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Acute Promyelocytic Leukemia: A History over 60 Years—From the Most Malignant to the most Curable Form of Acute Leukemia

Acute Promyelocytic Leukemia: A History over 60 Years—From the Most Malignant to the most Curable... Acute promyelocytic leukemia (APL) is a distinct subtype of acute myeloid leukemia (AML) that is cytogenetically characterized by a balanced reciprocal translocation between chromosomes 15 and 17, which results in the fusion of the promyelocytic leukemia (PML) and retinoic acid receptor alpha (RARα) genes. Because patients with APL present a tendency for severe bleeding, often resulting in an early fatal course, APL was historically considered to be one of the most fatal forms of acute leukemia. However, therapeutic advances, including anthracycline- and cytarabine-based chemotherapy, have significantly improved the outcomes of APL patients. Due to the further introduction of all-trans retinoic acid (ATRA) and—more recently—the development of arsenic trioxide (ATO)-containing regimens, APL is currently the most curable form of AML in adults. Treatment with these new agents has introduced the concept of cure through targeted therapy. With the advent of revolutionary ATRA-ATO combination therapies, chemotherapy can now be safely omitted from the treatment of low-risk APL patients. In this article, we review the six-decade history of APL, from its initial characterization to the era of chemotherapy-free ATRA-ATO, a model of cancer-targeted therapy. http://www.deepdyve.com/assets/images/DeepDyve-Logo-lg.png Oncology and Therapy Springer Journals

Acute Promyelocytic Leukemia: A History over 60 Years—From the Most Malignant to the most Curable Form of Acute Leukemia

Thomas, Xavier
Oncology and Therapy , Volume 7 (1) – Feb 5, 2019
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33 pages

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Publisher
Springer Journals
Copyright
Copyright © 2019 by The Author(s)
Subject
Medicine & Public Health; Internal Medicine
ISSN
2366-1070
eISSN
2366-1089
DOI
10.1007/s40487-018-0091-5
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Abstract

Acute promyelocytic leukemia (APL) is a distinct subtype of acute myeloid leukemia (AML) that is cytogenetically characterized by a balanced reciprocal translocation between chromosomes 15 and 17, which results in the fusion of the promyelocytic leukemia (PML) and retinoic acid receptor alpha (RARα) genes. Because patients with APL present a tendency for severe bleeding, often resulting in an early fatal course, APL was historically considered to be one of the most fatal forms of acute leukemia. However, therapeutic advances, including anthracycline- and cytarabine-based chemotherapy, have significantly improved the outcomes of APL patients. Due to the further introduction of all-trans retinoic acid (ATRA) and—more recently—the development of arsenic trioxide (ATO)-containing regimens, APL is currently the most curable form of AML in adults. Treatment with these new agents has introduced the concept of cure through targeted therapy. With the advent of revolutionary ATRA-ATO combination therapies, chemotherapy can now be safely omitted from the treatment of low-risk APL patients. In this article, we review the six-decade history of APL, from its initial characterization to the era of chemotherapy-free ATRA-ATO, a model of cancer-targeted therapy.

