Arsenic trioxide for management of acute promyelocytic leukemia: Current evidence on its role in front-line therapy and recurrent disease

Expert Opinion on Pharmacotherapy

Volumn 13, Issue 7, 2012, Pages 1031-1043

  Breccia, Massimo ^a   Lo Coco, Francesco ^b  

^a SAPIENZA UNIVERSITY OF ROME   (Italy)

^b UNIVERSITY OF ROME TOR VERGATA   (Italy)

Author keywords

Acute promyelocytic leukemia; APL relapses; Arsenic trioxide; ATRA; Chemotherapy

Indexed keywords

ANTINEOPLASTIC AGENT; ARSENATE SODIUM; ARSENIC ACID; ARSENIC DERIVATIVE; ARSENIC TRIOXIDE; ARSENITE SODIUM; ARSENOBETAINE; ARSENOCHOLINE; ARSINE DERIVATIVE; CACODYLIC ACID; CALCIUM; CYCLIC AMP DEPENDENT PROTEIN KINASE; CYTOTOXIC AGENT; DAUNORUBICIN; GEMTUZUMAB OZOGAMICIN; GRANULOCYTE COLONY STIMULATING FACTOR; IDARUBICIN; INTERFERON; MERCAPTOPURINE; METHANEARSONATE SODIUM; METHOTREXATE; MITOGEN ACTIVATED PROTEIN KINASE; MITOGEN ACTIVATED PROTEIN KINASE P38; RETINOIC ACID; RETINOID; STRESS ACTIVATED PROTEIN KINASE; TETRAMETHYLARSONIUM; TRIMETHYLARSINE OXIDE; TUMOR NECROSIS FACTOR; UNCLASSIFIED DRUG; UNINDEXED DRUG;

ALLOGENEIC STEM CELL TRANSPLANTATION; ANOREXIA; ANTINEOPLASTIC ACTIVITY; APOPTOSIS; ARTICLE; ATRIOVENTRICULAR BLOCK; BLOOD SAMPLING; BONE MARROW SUPPRESSION; BRAIN HEMORRHAGE; CANCER COMBINATION CHEMOTHERAPY; CANCER PATIENT; CANCER RADIOTHERAPY; CANCER RECURRENCE; CELL DIFFERENTIATION; CEREBROSPINAL FLUID; CHEMOTHERAPY INDUCED EMESIS; CHRONIC MYELOID LEUKEMIA; CLINICAL PROTOCOL; CLINICAL TRIAL (TOPIC); CONTINUOUS INFUSION; CYTOGENETICS; DIARRHEA; DISEASE FREE SURVIVAL; DIZZINESS; DRUG BLOOD LEVEL; DRUG EFFICACY; DRUG ELIMINATION; DRUG ERUPTION; DRUG EXCRETION; DRUG HALF LIFE; DRUG INDUCED HEADACHE; DRUG MECHANISM; DRUG SAFETY; DRUG SENSITIVITY; DRUG TOLERABILITY; DRUG URINE LEVEL; DRUG WITHDRAWAL; ECG ABNORMALITY; ERYTHEMA; ESOPHAGUS CANCER; EVENT FREE SURVIVAL; EVIDENCE BASED MEDICINE; FATIGUE; FEVER; GASTROINTESTINAL SYMPTOM; GENE EXPRESSION; GENE REPRESSION; HEART ARRHYTHMIA; HIGH PERFORMANCE LIQUID CHROMATOGRAPHY; HUMAN; HYPERGLYCEMIA; HYPERLEUKOCYTOSIS; HYPOKALEMIA; IN VITRO STUDY; IN VIVO STUDY; KIDNEY DYSFUNCTION; KIDNEY FAILURE; LEUKEMIA RELAPSE; LEUKEMIA REMISSION; LEUKOCYTOSIS; LIVER DYSFUNCTION; LIVER ENZYME BLOOD LEVEL; LOW DRUG DOSE; LUNG HEMORRHAGE; LYMPHOMA; MAXIMUM PLASMA CONCENTRATION; MULTIPLE CYCLE TREATMENT; MYALGIA; NAUSEA; NEUROPATHY; NEUTROPENIA; NONHUMAN; OVERALL SURVIVAL; PERIPHERAL NEUROPATHY; PRACTICE GUIDELINE; PROMYELOCYTIC LEUKEMIA; PROTEOMICS; PRURITUS; QT PROLONGATION; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; SEPSIS; SIDE EFFECT; SIGNAL TRANSDUCTION; SKIN ULCER; T WAVE; TIME TO MAXIMUM PLASMA CONCENTRATION; TREATMENT OUTCOME; TREATMENT RESPONSE; UNCERTAINTY;

