Prognostic factors in acute promyelocytic leukemia: strategies to define high-risk patients
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  • 作者:Ugo Testa ; Francesco Lo-Coco
  • 关键词:Acute myeloid leukemia ; Acute promyelocytic leukemia ; Prognosis ; Membrane markers
  • 刊名:Annals of Hematology
  • 出版年:2016
  • 出版时间:April 2016
  • 年:2016
  • 卷:95
  • 期:5
  • 页码:673-680
  • 全文大小:321 KB
  • 参考文献:1.Lo-Coco F, Hasan SK (2014) Understanding the molecular pathogenesis of acute promyelocytic leukemia. Best Pract Res Clin Haematol 27:3–9CrossRef PubMed
    2.Ablain J, De Thé H (2014) Retinoic acid signaling in cancer: the parable of acute promyelocytic leukemia. Int J Cancer 135:2262–2272CrossRef PubMed
    3.Vickers M, Jackson G, Taylor P (2000) The incidence of acute promyelocytic leukemia appears constant over most of a human lifespan, implying only one rate limiting mutation. Leukemia 17:722–726CrossRef
    4.Welch JS, Ley TJ, Link DC, Miller CA, Larson DE, Koboldt DC et al (2012) The origin and evolution of mutations in acute myeloid leukemia. Cell 150:264–278CrossRef PubMed PubMedCentral
    5.Cancer Gemome Atlas Research Network (2013) Genomic and epigenomic landscapes of adult de novo acute myeloid leukemia. N Engl J Med 368:2059–2074CrossRef
    6.Huang ME, Ye YC, Chen SR, Chai JR, Lu JX, Zhoa L et al (1988) Use of all-trans retinoic acid in the treatment of acute promyelocytic leukemia. Blood 72:567–572PubMed
    7.Mi JQ, Chen SJ, Zhou GB, Yan XJ, Chen Z (2015) Synergistic targeted therapy for acute promyelocyticleukaemia: a model of translational research in human cancer. J Int Med; in press
    8.Lo-Coco F, Avvisati G, Vignetti M, Thiede C, Orlando SM, Iacobelli S et al (2013) Retinoic acid and arsenic trioxide for acute promyelocytic leukemia. N Engl J Med 369:111–121CrossRef PubMed
    9.Lo-Coco F, Cicconi L (2014) What is the standard regimen for patients with acute promyelocytic leukemia? Curr Hematol Malig Rep 9:138–143CrossRef PubMed
    10.Muroi K, Fujiwara S, Tatara R, Sugimoto M, Yamamoto C, Uehara E et al (2013) CD56 expression in normal immature granulocytes after allogeneic hematopoietic stem cell transplantation. J Clin Exp Hematol 53:247–250CrossRef
    11.Montesinos P, Rayon C, Vellenga E, Brunet S, Gonzalez T, Gonzalez M et al (2011) Clinical significance of CD56 expression in patients with acute promyelocytic leukemia treated with all-trans retinoic acid and anthracycline-based regimes. Blood 117:1799–1805CrossRef PubMed
    12.Ono T, Takeshita A, Kishimoto Y, Kiyoi H, Okada M, Yamauchi T et al (2014) Expression of CD56 is an unfavorable prognostic factor for acute promyelocytic leukemia with higher initial white blood cell counts. Cancer Sci 105:97–104CrossRef PubMed PubMedCentral
    13.Breccia M, De Propris MS, Minotti C, Stefanizzi C, Raponi S, Colofigli G et al (2014) Aberrant phenotypic expression of CD15 and CD56 identifies poor prognostic acute promyelocytic leukemia patients. Leuk Res 38:194–197CrossRef PubMed
    14.Lou Y, Ma Y, Suo S, Ni W, Wang Y, Pan H et al (2015) Prognostic significance of patients with newly diagnosed acute promyelocytic leukemia treated with arsenic trioxide-based frontline. Leuk Res 39:938–944CrossRef PubMed
    15.De J, Zanjani R, Hibbard M, Dabis BH (2007) Immunophenotypic profile predictive of KIT activating mutations in AML1-ETO leukemia. Am J Pathol 128:550–557
    16.Raspadori D, Damiani D, Lenoci M, Rondelli D, Testoni N, Nasrdi G et al (2001) CD56 antigenic expression in acute myeloid leukemia identifies patients with poor clinical prognosis. Leukemia 15:1161–1164CrossRef PubMed
    17.Jiao B, Wu CF, Liang Y, Chen HM, Xiong SM, Chen B et al (2009) AML1-ETO9 is correlated with C-KIT overexpression/mutations and indicates poor disease outcome in t(8;21) acute myeloid leukemia. Leukemia 23:1598–1604CrossRef PubMed
