How I treat acute promyelocytic leukemia

Blood

Volumn 114, Issue 25, 2009, Pages 5126-5135

  Tallman, Martin S ^a   Altman, Jessica K ^a  

^a NORTHWESTERN UNIVERSITY   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ANTHRACYCLINE; ARSENIC TRIOXIDE; RETINOIC ACID; ANTINEOPLASTIC AGENT; ONCOPROTEIN; ORGANOARSENIC DERIVATIVE; OXIDE; PROMYELOCYTIC LEUKEMIA RETINOIC ACID RECEPTOR ALPHA FUSION ONCOPROTEIN; PROMYELOCYTIC LEUKEMIA-RETINOIC ACID RECEPTOR ALPHA FUSION ONCOPROTEIN;

BONE MARROW BIOPSY; CANCER COMBINATION CHEMOTHERAPY; CANCER DIAGNOSIS; CANCER EPIDEMIOLOGY; CANCER REGRESSION; CANCER RELAPSE; CANCER SURVIVAL; DRUG EFFICACY; GENETIC ANALYSIS; HISTOPATHOLOGY; HUMAN; HYPERLEUKOCYTOSIS; LEUKOCYTOSIS; MAINTENANCE THERAPY; MORPHOLOGY; OPTIMAL DRUG DOSE; PATIENT CARE; PRIORITY JOURNAL; PROMYELOCYTIC LEUKEMIA; REVIEW; SKIN BIOPSY; TREATMENT DURATION; ARTICLE; BONE MARROW EXAMINATION; DOSE RESPONSE; GENETICS; OUTCOME ASSESSMENT; PRACTICE GUIDELINE; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION;

ANTINEOPLASTIC AGENTS; ANTINEOPLASTIC COMBINED CHEMOTHERAPY PROTOCOLS; ARSENICALS; BONE MARROW EXAMINATION; DOSE-RESPONSE RELATIONSHIP, DRUG; HUMANS; LEUKEMIA, PROMYELOCYTIC, ACUTE; ONCOGENE PROTEINS, FUSION; OUTCOME ASSESSMENT (HEALTH CARE); OXIDES; PRACTICE GUIDELINES AS TOPIC; REVERSE TRANSCRIPTASE POLYMERASE CHAIN REACTION; TRETINOIN;

EID: 73949097021     PISSN: 00064971     EISSN: 15280020     Source Type: Journal    
DOI: 10.1182/blood-2009-07-216457     Document Type: Review

References (89)