ARSENICALS; CLINICAL TRIALS AS TOPIC; HUMANS; LEUKEMIA, PROMYELOCYTIC, ACUTE; OXIDES; RECURRENCE; TRETINOIN;

EID: 84860111877     PISSN: 14656566     EISSN: 17447666     Source Type: Journal    
DOI: 10.1517/14656566.2012.677436     Document Type: Article

References (84)

1. Grimwade D. The pathogenesis of acute promyelocytic leukemia: evaluation of the role of molecular diagnosis and monitoring in the management of the disease. Br J Haematol 1999;106:591-613 (Pubitemid 29434197)
2. Tallman MS, Nabhan C, Feusner JH, Rowe JM. Acute promyelocytic leukemia: evolving therapeutic strategies. Blood 2002;99:759-67 (Pubitemid 34525534)
3. Rowley JD, Golomb HM, Dougherty C. 15/17 translocation, a consistent chromosomal change in acute promyelocytic leukemia. Lancet 1977;1:549-50 (Pubitemid 8045927)
4. de The H, Chomienne C, Lanotte M, et al. The t(15;17) translocation of acute promyelocytic leukemia fuses the retinoic acid receptor alpha gene to a novel transcribed locus. Nature 1990;347:558-61
5. Mandelli F, Diverio D, Avvisati G, et al. Molecular remission in PML/RAR alpha-positive acute promyelocytic leukemia by combined all-trans retinoic acid and idarubicin (AIDA) therapy. Gruppo Italiano-Malattie Ematologiche Maligne dell'Adulto and Associazione Italiana di Ematologia ed Oncologia Pediatrica Cooperative groups. Blood 1997;90:1014-21
6. Tallman MS, Andersen JW, Schiffer CA, et al. All-trans-retinoic acid in acute promyelocytic leukemia. N Engl J Med 1997;337:1201-8
7. Fenaux P, Le Deley MC, Castaigne S, et al. Effect of all transretinoic acid in newly diagnosed acute promyelocytic leukemia. Results of a multicenter randomized trial. European APL 91 Group. Blood 1993;82:3241-9 (Pubitemid 23356690)
8. Sanz MA. Treatment of acute promyelocytic leukemia. Hematology 2006;147-55
9. Esteve J, Escoda L, Martin G, et al. Outcome of patients with acute promyelocytic leukemia failing to front-line treatment with all-trans retinoic acid and anthracycline-based chemotherapy (PETHEMA protocols LPA96 and LPA99): benefit of an early intervention. Leukemia 2007;21:446-52
10. Lo Coco F, Diverio D, Avvisati G, et al. Therapy off molecular relapse in acute promyelocytic leukemia. Blood 1999;94:2225-9 (Pubitemid 29467668)
11. Tallman MS. The expanding role of arsenic in acute promyelocytic leukemia. Semin Hematol 2008;45:S25-9
12. Emadi A, Gore SD. Arsenic trioxide-an old drug rediscovered. Blood Rev 2010;24:191-9
13. Sanz MA, Lo Coco F. Modern approaches to treating acute promyelocytic leukemia. J Clin Oncol 2011;29:495-503
14. Huff J, Chan P, Nyska A. Is the human carcinogen arsenic carcinogenic to laboratory animals? Toxicol Sci 2000;55:17-23 (Pubitemid 30252431)
15. Zhang TD, Chen GQ, Wang ZG, et al. Arsenic trioxide, a therapeutic agent for APL. Oncogene 2001;20:7146-53
16. Kwong YL, Todd D. Delicious poison: arsenic trioxide for the treatment of leukemia. Blood 1997;89:3487-8
17. Litzow MR. Arsenic trioxide. Expert Opin Pharmacother 2008;9:1773-85
18. Wang ZY, Chen Z. Acute promyelocytic leukemia: from highly fatal to highly curable. Blood 2008;111:2505-15
19. Chen GQ, Zhu J, Shi XG, et al. In vitro studies on cellular and molecular mechanisms of arsenic trioxide (As2O3) in the treatment of acute promyelocytic leukemia: as2O3 induces NB4 cell apoptosis with down-regulation of Bcl-2 expression and modulation of PML-RAR. Blood 1996;88:1052-61 (Pubitemid 26333331)
