Investigational non-JAK inhibitors for chronic phase myelofibrosis

References

• Tefferi A, Lasho TL, Jimma T, et al. One thousand patients with primary myelofibrosis: the mayo clinic experience. Mayo Clin Proc. 2012;87:25–33.
   PubMed Web of Science ®Google Scholar
• Cervantes F, Dupriez B, Pereira A, et al. New prognostic scoring system for primary myelofibrosis based on a study of the international working group for myelofibrosis research and treatment. Blood, J American Soc Hematol. 2009;113:2895–2901.
   Google Scholar
• Passamonti F, Cervantes F, Vannucchi AM, et al. A dynamic prognostic model to predict survival in primary myelofibrosis: a study by the IWG-MRT (international working group for myeloproliferative neoplasms research and treatment). Blood,JAmerican Soc Hematol. 2010;115:1703–1708.
   Google Scholar
• Gangat N, Caramazza D, Vaidya R, et al. DIPSS plus: a refined dynamic international prognostic scoring system for primary myelofibrosis that incorporates prognostic information from karyotype, platelet count, and transfusion status. J clin oncol. 2011;29:392–397.
   PubMed Web of Science ®Google Scholar
• Tefferi A, Guglielmelli P, Lasho TL, et al. MIPSS70+ version 2.0: mutation and karyotype-enhanced international prognostic scoring system for primary myelofibrosis. J Clin Oncol. 2018;36:1769.
   PubMed Web of Science ®Google Scholar
• Devlin R, Gupta V. Myelofibrosis: to transplant or not to transplant? Hematology Am Soc Hematol Educ Program. 2016;2016:543–551.
   PubMedGoogle Scholar
• Elliot Smith LL, Viswabandya A, Maze D, et al. Factors influencing selection of upfront hematopoietic stem cell transplantation versus best available non-transplant therapy in myelofibrosis. New York: 12th International Congress on Myeloproliferative Neoplasms; 2019.
   Google Scholar
• Blair HA. Fedratinib: first Approval. Drugs. 2019;79:1719–1725.
   PubMed Web of Science ®Google Scholar
• Mascarenhas J, Hoffman R. Ruxolitinib: the first FDA approved therapy for the treatment of myelofibrosis. Clin Cancer Res. 2012;18:3008–3014.
   PubMed Web of Science ®Google Scholar
• Palandri F, Breccia M, Bonifacio M, et al. Life after ruxolitinib: reasons for discontinuation, impact of disease phase, and outcomes in 218 patients with myelofibrosis. Cancer. 2020;126(6):1243-1252.
   Google Scholar
• Verstovsek S, Mesa RA, Gotlib J, et al. Long-term treatment with ruxolitinib for patients with myelofibrosis: 5-year update from the randomized, double-blind, placebo-controlled, phase 3 COMFORT-I trial. J Hematol Oncol. 2017;10:55.
   PubMed Web of Science ®Google Scholar
• Newberry KJ, Patel K, Masarova L, et al. Clonal evolution and outcomes in myelofibrosis after ruxolitinib discontinuation. Blood. 2017;130:1125–1131.
   PubMed Web of Science ®Google Scholar
• Tefferi A, Lasho TL, Jimma T, et al. One thousand patients with primary myelofibrosis: the mayo clinic experience. Mayo Clin Proc. 2012;87:25–33.
   PubMed Web of Science ®Google Scholar
• Mascarenhas J, Hoffman R, Talpaz M, et al. Pacritinib vs best available therapy, including ruxolitinib, in patients with myelofibrosis: a randomized clinical trial. JAMA Oncol. 2018;4:652–659.
   PubMed Web of Science ®Google Scholar
• Xu L, Feng J, Gao G, et al. Momelotinib for the treatment of myelofibrosis. Expert Opin Pharmacother. 2019;20:1943–1951.
   PubMed Web of Science ®Google Scholar
• Mullally A, Lane SW, Brumme K, et al. Myeloproliferative neoplasm animal models. Hematol/Oncol Clin. 2012;26:1065–1081.
   PubMed Web of Science ®Google Scholar
• Jain T, Mesa R. The development, safety and efficacy of pacritinib for the treatment of myelofibrosis. Expert Rev Anticancer Ther. 2016;16:1101–1108.