1. Rowley JD, Golomb HM, Dougherty C. 15/17 translocation, a consistent chromosomal change in acute promyelocytic leukaemia. Lancet. 1977; 1(8010):549-550.
2. Grignani F, Ferrucci PF, Testa U, et al. The acute promyelocytic leukemia-specific PML-RAR α fusion protein inhibits differentiation and promotes survival of myeloid precursor cells. Cell. 1993; 74(3):423-431.
3. Maule MM, Dama E, Mosso ML, Magnani C, Pastore G, Merletti F. High incidence of acute promyelocytic leukemia in children in northwest Italy, 1980-2003: a report from the Childhood Cancer Registry of Piedmont. Leukemia. 2008; 22(2):439-441.
4. Testi AM, Biondi A, Lo Coco F, et al. GIMEMA-AIEOPAIDA protocol for the treatment of newly diagnosed acute promyelocytic leukemia (APL) in children. Blood. 2005;106(2):447-453.
5. Melnick A, Licht JD. Deconstructing a disease: RARα, its fusion partners, and their roles in the pathogenesis of acute promyelocytic leukemia. Blood. 1999;93(10):3167-3215.
6. Tallman MS, Nabhan C, Feusner JH, Rowe JM. Acute promyelocytic leukemia: evolving therapeutic strategies. Blood. 2002;99(3):759-767.
7. Wang ZY, Chen Z. Acute promyelocytic leukemia: from highly fatal to highly curable. Blood. 2008; 111(5):2505-2515.
8. Degos L, Dombret H, Chomienne C, et al. All-trans-retinoic acid as a differentiating agent in the treatment of acute promyelocytic leukemia. Blood. 1995;85(10):2643-2653.
9. Bennett JM, Catovsky D, Daniel MT, et al. Hypergranular promyelocytic leukemia: correlation between morphology and chromosomal translocations including t(15;17) and t(11;17). Leukemia. 2000;14(7):1197-1200.
10. Tallman MS, Kim H, Schiffer CA, et al. Microgranular variant (M3V) of acute promyelocytic leukemia (APL) does not have a worse prognosis than classical APL in the atra era: a report of 153 patients treated on Intergroup 0129 and Pethema LPA96 and LPA99. Blood. 2004;104(11):116a.
11. Asou N, Kishimoto Y, Kiyoi H, et al. A randomized study with or without intensified maintenance chemotherapy in patients with acute promyelocytic leukemia who have become negative for PML-RARα transcript after consolidation therapy: the Japan Adult Leukemia Study Group (JALSG) APL97 study. Blood. 2007;110(1):59-66.
12. De la Serna J, Montesinos P, Vellenga E. Causes and prognostic factors of remission induction failure in patients with acute promyelocytic leukemia treated with all-trans retinoic acid and idarubicin. Blood. 2008;111:3395-3402.
13. Sanz MA, Martin G, Gonzalez M, et al. Riskadapted treatment of acute promyelocytic leukemia with all-trans-retinoic acid and anthracycline monochemotherapy: a multicenter study by the PETHEMA group. Blood. 2004;103(4):1237-1243.
14. de Botton S, Chevret S, Coiteux V, et al. Early onset of chemotherapy can reduce the incidence of ATRA syndrome in newly diagnosed acute promyelocytic leukemia (APL) with low white blood cell counts: results from APL 93 trial. Leukemia. 2003;17(2):339-342.
15. 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(16):2668-2676.
16. Saumet A, Vetter G, Bouttier M, et al. Transcriptional repression of microRNA genes by PMLRARA increases expression of key cancer proteins in acute promyelocytic leukemia. Blood. 2009;113(2):412-421.
17. Kurzrock R, Estey E, Talpaz M. All-trans retinoic acid: tolerance and biologic effects in myelodysplastic syndrome. J Clin Oncol. 1993;11(8):1489- 1495.
18. Hakimian D, Tallman MS, Zugerman C, Caro WA. Erythema nodosum associated with all-trans-retinoic acid in the treatment of acute promyelocytic leukemia. Leukemia. 1993;7(5):758-759.
19. Astudillo L, Loche F, Reynish W, Rigal-Huguet F, Lamant L, Pris J. Sweet's syndrome associated with retinoic acid syndrome in a patient with promyelocytic leukemia. Ann Hematol. 2002;81(2): 111-114.
20. Jagdeo J, Campbell R, Long T, Muglia J, Telang G, Robinson-Bostom L. Sweet's syndrome-like neutrophilic lobular panniculitis associated with all-trans-retinoic acid chemotherapy in a patient with acute promyelocytic leukemia. J Am Acad Dermatol. 2007;56(4):690-693.
21. Christ E, Linka A, Jacky E, Speich R, Marincek B, Schaffner A. Sweet's syndrome involving the musculoskeletal system during treatment of promyelocytic leukemia with all-trans retinoic acid. Leukemia. 1996;10(4):731-734.
