Advances in pharmacotherapy for myelofibrosis: what is the current state of play?

References

• Tefferi A. Primary myelofibrosis: 2023 update on diagnosis, risk-stratification, and management. Am J Hematol. 2023;98(5):801–821. doi: 10.1002/ajh.26857
   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. 2009;113(13):2895–2901. doi: 10.1182/blood-2008-07-170449
   PubMed Web of Science ®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. 2010;115(9):1703–1708. doi: 10.1182/blood-2009-09-245837
   PubMed Web of Science ®Google Scholar
• Guglielmelli P, Lasho TL, Rotunno G, et al. MIPSS70: mutation-enhanced international prognostic score system for transplantation-age patients with primary myelofibrosis. JCO. 2017;36(4):310–318. doi: 10.1200/JCO.2017.76.4886
   PubMed Web of Science ®Google Scholar
• Sureau L, Orvain C, Ianotto JC, et al. Efficacy and tolerability of janus kinase inhibitors in myelofibrosis: a systematic review and network meta-analysis. Blood Cancer J. 2021;11(7):135. doi: 10.1038/s41408-021-00526-z
   PubMed Web of Science ®Google Scholar
• Cervantes F, Vannucchi AM, Kiladjian JJ, et al. Three-year efficacy, safety and survival findings from COMFORT-II, a phase 3 study comparing ruxolitinib with best available therapy for myelofibrosis. Blood. 2013;122(25):4047–4053. doi: 10.1182/blood-2013-02-485888
   PubMed Web of Science ®Google Scholar
• Mascarenhas J, Hoffman R. A comprehensive review and analysis of the effect of ruxolitinib therapy on the survival of patients with myelofibrosis. Blood. 2013;121(24):4832–4837. doi: 10.1182/blood-2013-02-482232
   PubMed Web of Science ®Google Scholar
• Barosi G, Zhang MJ, Gale RP. Does ruxolitinib improve survival of persons with MPN-associated myelofibrosis? Should it? Leukemia. 2014;28(11):2267–2270. doi: 10.1038/leu.2014.220
   PubMed Web of Science ®Google Scholar
• Verstovsek S, Mesa RA, Gotlib J, et al. Efficacy, safety, and survival with ruxolitinib in patients with myelofibrosis: results of a median 3-year follow-up of COMFORT-I. Haematologica. 2015;100(4):479–488. doi: 10.3324/haematol.2014.115840
   PubMed Web of Science ®Google Scholar
• Harrison CN, Vannucchi A, Kiladjian JJ, et al. Long-term findings from COMFORT-II, a phase 3 study of ruxolitinib vs best available therapy for myelofibrosis. Leukemia. 2016;30(8):1701–1707. doi: 10.1038/leu.2016.148
   PubMed Web of Science ®Google Scholar
• Al-Ali HK, Griesshammer M, le Coutre P, et al. Safety and efficacy of ruxolitinib in an open-label, multicenter, single-arm phase 3b expanded-access study in patients with myelofibrosis: a snapshot of 1144 patients in the JUMP trial. Haematologica. 2016;101(9):1065–1073. doi: 10.3324/haematol.2016.143677
   PubMed Web of Science ®Google Scholar
• Palandri F, Palumbo GA, Bonifacio M, et al. Baseline factors associated with response to ruxolitinib: an independent study on 408 patients with myelofibrosis. Oncotarget. 2017;8(45):79073–79086. doi: 10.18632/oncotarget.18674
   PubMedGoogle Scholar
• Passamonti F, Heidel FH, Parikh RC, et al. Real-world clinical outcomes of patients with myelofibrosis treated with ruxolitinib: a medical record review. Future Oncol. 2022;18(18):2217–2231. doi: 10.2217/fon-2021-1358
   PubMed Web of Science ®Google Scholar