20. Fukai Y, Hirata M, Ueno M, et al. Clinical pharmacokinetic study of arsenic trioxide in an acute promyelocytic leukemia (APL) patient: speciation of arsenic metabolites in serum and urine. Biol Pharm Bull 2006;29:1022-7
21. Au WY, Tam S, Fong BM, Kwong YL. Determinants of cerebrospinal fluid arsenic concentration in patients with acute promyelocytic leukemia on oral arsenic trioxide therapy. Blood 2008;112:3587-90
22. Chen GQ, Shi XG, Tang W, et al. Use of arsenic trioxide (As203) in the treatment of acute promyelocytic leukemia (APL): clinical efficacy and pharmacokinetics in relapsed patients. Blood 1997;89:3354-60 (Pubitemid 27229822)
23. Soignet SL, Maslak P, Wang ZG, et al. Complete remission after treatment of acute promyelocytic leukemia with arsenic trioxide. N Engl J Med 1998;339:1341-8 (Pubitemid 28509343)
24. Lallemand-Breitenbach V, Guillemin MC, Janin A, et al. Retinoic acid and arsenic synergize to eradicate leukemic cells in a mouse model of acute promyelocytic leukemia. J Exp Med 1999;189:1043-52 (Pubitemid 29169821)
25. Kinjo K, Kikazi M, Muto A, et al. Arsenic trioxide (As2O3)-induced apoptosis and differentiation in retinoic acid-resistant acute promyelocytic leukemia model in hGM-CSF-producing transgenic SCID mice. Leukemia 2000;14:431-8 (Pubitemid 30142867)
26. Kitamura K, Minami Y, Yamamoto K, et al. Involvement of CD95-independent caspase 8 activation in arsenic trioxide-induced apoptosis. Leukemia 2000;14:1743-50
27. Gianni M, de The H. In acute promyelocytic leukemia NB4 cells, the synthetic retinoid CD437 induces contemporaneously apoptosis, a caspase-3 mediated degradation of PML/RARalpha protein and the PML retargeting on PML-nuclear bodies. Leukemia 1999;13:739-49 (Pubitemid 29243626)
28. Zhang W, Ohnisi K, Shigeno K, et al. The induction of apoptosis and cell cycle arrest by arsenic trioxide in lymphoid neoplasm's. Leukemia 1998;12:1383-91 (Pubitemid 28442384)
29. Wang ZG, Rivi R, Delva L, et al. Arsenic trioxide and melarsoprol induce programmed cell death in myeloid leukemia cell lines and function in a PML and PML-RARAalpha independent manner. Blood 1998;92:1497-504 (Pubitemid 28406851)
30. McCabe MJ, Singh KP, Reddy SA, et al. Sensitivity of myelomonocytic leukemia cells to arsenite-induced cell cycle disruption, apoptosis, and enhanced differentiation is dependent on the inter-relationship between arsenic concentration, duration of treatment, and cell cycle phase. J Pharmacol Exp Ther 2000;295:724-33
31. Miller WH Jr, Schipper HM, Lee JS, et al. Mechanisms of action of arsenic trioxide. Cancer Res 2002;62:3893-903 (Pubitemid 34792653)
32. Zhu J, Lallemand-Breitenbach V, de The H. Pathways of retinoic acid or arsenic trioxide-induced PML/RARalpha catabolism, role of oncogene degradation in disease remission. Oncogene 2001;20:7257-65 (Pubitemid 33107375)
33. Muller S, Miller WH, Dejean A. Trivalent antimonials induce degradation of the PML-RAR oncoprotein and reorganization of the promyelocytic leukemia nuclear bodies in acute promyelocytic leukemia NB4 cells. Blood 1998;92:4308-16 (Pubitemid 28544348)
34. Lallemand-Breitenbach V, Zhu J, Puvion F, et al. Role of promyelocytic leukemia (PML) sumolation in nuclear body formation, 11S proteasome recruitment, and As2O3-induced PML or PML/retinoic acid receptor alpha degradation. J Exp Med 2001;193:1361-71 (Pubitemid 32565965)