   PubMed Web of Science ®Google Scholar
• Fleming TR, DeMets DL. Surrogate end points in clinical trials: are we being misled? Ann Intern Med. 1996;125:605–613.
   PubMed Web of Science ®Google Scholar
• Mandrekar SJ, Sargent DJ. Randomized phase II trials: time for a new era in clinical trial design. J Thorac Oncol. 2010;5:932–934.
   PubMed Web of Science ®Google Scholar
• Vainchenker W, Kralovics R. Genetic basis and molecular pathophysiology of classical myeloproliferative neoplasms. Blood,JAmerican Soc Hematol. 2017;129:667–679.
   Google Scholar
• Zoi K, Cross N. Genomics of myeloproliferative neoplasms. J clin oncol. 2017;35:947–954.
   PubMed Web of Science ®Google Scholar
• Baxter EJ, Scott LM, Campbell PJ, et al. Acquired mutation of the tyrosine kinase JAK2 in human myeloproliferative disorders. Lancet. 2005;365:1054–1061.
   PubMed Web of Science ®Google Scholar
• Kralovics R, Passamonti F, Buser AS, et al. A gain-of-function mutation of JAK2 in myeloproliferative disorders. N Engl J Med. 2005;352:1779–1790.
   PubMed Web of Science ®Google Scholar
• Levine RL, Loriaux M, Huntly BJ, et al. The JAK2V617F activating mutation occurs in chronic myelomonocytic leukemia and acute myeloid leukemia, but not in acute lymphoblastic leukemia or chronic lymphocytic leukemia. Blood. 2005;106:3377–3379.
   PubMed Web of Science ®Google Scholar
• Nangalia J, Massie CE, Baxter EJ, et al. Somatic CALR mutations in myeloproliferative neoplasms with nonmutated JAK2. N Engl J Med. 2013;369:2391–2405.
   PubMed Web of Science ®Google Scholar
• Defour J-P, Itaya M, Gryshkova V, et al. Tryptophan at the transmembrane–cytosolic junction modulates thrombopoietin receptor dimerization and activation. Proceedings of the National Academy of Sciences 2013;110:2540–2545.
   Google Scholar
• Levine RL, Pardanani A, Tefferi A, et al. Role of JAK2 in the pathogenesis and therapy of myeloproliferative disorders. Nat Rev Cancer. 2007;7:673.
   PubMed Web of Science ®Google Scholar
• Lundberg P, Karow A, Nienhold R, et al. Clonal evolution and clinical correlates of somatic mutations in myeloproliferative neoplasms. Blood. 2014;123:2220–2228.
   PubMed Web of Science ®Google Scholar
• Wagner-Ballon O, Chagraoui H, Prina E, et al. Monocyte/macrophage dysfunctions do not impair the promotion of myelofibrosis by high levels of thrombopoietin. J Immunol. 2006;176:6425–6433.
   PubMed Web of Science ®Google Scholar
• Kleppe M, Koche R, Zou L, et al. Dual targeting of oncogenic activation and inflammatory signaling increases therapeutic efficacy in myeloproliferative neoplasms. Cancer Cell. 2018;33:29–43.e27.
   PubMed Web of Science ®Google Scholar
• Florence B, Faller DV. You bet-cha: a novel family of transcriptional regulators. Front Biosci. 2001;6:D1008–D1018.
   PubMed Web of Science ®Google Scholar
• Mascarenhas J, Kremyanskaya M, Hoffman R, et al. MANIFEST, a phase 2 study of cpi-0610, a bromodomain and extraterminal domain inhibitor (BETi), as monotherapy or” add-on” to ruxolitinib, in patients with refractory or intolerant advanced myelofibrosis. Blood. 2019;134(Supplement_1):670-670.
   Google Scholar
• Harrison CN, Patriarca A, Mascarenhas J, et al. Preliminary Report of MANIFEST, a phase 2 study of cpi-0610, a bromodomain and extraterminal domain inhibitor (BETi), in combination with ruxolitinib, in JAK inhibitor (JAKi) treatment naïve myelofibrosis patients. Blood. 2019;134(Supplement_1):4164-4164.
   Google Scholar
• Mesa RA, Verstovsek S, Rivera C, et al. 5-Azacitidine has limited therapeutic activity in myelofibrosis. Leukemia. 2009;23:180–182.
