New Food and Drug Administration–Approved and Emerging Novel Treatment Options for Acute Myeloid Leukemia

Diagnosis and management of AML in adults: 2017 ELN recommendations from an international expert panel

Döhner, H., Estey, E. H., Grimwade, D. J., Amadori, S., Appelbaum, F. R., Büchner, T., Dombret, H., Ebert, B. L., Fenaux, P., Larson, R. A., et al. (2016) · Blood · doi:10.1182/blood-2016-08-733196 · Cited by 5,780 · see citing works

Acute Myeloid Leukemia

Döhner, H., Weisdorf, D. J., & Bloomfield, C. D. (2015) · New England Journal of Medicine · doi:10.1056/nejmra1406184 · Cited by 3,354 · see citing works

The National Comprehensive Cancer Network (NCCN).

Wood, E. H. (2004) · Europe PMC (PubMed Central) · Cited by 2,843 · see citing works

Midostaurin plus Chemotherapy for Acute Myeloid Leukemia with a <i>FLT3</i> Mutation

Stone, R. M., Mandrekar, S. J., Sanford, B. L., Laumann, K., Geyer, S., Bloomfield, C. D., Thiede, C., Prior, T. W., Döhner, K., Marcucci, G., et al. (2017) · New England Journal of Medicine · doi:10.1056/nejmoa1614359 · Cited by 2,250 · see citing works

Prognostic Relevance of Integrated Genetic Profiling in Acute Myeloid Leukemia

Patel, J. P., Gönen, M., Figueroa, M. E., Fernandez, H. F., Sun, Z., Racevskis, J., Vlierberghe, P. V., Dolgalev, I., Thomas, S., Aminova, O., et al. (2012) · New England Journal of Medicine · doi:10.1056/nejmoa1112304 · Cited by 1,936 · see citing works

Enasidenib in mutant IDH2 relapsed or refractory acute myeloid leukemia

Stein, E. M., DiNardo, C. D., Pollyea, D. A., Fathi, A. T., Roboz, G. J., Altman, J. K., Stone, R. M., DeAngelo, D. J., Levine, R. L., Flinn, I. W., et al. (2017) · Blood · doi:10.1182/blood-2017-04-779405 · Cited by 1,516 · see citing works

Effect of gemtuzumab ozogamicin on survival of adult patients with de-novo acute myeloid leukaemia (ALFA-0701): a randomised, open-label, phase 3 study

Castaigné, S., Pautas, C., Terré, C., Raffoux, E., Bordessoule, D., Bastie, J.-N., Legrand, O., Thomas, X., Turlure, P., Reman, O., et al. (2012) · The Lancet · doi:10.1016/s0140-6736(12)60485-1 · Cited by 1,017 · see citing works

Efficacy and Safety of Gemtuzumab Ozogamicin in Patients With CD33-Positive Acute Myeloid Leukemia in First Relapse

Sievers, E. L., Larson, R. A., Stadtmauer, E. A., Estey, E. H., Löwenberg, B., Dombret, H., Karanes, C., Theobald, M., Bennett, J. M., Sherman, M. L., et al. (2001) · Journal of Clinical Oncology · doi:10.1200/jco.2001.19.13.3244 · Cited by 899 · see citing works

Prognostic significance of activating FLT3 mutations in younger adults (16 to 60 years) with acute myeloid leukemia and normal cytogenetics: a study of the AML Study Group Ulm

Fröhling, S., Schlenk, R. F., Breitruck, J., Benner, A., Kreitmeier, S., Tobis, K., Döhner, H., & Döhner, K. (2002) · Blood · doi:10.1182/blood-2002-05-1440 · Cited by 873 · see citing works

Targeted inhibition of mutant IDH2 in leukemia cells induces cellular differentiation

Wang, F., Travins, J., DeLaBarre, B., Penard-Lacronique, V., Schalm, S., Hansen, E., Straley, K., Kernytsky, A., Liu, W., Gliser, C., et al. (2013) · Science · doi:10.1126/science.1234769 · Cited by 804 · see citing works