• Guglielmelli P, Ghirardi A, Carobbio A, et al. Impact of ruxolitinib on survival of patients with myelofibrosis in the real world: update of the ERNEST Study. Blood Adv. 2022;6(2):373–375. doi: 10.1182/bloodadvances.2021006006
   PubMed Web of Science ®Google Scholar
• Verstovsek S, Parasuraman S, Yu J, et al. Real-world survival of US patients with intermediate- to high-risk myelofibrosis: impact of ruxolitinib approval. Ann Hematol. 2022;101(1):131–137. doi: 10.1007/s00277-021-04682-x
   PubMed Web of Science ®Google Scholar
• Verstovsek S, Mesa RA, Gotlib J, et al. A double-blind, placebo-controlled trial of ruxolitinib for myelofibrosis. N Engl J Med. 2012;366(9):799–807. doi: 10.1056/NEJMoa1110557
   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(20):1729–1738. doi: 10.1182/bloodadvances.2017009530
   PubMed Web of Science ®Google Scholar
• Gupta V, Harrison C, Hexner EO, et al. The impact of anemia on overall survival in patients with myelofibrosis treated with ruxolitinib in the COMFORT studies. Haematologica. 2016;101(12):e482. doi: 10.3324/haematol.2016.151449
   PubMed Web of Science ®Google Scholar
• Palandri F, Breccia M, Mazzoni C, et al. Ruxolitinib in cytopenic myelofibrosis: response, toxicity, drug discontinuation, and outcome. Cancer. 2023;129(11):1704–1713. doi: 10.1002/cncr.34722
   PubMed Web of Science ®Google Scholar
• Verstovsek S, Mesa RA, Livingston RA, et al. Ten Years of treatment with ruxolitinib for myelofibrosis: a review of safety. J Hematol Oncol. 2023;16(1):82. doi: 10.1186/s13045-023-01471-z
   PubMed Web of Science ®Google Scholar
• Bose P, Kuykendall AT, Miller C, et al. Moving beyond ruxolitinib failure in myelofibrosis: evolving strategies for second line therapy. Expert Opin Pharmacother. 2023;24(9):1091–1100. doi: 10.1080/14656566.2023.2213435
   PubMed Web of Science ®Google Scholar
• Bose P, Verstovsek S. JAK inhibition for the treatment of myelofibrosis: limitations and future perspectives. Hemasphere. 2020;4(4):e424. doi: 10.1097/HS9.0000000000000424
   PubMed Web of Science ®Google Scholar
• Maffioli M, Mora B, Ball S, et al. A prognostic model to predict survival after 6 months of ruxolitinib in patients with myelofibrosis. Blood Adv. 2022;6(6):1855–1864. doi: 10.1182/bloodadvances.2021006889
   PubMed Web of Science ®Google Scholar
• Pardanani A, Tefferi A, Masszi T, et al. Updated results of the placebo-controlled, phase III JAKARTA trial of fedratinib in patients with intermediate-2 or high-risk myelofibrosis. Br J Haematol. 2021;195(2):244–248. doi: 10.1111/bjh.17727
   PubMed Web of Science ®Google Scholar
• Harrison CN, Schaap N, Vannucchi AM, et al. Fedratinib in patients with myelofibrosis previously treated with ruxolitinib: an updated analysis of the JAKARTA2 study using stringent criteria for ruxolitinib failure. Am J Hematol. 2020;95(6):594–603. doi: 10.1002/ajh.25777
   PubMed Web of Science ®Google Scholar
• Mascarenhas J, Harrison C, Schuler TA, et al. Real-world use of fedratinib for myelofibrosis following prior ruxolitinib failure: patient characteristics, treatment patterns, and clinical outcomes. Clin Lymphoma Myeloma Leuk. 2024;24(2):122–132. doi: 10.1016/j.clml.2023.09.008
   PubMed Web of Science ®Google Scholar
• Gangat N, McCullough K, Al-Kali A, et al. Limited activity of fedratinib in myelofibrosis patients relapsed/refractory to ruxolitinib 20 mg twice daily or higher: a real-world experience. Br J Haematol. 2022;198(4):e54–e58. doi: 10.1111/bjh.18284
