goulden NJ, Hancock JP, Grandage VL, Harris EL, Garland RJ, Jones CG, Rowbottom AW, Hunt LP, Green AF, Clarke E, Lankester AW, Cornish JM, Pamphilon DH, Steward CG, Oakhill A (1998) Minimal residual disease status before allogeneic bone marrow transplantation is an important determinant of successful outcome for children and adolescents with acute lymphoblastic leukemia. Blood 92(11):4072–4079

    PubMed  CAS  Google Scholar 

  • Kurtzberg J, Prasad VK, Carter SL, Wagner JE, Baxter-Lowe LA, Wall D, Kapoor N, Guinan EC, Feig SA, Wagner EL, Kernan NA, Committee CS (2008) Results of the Cord Blood Transplantation Study (COBLT): clinical outcomes of unrelated donor umbilical cord blood transplantation in pediatric patients with hematologic malignancies. Blood 112(10):4318–4327. doi:10.1182/blood-2007-06-098020

    PubMed  CAS  Google Scholar 

  • Lange BJ, Smith FO, Feusner J, Barnard DR, Dinndorf P, Feig S, Heerema NA, Arndt C, Arceci RJ, Seibel N, Weiman M, Dusenbery K, Shannon K, Luna-Fineman S, Gerbing RB, Alonzo TA (2008) Outcomes in CCG-2961, a children’s oncology group phase 3 trial for untreated pediatric acute myeloid leukemia: a report from the children’s oncology group. Blood 111(3):1044–1053. doi:10.1182/blood-2007-04-084293

    PubMed  CAS  Google Scholar 

  • Leahey AM, Teunissen H, Friedman DL, Moshang T, Lange BJ, Meadows AT (1999) Late effects of chemotherapy compared to bone marrow transplantation in the treatment of pediatric acute myeloid leukemia and myelodysplasia. Med Pediatr Oncol 32(3):163–169

    PubMed  CAS  Google Scholar 

  • Lee SJ, Klein J, Haagenson M, Baxter-Lowe LA, Confer DL, Eapen M, Fernandez-Vina M, Flomenberg N, Horowitz M, Hurley CK, Noreen H, Oudshoorn M, Petersdorf E, Setterholm M, Spellman S, Weisdorf D, Williams TM, Anasetti C (2007) High-resolution donor-recipient HLA matching contributes to the success of unrelated donor marrow transplantation. Blood 110(13):4576–4583. doi:10.1182/blood-2007-06-097386, blood-2007-06-097386 [pii]

    PubMed  CAS  Google Scholar 

  • Lee JW, Jang PS, Chung NG, Cho B, Kim HK (2013) Treatment of children with acute myeloid leukaemia who relapsed after allogeneic haematopoietic stem cell transplantation. Br J Haematol 160(1):80–86. doi:10.1111/bjh.12074

    PubMed  Google Scholar 

  • Leung W, Hudson MM, Strickland DK, Phipps S, Srivastava DK, Ribeiro RC, Rubnitz JE, Sandlund JT, Kun LE, Bowman LC, Razzouk BI, Mathew P, Shearer P, Evans WE, Pui CH (2000) Late effects of treatment in survivors of childhood acute myeloid leukemia. J Clin Oncol 18(18):3273–3279

    PubMed  CAS  Google Scholar 

  • Leung W, Pui CH, Coustan-Smith E, Yang J, Pei D, Gan K, Srinivasan A, Hartford C, Triplett BM, Dallas M, Pillai A, Shook D, Rubnitz JE, Sandlund JT, Jeha S, Inaba H, Ribeiro RC, Handgretinger R, Laver JH, Campana D (2012) Detectable minimal residual disease before hematopoietic cell transplantation is prognostic but does not preclude cure for children with very-high-risk leukemia. Blood 120(2):468–472. doi:10.1182/blood-2012-02-409813

    PubMed  CAS  Google Scholar 

  • Levine RL, Gilliland DG (2008) Myeloproliferative disorders. Blood 112(6):2190–2198

    PubMed  CAS  Google Scholar 

  • Levine JE, Braun T, Penza SL, Beatty P, Cornetta K, Martino R, Drobyski WR, Barrett AJ, Porter DL, Giralt S, Leis J, Holmes HE, Johnson M, Horowitz M, Collins RH Jr (2002) Prospective trial of chemotherapy and donor leukocyte infusions for relapse of advanced myeloid malignancies after allogeneic stem-cell transplantation. J Clin Oncol 20(2):405–412

    PubMed  CAS  Google Scholar 

  • Levine JE, Barrett AJ, Zhang MJ, Arora M, Pulsipher MA, Bunin N, Fort J, Loberiza F, Porter D, Giralt S, Drobyski W, Wang D, Pavletic S, Ringden O, Horowitz MM, Collins R Jr (2008) Donor leukocyte infusions to treat hematologic malignancy relapse following allo-SCT in a pediatric population. Bone Marrow Transplant 42(3):201–205. doi:10.1038/bmt.2008.135, bmt2008135 [pii]

    PubMed  CAS  Google Scholar 

  • Liesner RJ, Leiper AD, Hann IM, Chessells JM (1994) Late effects of intensive treatment for acute myeloid leukemia and myelodysplasia in childhood. J Clin Oncol 12(5):916–924