   PubMed Web of Science ®Google Scholar
• Masarova L, Verstovsek S, Hidalgo-Lopez JE, et al., A phase 2 study of ruxolitinib in combination with azacitidine in patients with myelofibrosis. Blood. 2018;132(16): 1664–1674. .
   PubMed Web of Science ®Google Scholar
• Gao S-M, Chen C-Q, Wang L-Y, et al. Histone deacetylases inhibitor sodium butyrate inhibits JAK2/STAT signaling through upregulation of SOCS1 and SOCS3 mediated by HDAC8 inhibition in myeloproliferative neoplasms. Exp Hematol. 2013;41(261–270):e264. .
   Google Scholar
• Calzada AA, Pedrini O, Finazzi G, et al. Givinostat and hydroxyurea synergize in vitro to induce apoptosis of cells from JAK2V617F myeloproliferative neoplasm patients. Exp Hematol. 2013;41(253–260):e252.
   Google Scholar
• Fiskus W, Verstovsek S, Manshouri T, et al. Heat shock protein 90 inhibitor is synergistic with JAK2 inhibitor and overcomes resistance to JAK2-TKI in human myeloproliferative neoplasm cells. Clin Cancer Res. 2011;17:7347–7358.
   PubMed Web of Science ®Google Scholar
• Bose P, Swaminathan M, Pemmaraju N, et al. A phase 2 study of pracinostat combined with ruxolitinib in patients with myelofibrosis. Leuk Lymphoma. 2019;60:1767–1774.
   PubMed Web of Science ®Google Scholar
• Mascarenhas J, Marcellino B, Lu M, et al. A phase I study of panobinostat and ruxolitinib in patients with primary myelofibrosis (PMF) and post-polycythemia vera/essential thrombocythemia myelofibrosis (post-PV/ET MF). Leuk Res. 2020;88:106272.
   PubMed Web of Science ®Google Scholar
• Jean S, Kiger AA. Classes of phosphoinositide 3-kinases at a glance. Journal of Cell Science. 2014;127(5):923.
   Google Scholar
• Khan I, Huang Z, Wen Q, et al. AKT is a therapeutic target in myeloproliferative neoplasms. Leukemia. 2013;27:1882.
   PubMed Web of Science ®Google Scholar
• Choong ML, Pecquet C, Pendharkar V, et al. Combination treatment for myeloproliferative neoplasms using JAK and pan-class I PI3K inhibitors. J Cell Mol Med. 2013;17:1397–1409.
   PubMed Web of Science ®Google Scholar
• Vakkalanka S, Viswanadha S, Niecestro R, et al. TGR-1202 Suppresses AML and ALL Cells Via Selective Inhibition of PI3Kδ Kinase. Blood. 2012;120:2610.
   PubMedGoogle Scholar
• Moyo TK, Palmer J, Huang Y, et al. Resurrecting response to ruxolitinib: a phase I study of ruxolitinib and umbralisib (TGR-1202) in ruxolitinib-experienced myelofibrosis. HemaSphere. 2018;2(S1):19-20.
   Google Scholar
• Daver N, Dao K-H, Assad A, et al. 2 Study of the Safety and Efficacy of INCB050465 in Combination with Ruxolitinib in Patients with Myelofibrosis (MF). Clin Lymphoma Myeloma Leukemia. 2017;17:S351.
   Web of Science ®Google Scholar
• Durrant ST, Nagler A, Guglielmelli P, et al. Results from HARMONY: an open-label, multicentre, 2-arm, phase 1b, dose-finding study assessing the safety and efficacy of the oral combination of ruxolitinib and buparlisib in patients with myelofibrosis. Haematologica. 2019:haematol.2018.209965.
   Google Scholar
• Vainchenker W, Constantinescu SN. JAK/STAT signaling in hematological malignancies. Oncogene. 2013;32:2601.
   PubMed Web of Science ®Google Scholar
• Mazzacurati L, Collins RJ, Pandey G, et al. The pan-PIM inhibitor INCB053914 displays potent synergy in combination with ruxolitinib in models of MPN. Blood Adv. 2019;3:3503–3514.
   PubMed Web of Science ®Google Scholar
• Bhagwat N, Keller MD, Rampal RK, et al. Improved efficacy of combination of JAK2 and hedgehog inhibitors in myelofibrosis.  Blood. 2013;122(21):666-666.