   PubMed Web of Science ®Google Scholar
• Gupta V, Yacoub A, Verstovsek S, et al. Safety and efficacy of fedratinib in patients with primary (P), Post-Polycythemia Vera (Post-PV), and Post-essential thrombocythemia (Post-ET) myelofibrosis (MF) Previously treated with ruxolitinib: primary analysis of the FREEDOM trial. Blood (ASH Annual Meeting Abstracts). 2022;140(Suppl. Supplement 1):3935–3937. doi: 10.1182/blood-2022-156669
   Google Scholar
• Talpaz M, Kiladjian JJ. Fedratinib, a newly approved treatment for patients with myeloproliferative neoplasm-associated myelofibrosis. Leukemia. 2021;35(1):1–17. doi: 10.1038/s41375-020-0954-2
   PubMed Web of Science ®Google Scholar
• Hart S, Goh KC, Novotny-Diermayr V, et al. SB1518, a novel macrocyclic pyrimidine-based JAK2 inhibitor for the treatment of myeloid and lymphoid malignancies. Leukemia. 2011;25(11):1751–1759. doi: 10.1038/leu.2011.148
   PubMed Web of Science ®Google Scholar
• Oh ST, Mesa RA, Harrison CN, et al. Pacritinib is a potent ACVR1 inhibitor with significant anemia benefit in patients with myelofibrosis. Blood Adv. 2023;7(19):5835–5842. doi: 10.1182/bloodadvances.2023010151
   PubMed Web of Science ®Google Scholar
• Mesa RA, Vannucchi AM, Mead A, et al. Pacritinib versus best available therapy for the treatment of myelofibrosis irrespective of baseline cytopenias (PERSIST-1): an international, randomised, phase 3 trial. Lancet Haematol. 2017;4(5):e225–e236. doi: 10.1016/S2352-3026(17)30027-3
   PubMed Web of Science ®Google Scholar
• Verstovsek S, Mesa R, Talpaz M, et al. Retrospective analysis of pacritinib in patients with myelofibrosis and severe thrombocytopenia. Haematologica. 2022;107(7):1599–1607. doi: 10.3324/haematol.2021.279415
   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 Onc. 2018;4(5):652–659. doi: 10.1001/jamaoncol.2017.5818
   PubMed Web of Science ®Google Scholar
• Chifotides HT, Bose P, Verstovsek S. Momelotinib: an emerging treatment for myelofibrosis patients with anemia. J Hematol Oncol. 2022;15(1):7. doi: 10.1186/s13045-021-01157-4
   PubMed Web of Science ®Google Scholar
• Mesa RA, Kiladjian JJ, Catalano JV, et al. SIMPLIFY-1: a phase III randomized trial of momelotinib versus ruxolitinib in janus kinase inhibitor-naïve patients with myelofibrosis. J Clin Oncol. 2017;35(34):3844–3850. doi: 10.1200/JCO.2017.73.4418
   PubMed Web of Science ®Google Scholar
• Harrison CN, Vannucchi AM, Platzbecker U, et al. Momelotinib versus best available therapy in patients with myelofibrosis previously treated with ruxolitinib (SIMPLIFY 2): a randomised, open-label, phase 3 trial. Lancet Haematol. 2018;5(2):e73–e81. doi: 10.1016/S2352-3026(17)30237-5
   PubMed Web of Science ®Google Scholar
• Verstovsek S, Gerds AT, Vannucchi AM, et al. Momelotinib versus danazol in symptomatic patients with anaemia and myelofibrosis (MOMENTUM): results from an international, double-blind, randomised, controlled, phase 3 study. Lancet. 2023;401(10373):269–280. doi: 10.1016/S0140-6736(22)02036-0
   PubMed Web of Science ®Google Scholar
• Kiladjian JJ, Vannucchi AM, Gerds AT, et al. Momelotinib in myelofibrosis patients with thrombocytopenia: post hoc analysis from three randomized phase 3 trials. Hemasphere. 2023;7(11):e963. doi: 10.1097/HS9.0000000000000963
   PubMed Web of Science ®Google Scholar