    PubMed  CAS  Google Scholar 

  • Litzow MR, Perez WS, Klein JP, Bolwell BJ, Camitta B, Copelan EA, Gale RP, Giralt SA, Keating A, Lazarus HM, Marks DI, McCarthy PL, Miller CB, Milone G, Prentice HG, Russell JA, Schultz KR, Trigg ME, Weisdorf DJ, Horowitz MM (2002) Comparison of outcome following allogeneic bone marrow transplantation with cyclophosphamide-total body irradiation versus busulphan-cyclophosphamide conditioning regimens for acute myelogenous leukaemia in first remission. Br J Haematol 119(4):1115–1124

    PubMed  CAS  Google Scholar 

  • Locatelli F, Zecca M, Rondelli R, Bonetti F, Dini G, Prete A, Messina C, Uderzo C, Ripaldi M, Porta F, Giorgiani G, Giraldi E, Pession A (2000) Graft versus host disease prophylaxis with low-dose cyclosporine-A reduces the risk of relapse in children with acute leukemia given HLA-identical sibling bone marrow transplantation: results of a randomized trial. Blood 95(5):1572–1579

    PubMed  CAS  Google Scholar 

  • Lof CM, Winiarski J, Giesecke A, Ljungman P, Forinder U (2009) Health-related quality of life in adult survivors after paediatric allo-SCT. Bone Marrow Transplant 43(6):461–468. doi:10.1038/bmt.2008.338

    PubMed  CAS  Google Scholar 

  • Loken MR, Alonzo TA, Pardo L, Gerbing RB, Raimondi SC, Hirsch BA, Ho PA, Franklin J, Cooper TM, Gamis AS, Meshinchi S (2012) Residual disease detected by multidimensional flow cytometry signifies high relapse risk in patients with de novo acute myeloid leukemia: a report from Children’s Oncology Group. Blood 120(8):1581–1588. doi:10.1182/blood-2012-02-408336, blood-2012-02-408336 [pii]

    PubMed  Google Scholar 

  • MacMillan ML, Weisdorf DJ, Brunstein CG, Cao Q, DeFor TE, Verneris MR, Blazar BR, Wagner JE (2009) Acute graft-versus-host disease after unrelated donor umbilical cord blood transplantation: analysis of risk factors. Blood 113(11):2410–2415. doi:10.1182/blood-2008-07-163238

    PubMed  CAS  Google Scholar 

  • Maserati E, Aprili F, Vinante F, Locatelli F, Amendola G, Zatterale A, Milone G, Minelli A, Bernardi F, Lo Curto F, Pasquali F (2002) Trisomy 8 in myelodysplasia and acute leukemia is constitutional in 15-20% of cases. Genes Chromosomes Cancer 33(1):93–97. doi:10.1002/gcc.1214

    PubMed  CAS  Google Scholar 

  • Maserati E, Panarello C, Morerio C, Valli R, Pressato B, Patitucci F, Tassano E, Di Cesare-Merlone A, Cugno C, Balduini CL, Lo Curto F, Dufour C, Locatelli F, Pasquali F (2008) Clonal chromosome anomalies and propensity to myeloid malignancies in congenital amegakaryocytic thrombocytopenia (OMIM 604498). Haematologica 93(8):1271–1273. doi:10.3324/haematol.12748, haematol.12748 [pii]

    PubMed  Google Scholar 

  • Meshinchi S, Woods WG, Stirewalt DL, Sweetser DA, Buckley JD, Tjoa TK, Bernstein ID, Radich JP (2001) Prevalence and prognostic significance of Flt3 internal tandem duplication in pediatric acute myeloid leukemia. Blood 97(1):89–94

    PubMed  CAS  Google Scholar 

  • Meshinchi S, Leisenring WM, Carpenter PA, Woolfrey AE, Sievers EL, Radich JP, Sanders JE (2003) Survival after second hematopoietic stem cell transplantation for recurrent pediatric acute myeloid leukemia. Biol Blood Marrow Transplant 9(11):706–713. doi:10.1016/j.bbmt.2003.08.003, S108387910300291X [pii]

    PubMed  Google Scholar 

  • Meshinchi S, Alonzo TA, Stirewalt DL, Zwaan M, Zimmerman M, Reinhardt D, Kaspers GJ, Heerema NA, Gerbing R, Lange BJ, Radich JP (2006a) Clinical implications of FLT3 mutations in pediatric AML. Blood 108(12):3654–3661

    PubMed  CAS  Google Scholar 

  • Meshinchi S, Arceci RJ, Sanders JE, Smith FO, Woods WB, Radich JP, Alonzo TA (2006b) Role of allogeneic stem cell transplantation in FLT3/ITD-positive AML. Blood 108(1):400; author reply 400-401

    PubMed  CAS  Google Scholar 

  • Meshinchi S, Ries RE, Trevino LR, Hampton OA, Alonzo T, Farrar JE, Auvil JMG, Davidsen TM, Gesuwan P, Muzny DM, Gamis AS, Helton HL, Wheeler DA, Smith MA, Gerhard DS, Arceci RJ (2012) Identification of novel somatic mutations, regions of recurrent loss of heterozygosity (LOH) and significant clonal evolution from diagnosis to relapse in childhood AML determined by exome capture sequencing – an NCI/COG target AML study. ASH Ann Meet Abstr 120(21):123

