Contemporary treatment of APL

Current Hematologic Malignancy Reports

Volumn 9, Issue 2, 2014, Pages 193-201

  Cull, Elizabeth H ^a   Altman, Jessica K ^a  

^a NORTHWESTERN UNIVERSITY   (United States)

Author keywords

Acute promyelocytic leukemia; All trans retinoic acid; Arsenic trioxide; Early death rate

Indexed keywords

ARSENIC TRIOXIDE; RETINOIC ACID; RETINOIC ACID BINDING PROTEIN; TAMIBAROTENE; ANTINEOPLASTIC AGENT; ORGANOARSENIC DERIVATIVE; OXIDE;

ARTICLE; CANCER CHEMOTHERAPY; CANCER MORTALITY; CANCER THERAPY; CONSOLIDATION CHEMOTHERAPY; DRUG FORMULATION; DRUG TOXICITY; GERIATRIC PATIENT; HEMATOLOGIC DISEASE; HEMORRHAGIC COMPLICATION; HIGH RISK PATIENT; HUMAN; INDUCTION CHEMOTHERAPY; LEUKEMIA RELAPSE; LEUKEMIA REMISSION; MAINTENANCE CHEMOTHERAPY; PHASE 2 CLINICAL TRIAL (TOPIC); PHASE 3 CLINICAL TRIAL (TOPIC); PROMYELOCYTIC LEUKEMIA; RECURRENCE FREE SURVIVAL; RECURRENCE RISK; SENESCENCE; CLINICAL TRIAL (TOPIC); LEUKEMIA, PROMYELOCYTIC, ACUTE; RECURRENT DISEASE;

ANTINEOPLASTIC AGENTS; ANTINEOPLASTIC COMBINED CHEMOTHERAPY PROTOCOLS; ARSENICALS; CLINICAL TRIALS AS TOPIC; HUMANS; LEUKEMIA, PROMYELOCYTIC, ACUTE; OXIDES; RECURRENCE; TRETINOIN;

EID: 84903608950     PISSN: 15588211     EISSN: 1558822X     Source Type: Journal    
DOI: 10.1007/s11899-014-0205-6     Document Type: Article

References (76)