35. Hayakawa F, Privalsky ML. Phosphorylation of PML by mitogen-activated protein kinases plays a key role in arsenic trioxide-mediated apoptosis. Cancer Cell 2004;5:389-401 (Pubitemid 38482074)
36. Costantini P, Belzacq AS, Vieira HL, et al. Oxidation of a critical thiol residue of the adenine nucleotide translocator enforces Bcl-2 independent permeability transition pore opening and apoptosis. Oncogene 2000;19:307-14 (Pubitemid 30077228)
37. Larochette N, Decaudin D, Jacotot E, et al. Arsenite induces apoptosis via a direct effect on the mitochondrial permeability transition pore. Exp Cell Res 1999;249:413-21 (Pubitemid 29397949)
38. Li J, Chen P, Sinogeeva N, et al. Arsenic trioxide promotes histone H3 phosphoacetylation at the chromatin of CASPASE-10 in acute promyelocytic leukemia cells. J Biol Chem 2002;277:49504-10 (Pubitemid 36014387)
39. Davison K, Mann KK, Miller WH Jr, et al. Arsenic trioxide: mechanisms of action. Semin Hematol 2002;39:3-7
40. Bernardini S, Nuccettelli M, Noguera NI, et al. Role of GSTP-1 in mediating the effect of As2O3 in the acute promyelocytic leukemia cell line NB4. Ann Hematol 2006;85:681-7 (Pubitemid 44255038)
41. Ninomiya M, Kajiguchi T, Yamamoto K, et al. Increased oxidative DNA products in patients with acute promyelocytic leukemia during arsenic therapy. Haematologica 2006;91:1571-2 (Pubitemid 44736130)
42. Chou WC, Chen HY, Yu SL, et al. Arsenic suppresses gene expression in promyelocytic leukemia cells partly troughSp1 oxidation. Blood 2005;106:304-10
43. Mathieu J, Besancon F. Arsenic trioxide represses NF-kappaB activation and increases apoptosis in ATRA-treated APL cells. Ann NY Acad Sci 2006;1090:203-8 (Pubitemid 47092124)
44. Glienke W, Chow KU, Bauer N, et al. Down-regulation of Wt1 expression in leukemia cell lines as part of apoptotic effect in arsenic treatment using two compounds. Leuk Lymphoma 2006;47:1629-38 (Pubitemid 44364031)
45. Zheng PZ, Wang KK, Zhang QY, et al. Systems analysis of transcriptome and proteome in retinoic acid/arsenic trioxide-induced cell differentiation7apoptosis of promyelocytic leukemia. Proc Natl Acad Sci USA 2005;102:7653-8
46. Ghaffari SH, Bashash D, Dizaji MZ, et al. Alteration in miRNA gene expression pattern in acute promyelocytic leukemia cell induced by arsenic trioxide: a possible mechanism to explain arsenic multi-target action. Tumour Biol 2012;33:157-72
47. Jing Y, Wang L, Xia L, et al. Combined effect of all-trans retinoic acid and arsenic trioxide in acute promyelocytic leukemia cells in vitro and in vivo. Blood 2001;97:264-9 (Pubitemid 32061269)
48. Zhu Q, Zhang JW, Zhu HQ, et al. Synergic effects of arsenic trioxide and camp during acute promyelocytic leukemia cell maturation subtends a novel signalling cross-talk. Blood 2002;99:1014-22 (Pubitemid 34525566)
49. Nasr R, Guillemin MC, Ferhi O, et al. Eradication of acute promyelocytic leukemia-initiating cells through PML-RARA degradation. Nat Med 2008;14:1333-42
50. Leung J, Pang A, Yuen WH, et al. Relationship of expression of aquaglyceroporin 9 with arsenic uptake and sensitivity in leukemia cells. Blood 2007;109:740-6 (Pubitemid 46105976)
51. Sun HD, Ma L, Hu XC, et al. Ai-LinI treated 32 cases of acute promyelocytic leukemia. Chin J Integr Chin West Med 1992;12:170-1
52. Shen SZ, Chen GQ, Ni JH, et al. Use of arsenic trioxide (As2O3) in the treatment of acute promyelocytic leukemia (APL): clinical efficacy and pharmacokinetics in relapsed patients. Blood 1997;89:3354-60