22. Thepot SG, Chevret S, De Botton S, et al. Impact of ATRA duration during the induction treatment of newly diagnosed APL. Blood. 2008;112:139a.
23. Hatake K, Uwai M, Ohtsuki T, et al. Rare but important adverse effects of all-trans retinoic acid in acute promyelocytic leukemia and their management. Int J Hematol. 1997;66(1):13-19.
24. Castaigne S, Lefebvre P, Chomienne C, et al. Effectiveness and pharmacokinetics of low-dose all-trans retinoic acid (25 mg/m2) in acute promyelocytic leukemia. Blood. 1993;82(12):3560-3563.
25. Chen GQ, Shen ZX, Wu F, et al. Pharmacokinetics and efficacy of low-dose all-trans retinoic acid in the treatment of acute promyelocytic leukemia. Leukemia. 1996;10(5):825-828.
26. Nasr R, Guillemin MC, Ferhi O, et al. Eradication of acute promyelocytic leukemia-initiating cells through PML-RARA degradation. Nat Med. 2008; 14(12):1333-1342.
27. Jacomo RH, Melo RA, Souto FR, et al. Clinical features and outcomes of 134 Brazilians with acute promyelocytic leukemia who received ATRA and anthracyclines. Haematologica. 2007; 92(10):1431-1432.
28. Watanabe R, Murata M, Takayama N, et al. Longterm follow-up of hemostatic molecular markers during remission induction therapy with all-trans retinoic acid for acute promyelocytic leukemia: Keio Hematology-Oncology Cooperative Study Group (KHOCS). Thromb Haemost. 1997;77(4): 641-645.
29. 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(3):347-351.
30. Yanada M, Matsushita T, Asou N, et al. Severe hemorrhagic complications during remission induction therapy for acute promyelocytic leukemia: incidence, risk factors, and influence on outcome. Eur J Haematol. 2007;78(3):213-219.
31. Escudier SK, Kantarjian HM, Estey EH. Thrombosis in patients with acute promyelocytic leukemia treated with and without all-trans retinoic acid. Leuk Lymphoma. 1996;20(5):435-439.
32. Dally N, Hoffman R, Haddad N, Sarig G, Rowe JM, Brenner B. Predictive factors of bleeding and thrombosis during induction therapy in acute promyelocytic leukemia: a single center experience in 34 patients. Thromb Res. 2005;116(2):109-114.
33. Rodeghiero F, Avvisati G, Castaman G, Barbui T, Mandelli F. Early deaths and anti-hemorrhagic treatments in acute promyelocytic leukemia: a GIMEMA retrospective study in 268 consecutive patients. Blood. 1990;75(11):2112-2117.
34. Di Bona E, Avvisati G, Castaman G, et al. Early haemorrhagic morbidity and mortality during remission induction with or without all-trans retinoic acid in acute promyelocytic leukaemia. Br J Haematol. 2000;108(4):689-695.
35. Miller WH Jr, Kakizuka A, Frankel SR, et al. Reverse transcription polymerase chain reaction for the rearranged retinoic acid receptor α clarifies diagnosis and detects minimal residual disease in acute promyelocytic leukemia. Proc Natl Acad Sci U S A. 1992;89(7):2694-2698.
36. Burnett AK, Grimwade D, Solomon E, Wheatley K, Goldstone AH. 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. 1999;93(12): 4131-4143.
37. Gomis F, Sanz J, Sempere A, et al. Immunofluorescent analysis with the anti-PML monoclonal antibody PG-M3 for rapid and accurate genetic diagnosis of acute promyelocytic leukemia. Ann Hematol. 2004;83(11):687-690.
38. De Botton S, Dombret H, Sanz M, et al. Incidence, clinical features, and outcome of all transretinoic acid syndrome in 413 cases of newly diagnosed acute promyelocytic leukemia: the European APL Group. Blood. 1998;92(8):2712-2718.
39. Montesinos P, Bergua JM, Vellenga E, et al. Differentiation syndrome in patients with acute promyelocytic leukemia treated with all-trans retinoic acid and anthracycline chemotherapy: characteristics, outcome, and prognostic factors. Blood. 2009;113(4):775-783.
40. Frankel SR, Eardley A, Lauwers G, Weiss M, Warrell RP Jr. The "retinoic acid syndrome" in acute promyelocytic leukemia. Ann Intern Med. 1992;117(4):292-296.
41. Vahdat L, Maslak P, Miller WH Jr, et al. Early mortality and the retinoic acid syndrome in acute promyelocytic leukemia: impact of leukocytosis, lowdose chemotherapy, PMN/RAR-α isoform, and CD13 expression in patients treated with all-trans retinoic acid. Blood. 1994;84(11):3843-3849.