    18.Nishii K, Usui E, Katayama N, Lorenzo F, Nakase K, Kobayashi T et al (2003) Characteristics of t(8;21) acute myeloid leukemia (AML) with additional chromosomal abnormality: concomitant trisomy 4 may constitute a distinctive subtype of t(8;21) AML. Leukemia 17:731–737CrossRef PubMed
    19.Lin P, Hao S, Medeiros LJ, Estey EH, Pierce SA, Wang X et al (2004) Expression of CD2 in acute promyelocytic leukemia correlates with short form of PML-RARalpha transcripts and poorer prognosis. Am J Clin Pathol 121:402–407CrossRef PubMed
    20.Biondi A, Luciano A, Bassan R, Mininni D, Specchia G, Lanzi E et al (1995) CD2 expression in acute promyelocytic leukemia is associated with microgranular morphology (FAB M3v) but not with any PML gene breakpoint. Leukemia 9:1461–1466PubMed
    21.Albano F, Mestice A, Pannunzio A, Lanza F, Martino B, Pastore D et al (2006) The biological characteristics of CD34+CD2+ adult acute promyelocytic leukemia and the CD34−CD2− hyergranular and microgranular (M3v) phenotypes. Haematologica 91:311–316PubMed
    22.Xu F, Yin CX, Wang CL, Jiang XJ, Jiang L, Wang ZX et al (2014) Immunophenotypes and immune markers associated with acute promyelocytic leukemia prognosis. Dis Mark. art id 421906
    23.Foley R, Soamboonsrup P, Carter RF, Benger A, Meyer R, Walker I et al (2001) CD34-positive acute promyelocytic leukemia is associated with leukocytosis, microgranular/hypogranular morphology, expression of CD2 and bcr3 isoform. Am J Hematol 67:34–41CrossRef PubMed
    24.Guglielmi C, Martelli MP, Diverio D, Fenu S, Vegna ML, Cantu-Rajnoldi A et al (1998) Immunophenotype of adult and childhood acute promyelocytic leukemia: correlation with morphology, type of PML gene breakpoint and clinical outcome. A cooperative Italian study on 196 cases. Br J Haematol 102:1035–1041CrossRef PubMed
    25.Lee JJ, Cho D, Chung IJ, Cho SH, Park KS, Park MR et al (2003) CD34 expression is associated with poor clinical outcome in patients with acute promyelocytic leukemia. Am J Haematol 73:149–153CrossRef
    26.Paietta E, Golonbeva O, Neuberg D, Bennett JM, Gallagher R, Racevskis et al (2004) A surrogate marker profile of PML/RARalpha expressing acute promyelocytic leukemia and the association of immunophenotypic markers with morphologic and molecular subtypes. Cytometry Part B 59B:1–9CrossRef
    27.Breccia M, De Propris MS, Stefanizzi C, Raponi S, Modica M, Colafigli G et al (2014) Negative prognostic value of CD34 antigen also if expressed on a small population of acute promyelocytic leukemia cells. Ann Hematol 93:1819–1823CrossRef PubMed
    28.Chendamarai E, Ganesan S, Alex AA, Kamath V, Nair SC, Nellickai AJ et al (2015) Comparison of newly diagnosed and relapsed patients with acute promyelocytic leukemia treated with arsenic trioxide: insight into mechanisms of resistance. PLoS ONE 10, e0121912CrossRef PubMed PubMedCentral
    29.Tallman M, Lo-Coco F, Kwaan H, Sanz M, Gore S (2011) Clinical roundtable monograph. Early death in patients with acute promyelocytic leukemia. Clin Adv Hematol Oncol 9:1–16
    30.Park JH, Qiao B, Panageas KS, Schymura MJ, Jurcic JG, Rosenblat TL et al (2011) Early death rate in acute promyelocytic leukemia remains high despite all-trans retinoic acid. Blood 118:1248–1254CrossRef PubMed PubMedCentral
    31.Burnett AK, Grimwade D, Solomon E, Wheatley K, Goldstone AH (1999) Presenting white blood cell count and kinetics of molecular remission predict prognosis in acute promyelocytic leukemia treated with all-trans retinoic acid: result of the randomized MRC trial. Blood 93:4131–4143PubMed
    32.Sanz MA, Lo Coco F, Martin G, Avvisati G, Rayon C, Barbui T et al (2000) Definition of relapse risk and the role of non-anthracycline drugs for consolidation in patients with acute promyeloctic leukemia: a joint study of the PETHEMA and GIMEMA cooperative groups. Blood 96:1247–1253PubMed