• Mesa R, Harrison C, Oh ST, et al. Overall survival in the SIMPLIFY-1 and SIMPLIFY-2 phase 3 trials of momelotinib in patients with myelofibrosis. Leukemia. 2022;36(9):2261–2268. doi: 10.1038/s41375-022-01637-7
   PubMed Web of Science ®Google Scholar
• Oh ST, Verstovsek S, Gupta V, et al. Changes in bone marrow fibrosis during momelotinib or ruxolitinib therapy do not correlate with efficacy outcomes in patients with myelofibrosis. EJHaem. 2024;5(1):105–116. doi: 10.1002/jha2.854
   PubMedGoogle Scholar
• Tefferi A, Gangat N, Pardanani A. Jaktinib (JAK1/2 inhibitor): a momelotinib derivative with similar activity and optimized dosing schedule. Am J Hematol. 2022;97(12):1507–1509. doi: 10.1002/ajh.26712
   PubMed Web of Science ®Google Scholar
• Zhang Y, Zhou H, Jiang Z, et al. Safety and efficacy of jaktinib in the treatment of janus kinase inhibitor-naïve patients with myelofibrosis: results of a phase II trial. Am J Hematol. 2022;97(12):1510–1519. doi: 10.1002/ajh.26709
   PubMed Web of Science ®Google Scholar
• Zhang Y, Zhang Q, Liu Q, et al. Safety and efficacy of jaktinib (a novel JAK inhibitor) in patients with myelofibrosis who are relapsed or refractory to ruxolitinib: a single-arm, open-label, phase 2, multicenter study. Am J Hematol. 2023;98(10):1579–1587. doi: 10.1002/ajh.27031
   PubMed Web of Science ®Google Scholar
• Zhang Y, Zhou H, Duan M, et al. Safety and efficacy of jaktinib (a novel JAK inhibitor) in patients with myelofibrosis who are intolerant to ruxolitinib: a single-arm, open-label, phase 2, multicenter study. Am J Hematol. 2023;98(10):1588–1597. doi: 10.1002/ajh.27033
   PubMed Web of Science ®Google Scholar
• Taniguchi Y. The bromodomain and extra-terminal domain (BET) family: functional anatomy of BET paralogous proteins. Int J Mol Sci. 2016;17(11):1849. doi: 10.3390/ijms17111849
   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(1):29–43.e7. doi: 10.1016/j.ccell.2017.11.009
   PubMed Web of Science ®Google Scholar
• Kremyanskaya M, Mascarenhas J, Palandri F, et al. Pelabresib (CPI-0610) monotherapy in patients with Myelofibrosis – update of clinical and translational data from the ongoing MANIFEST trial. Blood (ASH Annual Meeting Abstracts). 2021;138(Suppl 1):141–144. doi: 10.1182/blood-2021-150172
   Google Scholar
• Harrison C, Kremyanskaya M, Bose P, et al. Pelabresib (CPI-0610) as add-on to ruxolitinib in myelofibrosis: durability of response and safety beyond week 24 in the phase 2 MANIFEST study. Blood (ASH Annual Meeting Abstracts). 2022;140(Suppl Supplement 1):9659–9662. doi: 10.1182/blood-2022-157735
   Google Scholar
• Mascarenhas J, Kremyanskaya M, Patriarca A, et al. MANIFEST: pelabresib in combination with ruxolitinib for janus kinase inhibitor treatment-naïve myelofibrosis. J Clin Oncol. 2023;41(32):4993–5004. doi: 10.1200/JCO.22.01972
   PubMed Web of Science ®Google Scholar
• Rampal R, Grosicki S, Chraniuk D, et al. Pelabresib in combination with ruxolitinib for janus kinase inhibitor treatment-naïve patients with myelofibrosis: results of the MANIFEST-2 randomized, double-blind, phase 3 study. Blood (ASH Annual Meeting Abstracts). 2023;142(Suppl Supplement 1):628. doi: 10.1182/blood-2023-179141
   Google Scholar
• Lavie D, Ribrag B, Loschi M, et al. BMS-986158, a Potent BET Inhibitor, in combination with ruxolitinib or fedratinib in patients (pts) with intermediate- or high-risk myelofibrosis (MF): updated results from a phase 1/2 study. Blood (ASH Annual Meeting Abstracts). 2023;142(Suppl 1):623. doi: 10.1182/blood-2023-179160
