Niemeyer CM, Flotho C. Juvenile myelomonocytic leukemia: who’s the driver at the wheel? Blood. 2019;133:1060–70.
Article CAS PubMed Google Scholar
Arber DA, Orazi A, Hasserjian RP, Borowitz MJ, Calvo KR, Kvasnicka H-M, et al. International Consensus Classification of Myeloid Neoplasms and Acute Leukemias: integrating morphologic, clinical, and genomic data. Blood. 2022;140:1200–28.
Article CAS PubMed PubMed Central Google Scholar
Niemeyer CM. JMML genomics and decisions. Hematol Am Soc Hematol Educ Program. 2018;2018:307–12.
Mayerhofer C, Niemeyer CM, Flotho C. Current treatment of juvenile myelomonocytic leukemia. J. Clin. Med. 2021;10:3084.
Schönung M, Meyer J, Nöllke P, Olshen AB, Hartmann M, Murakami N, et al. International consensus definition of DNA methylation subgroups in juvenile myelomonocytic leukemia. Clin Cancer Res. 2021;27:158–68.
Krombholz CF, Aumann K, Kollek M, Bertele D, Fluhr S, Kunze M, et al. Long-term serial xenotransplantation of juvenile myelomonocytic leukemia recapitulates human disease in Rag2-/-γc-/- mice. Haematologica. 2016;101:597–606.
Article PubMed PubMed Central Google Scholar
Krombholz CF, Gallego-Villar L, Sahoo SS, Panda PK, Wlodarski MW, Aumann K, et al. Azacitidine is effective for targeting leukemia-initiating cells in juvenile myelomonocytic leukemia. Leukemia. 2019;33:1805–10.
Niemeyer CM, Flotho C, Lipka DB, Starý J, Rössig C, Baruchel A, et al. Response to upfront azacitidine in juvenile myelomonocytic leukemia in the AZA-JMML-001 trial. Blood Adv. 2021;5:2901–8.
Article CAS PubMed PubMed Central Google Scholar
Zehnle PMA, Wu Y, Pommerening H, Erlacher M. Stayin’ alive: BCL-2 proteins in the hematopoietic system. Exp Hematol. 2022;110:1–12.
Article CAS PubMed Google Scholar
Roberts AW. Therapeutic development and current uses of BCL-2 inhibition. Hematol Am Soc Hematol Educ Program. 2020;2020:1–9.
Cook SJ, Stuart K, Gilley R, Sale MJ. Control of cell death and mitochondrial fission by ERK1/2 MAP kinase signalling. FEBS J. 2017;284:4177–95.
Article CAS PubMed PubMed Central Google Scholar
Oltersdorf T, Elmore SW, Shoemaker AR, Armstrong RC, Augeri DJ, Belli BA, et al. An inhibitor of Bcl-2 family proteins induces regression of solid tumours. Nature. 2005;435:677–81.
Article CAS PubMed Google Scholar
Goldman JP, Blundell MP, Lopes L, Kinnon C, Di Santo JP, Thrasher AJ. Enhanced human cell engraftment in mice deficient in RAG2 and the common cytokine receptor gamma chain. Br J Haematol. 1998;103:335–42.
Article CAS PubMed Google Scholar
Lapidot T, Grunberger T, Vormoor J, Estrov Z, Kollet O, Bunin N, et al. Identification of human juvenile chronic myelogenous leukemia stem cells capable of initiating the disease in primary and secondary SCID mice. Blood. 1996;88:2655–64.
Article CAS PubMed Google Scholar
Notta F, Mullighan CG, Wang JCY, Poeppl A, Doulatov S, Phillips LA, et al. Evolution of human BCR-ABL1 lymphoblastic leukaemia-initiating cells. Nature. 2011;469:362–7. https://pubmed.ncbi.nlm.nih.gov/21248843/.
Article CAS PubMed Google Scholar
Ianevski A, Giri AK, Aittokallio T. SynergyFinder 2.0: visual analytics of multi-drug combination synergies. Nucleic Acids Res. 2020;48:W488–W493.
Article CAS PubMed PubMed Central Google Scholar
Bolger AM, Lohse M, Usadel B. Trimmomatic: a flexible trimmer for Illumina sequence data. Bioinformatics. 2014;30:2114–20.
Article CAS PubMed PubMed Central Google Scholar
Dobin A, Davis CA, Schlesinger F, Drenkow J, Zaleski C, Jha S, et al. STAR: ultrafast universal RNA-seq aligner. Bioinformatics. 2013;29:15–21.
Article CAS PubMed Google Scholar
Ritchie ME, Phipson B, Wu D, Hu Y, Law CW, Shi W, et al. limma powers differential expression analyses for RNA-sequencing and microarray studies. Nucleic Acids Res. 2015;43:e47.
Article PubMed PubMed Central Google Scholar
Gibson L, Holmgreen SP, Huang DC, Bernard O, Copeland NG, Jenkins NA, et al. bcl-w, a novel member of the bcl-2 family, promotes cell survival. Oncogene. 1996;13:665–75.