    33.Fenaux P, Chastang C, Chevret S, Sanz M, Dombret H, Archimbaud E et al (1999) A randomized comparison of all transretinoic acid (ATRA) followed by chemotherapy and ATRA plus chemotherapy and the role of maintenance therapy in newly diagnosed acute promyelocytic leukemia. Eur APL Group Blood 94:1192–1200
    34.Ravandi F, Estey E, Jones D, Faderl S, O’Brien S, Fiorentino J et al (2009) Effective treatment of acute promyelocytic leukemia with all-trans-retinoic acid, arsenic trioxide, and gentuzumabozogamicin. J Clin Oncol 27:504–510CrossRef PubMed
    35.Mathews V, George B, Lakhmi KM, Viwabandya A, Bajel A, Balasubramanian P et al (2006) Single-agent arsenic trioxide in the treatment of newly diagnosed acute promyelocytic leukemia: durable remissions with minimal toxicity. Blood 107:2627–2632CrossRef PubMed
    36.Estey E, Garcia-Manero G, Ferrajoli A, Faderl S, Verstovek S, Jones D et al (2006) Use of all-trans retinoic acid plus arsenic trioxide ad an alternative to chemotherapy in untreated acute promyelocytic leukemia. Blood 107:3469–3473CrossRef PubMed
    37.Daver N, Kantarjian H, Marcucci G, Pierce S, Brandt M, Dinardo C et al (2015) Clinical characteristics and outcomes in patients with acute promyelocytic leukemia and hyperleukocytosis. Br J Haematol 168:646–653CrossRef PubMed PubMedCentral
    38.Montesinos P, Beagua J, Vellenga E, Rayen C, Parody R, de la Serna J et al (2009) Differentiation syndrome in patients with acute promyelocytic leukemia treated with all-trans retinoic acid and anthracycline chemotherapy: characteristics, outcome, and prognostic factors. Blood 113:775–783CrossRef PubMed
    39.Kiyoi H, Naoe T, Yokota N, Nakao M, Minami S, Kuriyama K et al (1997) Internal tandem duplication of FLT3 associated with leukocytosis in acute promyelocytic leukemia. Leukemia Study Group of the Ministry of Health and Welfare (KOHseisho). Leukemia 11:1447–1452CrossRef PubMed
    40.Moreno I, Martin G, Bolufer P, Barragan E, Rueda E, Roman J et al (2003) Incidence and prognostic value of FLT3 internal tandem duplication and D835 mutations in acute myeloid leukemia. Haematologica 88:19–24PubMed
    41.Kelly LM, Kutok JL, Williams IR, Boulton CL, Curley DP, Amaral SM et al (2002) PML/RARalpha and FLT3-ITD induce an APL-like disease in a mouse model. Proc Natl Acad Sci U S A 99:8283–8288CrossRef PubMed PubMedCentral
    42.Sohal J, Phan VT, Chan PV, Davis EM, Patel B, Kelly LM et al (2003) A model of APL with FLT3 mutation is responsive to retinoic acid and a receptor tyrosine kinase inhibitor, SU11657. Blood 101:3188–3197CrossRef PubMed
    43.Breccia M, Avvisati G, Latagliata R, Carmosino I, Guarini A, De Propris MS et al (2007) Occurrence of thrombotic events in acute promyelocytic leukemia correlate with consistent immunophenotypic and molecular features. Leukemia 21:79–83CrossRef PubMed
    44.Mitrovic M, Suvajdzic N, Elezovic I, Bogdanovic A, Djordjevic V, Miljic P et al (2015) Thrombotic events in acute promyelocytic leukemia. Thromb Res 135:588–593CrossRef PubMed
    45.Gale RE, Hills R, Pizzey A, Kottaridis PD, Swirsky D, Gilkes AF et al (2005) Relationship between FLT3 mutation status, biologic characteristics, and response to targeted therapy in acute promyelocytic leukemia. Blood 106:3768–3778CrossRef PubMed
    46.Souza Melo CP, Campos CB, Dutra AP, Neto JCA, Fenelon AJS, Neto AH et al (2015) Correlation between FLT3-ITD status and clinical, cellular and molecular profiles in promyelocytic acute leukemias. Leuk Res 39:131–137CrossRef PubMed
    47.Kutny MA, Moser BK, Laumann K, Feusner JH, Gamis A, Gregory J et al (2012) FLT3 mutation status is a predictor of early death in pediatric acute promyelocytic leukemia: a report from Children’s Oncology Group. Pediatr Blood Cancer 59:662–667CrossRef PubMed PubMedCentral
    48.Takenokuchi M, Kawano S, Nakamachi Y, Sakota Y, Syampurnawayi M, Saigo K et al (2012) FLT3/ITD associated with an immature immunophenotype in PML-RARα leukemia. Haematol Rep 4, e22