Treatment of acute myelocytic leukemia: a study by cancer and leukemia group B

Rai, K., Holland, J., Glidewell, O. J., Weinberg, V. E., Brunner, K. W., Obrecht, J., Preisler, H., Nawabi, I., Prager, D., Carey, R., et al. (1981) · Blood · doi:10.1182/blood.v58.6.1203.bloodjournal5861203 · Cited by 532 · see citing works

Selective inhibition of FLT3 by gilteritinib in relapsed or refractory acute myeloid leukaemia: a multicentre, first-in-human, open-label, phase 1–2 study

Perl, A. E., Altman, J. K., Cortes, J. E., Smith, C. C., Litzow, M. R., Baer, M. R., Claxton, D. F., Erba, H. P., Gill, S., Goldberg, S. L., et al. (2017) · The Lancet Oncology · doi:10.1016/s1470-2045(17)30416-3 · Cited by 493 · see citing works

Gemtuzumab Ozogamicin in Children and Adolescents With De Novo Acute Myeloid Leukemia Improves Event-Free Survival by Reducing Relapse Risk: Results From the Randomized Phase III Children's Oncology Group Trial AAML0531

Gamis, A. S., Alonzo, T. A., Meshinchi, S., Sung, L., Gerbing, R. B., Raimondi, S. C., Hirsch, B. A., Kahwash, S. B., Heerema‐McKenney, A., Winter, L., et al. (2014) · Journal of Clinical Oncology · doi:10.1200/jco.2014.55.3628 · Cited by 486 · see citing works

Phase I Study of Quizartinib Administered Daily to Patients With Relapsed or Refractory Acute Myeloid Leukemia Irrespective of FMS-Like Tyrosine Kinase 3–Internal Tandem Duplication Status

Cortes, J. E., Kantarjian, H. M., Foran, J. M., Ghirdaladze, D., Zodelava, M., Borthakur, G. M., Gammon, G. M., Trone, D., Armstrong, R. C., James, J., et al. (2013) · Journal of Clinical Oncology · doi:10.1200/jco.2013.48.8783 · Cited by 363 · see citing works

Gemtuzumab Ozogamicin Versus Best Supportive Care in Older Patients With Newly Diagnosed Acute Myeloid Leukemia Unsuitable for Intensive Chemotherapy: Results of the Randomized Phase III EORTC-GIMEMA AML-19 Trial

Amadori, S., Suciu, S., Selleslag, D., Aversa, F., Gaïdano, G., Musso, M., Annino, L., Venditti, A., Voso, M. T., Mazzone, C., et al. (2016) · Journal of Clinical Oncology · doi:10.1200/jco.2015.64.0060 · Cited by 362 · see citing works

Phase 2 trial of CPX-351, a fixed 5:1 molar ratio of cytarabine/daunorubicin, vs cytarabine/daunorubicin in older adults with untreated AML

Lancet, J. E., Cortes, J. E., Hogge, D. E., Tallman, M. S., Kovacsovics, T. J., Damon, L. E., Komrokji, R. S., Solomon, S. R., Kolitz, J. E., Cooper, M. A., et al. (2014) · Blood · doi:10.1182/blood-2013-12-540971 · Cited by 339 · see citing works

Acute Myeloid Leukemia

O'Donnell, M. R., Abboud, C. N., Altman, J. K., Appelbaum, F. R., Arber, D. A., Attar, E. C., Borate, U., Coutre, S. E., Damon, L. E., Goorha, S., et al. (2012) · Journal of the National Comprehensive Cancer Network · doi:10.6004/jnccn.2012.0103 · Cited by 323 · see citing works

First-In-Man Study of CPX-351: A Liposomal Carrier Containing Cytarabine and Daunorubicin in a Fixed 5:1 Molar Ratio for the Treatment of Relapsed and Refractory Acute Myeloid Leukemia

Feldman, E. J., Lancet, J. E., Kolitz, J. E., Ritchie, E. K., Roboz, G. J., List, A. F., Allen, S. L., Asatiani, E., Mayer, L. D., Swenson, C. E., et al. (2011) · Journal of Clinical Oncology · doi:10.1200/jco.2010.30.5961 · Cited by 310 · see citing works

