Acute promyelocytic leukemia historical perspective

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Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1] Shyam Patel [2]

Overview

Historical perspective

  • In 1882, A.C. Doyle reported on the efficacy of arsenic in acute promyelocytic leukemia.[1]
  • In 1957, Leif Hillestad described acute promyelocytic leukemia as a distinct clinical condition for the first time. He noted the syndrome of low fibrinogen levels, fibrin degradation, and life-threatening hemorrhage.[2] It was noted that this condition carried a high mortality rate due to severe hemorrhage.[3]
  • In 1973, Bernard and colleagues showed in clinical studies that therapeutic efficacy of daunorubicin, an chemotherapy agent of the anthracycline class. This medication was shown to induce remission in the majority of patients, with an increase in remission rate from 13% to 58%. The median duration of remission with daunorubicin was more than 2 years. Compared to 6-mercaptopurine, daunorubicin was shown to reduce mortality from bleeding after 5 days.[3]
  • In 1973, scientists in Harbin showed that arsenic trioxide and mercury chloride could treat acute and chronic leukemia.
  • In the early 1990s, it was noted that arsenic trioxide could induce remission in a high proportion of patients.[3][4]
  • In 1997, scientists in Harbin and Shanghai showed that single-agent arsenic trioxide could induce response rates of 90% in patients who relapsed after receiving all-trans retinoic acid and chemotherapy.[2]
  • In 1980, Breitman and colleagues showed that all-trans retinoic acid could lead to the differentiation of the HL-60 cell line of myeloid leukemia.[2] This was the first demonstration that differentiation therapy could treat leukemia. This concept was based on the fact that most cancers are primitive and stem-like, which leads to aggressive and chemo-resistant cellular behavior. Blockade of stemness and induction of differentiation could lead to anti-cancer effect.
  • In 1988, Huang and colleagues showed that all-trans retinoic acid could successfully treat acute promyelocytic leukemia in 24 patients. The complete remission rate was more tan 90%.[2]
  • In 1995, D. Head and colleagues showed that higher remission rates and higher survival rates could be achieved with higher doses of daunorubicin, with a survival rate of 61% after 9 years and a 0% relapse after 36 months.[5]
  • In 2001, Soignet and colleagues showed that a single, 5-week course of arsenic trioxide carried a complete remission rate of 50%. It was shown that 2 cycles of arsenic trioxide could induce remission in 86% of patients.[6]
  • In 2013, LoCoco and colleagues showed that, in a randomized phase 3 multicenter clinical trial, a non-chemotherapy-based regimen was superior to a chemotherapy-based regimen for low-risk acute promyelocytic leukemia.[7] Specifically, the combination of all-trans retinoic acid and arsenic trioxide resulted in improved overall survival compared to the combination of all-trans retinoic acid plus chemotherapy. This landmark clinical trial, which was conducted by the Italian and German-Austrian Leukemia Study Groups, altered the treatment paradigm for low-risk acute promyelocytic leukemia.[7]

References

  1. Falchi L, Verstovsek S, Ravandi-Kashani F, Kantarjian HM (2016). "The evolution of arsenic in the treatment of acute promyelocytic leukemia and other myeloid neoplasms: Moving toward an effective oral, outpatient therapy". Cancer. 122 (8): 1160–8. doi:10.1002/cncr.29852. PMC 5042140. PMID 26716387.
  2. 2.0 2.1 2.2 2.3 McCulloch D, Brown C, Iland H (2017). "Retinoic acid and arsenic trioxide in the treatment of acute promyelocytic leukemia: current perspectives". Onco Targets Ther. 10: 1585–1601. doi:10.2147/OTT.S100513. PMC 5359123. PMID 28352191.
  3. 3.0 3.1 3.2 Coombs CC, Tavakkoli M, Tallman MS (2015). "Acute promyelocytic leukemia: where did we start, where are we now, and the future". Blood Cancer J. 5: e304. doi:10.1038/bcj.2015.25. PMC 4450325. PMID 25885425.
  4. Park J, Jurcic JG, Rosenblat T, Tallman MS (2011). "Emerging new approaches for the treatment of acute promyelocytic leukemia". Ther Adv Hematol. 2 (5): 335–52. doi:10.1177/2040620711410773. PMC 3573416. PMID 23556100.
  5. Head D, Kopecky KJ, Weick J, Files JC, Ryan D, Foucar K; et al. (1995). "Effect of aggressive daunomycin therapy on survival in acute promyelocytic leukemia". Blood. 86 (5): 1717–28. PMID 7655004.
  6. Soignet SL, Frankel SR, Douer D, Tallman MS, Kantarjian H, Calleja E; et al. (2001). "United States multicenter study of arsenic trioxide in relapsed acute promyelocytic leukemia". J Clin Oncol. 19 (18): 3852–60. doi:10.1200/JCO.2001.19.18.3852. PMID 11559723.
  7. 7.0 7.1 Lo-Coco F, Avvisati G, Vignetti M, Thiede C, Orlando SM, Iacobelli S; et al. (2013). "Retinoic acid and arsenic trioxide for acute promyelocytic leukemia". N Engl J Med. 369 (2): 111–21. doi:10.1056/NEJMoa1300874. PMID 23841729.

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