   Google Scholar
• Couban S, Benevolo G, Donnellan W, et al. Phase 1b results of a study to assess the efficacy and safety of vismodegib in combination with ruxolitinib in patients with intermediate-or high-risk myelofibrosis. Blood. 2017;130(Supplement 1):4179-4179.
   Google Scholar
• Gupta V, Harrison CN, Hasselbalch H, et al. Phase 1b/2 study of the efficacy and safety of sonidegib (LDE225) in combination with ruxolitinib (INC424) in patients with myelofibrosis. Am Soc Hematology; 2015.
   Google Scholar
• Gerds AT, Tauchi T, Ritchie E, et al. Phase 1/2 trial of glasdegib in patients with primary or secondary myelofibrosis previously treated with ruxolitinib. Leuk Res. 2019;79:38–44.
   PubMed Web of Science ®Google Scholar
• Tognon R, Gasparotto EPL, Neves RP, et al. Deregulation of apoptosis-related genes is associated with PRV1 overexpression and JAK2 V617F allele burden in essential thrombocythemia and myelofibrosis. J Hematol Oncol. 2012;5:2.
   PubMed Web of Science ®Google Scholar
• Maiuri MC, Criollo A, Tasdemir E, et al. BH3-only proteins and BH3 mimetics induce autophagy by competitively disrupting the interaction between Beclin 1 and Bcl-2/Bcl-XL. Autophagy. 2007;3:374–376.
   PubMed Web of Science ®Google Scholar
• Waibel M, Solomon VS, Knight DA, et al. Combined targeting of JAK2 and Bcl-2/Bcl-xL to cure mutant JAK2-driven malignancies and overcome acquired resistance to JAK2 inhibitors. Cell Rep. 2013;5:1047–1059.
   PubMed Web of Science ®Google Scholar
• Harrison CN, Garcia JS, Mesa RA, et al. Results from a phase 2 study of navitoclax in combination with ruxolitinib in patients with primary or secondary myelofibrosis. Blood. 2019;134:671.
   Web of Science ®Google Scholar
• Quach H, Ritchie D, Stewart AK, et al. Mechanism of action of immunomodulatory drugs (IMiDS) in multiple myeloma. Leukemia. 2010;24:22.
   PubMed Web of Science ®Google Scholar
• Weinkove R, Reilly JT, McMullin MF, et al. Low-dose thalidomide in myelofibrosis. Haematologica. 2008;93:1100–1101.
   PubMed Web of Science ®Google Scholar
• Mesa RA, Pardanani AD, Hussein K, et al. Phase1/‐2 study of Pomalidomide in myelofibrosis. Am J Hematol. 2010;85:129–130.
   PubMed Web of Science ®Google Scholar
• Rampal RK, Verstovsek S, Devlin SM, et al. Safety and efficacy of combined ruxolitinib and thalidomide in patients with myelofibrosis: a phase II study. Blood. 2019;134(Supplement_1):4163-4163.
   Google Scholar
• Tefferi A, Al-Ali H, Barosi G, et al. A randomized study of pomalidomide vs placebo in persons with myeloproliferative neoplasm-associated myelofibrosis and RBC-transfusion dependence. Leukemia. 2017;31:896.
   PubMed Web of Science ®Google Scholar
• Stegelmann F, Koschmieder S, Isfort S, et al. Updated results from the german mpnsg-0212 combination trial: ruxolitinib plus pomalidomide in Myelofibrosis with Anemia. Blood. 2019;134(Supplement_1):672-672.
   Google Scholar
• Ceglia I, Dueck AC, Masiello F, et al. Preclinical rationale for TGF-β inhibition as a therapeutic target for the treatment of myelofibrosis. Exp Hematol. 2016;44(1138–1155):e1134. .
   Google Scholar
• Suragani RN, Cadena SM, Cawley SM, et al. Transforming growth factor-β superfamily ligand trap ACE-536 corrects anemia by promoting late-stage erythropoiesis. Nat Med. 2014;20:408.
   PubMed Web of Science ®Google Scholar
• Gerds AT, Vannucchi AM, Passamonti F, et al. A phase 2 study of luspatercept in patients with Myelofibrosis-Associated Anemia. Blood. 2019;134(Supplement_1):557-557.
