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Acute promyelocytic leukemia (APL): remaining challenges towards a cure for all

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Pages 3107-3115 | Received 15 Mar 2019, Accepted 24 Apr 2019, Published online: 16 Dec 2019

References

  • Grignani F, Ferrucci PF, Testa U, et al. The acute promyelocytic leukemia-specific PML-RAR alpha fusion protein inhibits differentiation and promotes survival of myeloid precursor cells. Cell. 1993;74:423–431.
  • Watts JM, Tallman MS. Acute promyelocytic leukemia: what is the new standard of care? Blood Rev. 2014;28:205–212.
  • 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;120:1570–1580.
  • Lo-Coco F, Avvisati G, Vignetti M, et al. Retinoic acid and arsenic trioxide for acute promyelocytic leukemia. N Engl J Med. 2013;369:111–121.
  • Tallman MS, Lo-Coco F, Kwaan HC, et al. Early death in patients with acute promyelocytic leukemia. Proceedings from a live roundtable at the 2010 American Society of Hematology Annual Meeting, December 4–7, 2010, Orlando, Florida. Clin Adv Hematol Oncol. 2011;9:1–16.
  • 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;108:689–695.
  • 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;111:3395–3402.
  • Tallman MS, Andersen JW, Schiffer CA, et al. All-trans-retinoic acid in acute promyelocytic leukemia. N Engl J Med. 1997;337:1021–1028.
  • 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;94:1192–1200.
  • 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/RARalpha-positive acute promyelocytic leukemia. PETHEMA group. Blood. 1999;94:3015–3021.
  • Asou N, Adachi K, Tamura J, et al. Analysis of prognostic factors in newly diagnosed acute promyelocytic leukemia treated with all-trans retinoic acid and chemotherapy. Japan Adult Leukemia Study Group. Jco. 1998;16:78–85.
  • Sanz MA, Montesinos P, Vellenga E, et al. Risk-adapted treatment of acute promyelocytic leukemia with all-trans retinoic acid and anthracycline monochemotherapy: long-term outcome of the LPA 99 multicenter study by the PETHEMA Group. Blood. 2008;112:3130–3134.
  • 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-RARalpha transcript after consolidation therapy: the Japan Adult Leukemia Study Group (JALSG) APL97 study. Blood. 2007;110:59–66.
  • Lengfelder E, Haferlach C, Saussele S, et al. High dose ara-C in the treatment of newly diagnosed acute promyelocytic leukemia: long-term results of the German AMLCG. Leukemia. 2009;23:2248–2258.
  • Avvisati G, Lo-Coco F, Paoloni FP, et al. AIDA 0493 protocol for newly diagnosed acute promyelocytic leukemia: very long-term results and role of maintenance. Blood. 2011;117:4716–4725.
  • Burnett AK, Russell NH, Hills RK, et al. Arsenic trioxide and all-trans retinoic acid treatment for acute promyelocytic leukaemia in all risk groups (AML17): results of a randomised, controlled, phase 3 trial. Lancet Oncol. 2015;16:1295–1305.
  • Jeddi R, Kacem K, Ben Neji H, et al. Predictive factors of all-trans-retinoic acid related complications during induction therapy for acute promyelocytic leukemia. Hematology. 2008;13:142–146.
  • 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;25:1128–1134.
  • McClellan JS, Kohrt HE, Coutre S, et al. Treatment advances have not improved the early death rate in acute promyelocytic leukemia. Haematologica. 2012;97:133–136.
  • Park JH, Qiao B, Panageas KS, et al. Early death rate in acute promyelocytic leukemia remains high despite all-trans retinoic acid. Blood. 2011;118:1248–1254.
  • 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;37:1004–1009.
  • Smith Giri KM, Vijaya Raj B. Overall survival (OS) of acute promyelocytic leukemia (APL) treated in academic (AC) versus non academic (NAC) centers. American Society of Clinical Oncology Meeting 2016, Abstract e18142; 2017.