    Google Scholar 

  • Messerer D, Engel J, Hasford J, Schaich M, Ehninger G, Sauerland C, Buchner T, Schumacher A, Krahl R, Niederwieser D, Krauter J, Ganser A, Creutzig U, Dohner H, Schlenk RF (2008) Impact of different post-remission strategies on quality of life in patients with acute myeloid leukemia. Haematologica 93(6):826–833. doi:10.3324/haematol.11987

    PubMed  Google Scholar 

  • Michel G, Gluckman E, Esperou-Bourdeau H, Reiffers J, Pico JL, Bordigoni P, Thuret I, Blaise D, Bernaudin F, Jouet JP et al (1994) Allogeneic bone marrow transplantation for children with acute myeloblastic leukemia in first complete remission: impact of conditioning regimen without total-body irradiation – a report from the Societe Francaise de Greffe de Moelle. J Clin Oncol 12(6):1217–1222

    PubMed  CAS  Google Scholar 

  • Michel G, Socie G, Gebhard F, Bernaudin F, Thuret I, Vannier JP, Demeocq F, Leverger G, Pico JL, Rubie H, Mechinaud F, Reiffers J, Gratecos N, Troussard X, Jouet JP, Simonin G, Gluckman E, Maraninchi D (1997) Late effects of allogeneic bone marrow transplantation for children with acute myeloblastic leukemia in first complete remission: the impact of conditioning regimen without total-body irradiation – a report from the Societe Francaise de Greffe de Moelle. J Clin Oncol 15(6):2238–2246

    PubMed  CAS  Google Scholar 

  • Molgaard-Hansen L, Glosli H, Jahnukainen K, Jarfelt M, Jonmundsson GK, Malmros-Svennilson J, Nysom K, Hasle H (2011) Quality of health in survivors of childhood acute myeloid leukemia treated with chemotherapy only: a NOPHO-AML study. Pediatr Blood Cancer 57(7):1222–1229. doi:10.1002/pbc.22931

    PubMed  Google Scholar 

  • Morishima Y, Sasazuki T, Inoko H, Juji T, Akaza T, Yamamoto K, Ishikawa Y, Kato S, Sao H, Sakamaki H, Kawa K, Hamajima N, Asano S, Kodera Y (2002) The clinical significance of human leukocyte antigen (HLA) allele compatibility in patients receiving a marrow transplant from serologically HLA-A, HLA-B, and HLA-DR matched unrelated donors. Blood 99(11):4200–4206

    PubMed  CAS  Google Scholar 

  • Mulcahy Levy JM, Tello T, Giller R, Wilkening G, Quinones R, Keating AK, Liu AK (2013) Late effects of total body irradiation and hematopoietic stem cell transplant in children under 3 years of age. Pediatr Blood Cancer 60(4):700–704. doi:10.1002/pbc.24252

    PubMed  Google Scholar 

  • Mulrooney DA, Dover DC, Li S, Yasui Y, Ness KK, Mertens AC, Neglia JP, Sklar CA, Robison LL, Davies SM (2008) Twenty years of follow-up among survivors of childhood and young adult acute myeloid leukemia: a report from the Childhood Cancer Survivor Study. Cancer 112(9):2071–2079. doi:10.1002/cncr.23405

    PubMed  Google Scholar 

  • Munoz A, Badell I, Olive T, Verdeguer A, Gomez P, Bureo E, Spanish Working Party for Bone Marrow Transplantation in Children (2002) Second allogeneic hematopoietic stem cell transplantation in hematologic malignancies in children: long-term results of a multicenter study of the Spanish Working Party for Bone Marrow Transplantation in Children (GETMON). Haematologica 87(3):331–332

    PubMed  Google Scholar 

  • Nakao M, Yokota S, Iwai T, Kaneko H, Horiike S, Kashima K, Sonoda Y, Fujimoto T, Misawa S (1996) Internal tandem duplication of the flt3 gene found in acute myeloid leukemia. Leukemia 10(12):1911–1918

    PubMed  CAS  Google Scholar 

  • Nanri T, Uike N, Kawakita T, Iwanaga E, Mitsuya H, Asou N (2010) A family harboring a germ-line N-terminal C/EBPalpha mutation and development of acute myeloid leukemia with an additional somatic C-terminal C/EBPalpha mutation. Genes Chromosomes Cancer 49(3):237–241. doi:10.1002/gcc.20734

    PubMed  CAS  Google Scholar 

  • Nash RA, Antin JH, Karanes C, Fay JW, Avalos BR, Yeager AM, Przepiorka D, Davies S, Petersen FB, Bartels P, Buell D, Fitzsimmons W, Anasetti C, Storb R, Ratanatharathorn V (2000) Phase 3 study comparing methotrexate and tacrolimus with methotrexate and cyclosporine for prophylaxis of acute graft-versus-host disease after marrow transplantation from unrelated donors. Blood 96(6):2062–2068