An overview on the role of FLT3-tyrosine kinase receptor in acute myeloid leukemia: biology and treatment

Grafone, T., Palmisano, M., Nicci, C., & Storti, S. (2012) · Oncology Reviews · doi:10.4081/oncol.2012.e8 · Cited by 279 · see citing works

Crenolanib is a potent inhibitor of FLT3 with activity against resistance-conferring point mutants

Galanis, A., Ma, H., Rajkhowa, T., Ramachandran, A., Small, D., Cortes, J. E., & Levis, M. J. (2013) · Blood · doi:10.1182/blood-2013-10-529313 · Cited by 258 · see citing works

Treatment of Relapsed/Refractory Acute Myeloid Leukemia

Bose, P., Vachhani, P. J., & Cortes, J. E. (2017) · Current Treatment Options in Oncology · doi:10.1007/s11864-017-0456-2 · Cited by 239 · see citing works

Preliminary Results of Southwest Oncology Group Study S0106: An International Intergroup Phase 3 Randomized Trial Comparing the Addition of Gemtuzumab Ozogamicin to Standard Induction Therapy Versus Standard Induction Therapy Followed by a Second Randomization to Post-Consolidation Gemtuzumab Ozogamicin Versus No Additional Therapy for Previously Untreated Acute Myeloid Leukemia.

Petersdorf, S. H., Kopecky, K. J., Stuart, R. K., Larson, R. A., Nevill, T. J., Stenke, L., Slovak, M. L., Tallman, M. S., Willman, C. L., Erba, H. P., et al. (2009) · Blood · doi:10.1182/blood.v114.22.790.790 · Cited by 184 · see citing works

Midostaurin, enasidenib, CPX-351, gemtuzumab ozogamicin, and venetoclax bring new hope to AML

Wei, A. H., & Tiong, I. S. (2017) · Blood · doi:10.1182/blood-2017-08-784066 · Cited by 127 · see citing works

Advances in targeted therapy for acute myeloid leukaemia

Käyser, S., & Levis, M. J. (2017) · British Journal of Haematology · doi:10.1111/bjh.15032 · Cited by 127 · see citing works

The importance of FLT3 mutational analysis in acute myeloid leukemia

Patnaik, M. M. (2017) · Leukemia & lymphoma/Leukemia and lymphoma · doi:10.1080/10428194.2017.1399312 · Cited by 97 · see citing works

New drugs in acute myeloid leukemia

Kadia, T. M., Ravandi, F., Cortes, J. E., & Kantarjian, H. M. (2016) · Annals of Oncology · doi:10.1093/annonc/mdw015 · Cited by 94 · see citing works

Evidence for Clinical Differentiation and Differentiation Syndrome in Patients With Acute Myeloid Leukemia and IDH1 Mutations Treated With the Targeted Mutant IDH1 Inhibitor, AG-120

Birendra, K. C., & Dinardo, C. D. (2016) · Clinical Lymphoma Myeloma & Leukemia · doi:10.1016/j.clml.2016.04.006 · Cited by 92 · see citing works

Investigation into CYP3A4-mediated drug–drug interactions on midostaurin in healthy volunteers

Dutreix, C., Munarini, F., Lorenzo, S., Roesel, J. L., & Wang, Y. (2013) · Cancer Chemotherapy and Pharmacology · doi:10.1007/s00280-013-2287-6 · Cited by 90 · see citing works

The role of FLT 3 inhibitors in the treatment of FLT 3‐mutated acute myeloid leukemia

Fathi, A. T., & Chen, Y. (2016) · European Journal Of Haematology · doi:10.1111/ejh.12841 · Cited by 77 · see citing works

Dose, schedule, safety, and efficacy of guadecitabine in relapsed or refractory acute myeloid leukemia