  • Anand Jillella MA. Asad Bashey et al Decreasing Early Deaths in Acute Promyelocytic Leukemia (APL) By Using a Simplified Treatment Algorithm and Establishing a Network with Academic and Community Centers in USA. Presented at the American Society of Hematology Meeting. 2015.
  • Choudhry A, DeLoughery TG. Bleeding and thrombosis in acute promyelocytic leukemia. Am J Hematol. 2012;87:596–603.
  • Chang H, Kuo MC, Shih LY, et al. Clinical bleeding events and laboratory coagulation profiles in acute promyelocytic leukemia. Eur J Haematol. 2012;88:321–328.
  • Falanga A, Alessio MG, Donati MB, et al. A new procoagulant in acute leukemia. Blood. 1988;71:870–875.
  • Koyama T, Hirosawa S, Kawamata N, et al. All-trans retinoic acid upregulates thrombomodulin and downregulates tissue-factor expression in acute promyelocytic leukemia cells: distinct expression of thrombomodulin and tissue factor in human leukemic cells. Blood. 1994;84:3001.
  • Falanga A, Iacoviello L, Evangelista V, et al. Loss of blast cell procoagulant activity and improvement of hemostatic variables in patients with acute promyelocytic leukemia administered all-trans-retinoic acid. Blood. 1995;86:1072–1081.
  • Bennett B, Booth NA, Croll A, et al. The bleeding disorder in acute promyelocytic leukaemia: fibrinolysis due to u-PA rather than defibrination. Br J Haematol. 1989;71:511–517.
  • Sakata Y, Murakami T, Noro A, et al. The specific activity of plasminogen activator inhibitor-1 in disseminated intravascular coagulation with acute promyelocytic leukemia. Blood. 1991;77:1949–1957.
  • Dombret H, Scrobohaci ML, Daniel MT, et al. In vivo thrombin and plasmin activities in patients with acute promyelocytic leukemia (APL): effect of all-trans retinoic acid (ATRA) therapy. Leukemia. 1995;9:19–24.
  • Stein E, McMahon B, Kwaan H, et al. The coagulopathy of acute promyelocytic leukaemia revisited. Best Pract Res Clin Haematol. 2009;22:153–163.
  • Lavallee VP, Chagraoui J, MacRae T, et al. Transcriptomic landscape of acute promyelocytic leukemia reveals aberrant surface expression of the platelet aggregation agonist Podoplanin. Leukemia. 2018;32:1349–1357.
  • Mantha S, Goldman DA, Devlin SM, et al. Determinants of fatal bleeding during induction therapy for acute promyelocytic leukemia in the ATRA era. Blood. 2017;129:1763–1767.
  • Breccia M, Avvisati G, Latagliata R, et al. Occurrence of thrombotic events in acute promyelocytic leukemia correlates with consistent immunophenotypic and molecular features. Leukemia. 2007;21:79–83.
  • Montesinos P, de la Serna J, Vellenga E, et al. Incidence and risk factors for thrombosis in patients with acute promyelocytic leukemia. experience of the PETHEMA LPA96 and LPA99 protocols. Blood. 2006;108:1503.
  • 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;113:775–783.
  • Sanz MA, Grimwade D, Tallman MS, et al. Management of acute promyelocytic leukemia: recommendations from an expert panel on behalf of the European Leukemia Net. Blood. 2009;113:1875–1891.
  • Sanz MA, Montesinos P. How we prevent and treat differentiation syndrome in patients with acute promyelocytic leukemia. Blood. 2014;123:2777–2782.
  • Gralnick HR, Bagley J, Abrell E. Heparin treatment for the hemorrhagic diathesis of acute promyelocytic leukemia. Am J Med. 1972;52:167–174.
  • Hoyle CF, Swirsky DM, Freedman L, et al. Beneficial effect of heparin in the management of patients with APL. Br J Haematol. 1988;68:283–289.
  • Avvisati G, ten Cate JW, Buller HR, et al. Tranexamic acid for control of haemorrhage in acute promyelocytic leukaemia. Lancet. 1989;2:122–124.