    PubMed  CAS  Google Scholar 

  • Nemecek ER, Guthrie KA, Sorror ML, Wood BL, Doney KC, Hilger RA, Scott BL, Kovacsovics TJ, Maziarz RT, Woolfrey AE, Bedalov A, Sanders JE, Pagel JM, Sickle EJ, Witherspoon R, Flowers ME, Appelbaum FR, Deeg HJ (2011) Conditioning with treosulfan and fludarabine followed by allogeneic hematopoietic cell transplantation for high-risk hematologic malignancies. Biol Blood Marrow Transplant 17(3):341–350. doi:10.1016/j.bbmt.2010.05.007

    PubMed  CAS  Google Scholar 

  • Neudorf S, Sanders J, Kobrinsky N, Alonzo TA, Buxton AB, Gold S, Barnard DR, Wallace JD, Kalousek D, Lange BJ, Woods WG (2004) Allogeneic bone marrow transplantation for children with acute myelocytic leukemia in first remission demonstrates a role for graft versus leukemia in the maintenance of disease-free survival. Blood 103(10):3655–3661. doi:10.1182/blood-2003-08-2705

    PubMed  CAS  Google Scholar 

  • Owen C, Barnett M, Fitzgibbon J (2008) Familial myelodysplasia and acute myeloid leukaemia – a review. Br J Haematol 140(2):123–132. doi:10.1111/j.1365-2141.2007.06909.x, BJH6909 [pii]

    PubMed  CAS  Google Scholar 

  • Pabst T, Eyholzer M, Haefliger S, Schardt J, Mueller BU (2008) Somatic CEBPA mutations are a frequent second event in families with germline CEBPA mutations and familial acute myeloid leukemia. J Clin Oncol 26(31):5088–5093. doi:10.1200/JCO.2008.16.5563, JCO.2008.16.5563 [pii]

    PubMed  CAS  Google Scholar 

  • Parsons SK, Barlow SE, Levy SL, Supran SE, Kaplan SH (1999) Health-related quality of life in pediatric bone marrow transplant survivors: according to whom? Int I Cancer Suppl Suppl 12:46–51

    Google Scholar 

  • Parsons SK, Phipps S, Sung L, Baker KS, Pulsipher MA, Ness KK (2012) NCI, NHLBI/PBMTC first international conference on late effects after pediatric hematopoietic cell transplantation: health-related quality of life, functional, and neurocognitive outcomes. Biol Blood Marrow Transplant 18(2):162–171. doi:10.1016/j.bbmt.2011.12.501

    PubMed  Google Scholar 

  • Pasquini M, Wang Z (2012) Current use and outcome of hematopoietic stem cell transplantation: CIBMTR summary slides. Available at: http://www.cibmtr.org

  • Pession A, Rizzari C, Putti MC (2009) Results of AIEOP AML 2002/01 study for treatment of children with acute myeloid leukemia. Blood 114:Abstract 117

    Google Scholar 

  • Petersdorf EW, Hansen JA, Martin PJ, Woolfrey A, Malkki M, Gooley T, Storer B, Mickelson E, Smith A, Anasetti C (2001) Major-histocompatibility-complex class I alleles and antigens in hematopoietic-cell transplantation. N Engl J Med 345(25):1794–1800. doi:10.1056/NEJMoa011826, 345/25/1794 [pii]

    PubMed  CAS  Google Scholar 

  • Pulsipher MA, Boucher KM, Wall D, Frangoul H, Duval M, Goyal RK, Shaw PJ, Haight AE, Grimley M, Grupp SA, Kletzel M, Kadota R (2009) Reduced-intensity allogeneic transplantation in pediatric patients ineligible for myeloablative therapy: results of the pediatric blood and marrow transplant consortium study ONC0313. Blood 114(7):1429–1436. doi:10.1182/blood-2009-01-196303

    PubMed  CAS  Google Scholar 

  • Pulsipher MA, Skinner R, McDonald GB, Hingorani S, Armenian SH, Cooke KR, Gracia C, Petryk A, Bhatia S,Bunin N, Nieder ML, Dvorak CC, Sung L, Sanders JE, Kurtzberg J, Baker KS (2012) National Cancer Institute, National Heart, Lung and Blood Institute/Pediatric Blood and Marrow Transplantation Consortium First International Consensus Conference on late effects after pediatric hematopoietic cell transplantation: the need for pediatric-specific long-term follow-up guidelines. Biol Blood Marrow Transplant 18(3):334–347. doi:10.1016/j.bbmt.2012.01.003

    PubMed  Google Scholar 

  • Puumala SE, Ross JA, Aplenc R, Spector LG (2013) Epidemiology of childhood acute myeloid leukemia. Pediatr Blood Cancer 60(5):728–733. doi:10.1002/pbc.24464

    PubMed  Google Scholar 

  • Ratanatharathorn V, Nash RA, Przepiorka D, Devine SM, Klein JL, Weisdorf D, Fay JW, Nademanee A, Antin JH, Christiansen NP, van der Jagt R, Herzig RH, Litzow MR, Wolff SN, Longo WL, Petersen FB, Karanes C, Avalos B, Storb R, Buell DN, Maher RM, Fitzsimmons WE, Wingard JR (1998) Phase III study comparing methotrexate and tacrolimus (prograf, FK506) with methotrexate and cyclosporine for graft-versus-host disease prophylaxis after HLA-identical sibling bone marrow transplantation. Blood 92(7):2303–2314