   Google Scholar
• Bose P, Pemmaraju N, Daver N, et al. Sotatercept (ace-011) in subjects with mpn-associated myelofibrosis and anemia: S829. Hemasphere. 2019;3:367–368.
   Google Scholar
• Cappellini MD, Porter J, Origa R, et al. Sotatercept, a novel transforming growth factor β ligand trap, improves anemia in β-thalassemia: a phase II, open-label, dose-finding study. Haematologica. 2019;104:477.
   PubMed Web of Science ®Google Scholar
• Xia Y, Schneyer AL. The biology of activin: recent advances in structure, regulation and function. J Endocrinol. 2009;202:1–12.
   PubMed Web of Science ®Google Scholar
• Ianotto JC, Kiladjian JJ, Demory JL, et al. PEG‐IFN‐α‐2a therapy in patients with myelofibrosis: A study of the French groupe d’etudes des myelofibroses (GEM) and France intergroupe des syndromes myéloprolifératifs (FIM). Br J Haematol. 2009;146:223–225.
   PubMed Web of Science ®Google Scholar
• Silver RT, Vandris K, Goldman JJ. Recombinant interferon-α may retard progression of early primary myelofibrosis: a preliminary report. Blood. 2011;117:6669–6672.
   PubMed Web of Science ®Google Scholar
• Mikkelsen SU, Kjær L, Bjørn ME, et al. Safety and efficacy of combination therapy of interferon-α2 and ruxolitinib in polycythemia vera and myelofibrosis. Cancer Med. 2018;7:3571–3581.
   PubMed Web of Science ®Google Scholar
• Kiladjian -J-J, Soret-Dulphy J, Resche-Rigon M, et al. Ruxopeg, a multi-center bayesian phase 1/2 adaptive randomized trial of the combination of ruxolitinib and pegylated interferon alpha 2a in patients with myeloproliferative neoplasm (MPN)-associated myelofibrosis. Blood. 2018;132:581.
   Web of Science ®Google Scholar
• Nakatake M, Monte-Mor B, Debili N, et al. JAK2V617F negatively regulates p53 stabilization by enhancing MDM2 via La expression in myeloproliferative neoplasms. Oncogene. 2012;31:1323–1333.
   PubMed Web of Science ®Google Scholar
• Spangrude GJ, Lewandowski D, Martelli F, et al. P-Selectin Sustains Extramedullary Hematopoiesis in the Gata1 low Model of Myelofibrosis. Stem Cells. 2016;34:67–82.
   PubMed Web of Science ®Google Scholar
• Kikushige Y, Shima T, Takayanagi S, et al. TIM-3 is a promising target to selectively kill acute myeloid leukemia stem cells. Cell Stem Cell. 2010;7:708–717.
   PubMed Web of Science ®Google Scholar
• Kikushige Y, Miyamoto T, Yuda J, et al. A TIM-3/Gal-9 autocrine stimulatory loop drives self-renewal of human myeloid leukemia stem cells and leukemic progression. Cell Stem Cell. 2015;17:341–352.
   PubMed Web of Science ®Google Scholar
• Ndhlovu LC, Lopez-Vergès S, Barbour JD, et al. Tim-3 marks human natural killer cell maturation and suppresses cell-mediated cytotoxicity. Blood. 2012;119:3734–3743.
   PubMed Web of Science ®Google Scholar
• Wang JC, Sindhu H, Chen C, et al. Immune derangements in patients with myelofibrosis: the role of Treg, Th17, and sIL2Rα. PloS One. 2015;10:e0116723.
   PubMed Web of Science ®Google Scholar
• Hasselbalch HC, Holmström MO. Perspectives on interferon-alpha in the treatment of polycythemia vera and related myeloproliferative neoplasms: minimal residual disease and cure? Semin Immunopathol. 2019 Jan;41(1):5-19.
   Google Scholar
• Kadia TM, Ma H, Zeng K, et al. Phase I clinical trial of CK0801 (cord blood regulatory T cells) in patients with bone marrow failure syndrome (BMF) including aplastic anemia, myelodysplasia and myelofibrosis. Blood. 2019;134(Supplement_1):1221-1221.
   Google Scholar
• Sprüssel A, Schulte J, Weber S, et al. Lysine-specific demethylase 1 restricts hematopoietic progenitor proliferation and is essential for terminal differentiation. Leukemia. 2012;26:2039.
