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Chapter 63  Myelodysplastic Syndromes and Myeloproliferative Neoplasms in Children  1004.e5


            183.  Passmore  SJ,  Chessells  JM,  Kempski  H,  et al:  Paediatric  myelodys-  204.  Patja K, Pukkala E, Sund R, et al: Cancer incidence of persons with
                plastic syndromes and juvenile myelomonocytic leukaemia in the UK:   Down  syndrome  in  Finland:  a  population-based  study.  Int  J  Cancer
                a  population-based  study  of  incidence  and  survival.  Br  J  Haematol   118:1769, 2006.
                121:758, 2003.                                    205.  Lange B: The management of neoplastic disorders of haematopoiesis in
            184.  Bresolin  S,  Zecca  M,  Flotho  C,  et al:  Gene  expression-based  classi-  children with Down’s syndrome. Br J Haematol 110:512, 2000.
                fication  as  an  independent  predictor  of  clinical  outcome  in  juvenile   206.  Zipursky  A:  Transient  leukaemia—a  benign  form  of  leukaemia  in
                myelomonocytic leukemia. J Clin Oncol 28:1919, 2010.  newborn infants with trisomy 21. Br J Haematol 120:930, 2003.
            185.  Yoshida N, Yagasaki H, Xu Y, et al: Correlation of clinical features with   207.  Gamis AS, Alonzo TA, Gerbing RB, et al: Natural history of transient
                the mutational status of GM-CSF signaling pathway-related genes in   myeloproliferative  disorder  clinically  diagnosed  in  Down  syndrome
                juvenile myelomonocytic leukemia. Pediatr Res 65:334, 2009.  neonates: a report from the Children’s Oncology Group Study A2971.
            186.  Niemeyer CM, Arico M, Basso G, et al: Chronic myelogenous leukemia   Blood 118:6752, 2011.
                in childhood: a retrospective analysis of 110 cases. European Working   208.  Bhatnagar N, Nizery L, Tunstall O, et al: Transient abnormal myelo-
                Group on Myelodysplastic Syndromes in Childhood (EWOG-MDS).   poiesis and AML in Down syndrome: an update. Curr Hematol Malig
                Blood 89:3534, 1997.                                  Rep 11:333, 2016.
            187.  Chan HS, Estrov Z, Weitzman SS, et al: The value of intensive com-  209.  Yoshida K, Toki T, Okuno Y, et al: The landscape of somatic mutations
                bination  chemotherapy  for  juvenile  chronic  myelogenous  leukemia.    in  Down  syndrome-related  myeloid  disorders.  Nat  Genet  45:1293,
                J Clin Oncol 5:1960, 1987.                            2013.
            188.  Chan GC, Wang WC, Raimondi SC, et al: Myelodysplastic syndrome   210.  Liu B, Filippi S, Roy A, et al: Stem and progenitor cell dysfunction in
                in  children:  differentiation  from  acute  myeloid  leukemia  with  a  low   human trisomies. EMBO Rep 16:44, 2015.
                blast count. Leukemia 11:206, 1997.               211.  Tunstall-Pedoe O, Roy A, Karadimitris A, et al: Abnormalities in the
            189.  Hasle  H,  Kerndrup  G,  Yssing  M,  et al:  Intensive  chemotherapy  in   myeloid progenitor compartment in Down syndrome fetal liver precede
                childhood  myelodysplastic  syndrome.  A  comparison  with  results  in   acquisition of GATA1 mutations. Blood 112:4507, 2008.
                acute myeloid leukemia. Leukemia 10:1269, 1996.   212.  Roy A, Cowen G, Mead AJ, et al: Perturbation of fetal liver hemato-
            190.  Estrov  Z,  Dube  ID,  Chan  HS,  et al:  Residual  juvenile  chronic   poietic stem and progenitor cell development by trisomy 21. Proc Natl
                myelogenous leukemia cells detected in peripheral blood during clinical   Acad Sci USA 109:17579, 2012.
                remission. Blood 70:1466, 1987.                   213.  Wechsler  J,  Greene  M,  McDevitt  MA,  et al:  Acquired  mutations  in
            191.  Festa RS, Shende A, Lanzkowsky P: Juvenile chronic myelocytic leuke-  GATA1  in  the  megakaryoblastic  leukemia  of  Down  syndrome.  Nat
                mia: experience with intensive combination chemotherapy. Med Pediatr   Genet 32:148, 2002.
                Oncol 18:311, 1990.                               214.  Groet J, McElwaine S, Spinelli M, et al: Acquired mutations in GATA1
            192.  Lilleyman  JS,  Harrison  JF,  Black  JA:  Treatment  of  juvenile  chronic   in  neonates  with  Down’s  syndrome  with  transient  myeloid  disorder.
                myeloid  leukemia  with  sequential  subcutaneous  cytarabine  and  oral   Lancet 361:1617, 2003.
                mercaptopurine. Blood 49:559, 1977.               215.  Hitzler  JK,  Cheung  J,  Li  Y,  et al:  GATA1  mutations  in  transient
            193.  Wasserman LR: The management of polycythaemia vera. Br J Haematol   leukemia  and  acute  megakaryoblastic  leukemia  of  Down  syndrome.
                21:371, 1971.                                         Blood 101:4301, 2003.