Roboz, G. J., Kantarjian, H. M., Yee, K. W. L., Kropf, P. L., O'Connell, C. L., Griffiths, E. A., Stock, W., Daver, N. G., Jabbour, E. J., Ritchie, E. K., et al. (2017) · Cancer · doi:10.1002/cncr.31138 · Cited by 66 · see citing works

Phase 1/2 Study of Venetoclax with Low-Dose Cytarabine in Treatment-Naive, Elderly Patients with Acute Myeloid Leukemia Unfit for Intensive Chemotherapy: 1-Year Outcomes

Wei, A. H., Strickland, S. A., Roboz, G. J., Hou, J., Fiedler, W., Lin, T. L., Walter, R. B., Enjeti, A. K., Chyla, B., Popovic, R., et al. (2017) · Blood · doi:10.1182/blood.v130.suppl_1.890.890 · Cited by 58 · see citing works

FLT3 Inhibitors for Treating Acute Myeloid Leukemia

Hassanein, M. F. R., Almahayni, M. H., Ahmed, S. O., Gaballa, S. R., & Fakih, R. O. E. (2016) · Clinical Lymphoma Myeloma & Leukemia · doi:10.1016/j.clml.2016.06.002 · Cited by 46 · see citing works

Low Relapse Rate in Younger Patients ≤ 60 Years Old with Newly Diagnosed FLT3-Mutated Acute Myeloid Leukemia (AML) Treated with Crenolanib and Cytarabine/Anthracycline Chemotherapy

Wang, E. S., Tallman, M. S., Stone, R. M., Walter, R. B., Karanes, C., Jain, V. K., & Collins, R. H. (2017) · Blood · doi:10.1182/blood.v130.suppl_1.566.566 · Cited by 38 · see citing works

A pharmacogenomic approach validates AG-221 as an effective and on-target therapy in IDH2 mutant AML

Kats, L. M., Vervoort, S. J., Cole, R., Rogers, A. J., Gregory, G. P., Vidacs, E., Li, J., Nagaraja, R., Yen, K. E., & Johnstone, R. W. (2017) · Leukemia · doi:10.1038/leu.2017.84 · Cited by 31 · see citing works

A Mechanism-Based Population Pharmacokinetic Model for Characterizing Time-Dependent Pharmacokinetics of Midostaurin and its Metabolites in Human Subjects

Yin, O. Q. P., Wang, Y., & SCHRAN, H. F. (2008) · Clinical Pharmacokinetics · doi:10.2165/0003088-200847120-00005 · Cited by 29 · see citing works

AG-221, a Small Molecule Mutant IDH2 Inhibitor, Remodels the Epigenetic State of IDH2-Mutant Cells and Induces Alterations in Self-Renewal/Differentiation in IDH2-Mutant AML Model in Vivo

Shih, A. H., SHANK, K. R., Meydan, C., Intlekofer, A. M., Ward, P. S., Thompson, C. B., Melnick, A. M., Travins, J. M., Straley, K., Gliser, C., et al. (2014) · Blood · doi:10.1182/blood.v124.21.437.437 · Cited by 28 · see citing works

Ivosidenib (AG-120) in Mutant IDH1 AML and Advanced Hematologic Malignancies: Results of a Phase 1 Dose Escalation and Expansion Study

Dinardo, C. D., de Botton, S., Stein, E. M., Roboz, G. J., Mims, A. S., Pollyea, D. A., Swords, R. T., Altman, J. K., Collins, R. H., Mannis, G. N., et al. (2017) · Blood · doi:10.1182/blood.v130.suppl_1.725.725 · Cited by 24 · see citing works

In Vitro Screening of Six Protein Kinase Inhibitors for Time‐Dependent Inhibition of CYP2C8 and CYP3A4: Possible Implications with regard to Drug–Drug Interactions

Filppula, A. M., Mustonen, T. M., & Backman, J. T. (2018) · Basic & Clinical Pharmacology & Toxicology · doi:10.1111/bcpt.13088 · Cited by 21 · see citing works

Phase 2 Study of Combination of Cytarabine, Idarubicin, and Nivolumab for Initial Therapy of Patients with Newly Diagnosed Acute Myeloid Leukemia

Ravandi, F., Daver, N. G., Garcia‐Manero, G., Benton, C. B., Thompson, P. A., Borthakur, G. M., Kadia, T. M., Boddu, P. C., Alvarado, Y., Jabbour, E. J., et al. (2017) · Blood · doi:10.1182/blood.v130.suppl_1.815.815 · Cited by 16 · see citing works

Differentiation syndrome associated with enasidenib, a selective inhibitor of mutant isocitrate dehydrogenase 2 (mIDH2).