Journal

Oncology and Therapy – Springer Journals

Published: Feb 5, 2019

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    Shen, Y; Shen, ZX; Yan, H
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    Kwong, YL; Au, WY; Chim, CS; Pang, A; Suen, C; Liang, R
  • Arsenic trioxide therapy for relapsed acute promyelocytic leukemia: a bridge to transplantation
    Leoni, F; Gianfaldoni, G; Annunziata, M
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    Ohnishi, K; Yoshida, H; Shigeno, K
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    Aribi, A; Kantarjian, HM; Estey, EH
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    Alimoghaddam, K; Ghavamzadeh, A; Jahani, M
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    Lengfelder, E; Hofmann, WK; Nowak, D
  • Arsenic trioxide and acute promyelocytic leukemia: clinical and biological
    Chen, Z; Zhao, WL; Shen, ZX
  • ATR, PML, and CHK2 play a role in arsenic trioxide-induced apoptosis
    Joe, Y; Jeong, JH; Yang, S
  • Leukocytosis and the retinoic acid syndrome in patients with acute promyelocytic leukemia treated with arsenic trioxide
    Camacho, LH; Soignet, SL; Chanel, S
  • Examen au microscope électronique de la tumeur VX2 du lapin domestique dérivée du papillome de Shope
    Thé, G; Rivière, M; Bernhard, W
  • Arsenic trioxide controls the fate of the PML-RARalpha oncoprotein by directly binding PML
    Zhang, XW; Yan, XJ; Zhou, ZR
  • Arsenic degrades PML or PML-RARalpha through a SUMO-triggered RNF4/ubiquitin-mediated pathway
    Lallemand-Breitenbach, V; Jeanne, M; Benhenda, S
  • RNF4 is a poly-SUMO-specific E3 ubiquitin ligase required for arsenic-induced PML degradation
    Tatham, MH; Geoffroy, MC; Shen, L
  • Activation of a promyelocytic leukemia-tumor protein 53 axis underlies acute promyelocytic leukemia cure
    Ablain, J; Rice, K; Soilihi, H
  • Phase II study of single-agent arsenic trioxide for the front-line therapy of acute promyelocytic leukemia
    Ghavamzadeh, A; Alimoghaddam, K; Rostami, S
  • Single-agent arsenic trioxide in the treatment of newly diagnosed acute promyelocytic leukemia: long-term follow-up data
    Mathews, V; George, B; Chendamarai, E
  • Therapy resistance in APL
    Lehmann-Che, J; Bailly, C; Thé, H
  • Synergy against PML-RARa: targeting transcription, proteolysis, differentiation, and self-renewal in acute promyelocytic leukemia
    DosSantos, GA; Kats, L; Pandolfi, PP
  • Elemental arsenic entered the cerebrospinal fluid during oral arsenic trioxide treatment of meningeal relapse of acute promyelocytic leukemia
    Au, WY; Tam, S; Fong, BM; Kwong, YL
  • Missense mutations in PML-RARA are critical for the lack of responsiveness to arsenic trioxide treatment
    Goto, E; Tomita, A; Hayakawa, F
  • Prevalence, management, and clinical consequences of QT interval prolongation during treatment with arsenic trioxide
    Roboz, GJ; Ritchie, EK; Carlin, RF
  • Speciation of arsenic trioxide penetrates into cerebrospinal fluid in patients with acute promyelocytic leukemia
    Kiguchi, T; Yoshino, Y; Yuan, B
  • Long-term efficacy and safety of all-trans retinoic acid/arsenic trioxide-based therapy in newly diagnosed acute promyelocytic leukemia
    Hu, J; Liu, YF; Wu, CF
  • Effective treatment of acute promyelocytic leukemia with all-trans retinoic acid, arsenic trioxide, and gemtuzumab ozogamicin
    Ravandi, F; Estey, E; Jones, D
  • Arsenic trioxide improves event-free and overall survival for adults with acute promyelocytic leukemia: North American Leukemia Intergroup Study C9710
    Powell, BL; Moser, B; Stock, W
  • Single cycle of arsenic trioxide-based consolidation chemotherapy spares anthracycline exposure in the primary management of acute promyelocytic leukemia