    PubMed  CAS  Google Scholar 

  • Ravandi F, Cortes JE, Jones D, Faderl S, Garcia-Manero G, Konopleva MY, O’Brien S, Estrov Z, Borthakur G, Thomas D, Pierce SR, Brandt M, Byrd A, Bekele BN, Pratz K, Luthra R, Levis M, Andreeff M, Kantarjian HM (2010) Phase I/II study of combination therapy with sorafenib, idarubicin, and cytarabine in younger patients with acute myeloid leukemia. J Clin Oncol 28(11):1856–1862. doi:10.1200/JCO.2009.25.4888, JCO.2009.25.4888 [pii]

    PubMed  CAS  Google Scholar 

  • Ravindranath Y, Yeager AM, Chang MN, Steuber CP, Krischer J, Graham-Pole J, Carroll A, Inoue S, Camitta B, Weinstein HJ (1996) Autologous bone marrow transplantation versus intensive consolidation chemotherapy for acute myeloid leukemia in childhood. Pediatric Oncology Group. N Engl J Med 334(22):1428–1434. doi:10.1056/NEJM199605303342203

    PubMed  CAS  Google Scholar 

  • Redaelli A, Stephens JM, Brandt S, Botteman MF, Pashos CL (2004) Short- and long-term effects of acute myeloid leukemia on patient health-related quality of life. Cancer Treat Rev 30(1):103–117. doi:10.1016/S0305-7372(03)00142-7

    PubMed  Google Scholar 

  • Reisner Y, Hagin D, Martelli MF (2011) Haploidentical hematopoietic transplantation: current status and future perspectives. Blood 118(23):6006–6017. doi:10.1182/blood-2011-07-338822, blood-2011-07-338822 [pii]

    PubMed  CAS  Google Scholar 

  • Ringden O, Horowitz MM, Sondel P, Gale RP, Biggs JC, Champlin RE, Deeg HJ, Dicke K, Masaoka T, Powles RL et al (1993) Methotrexate, cyclosporine, or both to prevent graft-versus-host disease after HLA-identical sibling bone marrow transplants for early leukemia? Blood 81(4):1094–1101

    PubMed  CAS  Google Scholar 

  • Rocha V, Wagner JE Jr, Sobocinski KA, Klein JP, Zhang MJ, Horowitz MM, Gluckman E (2000) Graft-versus-host disease in children who have received a cord-blood or bone marrow transplant from an HLA-identical sibling. Eurocord and International Bone Marrow Transplant Registry Working Committee on Alternative Donor and Stem Cell Sources. N Engl J Med 342(25):1846–1854. doi:10.1056/NEJM200006223422501

    PubMed  CAS  Google Scholar 

  • Rocha V, Cornish J, Sievers EL, Filipovich A, Locatelli F, Peters C, Remberger M, Michel G, Arcese W, Dallorso S, Tiedemann K, Busca A, Chan KW, Kato S, Ortega J, Vowels M, Zander A, Souillet G, Oakill A, Woolfrey A, Pay AL, Green A, Garnier F, Ionescu I, Wernet P, Sirchia G, Rubinstein P, Chevret S, Gluckman E (2001) Comparison of outcomes of unrelated bone marrow and umbilical cord blood transplants in children with acute leukemia. Blood 97(10):2962–2971

    PubMed  CAS  Google Scholar 

  • Rosenberg PS, Alter BP, Bolyard AA, Bonilla MA, Boxer LA, Cham B, Fier C, Freedman M, Kannourakis G, Kinsey S, Schwinzer B, Zeidler C, Welte K, Dale DC (2006) The incidence of leukemia and mortality from sepsis in patients with severe congenital neutropenia receiving long-term G-CSF therapy. Blood 107(12):4628–4635. doi:10.1182/blood-2005-11-4370, 2005-11-4370 [pii]

    PubMed  CAS  Google Scholar 

  • Rosenberg PS, Alter BP, Ebell W (2008) Cancer risks in Fanconi anemia: findings from the German Fanconi Anemia Registry. Haematologica 93(4):511–517. doi:10.3324/haematol.12234, haematol.12234 [pii]

    PubMed  Google Scholar 

  • Ross JA (2000) Dietary flavonoids and the MLL gene: a pathway to infant leukemia? Proc Natl Acad Sci U S A 97(9):4411–4413, 97/9/4411 [pii]

    PubMed  CAS  Google Scholar 

  • Rubinstein P, Carrier C, Scaradavou A, Kurtzberg J, Adamson J, Migliaccio AR, Berkowitz RL, Cabbad M, Dobrila NL, Taylor PE, Rosenfield RE, Stevens CE (1998) Outcomes among 562 recipients of placental-blood transplants from unrelated donors. N Engl J Med 339(22):1565–1577. doi:10.1056/NEJM199811263392201

    PubMed  CAS  Google Scholar 

  • Rubnitz JE, Inaba H, Dahl G, Ribeiro RC, Bowman WP, Taub J, Pounds S, Razzouk BI, Lacayo NJ, Cao X, Meshinchi S, Degar B, Airewele G, Raimondi SC, Onciu M, Coustan-Smith E, Downing JR, Leung W, Pui CH, Campana D (2010) Minimal residual disease-directed therapy for childhood acute myeloid leukaemia: results of the AML02 multicentre trial. Lancet Oncol 11(6):543–552. doi:10.1016/S1470-2045(10)70090-5, S1470-2045(10)70090-5 [pii]