1. Douer D. The epidemiology of acute promyelocytic leukemia. Balliere's Best Pract Clin Hematol. 2003;16:357-67.
2. Estey E, Thall P, Kantarjian H, et al. Association between increased body mass index and a diagnosis of acute promyelocytic leukemia in patients with acute myeloid leukemia. Leukemia. 1997;11:1661-4. (Pubitemid 27458604)
3. Scaglioni PP, Pandolfi PP. The theory of APL revisited. Curr Top Microbiol Immunol. 2007;313:85-100. (Pubitemid 47400334)
4. Huang ME, Ye YC, Chen SR, et al. Use of all-trans retinoic acid in the treatment of acute promyelocytic leukemia. Blood. 1988;72: 567-72.
5. Castaigne S, Chomienne C, DanielMT, et al. All-trans retinoic acid as a differentiation therapy for acute promyelocytic leukemia. I. Clinical results. Blood. 1990;76:1704-9.
6. Warrell Jr RP, Frankel SR, Miller Jr WH, et al. Differentiation therapy of acute promyelocytic leukemia with tretinoin (all-transretinoic acid). N Engl J Med. 1991;324:1385-93.
7. Chen ZX, Xue YQ, Zhang R, et al. A clinical and experimental study on all-trans retinoic acid-treated acute promyelocytic leukemia patients. Blood. 1991;78:1413-9.
8. Fenaux P, Castaigne S, Dombret H, et al. All-transretinoic acid followed by intensive chemotherapy gives a high complete remission rate and may prolong remissions in newly diagnosed acute promyelocytic leukemia: a pilot study on 26 cases. Blood. 1992;80: 2176-81.
9. Kanamaru A, Takemoto Y, Tanimoto M, et al. All-trans retinoic acid for the treatment of newly diagnosed acute promyelocytic leukemia. Japan Adult Leukemia Study Group. Blood. 1995;85:1202-6.
10. Tallman MS, Andersen JW, Schiffer CA, et al. All-trans-retinoic acid in acute promyelocytic leukemia. N Engl J Med. 1997;337: 1021-8. (Pubitemid 27430519)
11. Tallman MS, Nabhan C, Feusner JH, Rowe JM. Acute promyelocytic leukemia: evolving therapeutic strategies. Blood. 2002;99:759-67. (Pubitemid 34525534)
12. .•• Lo-Coco F, Avvisati G, Vignetti M, et al. Retinoic acid and arsenic trioxide for acute promyelocytic leukemia. N Engl J Med. 2013;369:111-21. Phase III trial that randomized standard risk patients with APL to treatment with ATRA/chemotherapy vs. ATRA/ATO. Results showed non-inferiority of ATRA/ATO regimen compared to ATRA/chemotherapy.
13. Fenaux P, Chastang C, Chevret S, et al. 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. The European APL Group. Blood. 1999;94:1192-200. (Pubitemid 29380399)
14. 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.
15. Sanz MA, Martin G, Rayon C, et al. A modified AIDA protocol with anthracycline-based consolidation results in high antileukemic efficacy and reduced toxicity in newly diagnosed PML/RARalphapositive acute promyelocytic leukemia. PETHEMA group. Blood. 1999;94:3015-21. (Pubitemid 29512705)
16. 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)
17. Ades L, Sanz MA, Chevret S, et al. Treatment of newly diagnosed acute promyelocytic leukemia (APL): a comparison of French- Belgian-Swiss and PETHEMA results. Blood. 2008;111:1078-84. (Pubitemid 351213385)
18. Sanz MA, Lo Coco F, Martin G, et al. Definition of relapse risk and role of nonanthracycline drugs for consolidation in patients with acute promyelocytic leukemia: a joint study of the PETHEMA and GIMEMA cooperative groups. Blood. 2000;96:1247-53. (Pubitemid 30658450)
19. Sanz MA, Martin G, Gonzalez M, et al. Risk-adapted treatment of acute promyelocytic leukemia with all-trans-retinoic acid and anthracycline monochemotherapy: a multicenter study by the PETHEMA group. Blood. 2004;103:1237-43. (Pubitemid 38168633)
20. Ades L, Chevret S, Raffoux E, et al. Is cytarabine useful in the treatment of acute promyelocytic leukemia? Results of a randomized trial from the European Acute Promyelocytic Leukemia Group. J Clin Oncol. 2006;24:5703-10. (Pubitemid 46631312)
21. 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.
22. 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)
23. 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)
24. 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.
25. .•• Iland HJ, Bradstock K, Supple SG, et al. All-trans-retinoic acid, idarubicin, and IV arsenic trioxide as initial therapy in acute promyelocytic leukemia (APML4). Blood. 2012;120:1570-80. They used a consolidation regimen of ATO/ATRA, eliminating chemotherapy from the consolidation phase and reported excellent response rates and OS.
26. .•• 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. They added ATO to the consolidation phase and reported improved outcomes, particularly in high risk APL patients.
27. Ravandi F, Estey E, Jones D, et al. Effective treatment of acute promyelocytic leukemia with all-trans-retinoic acid, arsenic trioxide, and gemtuzumab ozogamicin. J Clin Oncol. 2009;27:504-10.
28. 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 U S A. 2004;101:5328-35. (Pubitemid 38481160)
29. Iland HJ CM, Seymour J. The Australasian Leukaemia and lymphoma group APML4 trial - update on APL treatment down under [abstract]. In: 6th International Symposium on Acute Promyelocytic Leukemia Abstract Book 2013 Sept 29-Oct 2; Rome, Italy.