   PubMed Web of Science ®Google Scholar
• Jutzi JS, Kleppe M, Dias J, et al. LSD1 Inhibition prolongs survival in mouse models of MPN by selectively targeting the disease clone. Hemasphere. 2018;2:e54.
   PubMed Web of Science ®Google Scholar
• Pettit K, Gerds AT, Yacoub A, et al. A phase 2a study of the LSD1 inhibitor img-7289 (bomedemstat) for the treatment of myelofibrosis. Blood. 2019;134(Supplement_1):556-556. .
   Google Scholar
• Bernard L, Belisle C, Mollica L, et al. Telomere length is severely and similarly reduced in JAK2V617F-positive and-negative myeloproliferative neoplasms. Leukemia. 2009;23:287.
   PubMed Web of Science ®Google Scholar
• Wang X, Hu CS, Petersen B, et al. Imetelstat, a telomerase inhibitor, is capable of depleting myelofibrosis stem and progenitor cells. Blood Adv. 2018;2:2378–2388.
   PubMed Web of Science ®Google Scholar
• Mascarenhas J, Komrokji RS, Cavo M, et al. imetelstat is effective treatment for patients with intermediate-2 or high-risk myelofibrosis who have relapsed on or are refractory to janus kinase inhibitor therapy: results of a phase 2 randomized study of two dose levels. Blood. 2018;132:685.
   Web of Science ®Google Scholar
• Tefferi A, Lasho TL, Begna KH, et al. A pilot study of the telomerase inhibitor imetelstat for myelofibrosis. N Engl J Med. 2015;373:908–919.
   PubMed Web of Science ®Google Scholar
• Heaton WL, Senina AV, Pomicter AD, et al. Autocrine Tnf signaling favors malignant cells in myelofibrosis in a Tnfr2-dependent fashion. Leukemia. 2018;32:2399–2411.
   PubMed Web of Science ®Google Scholar
• Deveraux QL, Reed JC. IAP family proteins–suppressors of apoptosis. Genes Dev. 1999;13:239–252.
   PubMed Web of Science ®Google Scholar
• Du C, Fang M, Li Y, et al. Smac, a mitochondrial protein that promotes cytochrome c–dependent caspase activation by eliminating IAP Inhibition. Cell. 2000;102:33–42.
   PubMed Web of Science ®Google Scholar
• Pemmaraju N, Carter BZ, Kantarjian HM, et al. Final results of phase 2 clinical trial of LCL161, a novel oral SMAC mimetic/IAP antagonist, for patients with intermediate to high risk myelofibrosis. Blood. 2019;134:555.
   Web of Science ®Google Scholar
• Pastore F, Bhagwat N, Krishnan A, et al. PRMT5 inhibition modulates E2F1 methylation and gene regulatory networks leading to therapeutic efficacy in JAK2VF mutant MPN. Blood. 2019;134(Supplement_1):473-473.
   Google Scholar
• Yan D, Pomicter AD, Tantravahi S, et al. Nuclear–Cytoplasmic Transport Is a Therapeutic Target in Myelofibrosis. Clin Cancer Res. 2019;25:2323–2335.
   PubMed Web of Science ®Google Scholar
• Marubayashi S, Koppikar P, Taldone T, et al. HSP90 is a therapeutic target in JAK2-dependent myeloproliferative neoplasms in mice and humans. J Clin Invest. 2010;120:3578–3593.
   PubMed Web of Science ®Google Scholar
• Bhagwat N, Koppikar P, Keller M, et al. Improved targeting of JAK2 leads to increased therapeutic efficacy in myeloproliferative neoplasms. Blood. 2014;123:2075–2083.
   PubMed Web of Science ®Google Scholar
• Bagley CJ, Woodcock JM, Stomski FC, et al. The structural and functional basis of cytokine receptor activation: lessons from the common β subunit of the granulocyte-macrophage colony-stimulating factor, interleukin-3 (IL-3), and IL-5 receptors. Blood,JAmerican Soc Hematol. 1997;89:1471–1482.
   Google Scholar
• Jin L, Lee EM, Ramshaw HS, et al. Monoclonal antibody-mediated targeting of CD123, IL-3 receptor α chain, eliminates human acute myeloid leukemic stem cells. Cell Stem Cell. 2009;5:31–42.
