New and Emerging Targeted Therapies for Pediatric Acute Myeloid Leukemia (AML)

被引：26

作者：

Chen, Jing ^{[1
]}

Glasser, Chana L. ^{[2
]}

机构：

[1] Hackensack Univ Med Ctr, Div Pediat Hematol Oncol, Hackensack, NJ 07601 USA

[2] NYU Winthrop Hosp, Div Pediat Hematol Oncol, Mineola, NY 11501 USA

来源：

CHILDREN-BASEL | 2020年 / 7卷 / 02期

关键词：

acute myeloid leukemia; antibody drug conjugate; ANTIBODY-DRUG CONJUGATE; OPEN-LABEL; GEMTUZUMAB OZOGAMICIN; MOLECULAR LANDSCAPE; MDM2; ANTAGONISTS; INHIBITION; CHEMOTHERAPY; MULTICENTER; ACTIVATION; YOUNGER;

D O I：

10.3390/children7020012

中图分类号：

R72 [儿科学];

学科分类号：

100202 ;

摘要：

The relapse rate for children with acute myeloid leukemia (AML) remains high despite advancements in risk classification, multi-agent chemotherapy intensification, stem cell transplantation, and supportive care guidelines. Prognosis for this subgroup of children with relapsed/refractory AML remains poor. It is well known that the ceiling of chemotherapy intensification has been reached, limited by acute and chronic toxicity, necessitating alternative treatment approaches. In the last several years, our improved understanding of disease biology and critical molecular pathways in AML has yielded a variety of new drugs to target these specific pathways. This review provides a summary of antibody drug conjugates (ADCs), small molecule inhibitors, and tyrosine kinase inhibitors with an emphasis on those that are currently under clinical evaluation or soon to open in early phase trials for children with relapsed/refractory AML.

引用

页数：15

共 91 条

[1] Small molecule inhibitors in acute myeloid leukemia: from the bench to the clinic [J].

Al-Hussaini, Muneera ;

DiPersio, John F. .

EXPERT REVIEW OF HEMATOLOGY, 2014, 7 (04) :439-464

[2]

[Anonymous], BLOOD S1, DOI DOI 10.1182/BLOOD-2018-99-118966

[3] The role of TP53 in acute myeloid leukemia: Challenges and opportunities [J].

Barbosa, Karina ;

Li, Sha ;

Adams, Peter D. ;

Deshpande, Aniruddha J. .

GENES CHROMOSOMES & CANCER, 2019, 58 (12) :875-888

[4]

BASSET J, 2018, XENOPHORA JAN, P14

[5] MLL-Rearranged Leukemia Is Dependent on Aberrant H3K79 Methylation by DOT1L [J].

Bernt, Kathrin M. ;

Zhu, Nan ;

Sinha, Amit U. ;

Vempati, Sridhar ;

Faber, Joerg ;

Krivtsov, Andrei V. ;

Feng, Zhaohui ;

Punt, Natalie ;

Daigle, Amanda ;

Bullinger, Lars ;

Pollock, Roy M. ;

Richon, Victoria M. ;

Kung, Andrew L. ;

Armstrong, Scott A. .

CANCER CELL, 2011, 20 (01) :66-78

[6] New Targeted Agents in Acute Myeloid Leukemia: New Hope on the Rise [J].

Bohl, Stephan R. ;

Bullinger, Lars ;

Ruecker, Frank G. .

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES, 2019, 20 (08)

[7] The molecular landscape of pediatric acute myeloid leukemia reveals recurrent structural alterations and age-specific mutational interactions (vol 24, pg 103, 2017) [J].

Bolouri, Hamid ;

Farrar, Jason E. ;

Triche, Timothy, Jr. ;

Ries, Rhonda E. ;

Lim, Emilia L. ;

Alonzo, Todd A. ;

Ma, Yussanne ;

Moore, Richard ;

Mungall, Andrew J. ;

Marra, Marco A. ;

Zhang, Jinghui ;

Ma, Xiaotu ;

Liu, Yu ;

Liu, Yanling ;

Auvil, Jaime M. Guidry ;

Davidsen, Tanja M. ;

Gesuwan, Patee ;

Hermida, Leandro C. ;

Salhia, Bodour ;

Capone, Stephen ;

Ramsingh, Giridharan ;

Zwaan, Christian Michel ;

Noort, Sanne ;

Piccolo, Stephen R. ;

Kolb, E. Anders ;

Gamis, Alan S. ;

Smith, Malcolm A. ;

Gerhard, Daniela S. ;

Meshinchi, Soheil .

NATURE MEDICINE, 2018, 24 (01) :103-+

[8] Pharmacologic Inhibition of the Menin-MLL Interaction Blocks Progression of MLL Leukemia In Vivo [J].

Borkin, Dmitry ;

He, Shihan ;

Miao, Hongzhi ;

Kempinska, Katarzyna ;

Pollock, Jonathan ;

Chase, Jennifer ;

Purohit, Trupta ;

Malik, Bhavna ;

Zhao, Ting ;

Wang, Jingya ;

Wen, Bo ;

Zong, Hongliang ;

Jones, Morgan ;

Danet-Desnoyers, Gwenn ;

Guzman, Monica L. ;

Talpaz, Moshe ;

Bixby, Dale L. ;

Sun, Duxin ;

Hess, Jay L. ;

Muntean, Andrew G. ;

Maillard, Ivan ;

Cierpicki, Tomasz ;

Grembecka, Jolanta .

CANCER CELL, 2015, 27 (04) :589-602

[9] Activity of the oral mitogen-activated protein kinase kinase inhibitor trametinib in RAS-mutant relapsed or refractory myeloid malignancies [J].

Borthakur, Gautam ;

Popplewell, Leslie ;

Boyiadzis, Michael ;

Foran, James ;

Platzbecker, Uwe ;

Vey, Norbert ;

Walter, Roland B. ;

Olin, Rebecca ;

Raza, Azra ;

Giagounidis, Aristoteles ;

Al-Kali, Aref ;

Jabbour, Elias ;

Kadia, Tapan ;

Garcia-Manero, Guillermo ;

Bauman, John W. ;

Wu, Yuehui ;

Liu, Yuan ;

Schramek, Dan ;

Cox, Donna S. ;

Wissel, Paul ;

Kantarjian, Hagop .

CANCER, 2016, 122 (12) :1871-1879

[10] TP53 Variations in Human Cancers: New Lessons from the IARC TP53 Database and Genomics Data [J].

Bouaoun, Liacine ;

Sonkin, Dmitriy ;

Ardin, Maude ;

Hollstein, Monica ;

Byrnes, Graham ;

Zavadil, Jiri ;

Olivier, Magali .

HUMAN MUTATION, 2016, 37 (09) :865-876

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