    49.Noguera NI, Breccia M, Divona M, Diverio D, Costa V, De Santis S et al (2002) Alterations of the FLT3 gene in acute promyelocyticleukemia: association with diagnostic characteristics and analysis of clinical outcome in patients treated with the Italian AIDA protocol. Leukemia 16:2185–2189CrossRef PubMed
    50.Molica M, Breccia M (2015) FLT3-ITD in acute promyelocytic leukemia: clinical distinct profile but still controversial prognosis. Leuk Res 39:397–399CrossRef PubMed
    51.Lucena-Araujo AR, Kim HT, Jacomo RH, Melo RA, Bittencourt R, Pasquini R et al (2014) Internal tandem duplication of the FLT3 gene confer poor overall survival in patients with acute promyelocytic leukemia treated with all-trans retinoic acid and anthracycline-based chemotherapy: an International Consortium on acute promyelocytic leukemia study. Ann Hematol 93:2011–2010CrossRef
    52.Chillon MC, Santamaria C, Garcia-Sanz R, Balanzategui A, Saraquete ME, Alcocebeda M et al (2010) Log FLT3 internal tandem duplications and reduced PML-RARalpha expression at diagnosis characterize a high-risk subgroup of acute promyelocytic leukemia patients. Hematologica 95:745–752CrossRef
    53.Iland HJ, Bradstock K, Supple SG, Catalano A, Collins M, Hertzberg M et al (2012) All-trans-retinoic acid, idarubicin, and IV arsenic trioxide as initial therapy in acute promyelocytic leukemia (APML4). Blood 120:1570–1580CrossRef PubMed
    54.Shen Y, Fu YK, Zhu YM, Lou YJ, Gu ZH, Shi JY et al (2015) Mutations of epigenetic modifier genes as a poor prognostic factor in acute promyelocytic leukemia under treatment with all-trans retinoic acid and arsenic trioxide. Electromagn Biol Med 2:563–571
    55.Gaur GC, Ramadan S, Cicconi L, Noguera NI, Luna I, Such E et al (2012) Analysis of mutational status, SNP rs16754, and expression levels of Wilms tumor 1 (WT1) gene in acute promyelocyticleukemia. Ann Hematol 91:1855–1860CrossRef PubMed
    56.Krauth MT, Alpermann T, Bacher U, Eder C, Dicker F, Ulke M et al (2015) WT1 mutations are secondary events in AML, show varying frequencies and impact on prognosis between genetic subgroups. Leukemia 29:660–667CrossRef PubMed
    57.Cervera J, Montesinos P, Hernandez-Rivas J, Calosanz MJ, Aventin A, Ferro MT et al (2010) Additional chromosomal abnormalities treated with all-trans retinoic acid and chemotherapy. Haematologica 95:424–431CrossRef PubMed PubMedCentral
    58.De Botton S, Chevret S, Canz M, Dombret H, Thomas X, Guerci A et al (2010) Additional chromosomal abnormalities in patients with acute promyelocytic leukemia (APL) do not confer poor prognosis: results of APL 93 trial. Br J Haematol 111:801–806
    59.Albano F, Zagaria A, Anelli L, Orsini P, Minervini CP, Impera L et al (2013) Lymphoid enhancer binding factor-1 (LEF1) expression as a prognostic factor in adult acute promyelocytic leukemia. Oncotarget 5:649–658CrossRef PubMedCentral
    60.Hecht A, Nowak D, Nowak V, Hanfstein B, Faldum A, Buchner T et al (2013) High expression of the Ets-related gene (ERG) is an independent prognostic marker for relapse-free survival. Ann Hematol 92:443–449CrossRef PubMed
    61.Lucena-Araujo AR, Kim H, Jacomo RH, Melo RA, Bittencourt R, Pasquini R et al (2014) Prognostic impact of KMT2E transcript level on outcome of patients with acute promyelocytic leukemia treated with all-trans retinoic acid and anthracycline-based chemotherapy: an International Consortium of Acute Promyelocytic Leukemia study. Br J Haematol 166:540–549CrossRef PubMed
    62.Lucena-Araujo AR, Kim H, Thomé C, Jacomo RH, Melo RA, Bittencourt R et al (2015) High ΔNp73/Tap73 ratio is associated with poor prognosis in acute promyelocytic leukemia. Blood 126:2302–2306CrossRef PubMed PubMedCentral
    63.Sunter NJ, Scott K, Hills R, Grimwade D, Taylor S, Worrilow LJ et al (2012) A functional variant in the core promoter of the CD95 cell death receptor gene predicts prognosis in acute promyelocytic leukemia. Blood 119:196–205CrossRef PubMed