   Google Scholar
• Pullarkat V, Cruz-Chacon A, Gangatharan S, et al. P1070: Navitoclax monotherapy in patients with myelofibrosis previously treated with jak-2 inhibitors: safety and tolerability. Hemasphere. 2022;6:960–961. doi: 10.1097/01.HS9.0000847148.78233.c8
   Google Scholar
• Harrison CN, Garcia JS, Somervaille TCP, et al. Addition of Navitoclax to ongoing ruxolitinib therapy for patients with myelofibrosis with progression or suboptimal response: phase II safety and efficacy. J Clin Oncol. 2022;40(15):1671–1680. doi: 10.1200/JCO.21.02188
   PubMed Web of Science ®Google Scholar
• Pemmaraju N, Garcia JS, Potluri J, et al. Addition of navitoclax to ongoing ruxolitinib treatment in patients with myelofibrosis (REFINE): a post-hoc analysis of molecular biomarkers in a phase 2 study. Lancet Haematol. 2022;9(6):e434–e444. doi: 10.1016/S2352-3026(22)00116-8
   PubMed Web of Science ®Google Scholar
• Passamonti F, Foran JM, Tandra A, et al. The combination of navitoclax and ruxolitinib in JAK inhibitor-naive patients with myelofibrosis mediates responses suggestive of disease modification. Blood (ASH Annual Meeting Abstracts). 2022;140(suppl 1):583–585. doi: 10.1182/blood-2022-157949
   Google Scholar
• Pemmaraju N, Mead AJ, Somervaille TCP, et al. TRANSFORM-1: a randomized, double-blind, placebo-controlled, multicenter, international phase 3 study of navitoclax in combination with ruxolitinib versus ruxolitinib plus placebo in patients with untreated myelofibrosis. Blood (ASH Annual Meeting Abstracts). 2023;142(Suppl Supplement 1):620. doi: 10.1182/blood-2023-173509
   Google Scholar
• Yan D, Pomicter AD, Tantravahi SK, et al. Nuclear-cytoplasmic transport is a therapeutic target in myelofibrosis. Clin Cancer Res. 2019;25(7):2323–2335. doi: 10.1158/1078-0432.CCR-18-0959
   PubMed Web of Science ®Google Scholar
• Tantravahi SK, Kim SJ, Syundar D, et al. A phase 2 study to evaluate the efficacy and safety of selinexor in patients with myelofibrosis refractory of intolerant to JAK inhibitors. Blood (ASH Annual Meeting Abstracts). 2021;138(Suppl 1):143. doi: 10.1182/blood-2021-153940
   Google Scholar
• Duan M, Ma L, Wu 1, et al. The efficacy and safety of selinexor in combination with ruxolitinib in ruxolitinib-treated myelofibrosis patients: the interim analysis of a prospective, open-label, multicenter, parallel-cohort, phase 2 study. Blood (ASH Annual Meeting Abstracts). 2023;142(Suppl 1):4578. doi: 10.1182/blood-2023-188037
   Google Scholar
• Ali H, Kishtagari A, Maher K, et al. Selinexor (SEL) plus ruxolitinib (RUX) in JAK Inhibitor (JAKi) treatment-naïve patients with myelofibrosis: updated results from XPORT-MF-034. J Clin Oncol. 2023;41(16 Suppl):7063. doi: 10.1200/JCO.2023.41.16_suppl.7063
   Web of Science ®Google Scholar
• Tantravahi SK, Kishtagari A, Maher K, et al. Selinexor plus ruxolitinib in JAK Inhibitor (JAKi)-naïve patients with myelofibrosis: long term follow up from XPORT-MF-034 suggestive of disease modification. Blood (ASH Annual Meeting Abstracts). 2023;142(Suppl Supplement 1):622. doi: 10.1182/blood-2023-180844
   Google Scholar
• Durrant ST, Nagler A, Guglielmelli P, et al. Results from HARMONY: an open-label, multicenter, 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;104(12):e551–e554. doi: 10.3324/haematol.2018.209965