53. Niu C, Yan H, Yu T, et al. Studies on treatment of acute promyelocytic leukemia with arsenic trioxide: remission induction, follow-up and molecular monitoring in 11 newly diagnosed and 47 relapsed acute promyelocytic leukemia patients. Blood 1999;94:3315-24 (Pubitemid 29536124)
54. Soignet SL, Frankel SR, Douer D, et al. United States multicenter study of arsenic trioxide in relapsed acute promyelocytic leukemia. J Clin Oncol 2001;19:3852-60 (Pubitemid 32880064)
55. Lazo G, Kantarjian H, Estey E, et al. Use of arsenic trioxide (As2O3) in the treatment of patients with acute promyelocytic leukemia: the M. D. Anderson experience. Cancer 2003;97:2218-24
56. Au WY, Lie AK, Chim CS, et al. Arsenic trioxide in comparison with chemotherapy and bone marrow transplantation for the treatment of relapsed acute promyelocytic leukemia. Ann Oncol 2003;14:752-7 (Pubitemid 36621776)
57. Raffoux E, Rousselot P, Poupon J, et al. Combined treatment with arsenic trioxide and all-trans retinoic acid in patients with relapsed acute promyelocytic leukemia. J Clin Oncol 2003;21:2326-34 (Pubitemid 46621871)
58. Carmosino I, Latagliata R, Avvisati G, et al. Arsenic trioxide in the treatment of advanced acute promyelocytic leukemia. Haematologica 2004;89:615-17 (Pubitemid 38691254)
59. Shigeno K, Naito K, Sahara N, et al. Arsenic trioxide therapy in relapsed or refractory Japanese patients with acute promyelocytic leukemia: updates outcomes of the phase II study and postremission therapies. Int J Hematol 2005;82:224-9 (Pubitemid 41552328)
60. Aribi A, Kantarjian H, Estey E, et al. Combination therapy with arsenic trioxide, all-trans retinoic acid, and gentuzumab ozogamicin in recurrent acute promyelocytic leukemia. Cancer 2007;109:1355-9 (Pubitemid 46466562)
61. Shen ZX, Shi ZZ, Fang J, et al. All-trans retinoic acid/As2O3 combination yields a high quality remission and survival in newly diagnosed acute promyelocytic leukemia. Proc Natl Acad Sci USA 2004;101:5328-35 (Pubitemid 38481160)
62. Hu J, Liu YF, Wu CF, et al. Long-term efficacy and safety of all-trans retinoic acid/arsenic trioxide-based therapy in newly diagnosed acute promyelocytic leukemia. Proc Natl Acad Sci USA 2009;106:3342-7
63. Estey E, Garcia-Manero G, Ferrajoli A, et al. Use of all-trans retinoic acid plus arsenic trioxide as an alternative to chemotherapy in untreated acute promyelocytic leukemia. Blood 2006;107:3469-73
64. Mathews V, George B, Lakshmi KM, et al. Single-agent arsenic trioxide in the treatment of newly diagnosed acute promyelocytic leukemia: durable remissions with minimal toxicity. Blood 2006;107:2627-32
65. Mathews V, Thomas M, Srivastava VM, et al. Impact of FLT3 mutations and secondary cytogenetic changes on the outcome of patients with newly diagnosed acute promyelocytic leukemia treated with a single agent arsenic trioxide regimen. Haematologica 2007;92:994-5 (Pubitemid 350144251)
66. Mathews V, George B, Chendamarai E, et al. Single-agent arsenic trioxide in the treatment of newly diagnosed acute promyelocytic leukemia. long-term follow-up data. J Clin Oncol 2010;28:3866-71
67. Ghavamzadeh A, Alimoghaddam K, Ghaffari SH, et al. Treatment of acute promyelocytic leukemia with arsenic trioxide without ATRA and/or chemotherapy. Ann Oncol 2006;17:131-4 (Pubitemid 43033852)