    PubMed  CAS  Google Scholar 

  • Russell JA, Tran HT, Quinlan D, Chaudhry A, Duggan P, Brown C, Stewart D, Ruether JD, Morris D, Glick S, Gyonyor E, Andersson BS (2002) Once-daily intravenous busulfan given with fludarabine as conditioning for allogeneic stem cell transplantation: study of pharmacokinetics and early clinical outcomes. Biol Blood Marrow Transplant 8(9):468–476

    PubMed  CAS  Google Scholar 

  • Sander A, Zimmermann M, Dworzak M, Fleischhack G, von Neuhoff C, Reinhardt D, Kaspers GJ, Creutzig U (2010) Consequent and intensified relapse therapy improved survival in pediatric AML: results of relapse treatment in 379 patients of three consecutive AML-BFM trials. Leukemia 24(8):1422–1428. doi:10.1038/leu.2010.127

    PubMed  CAS  Google Scholar 

  • Sanders JE, Thomas ED, Buckner CD, Flournoy N, Stewart PS, Clift RA, Lum L, Bensinger WI, Storb R, Appelbaum FR et al (1985) Marrow transplantation for children in first remission of acute nonlymphoblastic leukemia: an update. Blood 66(2):460–462

    PubMed  CAS  Google Scholar 

  • Santos GW, Tutschka PJ, Brookmeyer R, Saral R, Beschorner WE, Bias WB, Braine HG, Burns WH, Elfenbein GJ, Kaizer H et al (1983) Marrow transplantation for acute nonlymphocytic leukemia after treatment with busulfan and cyclophosphamide. N Engl J Med 309(22):1347–1353. doi:10.1056/NEJM198312013092202

    PubMed  CAS  Google Scholar 

  • Schmiegelow K, Al-Modhwahi I, Andersen MK, Behrendtz M, Forestier E, Hasle H, Heyman M, Kristinsson J, Nersting J, Nygaard R, Svendsen AL, Vettenranta K, Weinshilboum R (2009) Methotrexate/6-mercaptopurine maintenance therapy influences the risk of a second malignant neoplasm after childhood acute lymphoblastic leukemia: results from the NOPHO ALL-92 study. Blood 113(24):6077–6084. doi:10.1182/blood-2008-11-187880, blood-2008-11-187880 [pii]

    PubMed  CAS  Google Scholar 

  • Severson RK, Buckley JD, Woods WG, Benjamin D, Robison LL (1993) Cigarette smoking and alcohol consumption by parents of children with acute myeloid leukemia: an analysis within morphological subgroups – a report from the Childrens Cancer Group. Cancer Epidemiol Biomarkers Prev 2(5):433–439

    PubMed  CAS  Google Scholar 

  • Shaw PJ, Kan F, Woo Ahn K, Spellman SR, Aljurf M, Ayas M, Burke M, Cairo MS, Chen AR, Davies SM, Frangoul H, Gajewski J, Gale RP, Godder K, Hale GA, Heemskerk MB, Horan J, Kamani N, Kasow KA, Chan KW, Lee SJ, Leung WH, Lewis VA, Miklos D, Oudshoorn M, Petersdorf EW, Ringden O, Sanders J, Schultz KR, Seber A, Setterholm M, Wall DA, Yu L, Pulsipher MA (2010) Outcomes of pediatric bone marrow transplantation for leukemia and myelodysplasia using matched sibling, mismatched related, or matched unrelated donors. Blood 116(19):4007–4015. doi:10.1182/blood-2010-01-261958, blood-2010-01-261958 [pii]

    PubMed  CAS  Google Scholar 

  • Shinawi M, Erez A, Shardy DL, Lee B, Naeem R, Weissenberger G, Chinault AC, Cheung SW, Plon SE (2008) Syndromic thrombocytopenia and predisposition to acute myelogenous leukemia caused by constitutional microdeletions on chromosome 21q. Blood 112(4):1042–1047. doi:10.1182/blood-2008-01-135970, blood-2008-01-135970 [pii]

    PubMed  CAS  Google Scholar 

  • Shu XO, Reaman GH, Lampkin B, Sather HN, Pendergrass TW, Robison LL (1994) Association of paternal diagnostic X-ray exposure with risk of infant leukemia. Investigators of the Childrens Cancer Group. Cancer Epidemiol Biomarkers Prev 3(8):645–653

    PubMed  CAS  Google Scholar 

  • Smith OP (2002) Shwachman-Diamond syndrome. Semin Hematol 39(2):95–102. doi:S0037196302500196 [pii]

    PubMed  CAS  Google Scholar 

  • Sorrell AD, Alonzo TA, Hilden JM, Gerbing RB, Loew TW, Hathaway L, Barnard D, Taub JW, Ravindranath Y, Smith FO, Arceci RJ, Woods WG, Gamis AS (2012) 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 118(19):4806–4814. doi:10.1002/cncr.27484