    Gore, SD; Gojo, I; Sekeres, MA
  • Arsenic trioxide is required in the treatment of newly diagnosed acute promyelocytic leukemia. Analysis of a randomized trial (APL 2006) by the French-Belgian-Swiss APL group
    Adès, L; Thomas, X; Guerci Bresler, A
  • Retinoic acid and arsenic synergize to eradicate leukemic cells in a mouse model of acute promyelocytic leukemia
    Lallemand-Breitenbach, V; Guillemin, MC; Janin, A
  • All-trans retinoic acid/AS2O3 combination yields a high quality remission and survival in newly diagnosed acute promyelocytic leukemia
    Shen, ZX; Shi, ZZ; Fang, J
  • Long-term outcome of acute promyelocytic leukemia treated with all-trans retinoic acid, arsenic trioxide, and gemtuzumab
    Abaza, Y; Kantarjian, H; Garcia-Manero, G
  • Improved outcomes with retinoic acid and arsenic trioxide compared with retinoic acid and chemotherapy in non-high-risk acute promyelocytic leukemia: final results of the randomized Italian-German APL0406 trial
    Platzbecker, U; Avvisati, G; Cicconi, L
  • Arsenic trioxide and mannitol for the treatment of acute promyelocytic leukemia relapse in the central nervous system
    Wang, H; Cao, FL; Li, JM
  • Acute promyelocytic leukaemia: novel insights into the mechanisms of cure
    Thé, H; Chen, Z
  • Impairment of p53 acetylation, stability and function by an oncogenic transcription factor
    Insinga, A; Monestiroli, S; Ronzoni, S
  • PML nuclear bodies
    Lallemand-Breitenbach, V; Thé, H
  • The histone demethylase PHF8 governs retinoic acid response in acute promyelocytic leukemia
    Arteaga, MF; Mikesch, JH; Qiu, J
  • PML-RARA requires DNA methyltransferase 3A to initiate acute promyelocytic leukemia
    Cole, CB; Verdoni, AM; Ketkar, S
  • TRIB3 promotes APL progression through stabilization of the oncoprotein PML-RARalpha and inhibition of p53-mediated senescence
    Li, K; Wang, F; Cao, WB
  • HDAC3 activity is required for initiation of leukemogenesis in acute promyelocytic leukemia
    Mehdipour, P; Santoro, F; Botrugno, OA
  • Disruption of the PHRF1 tumor suppressor network by PML-RARalpha drives acute promyelocytic leukemia pathogenesis
    Prunier, C; Zhang, MZ; Kumar, S
  • Pathways of retinoic acid- or arsenic trioxide-induced PML/RARalpha catabolism, role of oncogene degradation in disease remission
    Zhu, J; Lallemand-Breitenbach, V; Thé, H
  • Histiocytic clearance of neoplastic cells in acute promyelocytic leukemia
    Schafernak, KT; Corey, SJ
  • The PML domain of PML-RARalpha blocks senescence to promote leukemia
    Korf, K; Wodrich, H; Haschke, A
  • Pro-senescence therapy for cancer treatment
    Nardella, C; Clohessy, JG; Alimonti, A; Pandolfi, PP
  • Forced retinoic acid receptor a homodimer prime mice for APL-like leukemia
    Sterndorf, T; Phan, VT; Maunakea, ML
  • Oligomerization of RAR and AML1 transcription factors as a novel mechanism of oncogenic activation
    Minucci, S; Maccarana, M; Cioce, M
  • The self-association coiled-coil domain of PML is sufficient for the oncogenic conversion of the retinoic acid receptor (RAR) alpha
    Occhionorelli, M; Santoro, F; Pallavicini, I
  • Active Pin1 is a key target of all-trans retinoic acid in acute promyelocytic leukemia and breast cancer
    Wei, S; Kozono, S; Kats, L
  • Continuing high early death rate in acute promyelocytic leukemia: a population-based report from the Swedish Adult Acute Leukemia Registry
    Lehmann, S; Ravn, A; Carlsson, L
  • 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
    Paulson, K; Serebrin, A; Lambert, P
  • Early death in acute promyelocytic leukemia (APL) in French centers: a multicenter study in 399 patients
    Rahmé, R; Thomas, X; Recher, C