            194.  Maybee  D,  Dubowy  R:  Toxicity  of  high-dose  alpha  inferferon  in   216.  Mundschau G, Gurbuxani S, Gamis AS, et al: Mutagenesis of GATA1
                children with Philadelphia chromosome-positive chronic myelogenous   is  an  initiating  event  in  Down  syndrome  leukemogenesis.  Blood
                leukemia: a Pediatric Oncology Group study. Proc Natl Acad Sci USA   101:4298, 2003.
                12:323, 1993.                                     217.  Xu  G,  Nagano  M,  Kanezaki  R,  et al:  Frequent  mutations  in  the
            195.  Stieglitz  E,  Ward  AF,  Gerbing  RB,  et al:  Phase  II/III  trial  of  a  pre-  GATA-1  gene  in  the  transient  myeloproliferative  disorder  of  Down
                transplant  farnesyl  transferase  inhibitor  in  juvenile  myelomonocytic   syndrome. Blood 102:2960, 2003.
                leukemia: a report from the Children’s Oncology Group. Pediatr Blood   218.  Calligaris R, Bottardi S, Cogoi S, et al: Alternative translation initiation
                Cancer 62:629, 2015.                                  site  usage  results  in  two  functionally  distinct  forms  of  the  GATA-1
            196.  Archambeault  S,  Flores  NJ,  Yoshimi  A,  et al:  Development  of  an   transcription factor. Proc Natl Acad Sci USA 92:11598, 1995.
                allele-specific minimal residual disease assay for patients with juvenile   219.  Kuhl C, Atzberger A, Iborra F, et al: GATA1-mediated megakaryocyte
                myelomonocytic leukemia. Blood 111:1124, 2008.        differentiation and growth control can be uncoupled and mapped to
            197.  Locatelli F, Nollke P, Zecca M, et al: Hematopoietic stem cell trans-  different domains in GATA1. Mol Cell Biol 25:8592, 2005.
                plantation (HSCT) in children with juvenile myelomonocytic leukemia   220.  Li Z, Godinho FJ, Klusmann JH, et al: Developmental stage-selective
                (JMML):  results  of  the  EWOG-MDS/EBMT  trial.  Blood  105:410,   effect of somatically mutated leukemogenic transcription factor GATA1.
                2005.                                                 Nat Genet 37:613, 2005.
            198.  Manabe A, Okamura J, Yumura-Yagi K, et al: Allogeneic hematopoietic   221.  Muntean AG, Crispino JD: Differential requirements for the activation
                stem cell transplantation for 27 children with juvenile myelomonocytic   domain  and  FOG-interaction  surface  of  GATA-1  in  megakaryocyte
                leukemia diagnosed based on the criteria of the International JMML   gene expression and development. Blood 106:1223, 2005.
                Working Group. Leukemia 16:645, 2002.             222.  Mateow MK, Barbaric D, Byatt S-A, et al: Down syndrome and leu-
            199.  Yoshimi A, Niemeyer CM, Bohmer V, et al: Chimaerism analyses and   kemia:  insights  into  leukemiogenesis  and  translationsl  targets.  Transl
                subsequent immunological intervention after stem cell transplantation   Pediatr 4:76, 2015.
                in  patients  with  juvenile  myelomonocytic  leukaemia.  Br  J  Haematol   223.  Walters DK, Mercher T, Gu TL, et al: Activating alleles of JAK3 in
                129:542, 2005.                                        acute megakaryoblastic leukemia. Cancer Cell 10:65, 2006.
            200.  Inagaki J, Fukano R, Nishikawa T, et al: Outcomes of immunologi-  224.  Kiyoi  H, Yamaji  S,  Kojima  S,  et al:  JAK3  mutations  occur  in  acute
                cal interventions for mixed chimerism following allogeneic stem  cell   megakaryoblastic leukemia both in Down syndrome children and non-
                transplantation  in  children  with  juvenile  myelomonocytic  leukemia.   Down syndrome adults. Leukemia 21:574, 2007.
                Pediatr Blood Cancer 60:116, 2013.                225.  De  Vita  S,  Mulligan  C,  McElwaine  S,  et al:  Loss-of-function  JAK3
            201.  Faraci M, Micalizzi C, Lanino E, et al: Three consecutive related bone   mutations  in TMD  and  AMKL  of  Down  syndrome.  Br  J  Haematol
                marrow  transplants  for  juvenile  myelomonocytic  leukaemia.  Pediatr   137:337, 2007.
                Transplant 9:797, 2005.                           226.  Karandikar  NJ,  Aquino  DB,  McKenna  RW,  et al:  Transient  myelo-
            202.  Smith  FO,  King  R,  Nelson  G,  et al:  Unrelated  donor  bone  marrow   proliferative  disorder  and  acute  myeloid  leukemia  in  Down  syn-
                transplantation for children with juvenile myelomonocytic leukaemia.   drome.  An  immunophenotypic  analysis.  Am  J  Clin  Pathol  116:204,
                Br J Haematol 116:716, 2002.                          2001.
            203.  Yoshimi A, Bader P, Matthes-Martin S, et al: Donor leukocyte infusion   227.  Tchernia G, Lejeune F, Boccara JF, et al: Erythroblastic and/or mega-
                after hematopoietic stem cell transplantation in patients with juvenile   karyoblastic  leukemia  in  Down  syndrome:  treatment  with  low-dose
                myelomonocytic leukemia. Leukemia 19:971, 2005.       arabinosyl cytosine. J Pediatr Hematol Oncol 8:59, 1996.
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