  • Keane TJ, Gorman AM, O’Connell LG, et al. epsilon-Amino-caproic acid in the management of acute promyelocytic leukaemia. Acta Haematol. 1976;56:202–204.
  • Rodeghiero F, Avvisati G, Castaman G, et al. Early deaths and anti-hemorrhagic treatments in acute promyelocytic leukemia. A GIMEMA retrospective study in 268 consecutive patients. Blood. 1990;75:2112–2117.
  • Pemmaraju N, Sasaki K, Johnson D, et al. Successful treatment of intracranial hemorrhage with recombinant activated factor VII in a patient with newly diagnosed acute myeloid leukemia: a case report and review of the literature. Front Oncol. 2015;5:29.
  • Nosari A, Caimi TM, Zilioli V, et al. Cerebral hemorrhage treated with NovoSeven in acute promyelocytic leukemia. Leuk Lymphoma. 2012;53:160.
  • Zver S, Andoljsek D, Cernelc P. Effective treatment of life-threatening bleeding with recombinant activated factor VII in a patient with acute promyelocytic leukaemia. Eur J Haematol. 2004;72:455–456.
  • Platzbecker U, Avvisati G, Cicconi L, et al. 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. JCO. 2017;35:605–612.
  • Zhu HH, Huang XJ. Oral arsenic and retinoic acid for non-high-risk acute promyelocytic leukemia. N Engl J Med. 2014;371:2239–2241.
  • O'Donnell MR, Tallman MS, Abboud CN, et al. Acute Myeloid Leukemia, Version 3.2017, NCCN Clinical Practice Guidelines in Oncology. J Natl Compr Canc Netw. 2017.
  • 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;103:1237–1243.
  • 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;115:5137–5146.
  • Iland HJ, Collins M, Bradstock K, et al. Use of arsenic trioxide in remission induction and consolidation therapy for acute promyelocytic leukaemia in the Australasian Leukaemia and Lymphoma Group (ALLG) APML4 study: a non-randomised phase 2 trial. Lancet Haematol. 2015;2:e357–e366.
  • 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;107:3469–3473.
  • Zhu HH, Liu YR, Jia JS, et al. Oral arsenic and all-trans retinoic acid for high-risk acute promyelocytic leukemia. Blood. 2018;131:2987–2989.
  • 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;14:752–757.
  • 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;21:2326–2334.
  • 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;82:224–229.
  • Ding W, Li YP, Nobile LM, et al. Leukemic cellular retinoic acid resistance and missense mutations in the PML-RARalpha fusion gene after relapse of acute promyelocytic leukemia from treatment with all-trans retinoic acid and intensive chemotherapy. Blood. 1998;92:1172–1183.
  • Fasan A, Haferlach C, Perglerova K, et al. Molecular landscape of acute promyelocytic leukemia at diagnosis and relapse. Haematologica. 2017;102:e222–e224.
  • 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;15:1479–1484.
  • Meloni G, Diverio D, Vignetti M, et al. Autologous bone marrow transplantation for acute promyelocytic leukemia in second remission: prognostic relevance of pretransplant minimal residual disease assessment by reverse-transcription polymerase chain reaction of the PML/RAR alpha fusion gene. Blood. 1997;90:1321–1325.
  • Ramadan SM, Di Veroli A, Camboni A, et al. Allogeneic stem cell transplantation for advanced acute promyelocytic leukemia in the ATRA and ATO era. Haematologica. 2012;97:1731–1735.
  • Petti MC, Pinazzi MB, Diverio D, et al. Prolonged molecular remission in advanced acute promyelocytic leukaemia after treatment with gemtuzumab ozogamicin (Mylotarg CMA-676). Br J Haematol. 2001;115:63–65.
  • Akihiro Takeshita, Takaaki Ono, Yumi Kojima, et al. Efficacy of Gemtuzumab Ozogamicin (GO) Monotherapy on Relapsed/Refractory Acute Promyelocytic Leukemia (APL). Presented at American Society of Hematology (ASH) Annual meeting 2011; 2011.

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