    PubMed  CAS  Google Scholar 

  • Spiekermann K, Dirschinger RJ, Schwab R, Bagrintseva K, Faber F, Buske C, Schnittger S, Kelly LM, Gilliland DG, Hiddemann W (2003) The protein tyrosine kinase inhibitor SU5614 inhibits FLT3 and induces growth arrest and apoptosis in AML-derived cell lines expressing a constitutively activated FLT3. Blood 101(4):1494–1504

    PubMed  CAS  Google Scholar 

  • Stahnke K, Boos J, Bender-Gotze C, Ritter J, Zimmermann M, Creutzig U (1998) Duration of first remission predicts remission rates and long-term survival in children with relapsed acute myelogenous leukemia. Leukemia 12(10):1534–1538

    PubMed  CAS  Google Scholar 

  • Stevens RF, Hann IM, Wheatley K, Gray RG (1998) Marked improvements in outcome with chemotherapy alone in paediatric acute myeloid leukemia: results of the United Kingdom Medical Research Council’s 10th AML trial. MRC Childhood Leukaemia Working Party. Br J Haematol 101(1):130–140

    PubMed  CAS  Google Scholar 

  • Storb R, Deeg HJ, Pepe M, Appelbaum F, Anasetti C, Beatty P, Bensinger W, Berenson R, Buckner CD, Clift R et al (1989) Methotrexate and cyclosporine versus cyclosporine alone for prophylaxis of graft-versus-host disease in patients given HLA-identical marrow grafts for leukemia: long-term follow-up of a controlled trial. Blood 73(6):1729–1734

    PubMed  CAS  Google Scholar 

  • Swerdlow SH, International Agency for Research on Cancer, World Health Organization (2008) WHO classification of tumours of haematopoietic and lymphoid tissues. World Health Organization classification of tumours, 4th edn. International Agency for Research on Cancer, Lyon

    Google Scholar 

  • The Cancer Genome Atlas Research Network (2013) Genomic and epigenomic landscapes of adult de novo acute myeloid leukemia. N Engl J Med 368(22):2059–2074. doi:10.1056/NEJMoa1301689

    Google Scholar 

  • Thomas ED, Buckner CD, Banaji M, Clift RA, Fefer A, Flournoy N, Goodell BW, Hickman RO, Lerner KG, Neiman PE, Sale GE, Sanders JE, Singer J, Stevens M, Storb R, Weiden PL (1977) One hundred patients with acute leukemia treated by chemotherapy, total body irradiation, and allogeneic marrow transplantation. Blood 49(4):511–533

    PubMed  CAS  Google Scholar 

  • Tsukimoto I, Tawa A, Horibe K, Tabuchi K, Kigasawa H, Tsuchida M, Yabe H, Nakayama H, Kudo K, Kobayashi R, Hamamoto K, Imaizumi M, Morimoto A, Tsuchiya S, Hanada R (2009) Risk-stratified therapy and the intensive use of cytarabine improves the outcome in childhood acute myeloid leukemia: the AML99 trial from the Japanese Childhood AML Cooperative Study Group. J Clin Oncol 27(24):4007–4013. doi:10.1200/JCO.2008.18.7948, JCO.2008.18.7948 [pii]

    PubMed  CAS  Google Scholar 

  • Vardiman JW, Thiele J, Arber DA, Brunning RD, Borowitz MJ, Porwit A, Harris NL, Le Beau MM, Hellstrom-Lindberg E, Tefferi A, Bloomfield CD (2009) The 2008 revision of the World Health Organization (WHO) classification of myeloid neoplasms and acute leukemia: rationale and important changes. Blood 114(5):937–951. doi:10.1182/blood-2009-03-209262, blood-2009-03-209262 [pii]

    PubMed  CAS  Google Scholar 

  • Vlachos A, Ball S, Dahl N, Alter BP, Sheth S, Ramenghi U, Meerpohl J, Karlsson S, Liu JM, Leblanc T, Paley C, Kang EM, Leder EJ, Atsidaftos E, Shimamura A, Bessler M, Glader B, Lipton JM (2008) Diagnosing and treating Diamond Blackfan anaemia: results of an international clinical consensus conference. Br J Haematol 142(6):859–876. doi:10.1111/j.1365-2141.2008.07269.x, BJH7269 [pii]

    PubMed  CAS  Google Scholar 

  • Wagner JE, Thompson JS, Carter SL, Kernan NA, Unrelated Donor Marrow Transplantation Trial (2005) Effect of graft-versus-host disease prophylaxis on 3-year disease-free survival in recipients of unrelated donor bone marrow (T-cell Depletion Trial): a multi-centre, randomised phase II-III trial. Lancet 366(9487):733–741. doi:10.1016/S0140-6736(05)66996-6

    PubMed  CAS  Google Scholar 

  • Wagner J, Eapen M, Carter S, Haut P, Peres E, Schultz K, Thompson J, Wall D, Kurtzberg J (2012) No survival advantage after double umbilical cord blood (UCB) compared to single UCB transplant in children with hematological malignancy: results of the blood and marrow transplant clinical trials network (BMT CTN 0501) randomized trial. ASH Ann Meet. Abstr 120(21):359

    Google Scholar 

  • Webb DK, Wheatley K, Harrison G, Stevens RF, Hann IM (1999) Outcome for children with relapsed acute myeloid leukaemia following initial therapy in the Medical Research Council (MRC) AML 10 trial. MRC Childhood Leukaemia Working Party. Leukemia 13(1):25–31