   PubMed Web of Science ®Google Scholar
• Yacoub A, Borate U, Rampal RK, et al. Phase 2 study of add-on parsaclisib in myelofibrosis patients with suboptimal response to ruxolitinib: final results. Blood Adv. 2024;8(6):1515–1528. doi: 10.1182/bloodadvances.2023011620
   PubMed Web of Science ®Google Scholar
• Kiladjian JJ, Ianotto JC, Soret J, et al. Final results of ruxopeg, a phase 1/2 adaptive randomized trial of ruxolitinib (rux) and pegylated interferon alpha (IFNa) 2a in patients with myelofibrosis (MF). Blood (ASH Annual Meeting Abstracts). 2022;140(suppl 1):577‐578. doi: 10.1182/blood-2022-156389
   Google Scholar
• Sørensen AL, Mikkelsen SU, Knudsen TA, et al. Ruxolitinib and interferon-α2 combination therapy for patients with polycythemia vera or myelofibrosis: a phase II study. Haematologica. 2020;105(9):2262–2272. doi: 10.3324/haematol.2019.235648
   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. doi: 10.1182/blood-2018-04-846626
   PubMed Web of Science ®Google Scholar
• Hernández-Boluda JC, Correa JG, García-Delgado R, et al. Predictive factors for anemia response to erythropoiesis-stimulating agents in myelofibrosis. Eur J Haematol. 2017 Apr;98(4):407–414. doi: 10.1111/ejh.12846
   PubMed Web of Science ®Google Scholar
• Cervantes F, Isola IM, Alvarez-Larrán A, et al. Danazol therapy for the anemia of myelofibrosis: assessment of efficacy with current criteria of response and long-term results. Ann Hematol. 2015 Nov;94(11):1791–1796. doi: 10.1007/s00277-015-2435-7
   PubMed Web of Science ®Google Scholar
• Castillo-Tokumori F, Talati C, Al Ali N, et al. Retrospective analysis of the clinical use and benefit of lenalidomide and thalidomide in myelofibrosis. Clin Lymphoma Myeloma Leuk. 2020 Dec;20(12):e956–e960. doi: 10.1016/j.clml.2020.07.006
   PubMed Web of Science ®Google Scholar
• Qu S, Xu Z, Qin T, et al. Ruxolitinib combined with prednisone, thalidomide and danazol in patients with myelofibrosis: results of a pilot study. Hematol Oncol. 2022 Oct;40(4):787–795. doi: 10.1002/hon.3026
   PubMed Web of Science ®Google Scholar
• Gerds AT, Vannucchi AM, Passamonti F, et al. Duration of response to Luspatercept in patients (pts) requiring red blood cell (RBC) transfusions with myelofibrosis (MF) - updated data from the phase 2 ACE-536-MF-001 study. Blood (ASH Annual Meeting Abstracts). 2020;136(suppl Supplement 1):47–48. doi: 10.1182/blood-2020-137265
   Google Scholar
• Harrison C, Ross DM, Chee LCY, et al. Modulation of TGF-b superfamily signaling by KER-050 (elritercept) demonstrates potential to treat myelofibrosis and mitigate ruxolitinib-associated cytopenias. Blood (ASH Annual Meeting Abstracts). 2023;142(Suppl 1):3185. doi: 10.1182/blood-2023-187099
   Google Scholar
• Mohan S, Oh ST, Kiladjian JJ, et al. Phase 1/2 study of the activin receptor-like kinase-2 inhibitor zilurgisertib (INCB000928, LIMBER-104) as monotherapy or with ruxolitinib in patients with anemia due to myelofibrosis. Blood (ASH Annual Meeting Abstracts). 2023;142(Suppl Supplement 1):624. doi: 10.1182/blood-2023-179291
   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(18):2378–2388. doi: 10.1182/bloodadvances.2018022012
   PubMed Web of Science ®Google Scholar
