Are we witnessing the start of a therapeutic revolution in acute myeloid leukemia?

被引：26

作者：

Bewersdorf, Jan Philipp ^{[1
]}

Stahl, Maximilian ^{[2
]}

Zeidan, Amer M. ^{[1
]}

机构：

[1] Yale Univ, Sch Med, Dept Internal Med, Sect Hematol, New Haven, CT 06510 USA

[2] Mem Sloan Kettering Canc Ctr, Dept Med, Div Hematol Malignancies, 1275 York Ave, New York, NY 10021 USA

来源：

LEUKEMIA & LYMPHOMA | 2019年 / 60卷 / 06期

关键词：

Acute myelogenous leukemia; novel therapies; future directions; targeted therapy; FLT3; inhibitor; epigenetic therapy; INTERNAL TANDEM DUPLICATION; HISTONE DEACETYLASE INHIBITOR; CONVENTIONAL CARE REGIMENS; LOW-DOSE CYTARABINE; GEMTUZUMAB OZOGAMICIN; MYELODYSPLASTIC SYNDROMES; OPEN-LABEL; PHASE-I; ADULT PATIENTS; KINASE INHIBITOR;

D O I：

10.1080/10428194.2018.1546854

中图分类号：

R73 [肿瘤学];

学科分类号：

100214 ;

摘要：

The 5-year overall survival rate of AML patients remains 25-40%. The prognosis is even more dismal for older patients who are ineligible for intensive chemotherapy and patients with secondary or relapsed/refractory AML. In 2017, 4 new drugs were approved by the US Food and Drug Administration for AML treatment: The FLT3 inhibitor midostaurin, the isocitrate dehydrogenase (IDH)-2 inhibitor enasidenib, a liposomal formulation of cytarabine and daunorubicin (CPX-351), and the anti-CD33 antibody gemtuzumab ozogamicin. Additionally, the IDH1 inhibitor ivosidenib has received FDA approval in July 2018. However, all these drugs were approved in certain settings and/or for certain subsets of AML patients. Herein, we review the mechanisms of actions and preclinical data, highlight pivotal clinical trial data, and discuss future directions and challenges for further development of these 5 novel therapeutics. Finally, we briefly overview some of the highly promising agents that are currently in advanced stages of clinical development.

引用

页码：1354 / 1369

页数：16

共 144 条

[1] Phase 1 dose escalation multicenter trial of pracinostat alone and in combination with azacitidine in patients with advanced hematologic malignancies [J].

Abaza, Yasmin M. ;

Kadia, Tapan M. ;

Jabbour, Elias J. ;

Konopleva, Marina Y. ;

Borthakur, Gautam ;

Ferrajoli, Alessandra ;

Estrov, Zeev ;

Wierda, William G. ;

Alfonso, Ana ;

Chong, Toh Han ;

Chuah, Charles ;

Koh, Liang-Piu ;

Goh, Boon-Cher ;

Chang, Julie E. ;

Durkes, Daniel E. ;

Foudray, Maria Cielo ;

Kantarjian, Hagop M. ;

Dong, Xiao Qin ;

Garcia-Manero, Guillermo .

CANCER, 2017, 123 (24) :4851-4859

[2] The Combination of Quizartinib with Azacitidine or Low Dose Cytarabine Is Highly Active in Patients (Pts) with FLT3-ITD Mutated Myeloid Leukemias: Interim Report of a Phase I/II Trial [J].

Abdelall, Waleed ;

Kantarjian, Hagop M. ;

Borthakur, Gautam ;

Garcia-Manero, Guillermo ;

Patel, Keyur P. ;

Jabbour, Elias J. ;

Daver, Naval G. ;

Kadia, Tapan ;

Gborogen, Rosilyn A. ;

Konopleva, Marina ;

Ravandi, Farhad ;

Andreef, Michael ;

Cortes, Jorge E. .

BLOOD, 2016, 128 (22)

[3] Prediction of acute myeloid leukaemia risk in healthy individuals [J].

Abelson, Sagi ;

Collord, Grace ;

Ng, Stanley W. K. ;

Weissbrod, Omer ;

Cohen, Netta Mendelson ;

Niemeyer, Elisabeth ;

Barda, Noam ;

Zuzarte, Philip C. ;

Heisler, Lawrence ;

Sundaravadanam, Yogi ;

Luben, Robert ;

Hayat, Shabina ;

Wang, Ting Ting ;

Zhao, Zhen ;

Cirlan, Julia ;

Pugh, Trevor J. ;

Soave, David ;

Ng, Karen ;

Latimer, Calli ;

Hardy, Claire ;

Raine, Keiran ;

Jones, David ;

Hoult, Diana ;

Britten, Abigail ;

McPherson, John D. ;

Johansson, Mattias ;

Mbabaali, Faridah ;

Eagles, Jenna ;

Millers, Jessica K. ;

Pasternack, Danielle ;

Timms, Lee ;

Krzyzanowski, Paul ;

Awadalla, Philip ;

Costa, Rui ;

Segal, Eran ;

Bratman, Scott, V ;

Beer, Philip ;

Behjati, Sam ;

Martincorena, Inigo ;

Wang, Jean C. Y. ;

Bowles, Kristian M. ;

Ramon Quiros, J. ;

Karakatsani, Anna ;

La Vecchia, Carlo ;

Trichopoulou, Antonia ;

Salamanca-Fernandez, Elena ;

Huerta, Jose M. ;

Barricarte, Aurelio ;

Travis, Ruth C. ;

Tumino, Rosario .

NATURE, 2018, 559 (7714) :400-+

[4] Enhancing SHP-1 expression with 5-azacytidine may inhibit STAT3 activation and confer sensitivity in lestaurtinib (CEP-701)-resistant FLT3-ITD positive acute myeloid leukemia [J].

Al-Jamal, Hamid Ali Nagi ;

Jusoh, Siti Asmaa Mat ;

Hassan, Rosline ;

Johan, Muhammad Farid .

BMC CANCER, 2015, 15

[5] Phase 1 study of quizartinib in combination with induction and consolidation chemotherapy in patients with newly diagnosed acute myeloid leukemia [J].

Altman, Jessica K. ;

Foran, James M. ;

Pratz, Keith W. ;

Trone, Denise ;

Cortes, Jorge E. ;

Tallman, Martin S. .

AMERICAN JOURNAL OF HEMATOLOGY, 2018, 93 (02) :213-221

[6] Enasidenib induces acute myeloid leukemia cell differentiation to promote clinical response [J].

Amatangelo, Michael D. ;

Quek, Lynn ;

Shih, Alan ;

Stein, Eytan M. ;

Roshal, Mikhail ;

David, Muriel D. ;

Marteyn, Benoit ;

Farnoud, Noushin Rahnamay ;

de Botton, Stephane ;

Bernard, Olivier A. ;

Wu, Bin ;

Yen, Katharine E. ;

Tallman, Martin S. ;

Papaemmanuil, Elli ;

Penard-Lacronique, Virginie ;

Thakurta, Anjan ;

Vyas, Paresh ;

Levine, Ross L. .

BLOOD, 2017, 130 (06) :732-741

[7]

[Anonymous], BLOOD

[8]

[Anonymous], ONCOTARGET

[9]

[Anonymous], 2018 ASCO ANN M JUN

[10]

[Anonymous], BLOOD S1

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