   PubMed Web of Science ®Google Scholar
• Lasho T, Finke C, Kimlinger TK, et al. Expression of CD123 (IL-3R-alpha), a therapeutic target of SL-401, on myeloproliferative neoplasms. Am Soc Hematology; 2014.
   Google Scholar
• Testa U, Riccioni R, Biffoni M, et al. Diphtheria toxin fused to variant human interleukin-3 induces cytotoxicity of blasts from patients with acute myeloid leukemia according to the level of interleukin-3 receptor expression. Blood. 2005;106:2527–2529.
   PubMed Web of Science ®Google Scholar
• Pemmaraju N, Gupta V, Ali H, et al. Results from a phase 1/2 clinical trial of tagraxofusp (SL-401) in patients with intermediate, or high risk, relapsed/refractory myelofibrosis. Blood. 2019;134(Supplement_1):558-558.
   Google Scholar
• Zhang Y, Nagata Y, Yu G, et al. Aberrant quantity and localization of Aurora-B/AIM-1 and survivin during megakaryocyte polyploidization and the consequences of Aurora-B/AIM-1–deregulated expression. Blood. 2004;103:3717–3726.
   PubMed Web of Science ®Google Scholar
• Wen Q, Goldenson B, Silver SJ, et al. Identification of regulators of polyploidization presents therapeutic targets for treatment of AMKL. Cell. 2012;150:575–589.
   PubMed Web of Science ®Google Scholar
• Gangat N, Marinaccio C, Swords R, et al. Aurora kinase A inhibition provides clinical benefit, normalizes megakaryocytes and reduces bone marrow fibrosis in patients with myelofibrosis. Clin Cancer Res. 2019 Aug 15;25(16):4898-4906.
   Google Scholar
• Castaño AP, Lin S-L, Surowy T, et al. Serum amyloid P inhibits fibrosis through FcγR-dependent monocyte-macrophage regulation in vivo. Sci Transl Med. 2009;1:5ra13–15ra13.
   PubMed Web of Science ®Google Scholar
• Verstovsek S, Talpaz M, Wadleigh M, et al. A randomized, double blind phase 2 study of 3 different doses of prm-151 in patients with myelofibrosis who were previously treated with or ineligible for ruxolitinib: S828. Hemasphere. 2019;3:367.
   Google Scholar
• Vannucchi AM, Bianchi L, Paoletti F, et al. A pathobiologic pathway linking thrombopoietin, GATA-1, and TGF-beta1 in the development of myelofibrosis. Blood. 2005;105:3493–3501.
   PubMed Web of Science ®Google Scholar
• Drexler B, Passweg JR, Tzankov A, et al. The sympathomimetic agonist mirabegron did not lower JAK2-V617F allele burden, but restored nestin-positive cells and reduced reticulin fibrosis in patients with myeloproliferative neoplasms: results of phase II study SAKK 33/14. Haematologica. 2019;104:710–716.
   PubMed Web of Science ®Google Scholar
• Arranz L, Sanchez-Aguilera A, Martin-Perez D, et al. Neuropathy of haematopoietic stem cell niche is essential for myeloproliferative neoplasms. Nature. 2014;512:78–81.
   PubMed Web of Science ®Google Scholar
• Prestipino A, Emhardt AJ, Aumann K, et al. Oncogenic JAK2(V617F) causes PD-L1 expression, mediating immune escape in myeloproliferative neoplasms. Science Translational Medicine. 2018;10(429):eaam7729.
   Google Scholar
• Holmström MO, Ahmad SM, Klausen U, et al. High frequencies of circulating memory T cells specific for calreticulin exon 9 mutations in healthy individuals. Blood Cancer J. 2019;9:8.
   PubMed Web of Science ®Google Scholar
• Spiegel JY, McNamara C, Kennedy JA, et al. Impact of genomic alterations on outcomes in myelofibrosis patients undergoing JAK1/2 inhibitor therapy. Blood Adv. 2017;1:1729–1738.
   PubMed Web of Science ®Google Scholar
• Patel KP, Newberry KJ, Luthra R, et al. Correlation of mutation profile and response in patients with myelofibrosis treated with ruxolitinib. Blood. 2015;126:790–797.
   PubMed Web of Science ®Google Scholar