Fathi, A. T., Dinardo, C. D., Kline, I., Kenvin, L., Gupta, I. V., Attar, E. C., Stein, E. M., de Botton, S., & On Behalf of the AG-221-C-001 Study Investigators (2017) · Journal of Clinical Oncology · doi:10.1200/jco.2017.35.15_suppl.7015 · Cited by 11 · see citing works

The Role of Reactive Oxygen Species in Acute Myeloid Leukaemia

Sillar, J. R., Germon, Z. P., Iuliis, G. N. D., & Dun, M. D. (2019) · International Journal of Molecular Sciences · doi:10.3390/ijms20236003 · Cited by 136 · see citing works

Advances and challenges in retinoid delivery systems in regenerative and therapeutic medicine

Ferreira, R., Napoli, J., Enver, T., Bernardino, L., & Ferreira, L. (2020) · Nature Communications · doi:10.1038/s41467-020-18042-2 · Cited by 138 · see citing works

Pharmacokinetics of trastuzumab deruxtecan (T-DXd), a novel anti-HER2 antibody-drug conjugate, in HER2-positive tumour-bearing mice

Okamoto, H., Oitate, M., Hagihara, K., Shiozawa, H., Furuta, Y., Ogitani, Y., & Kuga, H. (2020) · Xenobiotica · doi:10.1080/00498254.2020.1755909 · Cited by 82 · see citing works

LILRB3 supports acute myeloid leukemia development and regulates T-cell antitumor immune responses through the TRAF2–cFLIP–NF-κB signaling axis

Wu, G., Xu, Y., Schultz, R. D., Chen, H., Xie, J., Deng, M., Liu, X., Gui, X., John, S., Lu, Z., et al. (2021) · Nature Cancer · doi:10.1038/s43018-021-00262-0 · Cited by 75 · see citing works

Natural Products and Acute Myeloid Leukemia: A Review Highlighting Mechanisms of Action

Hwang, D., Kim, M., Park, H., Jeong, M. I., Jung, W., & Kim, B. (2019) · Nutrients · doi:10.3390/nu11051010 · Cited by 64 · see citing works

Age at Diagnosis and Patient Preferences for Treatment Outcomes in AML: A Discrete Choice Experiment to Explore Meaningful Benefits

Richardson, D. R., Crossnohere, N. L., Seo, J., Estey, E. H., O’Donoghue, B., Smith, B. D., & Bridges, J. F. P. (2020) · Cancer Epidemiology Biomarkers & Prevention · doi:10.1158/1055-9965.epi-19-1277 · Cited by 42 · see citing works

Evaluating venetoclax and its potential in treatment-naïve acute myeloid leukemia

Knight, T. E., Edwards, H. J., Taub, J. W., & Ge, Y. (2019) · Cancer Management and Research · doi:10.2147/cmar.s180724 · Cited by 25 · see citing works

Investigation of pathogenesis and therapeutic targets of acute myeloid leukemia based on untargeted plasma metabolomics and network pharmacology approach

Yuan, Y., Wu, Q., Zhao, J., Feng, Z., Dong, J., An, M., Wu, G., Qin, F., & Zhao, L. (2020) · Journal of Pharmaceutical and Biomedical Analysis · doi:10.1016/j.jpba.2020.113824 · Cited by 17 · see citing works