O’Reilly LA, Print C, Hausmann G, Moriishi K, Cory S, Huang DC, et al. Tissue expression and subcellular localization of the pro-survival molecule Bcl-w. Cell Death Differ. 2001;8:486–94.
Hofmans M, Lammens T, Depreter B, Wu Y, Erlacher M, Caye A, et al. Long non-coding RNAs as novel therapeutic targets in juvenile myelomonocytic leukemia. Sci Rep. 2021;11:2801 https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7854679/.
Article CAS PubMed PubMed Central Google Scholar
DiNardo CD, Pratz K, Pullarkat V, Jonas BA, Arellano M, Becker PS, et al. Venetoclax combined with decitabine or azacitidine in treatment-naive, elderly patients with acute myeloid leukemia. Blood. 2019;133:7–17.
Article CAS PubMed PubMed Central Google Scholar
Locatelli F, Nöllke P, Zecca M, Korthof E, Lanino E, Peters C, et al. Hematopoietic stem cell transplantation (HSCT) in children with juvenile myelomonocytic leukemia (JMML): results of the EWOG-MDS/EBMT trial. Blood. 2005;105:410–9. https://pubmed.ncbi.nlm.nih.gov/15353481/.
Article CAS PubMed Google Scholar
Scheid MP, Schubert KM, Duronio V. Regulation of bad phosphorylation and association with Bcl-x(L) by the MAPK/Erk kinase. J Biol Chem. 1999;274:31108–13.
Article CAS PubMed Google Scholar
Ley R, Balmanno K, Hadfield K, Weston C, Cook SJ. Activation of the ERK1/2 signaling pathway promotes phosphorylation and proteasome-dependent degradation of the BH3-only protein, Bim. J Biol Chem. 2003;278:18811–6.
Article CAS PubMed Google Scholar
Yang Z, Li Y, Yin F, Chan RJ. Activating PTPN11 mutants promote hematopoietic progenitor cell-cycle progression and survival. Exp Hematol. 2008;36:1285–96.
Article CAS PubMed PubMed Central Google Scholar
Chan G, Cheung LS, Yang W, Milyavsky M, Sanders AD, Gu S, et al. Essential role for Ptpn11 in survival of hematopoietic stem and progenitor cells. Blood. 2011;117:4253–61.
Article CAS PubMed PubMed Central Google Scholar
Hanahan D, Weinberg RA. Hallmarks of cancer: the next generation. Cell. 2011;144:646–74.
Article CAS PubMed Google Scholar
Certo M, Del Gaizo Moore V, Nishino M, Wei G, Korsmeyer S, Armstrong SA, et al. Mitochondria primed by death signals determine cellular addiction to antiapoptotic BCL-2 family members. Cancer Cell. 2006;9:351–65.
Article CAS PubMed Google Scholar
Potter DS, Letai A. To prime, or not to prime: that is the question. Cold Spring Harb Symp Quant Biol. 2016;81:131–40.
Montero J, Letai A. Why do BCL-2 inhibitors work and where should we use them in the clinic? Cell Death Differ. 2018;25:56–64.
Article CAS PubMed Google Scholar
Flotho C. Gene mutations do not operate in a vacuum: the increasing importance of epigenetics in juvenile myelomonocytic leukemia. Epigenetics. 2019;14:236–44.
Article PubMed PubMed Central Google Scholar
Šimoničová K, Janotka Ľ, Kavcová H, Sulová Z, Breier A, Messingerova L. Different mechanisms of drug resistance to hypomethylating agents in the treatment of myelodysplastic syndromes and acute myeloid leukemia. Drug Resist Updates: Rev Commentaries Antimicrobial Anticancer Chemother. 2022;61:100805. Available from: URL https://pubmed.ncbi.nlm.nih.gov/35227933/.
Kaminskas E, Farrell AT, Wang Y-C, Sridhara R, Pazdur R. FDA drug approval summary: azacitidine (5-azacytidine, Vidaza) for injectable suspension. Oncologist. 2005;10:176–82.
Article CAS PubMed Google Scholar
Wilson WH, O’Connor OA, Czuczman MS, LaCasce AS, Gerecitano JF, Leonard JP, et al. Navitoclax, a targeted high-affinity inhibitor of BCL-2, in lymphoid malignancies: a phase 1 dose-escalation study of safety, pharmacokinetics, pharmacodynamics, and antitumour activity. Lancet Oncol. 2010;11:1149–59.
Article CAS PubMed PubMed Central Google Scholar
Roberts AW, Seymour JF, Brown JR, Wierda WG, Kipps TJ, Khaw SL, et al. Substantial susceptibility of chronic lymphocytic leukemia to BCL2 inhibition: results of a phase I study of navitoclax in patients with relapsed or refractory disease. J Clin Oncol. 2012;30:488–96.
Comments (0)