• Mascarenhas J, Komrokji RS, Palandri F, et al. Randomized, Single-Blind, multicenter phase II study of two doses of imetelstat in relapsed or refractory myelofibrosis. J Clin Oncol. 2021;39(26):2881–2892. doi: 10.1200/JCO.20.02864
   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. doi: 10.1002/cncr.32664
   PubMed Web of Science ®Google Scholar
• Kuykendall AT, Sun L, Mascarenhas J, et al. Favorable overall survival with imetelstat in relapsed/refractory myelofibrosis patients compared with real-world data. Ann Hematol. 2022;101(1):139–146. doi: 10.1007/s00277-021-04683-w
   PubMed Web of Science ®Google Scholar
• Gill H, Yacoub A, Pettit K, et al. A phase 2 study of IMG-7289 (bomedemstat) for the treatment of advanced myelofibrosis. Hemasphere. 2022;6:941–942. doi: 10.1097/01.HS9.0000847072.29067.d6
   Google Scholar
• Gill H, Au L, Leung G, et al. Phase 2 study to assess the safety and efficacy of bomedemstat (MK3543) in combination with ruxolitinib in patients with myelofibrosis. Blood (ASH Annual Meeting Abstracts). 2023;142(Suppl Supplement 1):621. doi: 10.1182/blood-2023-181971
   PubMedGoogle Scholar
• Al-Ali H, Garcia Delgado R, Lange A, et al. KRT-232, a first-in-class, murine double minute 2 inhibitor (MDM2I), for myelofibrosis (MF) relapsed or refractory (R/R) to janus-associated kinase inhibitor (JAKI) treatment (TX). Hemasphere. 2020;4:65.
   Google Scholar
• Vachani P, Lange A, Garcia Delgado R, et al. Potential disease-modifying activity of navtemadlin (KRT-232), a first-in-class MDM2 inhibitor, correlates with clinical benefits in relapsed/refractory myelofibrosis (MF). Blood (ASH Annual Meeting Abstracts). 2021;138(Suppl Supplement 1):3581. doi: 10.1182/blood-2021-147543
   Google Scholar
• Mascarenhas J, Jain T, Otoukesh S, et al. An open-label, global, phase (Ph) 1b/2 study adding navtemadlin (NVTM) to ruxolitinib (RUX) in patients (pts) with primary or secondary myelofibrosis (MF) who have a suboptimal response to RUX. Hemasphere. 2023;7(Suppl 3):S210. doi: 10.1097/01.HS9.0000967752.72578.ff
   Google Scholar
• Yacoub A, Patnaik MM, Ali H, et al. A phase 1/2 study of Single Agent Tagraxofusp, a first-in-class CD123-targeted therapy, in patients with myelofibrosis that is Relapsed/Refractory following JAK inhibitor therapy. Patients With Myelofibrosis That Is Relapsed/Refractory Following JAK Inhibitor Therapy Blood (ASH Annual Meeting Abstracts). 2021;138(Suppl Supplement 1):140. doi: 10.1182/blood-2021-145276
   Google Scholar
• Mascarenhas J, Migliaccio AR, Kosiorek H, et al. A phase ib trial of AVID200, a TGFβ 1/3 Trap, in patients with myelofibrosis. Clin Cancer Res. [2023 Sep 15];29(18):3622–3632. doi: 10.1158/1078-0432.CCR-23-0276
   PubMed Web of Science ®Google Scholar
• Passamonti F, Mora B. Myelofibrosis. Blood. 2023;141(16):1954–1970. doi: 10.1182/blood.2022017423
   PubMed Web of Science ®Google Scholar
• Talpaz M, Prchal J, Afrin L, et al. Safety and efficacy of ruxolitinib in patients with Myelofibrosis and low platelet counts (50 - 100 × 109/L): final analysis of an open-label phase 2 study. Clin Lymphoma Myeloma Leuk. 2022 May;22(5):336–346. doi: 10.1016/j.clml.2021.10.016
   PubMed Web of Science ®Google Scholar
• Tiribelli M, Palandri F, Sant’antonio E, et al. The role of allogeneic stem-cell transplant in myelofibrosis in the era of JAK inhibitors: a case-based review. Bone Marrow Transplant. 2020;55(4):708–716. doi: 10.1038/s41409-019-0683-1
   PubMed Web of Science ®Google Scholar