    PubMed  CAS  Google Scholar 

  • Weisberg E, Boulton C, Kelly LM, Manley P, Fabbro D, Meyer T, Gilliland DG, Griffin JD (2002) Inhibition of mutant FLT3 receptors in leukemia cells by the small molecule tyrosine kinase inhibitor PKC412. Cancer Cell 1(5):433–443

    PubMed  CAS  Google Scholar 

  • Wheatley K, Burnett AK, Goldstone AH, Gray RG, Hann IM, Harrison CJ, Rees JK, Stevens RF, Walker H (1999) A simple, robust, validated and highly predictive index for the determination of risk-directed therapy in acute myeloid leukaemia derived from the MRC AML 10 trial. United Kingdom Medical Research Council’s Adult and Childhood Leukaemia Working Parties. Br J Haematol 107(1):69–79

    PubMed  CAS  Google Scholar 

  • Woods WG, Neudorf S, Gold S, Sanders J, Buckley JD, Barnard DR, Dusenbery K, DeSwarte J, Arthur DC, Lange BJ, Kobrinsky NL, Children’s Cancer Group (2001) A comparison of allogeneic bone marrow transplantation, autologous bone marrow transplantation, and aggressive chemotherapy in children with acute myeloid leukemia in remission. Blood 97(1):56–62

    PubMed  CAS  Google Scholar 

  • Yakoub-Agha I, Mesnil F, Kuentz M, Boiron JM, Ifrah N, Milpied N, Chehata S, Esperou H, Vernant JP, Michallet M, Buzyn A, Gratecos N, Cahn JY, Bourhis JH, Chir Z, Raffoux C, Socie G, Golmard JL, Jouet JP (2006) Allogeneic marrow stem-cell transplantation from human leukocyte antigen-identical siblings versus human leukocyte antigen-allelic-matched unrelated donors (10/10) in patients with standard-risk hematologic malignancy: a prospective study from the French Society of Bone Marrow Transplantation and Cell Therapy. J Clin Oncol 24(36):5695–5702. doi:10.1200/JCO.2006.08.0952, JCO.2006.08.0952 [pii]

    PubMed  Google Scholar 

  • Yamamoto Y, Kiyoi H, Nakano Y, Suzuki R, Kodera Y, Miyawaki S, Asou N, Kuriyama K, Yagasaki F, Shimazaki C, Akiyama H, Saito K, Nishimura M, Motoji T, Shinagawa K, Takeshita A, Saito H, Ueda R, Ohno R, Naoe T (2001) Activating mutation of D835 within the activation loop of FLT3 in human hematologic malignancies. Blood 97(8):2434–2439

    PubMed  CAS  Google Scholar 

  • Yeager AM, Wagner JE Jr, Graham ML, Jones RJ, Santos GW, Grochow LB (1992) Optimization of busulfan dosage in children undergoing bone marrow transplantation: a pharmacokinetic study of dose escalation. Blood 80(9):2425–2428

    PubMed  CAS  Google Scholar 

Download references

Author information

Authors and Affiliations

  1. Aflac Cancer and Blood Disorders Center, Emory University, Children’s Healthcare of Atlanta, Atlanta, GA, USA

    John Horan

  2. Department of Pediatrics, Aarhus University Hospital, Skejby, Denmark

    Henrik Hasle

  3. Clinical Research Division, Fred Hutchinson Cancer Research Center, Department of Pediatrics, University of Washington School of Medicine, Seattle, WA, USA

    Soheil Meshinchi

Authors
  1. John Horan
    View author publications

    You can also search for this author in PubMed Google Scholar

  2. Henrik Hasle
    View author publications

    You can also search for this author in PubMed Google Scholar

  3. Soheil Meshinchi
    View author publications

    You can also search for this author in PubMed Google Scholar

Corresponding author

Correspondence to John Horan .

Editor information

Editors and Affiliations

  1. University of Cincinnati, Cincinnati, Ohio, USA

    Franklin O. Smith

  2. George Washington University, Washington, Washington, USA

    Gregory H. Reaman

  3. Division of Hematology/Oncology, Dept. of Internal Medicine, University of Cincinnati College of Medicine, Madeira, Ohio, USA

    Judy M. Racadio

Rights and permissions

Reprints and permissions

Copyright information

© 2014 Springer-Verlag Berlin Heidelberg

About this chapter

Cite this chapter

Horan, J., Hasle, H., Meshinchi, S. (2014). Acute Myeloid Leukemia. In: Smith, F., Reaman, G., Racadio, J. (eds) Hematopoietic Cell Transplantation in Children with Cancer. Pediatric Oncology. Springer, Berlin, Heidelberg. https://doi.org/10.1007/978-3-642-39920-6_9

Download citation

  • DOI: https://doi.org/10.1007/978-3-642-39920-6_9

  • Published:

  • Publisher Name: Springer, Berlin, Heidelberg

  • Print ISBN: 978-3-642-39919-0

  • Online ISBN: 978-3-642-39920-6

  • eBook Packages: MedicineMedicine (R0)

Publish with us

Policies and ethics

Search

Navigation