How Precision Medicine Is Changing Acute Myeloid Leukemia Therapy

Heuser, M., Mina, A. A., Stein, E. M., & Altman, J. K. (2019) · American Society of Clinical Oncology Educational Book · doi:10.1200/edbk_238687 · Cited by 15 · see citing works

miRNA-222-3p enhances the proliferation and suppresses the apoptosis of acute myeloid leukemia cells by targeting Axin2 and modulating the Wnt/β-catenin pathway

Liu, Z., Zhong, L., Dan, W., Chu, X., Liu, C., Luo, X. W., Zhang, Z., Lu, Y., Wan, P., Wang, X., et al. (2022) · Biochemical and Biophysical Research Communications · doi:10.1016/j.bbrc.2022.06.054 · Cited by 7 · see citing works

CDK12 /13 dual inhibitors are potential therapeutics for acute myeloid leukemia

Savoy, L., Long, N., Lee, H., Chen, R., Allen, B., Lin, H., Tognon, C. E., Malhotra, S. V., Tyner, J. W., & Zhang, H. (2023) · British Journal of Haematology · doi:10.1111/bjh.18843 · Cited by 6 · see citing works

Acute myeloid leukemia drug development in the post‐venetoclax era

Pollyea, D. A. (2019) · American Journal of Hematology · doi:10.1002/ajh.25556 · Cited by 6 · see citing works

A Phase 1, Open-Label Study in Healthy Subjects to Evaluate the Absolute Bioavailability of AG-221 by a Microtracer Approach

Wang, X., Chen, J., Reyes, J. F., Zhou, S. Y., Palmisano, M., & Li, Y. (2019) · Oncology and Therapy · doi:10.1007/s40487-019-0097-7 · Cited by 5 · see citing works

Clinical Trials of Repurposing Medicines in Acute Myeloid Leukemia

Andresen, V., & Gjertsen, B. T. (2019) · The Cancer Journal · doi:10.1097/ppo.0000000000000363 · Cited by 4 · see citing works

EVI-1-Mediated Up-Regulation of lncRNA TUG1 Interacts with miR-186-5p to Promote Proliferation and Drug Resistance in Pediatric Acute Myeloid LeukemiaPreprint

Li, E., Yu, L., Zhang, H., Zhang, L., Tang, Y., & Wanyan, Y. (2021) · Research Square · doi:10.21203/rs.3.rs-762990/v1

Research Strategy for the Development of a Quality-of-Life Decision-Making Model for Older Patients With Acute Myeloid Leukemia

Sara M. Tinsely-Vance, P. (2023) · Journal of the Advanced Practitioner in Oncology · doi:10.6004/jadpro.2023.14.4.2

Developing and validating a sensitive and fast UPLC-MS/MS method for estimating the in vitro metabolic stability of crenolanib in HLMs: identification of structural alarms related to the in silico toxicity and metabolic lability

Attwa, M. W., Ali, A. M., Alrabiah, H. K., & Kadi, A. A. (2025) · Analytical Methods · doi:10.1039/d5ay01691f

[Incorporation of novel agents into the treatment for acute myeloid leukemia].

Yamauchi, T. (2018) · PubMed · doi:10.11406/rinketsu.59.1988 · Cited by 2 · see citing works

Novel Agents for Acute Myeloid Leukemia

Luppi, M., Fabbiano, F., Visani, G., Martinelli, G., & Venditti, A. (2018) · Cancers · doi:10.3390/cancers10110429 · Cited by 44 · see citing works

Incorporating newer agents in the treatment of acute myeloid leukemia

Raj, R. V., Abedin, S. M., & Atallah, E. L. (2018) · Leukemia Research · doi:10.1016/j.leukres.2018.10.008 · Cited by 9 · see citing works

New Targeted Agents in Acute Myeloid Leukemia: New Hope on the Rise

Bohl, S., Bullinger, L., & Rücker, F. G. (2019) · International Journal of Molecular Sciences · doi:10.3390/ijms20081983 · Cited by 86 · see citing works

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

Bewersdorf, J. P., Stahl, M. F., & Zeidan, A. M. (2019) · Leukemia & lymphoma/Leukemia and lymphoma · doi:10.1080/10428194.2018.1546854 · Cited by 30 · see citing works