  • Management and treatment results in patients with acute promyelocytic leukaemia (APL) not enrolled in clinical trials
    Micol, JB; Raffoux, E; Boissel, N
  • The global problem of early deaths in acute promyelocytic leukemia: a strategy to decrease induction mortality in the most curable leukemia
    Jillela, AP; Kota, VK
  • Determinants of fatal bleeding during induction therapy for acute promyelocytic leukemia in the ATRA era
    Mantha, S; Goldman, DA; Devlin, SM
  • Administration of ATRA to newly diagnosed patients with acute promyelocytic leukemia is delayed contributing to early hemorrhagic death
    Altman, JK; Rademaker, A; Cull, E
  • Early hemorrhagic death before starting therapy in acute promyelocytic leukemia: association with high WBC count, late diagnosis and delayed treatment initiation
    Breccia, M; Latagliata, R; Cannella, L
  • Development of a risk grading system to identify patients with acute promyelocytic leukemia at high risk of early death
    Zhang, Y; Hou, W; Wang, P
  • Management of patients with acute promyelocytic leukemia
    Kayser, S; Schlenk, RF; Platzbecker, U
  • Synergistic targeted therapy for acute promyelocytic leukaemia: a model of translational research in human cancer
    Mi, JQ; Chen, SJ; Zhou, GB
  • Long-term prognosis of childhood acute promyelocytic leukaemia with arsenic trioxide administration in induction and consolidation chemotherapy phases: a single-center experience
    Cheng, Y; Zhang, L; Wu, J
  • Gemtuzumab ozogamicin (Mylotarg) as a single agent for molecularly relapsed acute promyelocytic leukemia
    Lo-Coco, F; Cimino, G; Breccia, M
  • A phase 1 trial of vadastuximab talirine as monotherapy in patients with CD33-positive acute myeloid leukemia
    Stein, EM; Walter, RB; Erba, HP
  • Molecular remission induction with retinoic acid and anti-CD33 monoclonal antibody HuM195 in acute promyelocytic leukemia
    Jurcic, JG; DeBlasio, T; Dumont, L; Yao, TJ; Scheinberg, DA
  • A model of APL with FLT3 mutation is responsive to retinoic acid and a receptor tyrosine kinase inhibitor, SU11657
    Sohal, J; Phan, VT; Chan, PV
  • Tamibarotene maintenance improved relapse-free survival of acute promyelocytic leukemia: a final result of prospective, randomized, JALSG-APL204 study
    Takeshita, A; Asou, N; Atsuta, Y
  • Systemic availability of arsenic from oral arsenic-trioxide used to treat patients with hematological malignancies
    Kumana, CR; Au, WY; Lee, NS
  • Effects of oral arsenic trioxide therapy on QT intervals in patients with acute promyelocytic leukemia: implications for long-term cardiac safety
    Siu, CW; Au, WY; Yung, C
  • Oral arsenic and all-trans retinoic acid for high-risk acute promyelocytic leukemia
    Zhu, HH; Liu, YR; Jia, JS
  • Oral arsenic plus retinoic acid versus intravenous arsenic plus retinoic acid for non-high-risk acute promyelocytic leukaemia: a non-inferiority, randomized phase 3 trial
    Zhu, HH; Wu, DP; Du, X
  • Oral arsenic trioxide in the treatment of relapsed acute promyelocytic leukemia
    Au, WY; Kumana, CR; Kou, M
  • Oral arsenic and retinoic acid for non-high-risk acute promyelocytic leukemia
    Zhu, HH; Huang, XJ
  • Oral arsenic trioxide-based maintenance regimens for first complete remission of acute promyelocytic leukemia: a 10-year follow-up study
    Au, WY; Kumana, CR; Lee, HK
  • Role of arsenic trioxide in acute promyelocytic leukemia
    Iland, HJ; Seymour, JF
  • Molecular remission as a therapeutic objective in acute promyelocytic leukemia
    Cicconi, L; Fenaux, P; Kantarjian, H; Tallman, M; Sanz, MA; Lo-Coco, F
  • Treating acute promyelocytic leukaemia in Latin America: lessons from the International Consortium on Acute Leukaemia experience
    Correa de Araujo Koury, L; Ganser, A; Berliner, N