42. Tallman MS, Andersen JW, Schiffer CA, et al. Clinical description of 44 patients with acute promyelocytic leukemia who developed the retinoic acid syndrome. Blood. 2000;95(1):90-95.
43. Raanani P, Segal E, Levi I, et al. Diffuse alveolar hemorrhage in acute promyelocytic leukemia patients treated with ATRA: a manifestation of the basic disease or the treatment. Leuk Lymphoma. 2000;37(5):605-610.
44. Wiley JS, Firkin FC. Reduction of pulmonary toxicity by prednisolone prophylaxis during all-trans retinoic acid treatment of acute promyelocytic leukemia: Australian Leukaemia Study Group. Leukemia. 1995;9(5):774-778.
45. 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(2): 266-270.
46. 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(1):35-41.
47. 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(1):120-126.
48. Dvorak CC, Agarwal R, Dahl GV, Gregory JJ, Feusner JH. Hematopoietic stem cell transplant for pediatric acute promyelocytic leukemia. Biol Blood Marrow Transplant. 2008;14(7):824-830.
49. Linker CA, Owzar K, Powell B, et al. Auto-SCT for AML in second remission: CALGB Study 9620. Bone Marrow Transplant. 2009;44(6):353-359.
50. 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(4):1192-1200.
51. Avvisati G, Lo Coco F, Diverio D, et al. AIDA (all-trans retinoic acid + idarubicin) in newly diagnosed acute promyelocytic leukemia: a Gruppo Italiano Malattie Ematologiche Maligne dell'Adulto (GIMEMA) pilot study. Blood. 1996; 88(4):1390-1398.
52. Mandelli F, Diverio D, Avvisati G, et al. Molecular remission in PML/RAR α-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(3):1014-1021.
53. Tallman MS, Andersen JW, Schiffer CA, et al. All-trans-retinoic acid in acute promyelocytic leukemia. N Engl J Med. 1997;337(15):1021-1028.
54. 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/RARα-positive acute promyelocytic leukemia: PETHEMA group. Blood. 1999;94(9):3015-3021.
55. Lengfelder E, Reichert A, Schoch C, et al. Double induction strategy including high dose cytarabine in combination with all-trans retinoic acid: effects in patients with newly diagnosed acute promyelocytic leukemia: German AML Cooperative Group. Leukemia. 2000;14(8):1362-1370.
56. Hsu HC, Tsai WH, Chen PG, Hsu ML, Ho CK, Wang SY. In vitro effect of granulocyte-colony stimulating factor and all-trans retinoic acid on the expression of inflammatory cytokines and adhesion molecules in acute promyelocytic leukemic cells. Eur J Haematol. 1999;63(1):11-18.
57. Specchia G, Lo Coco F, Vignetti M, et al. Extramedullary involvement at relapse in acute promyelocytic leukemia patients treated or not with all-trans retinoic acid: a report by the Gruppo Italiano Malattie Ematologiche dell'Adulto. J Clin Oncol. 2001;19(20):4023-4028.
58. Lobe I, Rigal-Huguet F, Vekhoff A, et al. Myelodysplastic syndrome after acute promyelocytic leukemia: the European APL group experience. Leukemia. 2003;17(8):1600-1604.
59. Thomas X, Le QH, Fiere D. Anthracycline-related toxicity requiring cardiac transplantation in longterm disease-free survivors with acute promyelocytic leukemia. Ann Hematol. 2002;81(9):504-507.
60. Manna A, Cadenotti L, Motto A, Ballo P. Reversible cardiac dysfunction without myocytolysis related to all-trans retinoic acid administration during induction therapy of acute promyelocytic leukemia. Ann Hematol. 2009;88(1):91-92.
61. Hu J, Liu YF, Wu CF, et al. Long-term efficacy and safety of all-trans retinoic acid/arsenic trioxidebased therapy in newly diagnosed acute promyelocytic leukemia. Proc Natl Acad Sci U S A. 2009;106(9):3342-3347.
62. 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(4):504-510.
63. 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(7):2627-2632.
64. 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(1):131-134.
65. 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):1-8.
66. George B, Mathews V, Poonkuzhali B, Shaji RV, Srivastava A, Chandy M. Treatment of children with newly diagnosed acute promyelocytic leukemia with arsenic trioxide: a single center experience. Leukemia. 2004;18(10):1587-1590.