30. National Comprehensive Cancer Network. Acute Myeloid Leukemia version 2.2013. http://www.nccn.org/professionals/physician-gls/pdf/aml.pdf.
31. Sanz MA, Montesinos P, Rayon C, et al. Risk-adapted treatment of acute promyelocytic leukemia based on all-trans retinoic acid and anthracycline with addition of cytarabine in consolidation therapy for high-risk patients: further improvements in treatment outcome. Blood. 2010;115:5137-46.
32. .• Coutre S OM, Powell B, et al. A phase III randomized trial (S0521) of maintenance versus observation for patients with previously untreated low/intermediate risk acute promyelocytic leukemia. In: 6th International Symposium on Acute Promyelocytic Leukemia Abstract Book 2013 Sept 29-Oct 2. Phase III randomized trial that compared maintenance therapy to no maintenance therapy in standard risk patients and reported no differences in relapse rates or OS.
33. de la Serna J, Montesinos P, Vellenga E, et al. Causes and prognostic factors of remission induction failure in patients with acute promyelocytic leukemia treated with all-trans retinoic acid and idarubicin. Blood. 2008;111:3395-402.
34. Micol JB Raffoux E, Boissel N, et al. Do early events excluding patients with acute promyelocytic leukemia (APL) from trial enrollment modify treatment result evaluation? Real-life management of 100 patients referred to the University Hospital Saint-Louis between 2000 and 2010. Blood. 2010;116:473. ASH Annual Meeting Abstracts.
35. Lehmann S, Ravn A, Carlsson L, et al. Continuing high early death rate in acute promyelocytic leukemia: a population-based report from the Swedish Adult Acute Leukemia Registry. Leukemia. 2011;25:1128-34.
36. Altman JK, Rademaker A, Cull E, et al. Administration of ATRA to newly diagnosed patients with acute promyelocytic leukemia is delayed contributing to early hemorrhagic death. Leuk Res. 2013;37:1004-9.
37. Rahmé R, Thomas X, Recher C, et al. Early Deaths (ED) in Acute Promyelocytic Leukemia (APL) in France: a retrospective multicenter study in 355 patients (pts). Blood. 2012;120:890. ASH annual Meeting Abstracts.
38. Tallman MS, Altman JK. How I treat acute promyelocytic leukemia. Blood. 2009;114:5126-35.
39. Tallman MS, Brenner B, Serna Jde L, et al. Meeting report. Acute promyelocytic leukemia-associated coagulopathy, 21 January 2004, London, United Kingdom. Leuk Res. 2005;29:347-51.
40. Barbui T, Finazzi G, Falanga A. The impact of all-trans-retinoic acid on the coagulopathy of acute promyelocytic leukemia. Blood. 1998;91:3093-102. (Pubitemid 28193856)
41. Kelaidi C, Ades L, Fenaux P. Treatment of acute promyelocytic leukemia with high white cell blood counts. Mediterr J Hematol Infect Dis. 2011;3:e2011038.
42. Frankel SR, Eardley A, Lauwers G, et al. The "retinoic acid syndrome"in acute promyelocytic leukemia. Ann Intern Med. 1992;117:292-6.
43. Kelaidi C, Chevret S, De Botton S, et al. Improved outcome of acute promyelocytic leukemia with high WBC counts over the last 15 years: the European APL Group experience. J Clin Oncol. 2009;27:2668-76.
44. Vahdat L, Maslak P, Miller Jr WH, et al. Early mortality and the retinoic acid syndrome in acute promyelocytic leukemia: impact of leukocytosis, low-dose chemotherapy, PMN/RAR-alpha isoform, and CD13 expression in patients treated with all-trans retinoic acid. Blood. 1994;84:3843-9.
45. de Botton S, Sanz MA, Chevret S, et al. Extramedullary relapse in acute promyelocytic leukemia treated with all-trans retinoic acid and chemotherapy. Leukemia. 2006;20:35-41.
46. Sanz MA, Grimwade D, Tallman MS, et al. Management of acute promyelocytic leukemia: recommendations from an expert panel on behalf of the European LeukemiaNet. Blood. 2009;113:1875-91.
47. Breccia M, Carmosino I, Diverio D, et al. Early detection of meningeal localization in acute promyelocytic leukaemia patients with high presenting leucocyte count. Br J Haematol. 2003;120: 266-70. (Pubitemid 36194175)
48. Lengfelder E, Hofmann WK, Nolte F. Management of elderly patients with acute promyelocytic leukemia: progress and problems. Ann Hematol. 2013;92:1181-8.
49. Park JH, Qiao B, Panageas KS, et al. Early death rate in acute promyelocytic leukemia remains high despite all-trans retinoic acid. Blood. 2011;118:1248-54.
50. Ades L, Chevret S, De Botton S, et al. Outcome of acute promyelocytic leukemia treated with all trans retinoic acid and chemotherapy in elderly patients: the European group experience. Leukemia. 2005;19:230-3. (Pubitemid 40220587)
51. Ono T, Takeshita A, Kishimoto Y, et al. Long-term outcome and prognostic factors of elderly patients with acute promyelocytic leukemia. Cancer Sci. 2012;103:1974-8.
52. Sanz MA, Vellenga E, Rayon C, et al. All-trans retinoic acid and anthracycline monochemotherapy for the treatment of elderly patients with acute promyelocytic leukemia. Blood. 2004;104:3490-3. (Pubitemid 39564417)