68. Ghavamzadeh A, Alimoghaddam K, Rostami S, et al. Phase II study of single-agent arsenic trioxide for the front-line therapy of acute promyelocytic leukemia. J Clin Oncol 2011;29:2753-7
69. Dai CW, Zhang GS, Shen JK, et al. Use of all-trans retinoic acid in combination with arsenic trioxide for remission induction in patients with newly diagnosed acute promyelocytic leukemia and for consolidation/maintenance in CR patients. Acta Haematol 2009;121:1-8
70. Ravandi F, Estey E, Jones D, et al. Effective treatment of acute promyelocytic leukemia with all-trans retinoic acid, arsenic trioxide, and gentuzumab ozogamicin. J Clin Oncol 2009;27:504-10
71. Powell BL, Moser B, Stock W, et al. Arsenic trioxide improves event-free and overall survival for adults with acute promyelocytic leukemia: north American Leukemia Intergroup Study C9710. Blood 2010;116:3751-7
72. Gore SD, Gojo I, Sekeres MA, et al. Single cycle of arsenic trioxide-based consolidation chemotherapy spares anthracycline exposure in the primary management of acute promyelocytic leukemia. J Clin Oncol 2010;28:1047-53
73. George B, Mathews V, Poonkuzhali B, et al. Treatment of children with newly diagnosed acute promyelocytic leukemia with arsenic trioxide: a single center experience. Leukemia 2004;18:1587-90 (Pubitemid 39386334)
74. Zhou J, Zhang Y, Li J, et al. Single-agent arsenic trioxide in the treatment of children with newly diagnosed acute promyelocytic leukemia. Blood 2010;115:1697-702
75. Zhang L, Zhu X, Zou Y, et al. Effect of arsenic trioxide on the treatment of children with newly diagnosed acute promyelocytic leukemia in China. Int J Hematol 2011;93:199-205
76. Breccia M, Cicconi L, Minotti C, et al. Efficacy of prolonged therapy with combined arsenic trioxide and ATRA for relapse of acute promyelocytic leukemia. Haematologica 2011; Epub ahead of print
77. Huang SL, Guo AX, Xiang Y, et al. Clinical study on the treatment of acute promyelocytic leukemia with composite Indigo Naturalis tablets. Chin J Hematol 1995;16:26-8
78. Lu DP, Qiu JY, Jiang B, et al. Tetra-arsenic tetra-sulfide for the treatment of acute promyelocytic leukemia: a pilot report. Blood 2002;99:3136-43 (Pubitemid 34525290)
79. Wu T, Zhao J, Jiang B, et al. Tetra-arsenic tetra-sulfide containing triple-agent regimen as the first-line therapy for acute promyelocytic leukemia: expeditiously consecutive complete remission and improved disease-free survival. Blood 2007;110:abstract 591
80. Ghaffari SH, Shayan-Asl N, Jamialahmadi AH, et al. Telomerase activity and telomere length in patients with acute promyelocytic leukemia: indicative of proliferative activity, disease progression, and overall survival. Ann Oncol 2008;19:1927-34
81. Alimoghaddam K, Ghavamzadeh A, Rostami S, et al. Risk factors for early mortality, relapse and overall survival in new cases of APL treated with arsenic trioxide. J Clin Oncol 2007;25:abstract 374
82. Tallman MS. What is the role of arsenic in newly diagnosed APL? Best Pract Res Clin Haematol 2008;21:659-66
83. Liu YJ, Wu DP, Liang JY, et al. Long-term survey of outcome in acute promyelocytic leukemia: a single center experience in 340 patients. Med Oncol 2010; Epub ahead of print
84. Ferrara F, Finizio O, Izzo T, et al. Autologous stem cell transplantation for patients with acute promyelocytic leukemia in second molecular remission. Anticancer Res 2010;30:3845-9