67. Mathews V, Thomas M, Srivastava VM, George B, Srivastava A, Chandy M. 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(7):994-995.
68. Powell BL. Effect of consolidation with arsenic trioxide (As2O3) on event-free survival (EFS) and overall survival (OS) among patients with newly diagnosed acute promyelocytic leukemia (APL): North American Intergroup Protocol C9710. Proc Am Soc Clin Oncol. 2007;25:1S.
69. Avvisati G, Petti M, Lo Coco F, Testi A, Fazi P. AIDA: the Italian way of treating acute promyelocytic leukemia (APL), final act. Blood. 2003; 102(11):142a.
70. Detourmignies L, Castaigne S, Stoppa AM, et al. Therapy-related acute promyelocytic leukemia: a report on 16 cases. J Clin Oncol. 1992;10(9): 1430-1435.
71. Pollicardo N, O'Brien S, Estey EH, et al. Secondary acute promyelocytic leukemia: characteristics and prognosis of 14 patients from a single institution. Leukemia. 1996;10(1):27-31.
72. Latagliata R, Petti MC, Fenu S, et al. Therapyrelated myelodysplastic syndrome-acute myelogenous leukemia in patients treated for acute promyelocytic leukemia: an emerging problem. Blood. 2002;99(3):822-824.
73. Pulsoni A, Pagano L, Lo Coco F, et al. Clinicobiological features and outcome of acute promyelocytic leukemia occurring as a second tumor: the GIMEMA experience. Blood. 2002;100(6):1972-1976.
74. Gale RE, Hills R, Pizzey AR, et al. Relationship between FLT3 mutation status, biologic characteristics, and response to targeted therapy in acute promyelocytic leukemia. Blood. 2005; 106(12):3768-3776.
75. Grimwade D, Lo Coco F. Acute promyelocytic leukemia: a model for the role of molecular diagnosis and residual disease monitoring in directing treatment approach in acute myeloid leukemia. Leukemia. 2002;16(10):1959-1973.
76. Kelaidi C, Ades L, Chevret S, et al. Late first relapses in APL treated with all-trans-retinoic acidand anthracycline-based chemotherapy: the European APL group experience (APL 91 and APL 93 trials). Leukemia. 2006;20(5):905-907.
77. 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(10):3315-3324.
78. 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(18):3852-3860.
79. Ohnishi K, Yoshida H, Shigeno K, et al. Arsenic trioxide therapy for relapsed or refractory Japanese patients with acute promyelocytic leukemia: need for careful electrocardiogram monitoring. Leukemia. 2002;16(4):617-622.
80. 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(12):2326-2334.
81. Douer D, Ramezani L, Watkins K, Louie R, Tallman MS. Durable molecular remission in two acute promyelocytic leukemia (APL) patients treated with arsenic trioxide at first molecular relapse. Blood. 2005;106:1847a.
82. 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(7):1995-1999.
83. Breccia M, Cimino G, Diverio D, Gentilini F, Mandelli F, Lo Coco F. Sustained molecular remission after low dose gemtuzumab-ozogamicin in elderly patients with advanced acute promyelocytic leukemia. Haematologica. 2007;92(9):1273-1274.
84. Estey EH, Giles FJ, Beran M, et al. Experience with gemtuzumab ozogamycin ("mylotarg") and all-trans retinoic acid in untreated acute promyelocytic leukemia. Blood. 2002;99(11):4222-4224.
85. Thomas X, Pigneux A, Raffoux E, Huguet F, Caillot D, Fenaux P. Superiority of an arsenic trioxidebased regimen over a historic control combining all-trans retinoic acid plus intensive chemotherapy in the treatment of relapsed acute promyelocytic leukemia. Haematologica. 2006;91(7): 996-997.
86. 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(9): 3587-3590.
87. Au WY, Tam S, Kwong YL. Entry of elemental arsenic into the central nervous system in patients with acute promyelocytic leukemia during arsenic trioxide treatment. Leuk Res. 2008;32(2):357-358.
88. Lu DP, Qiu JY, Jiang B, et al. Tetra-arsenic tetrasulfide for the treatment of acute promyelocytic leukemia: a pilot report. Blood. 2002;99(9):3136-3143.
89. Takeuchi M, Yano T, Omoto E, et al. Relapsed acute promyelocytic leukemia previously treated with all-trans retinoic acid: clinical experience with a new synthetic retinoid, Am-80. Leuk Lymphoma. 1998;31(5):441-451.