53. Latagliata R, Breccia M, Fazi P, et al. GIMEMA AIDA 0493 amended protocol for elderly patients with acute promyelocytic leukaemia. Long-term results and prognostic factors. Br J Haematol. 2011;154:564-8.
54. Zhang Y, Zhang Z, Li J, et al. Long-term efficacy and safety of arsenic trioxide for first-line treatment of elderly patients with newly diagnosed acute promyelocytic leukemia. Cancer. 2013;119:115-25.
55. Lo-Coco F, Cimino G, Breccia M, et al. Gemtuzumab ozogamicin (Mylotarg) as a single agent for molecularly relapsed acute promyelocytic leukemia. Blood. 2004;104:1995-9. (Pubitemid 39297848)
56. Breccia M, Cimino G, Diverio D, et al. Sustained molecular remission after low dose gemtuzumab-ozogamicin in elderly patients with advanced acute promyelocytic leukemia. Haematologica. 2007;92:1273-4. (Pubitemid 350144194)
57. Lo-Coco F, Orlando SM, Platzbecker U. Treatment of acute promyelocytic leukemia. N Engl J Med. 2013;369:1472.
58. 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)
59. 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 leukaemia. Ann Oncol. 2003;14:752-7. (Pubitemid 36621776)
60. 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)
61. Shigeno K, Naito K, Sahara N, et al. Arsenic trioxide therapy in relapsed or refractory Japanese patients with acute promyelocytic leukemia: updated outcomes of the phase II study and postremission therapies. Int J Hematol. 2005;82:224-9. (Pubitemid 41552328)
62. Aribi A, Kantarjian HM, Estey EH, et al. Combination therapy with arsenic trioxide, all-trans retinoic acid, and gemtuzumab ozogamicin in recurrent acute promyelocytic leukemia. Cancer. 2007;109:1355-9. (Pubitemid 46466562)
63. Lu J, Huang X, Bao L, et al. Treatment outcomes in relapsed acute promyelocytic leukemia patients initially treated with all- retinoic acid and arsenic compound-based combined therapies. Oncol Lett. 2014;7:177-82.
64. Thirugnanam R, George B, Chendamarai E, et al. Comparison of clinical outcomes of patients with relapsed acute promyelocytic leukemia induced with arsenic trioxide and consolidated with either an autologous stem cell transplant or an arsenic trioxide-based regimen. Biol Blood Marrow Transplant. 2009;15:1479-84.
65. de Botton S, Fawaz A, Chevret S, et al. Autologous and allogeneic stem-cell transplantation as salvage treatment of acute promyelocytic leukemia initially treated with all-trans-retinoic acid: a retrospective analysis of the European acute promyelocytic leukemia group. J Clin Oncol. 2005;23:120-6. (Pubitemid 41702630)
66. Pemmaraju N, Tanaka MF, Ravandi F, et al. Outcomes in patients with relapsed or refractory acute promyelocytic leukemia treated with or without autologous or allogeneic hematopoietic stem cell transplantation. Clin Lymphoma Myeloma Leuk. 2013;13:485-92.
67. .•• Zhu HH,Wu DP, Jin J, et al. Oral tetra-arsenic tetra-sulfide formula versus intravenous arsenic trioxide as first-line treatment of acute promyelocytic leukemia: a multicenter randomized controlled trial. J Clin Oncol. 2013;31:4215-21. This is a randomized controlled trial comparing outcomes with IVarsenic vs. a novel form of oral arsenic. Demonstrated no difference in outcomes with the new formulation.
68. Ozpolat B, Lopez-Berestein G, Adamson P, et al. Pharmacokinetics of intravenously administered liposomal all-trans-retinoic acid (ATRA) and orally administered ATRA in healthy volunteers. J Pharm Pharm Sci. 2003;6:292-301. (Pubitemid 37207235)
69. Tsimberidou AM, Tirado-Gomez M, Andreeff M, et al. Singleagent liposomal all-trans retinoic acid can cure some patients with untreated acute promyelocytic leukemia: an update of The University of Texas M. D. Anderson Cancer Center Series. Leuk Lymphoma. 2006;47:1062-8. (Pubitemid 44102970)
70. Jain P, Kantarjian H, Estey E et al. Single-Agent Liposomal All- Trans-Retinoic Acid as Initial Therapy for Acute Promyelocytic Leukemia: 13-Year Follow-Up Data. Clin Lymphoma Myeloma Leuk 2013.
71. Warrell Jr RP. Retinoid resistance in acute promyelocytic leukemia: new mechanisms, strategies, and implications. Blood. 1993;82: 1949-53. (Pubitemid 23293971)
72. Shao W, Benedetti L, Lamph WW, et al. A retinoid-resistant acute promyelocytic leukemia subclone expresses a dominant negative PML-RAR alpha mutation. Blood. 1997;89:4282-9. (Pubitemid 27260235)
73. Ohnishi K. PML-RARalpha inhibitors (ATRA, tamibaroten, arsenic troxide) for acute promyelocytic leukemia. Int J Clin Oncol. 2007;12:313-7. (Pubitemid 47573414)
74. Tobita T, Takeshita A, Kitamura K, et al. Treatment with a new synthetic retinoid,Am80, of acute promyelocytic leukemia relapsed from complete remission induced by all-trans retinoic acid. Blood. 1997;90:967-73. (Pubitemid 27314129)
75. Naina HV, Levitt D, Vusirikala M, et al. Successful treatment of relapsed and refractory extramedullary acute promyelocytic leukemia with tamibarotene. J Clin Oncol. 2011;29:e534-6.
76. Di Veroli A, Ramadan SM, Divona M, et al. Molecular remission in advanced acute promyelocytic leukaemia after treatment with the oral synthetic retinoid Tamibarotene. Br J Haematol. 2010;151:99-101.