    64.Breccia M, Lo-Coco F (2014) Thrombo-hemorrhagic deaths in acute promyelocytic leukemia. Thromb Res 133(S2):S112–S116CrossRef PubMed
    65.Lehmann S, Ravn A, Carlsson L, Antunovic P, Deneberg S, Mollgard S et al (2011) Continuing high early death rate in acute promyelocytic leukemia: a population-based report from the Swedish Adult Acute Leukemia Registry. Leukemia 25:1128–1134CrossRef PubMed
    66.McClellan JS, Kohrt HE, Coutre S, Gotlib JR, Majeti R, Alizadeh AA et al (2012) Treatment advances have not improved the early death rate in acute promyelocytic leukemia. Haematologica 97:133–136CrossRef PubMed PubMedCentral
    67.Altman JK, Rademaker A, Cull E, Weitner BB, Ofran Y, Rosenblat TL et al (2013) Administration of ATRA to newly diagnosed patients with acute promyelocytic leukemia is delayed contributing to early hemorrhagic death. Leuk Res 37:1004–1009CrossRef PubMed
    68.Paulson K, Serebrin A, Lambert P, Bergeron J, Everett J, Kew A et al (2014) Acute promyelocytic leukemia is characterized by stable incidence and improved survival that is restricted to patients managed in leukemia referral centres: a pan-Canadian epidemiological study. Br J Haematol 166:660–666CrossRef PubMed
    69.de la Serna J, Montesinos P, Vellenga E, Rayon C, Parody R, Leon A et al (2008) Causes and prognostic factors of remission induction failure in patients with acute promyelocytic leukemia treated with all-trans retinoic acid and idarubicin. Blood 111:3395–3402CrossRef PubMed
    70.Burnett A, Hunter A, Khwaja A, Bowen D, Grimwade D, Hills R et al (2015) APL of all risk groups is highly curable with a chemo-free combination of attenuated arsenic trioxide and ATRA. EHA Meeting, abstract LB 2067
  • 作者单位:Ugo Testa (1)
    Francesco Lo-Coco (2) (3)

    1. Department of Hematology, Oncology and Molecular Medicine, Istituto Superiore di Sanità, Rome, Italy
    2. Department of Biomedicine and Prevention, University Tor Vergata, Rome, Italy
    3. Laboratory of Neuro-Oncohematology, Santa Lucia Foundation, Rome, Italy
  • 刊物类别:Medicine
  • 刊物主题:Medicine & Public Health
    Hematology
    Oncology
  • 出版者:Springer Berlin / Heidelberg
  • ISSN:1432-0584
文摘
All trans retinoic acid (ATRA) has revolutionized the therapy of acute promyelocytic leukemia (APL). Treatment of this leukemia with ATRA in combination with chemotherapy has resulted in complete remission rates >90 % and long-term remission rates above 80 %. Furthermore, the combination of ATRA and arsenic trioxide (ATO) was shown to be safe and effective in frontline treatment and, for patients with low and intermediate risk disease, possibly superior to the standard ATRA and anthracycline-based regimen. However, in spite of this tremendous progress, APL still remains associated with a high incidence of early death due to the frequent occurrence of an abrupt bleeding diathesis. This hemorrhagic syndrome more frequently develops in high-risk APL patients, currently defined as those exhibiting >10 × 109/L WBC at presentation. In addition to high WBC count, other molecular and immunophenotypic features have been associated with high-risk APL. Among them, the expression in APL blasts of the stem/progenitor cell antigen CD34, the neural adhesion molecule (CD56), and the T cell antigen CD2 help to identify a subset of patients at higher risk of relapse and often the expression of these markers is associated with high WBC count. At the molecular level, the short PML/RARA isoform and FLT3-internal tandem duplication (ITD) mutations have been associated with increased relapse risk. These observations indicate that extended immunophenotypic and molecular characterization of APL at diagnosis including evaluation of CD2, CD56, and CD34 antigens and of FLT3 mutations may help to better design risk-adapted treatment in this disease.

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